Colorized scanning electron micrograph of a cultured human neuron. Photo credit: Thomas Deerinck, UC San Diego National Center for Microscopy and Imaging

Using stem cells to restore lost functions due to spinal cord injury (SCI) has long been an ambition of scientists and doctors. Nearly 18,000 people in the United States suffer SCIs each year, with another 294,000 persons living with an SCI, usually involving some degree of permanent paralysis or diminished physical function, such as bladder control or difficulty breathing.

In a new study, published August 5, 2020 in Cell Stem Cell, researchers at University of California San Diego School of Medicine report successfully implanting highly specialized grafts of neural stem cells directly into spinal cord injuries in mice, then documenting how the grafts grew and filled the injury sites, integrating with and mimicking the animals existing neuronal network.

Until this study, said the studys first author Steven Ceto, a postdoctoral fellow in the lab of Mark H. Tuszynski, MD, PhD, professor of neurosciences and director of the Translational Neuroscience Institute at UC San Diego School of Medicine, neural stem cell grafts being developed in the lab were sort of a black box.

Although previous research, including published work by Tuszynski and colleagues, had shown improved functioning in SCI animal models after neural stem cell grafts, scientists did not know exactly what was happening.

We knew that damaged host axons grew extensively into (injury sites), and that graft neurons in turn extended large numbers of axons into the spinal cord, but we had no idea what kind of activity was actually occurring inside the graft itself, said Ceto. We didnt know if host and graft axons were actually making functional connections, or if they just looked like they could be.

Ceto, Tuszynski and colleagues took advantage of recent technological advances that allow researchers to both stimulate and record the activity of genetically and anatomically defined neuron populations with light rather than electricity. This ensured they knew exactly which host and graft neurons were in play, without having to worry about electric currents spreading through tissue and giving potentially misleading results.

They discovered that even in the absence of a specific stimulus, graft neurons fired spontaneously in distinct clusters of neurons with highly correlated activity, much like in the neural networks of the normal spinal cord. When researchers stimulated regenerating axons coming from the animals brain, they found that some of the same spontaneously active clusters of graft neurons responded robustly, indicating that these networks receive functional synaptic connections from inputs that typically drive movement. Sensory stimuli, such as a light touch and pinch, also activated graft neurons.

We showed that we could turn on spinal cord neurons below the injury site by stimulating graft axons extending into these areas, said Ceto. Putting all these results together, it turns out that neural stem cell grafts have a remarkable ability to self-assemble into spinal cord-like neural networks that functionally integrate with the host nervous system. After years of speculation and inference, we showed directly that each of the building blocks of a neuronal relay across spinal cord injury are in fact functional.

Tuszynski said his team is now working on several avenues to enhance the functional connectivity of stem cell grafts, such as organizing the topology of grafts to mimic that of the normal spinal cord with scaffolds and using electrical stimulation to strengthen the synapses between host and graft neurons.

While the perfect combination of stem cells, stimulation, rehabilitation and other interventions may be years off, patients are living with spinal cord injury right now, Tuszynski said. Therefore, we are currently working with regulatory authorities to move our stem cell graft approach into clinical trials as soon as possible. If everything goes well, we could have a therapy within the decade.

Co-authors of the study are Kohel J. Sekiguchi and Axel Nimmerjahn, Salk Institute for Biological Studies and Yoshio Takashima, UC San Diego and Veterans Administration Medical Center, San Diego.

Funding for this research came, in part, from Wings for Life, the University of California Frontiers of Innovation Scholars Program, the Veterans Administration (Gordon Mansfield Spinal Cord Injury Collaborative Consortium, RR&D B7332R), the National Institutes of Health (grants NS104442 and NS108034), The Craig H. Neilsen Foundation, the Kakajima Foundation, the Bernard and Anne Spitzer Charitable Trust and the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation.

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Implanted Neural Stem Cell Grafts Show Functionality in ...

Spinal Cord Stem Cells Comments Off on Implanted Neural Stem Cell Grafts Show Functionality in … | August 12th, 2020

Although spinal cord injuries (SCI) are not as prevalent as other debilitating conditions, they can be particularly devastating. Patients often lose motor control and sensibility and require assistance with everyday tasks. Most people are familiar with the case of Christopher Reeve, an American actor who played Superman in the 70s and 80s. He suffered a cervical spinal cord injury and was left paralyzed from the neck down. Reeve became an advocate for research into a potential cure using stem cells.

The World Health Organization estimates that every year up to 500,000 people suffer this type of injury worldwide. In Canada, approximately 85,000 people are currently living with some type of SCI.

The possibility of repairing damage sustained by the spinal cord is one of the most exciting potential applications of regenerative medicine. There have been promising advancements in this field and it is just a matter of time before they give way to actual treatments. Access to these treatments is just one of the many advantages of cell banking.

Inside the spinal column, which runs from the base of the skull to the coccyx, lies a fragile structure made up of nervous tissue. This is the spinal cord. Its job is to carry nerve signals from the brain to the rest of the body.

The spinal cord is very delicate and while it is protected by the vertebrae, it can easily be damaged either by trauma or disease. Injuries are graded according to their severity on a scale designed by the American Spinal Injury Association (ASIA) that goes from A to E (A being a complete injury, where all motor and sensory function is lost. E represents normal function). The severity of the injury is inversely correlated with the probability of recovery. According to the American Association of Neurological Surgeons, nearly half of all spinal cord injuries are complete.

In addition to the physical damage, spinal cord injuries are incredibly challenging in psychological terms. The more severe the injury, the more likely it is that the individual will lose the ability to care for himself. As reported in Mayo Clinic Proceedings, adults with spinal cord injury are at a higher risk of developing mental health disorders. Additionally, the rate of suicide increases three-fold among patients with this type of injury compared to the general population.

In a study published in Topics in Spinal Cord Injury Rehabilitation, researchers from the University of Alabama analyzed data from patients that sustained spinal cord injuries from 2005 to 2011. They found that automobile crashes (31.5% of cases) and falls (25.3%) account for more than half of all incidents. Other common causes are gunshot wounds, motorcycle crashes, and diving accidents. Although not frequent, diseases can cause spinal cord injury as well, specifically cancer, and osteoporosis.

Considering all causes, men account for 8 out of every 10 cases of spinal cord injury. Additionally, according to the Mayo Clinic, people are more likely to suffer traumatic cord injuries between the ages of 16 and 30. Avoiding risky behavior is the most effective strategy for preventing spinal cord injury.

In addition to the ASIA scale, which ranks injury by the severity of the damage, spinal cord injuries can be classified depending on the area affected. There are four types of spinal cord injury: cervical, thoracic, lumbar, and sacral.

The uppermost portion of the spine (vertebrae C1 to C7) is called the cervical section. Traumatic cord injuries in this area can lead to quadriplegia or full paralysis. This is the sort of injury that actor Christopher Reeve sustained. He shattered his C-1 and C-2 vertebrae in a horseback riding accident. Baseball player Roy Campanella damaged his C-5 and C-6 vertebrae in an automobile accident.

The thoracic spine is comprised of 12 vertebrae (T-1 to T-12) and it is located below the cervical section. An injury to this area could result in loss of use of the chest, upper back, and abdominals.

The lumbar section of the spine is located in the lower back. It comprises five vertebrae (L1 to L5). An injury to this area can leave an individual paraplegic, unable to move or feel anything below the point of injury. Deng Pufang, son of Chinas former leader Deng Xiaoping, suffered a lumbar spinal cord injury and became paralyzed.

The sacral section is located between the lumbar section and the coccyx. Injury to this area may cause loss of function in the hips and legs. Bladder function may be compromised as well. Injuries to this section of the spine are less common than cervical, thoracic, or lumbar injuries.

A discovery made by researchers John B. Gurdon and Shinya Yamanaka, for which they won the 2012 Nobel Prize in Medicine, may hold the key to repairing spinal cord injury. They found a way to induce adult cells, like those located in your hair follicles, to become pluripotent. Once this happens, these cells, called induced pluripotent stem cells (iPSCs), can become any cell type in the body.

A team of researchers from Keio University in Japan injected mice that had suffered spinal cord injury with neural cells derived from human iPSCs. These cells were able to successfully migrate and differentiate into their appropriate neural lineages, and they performed synapses. This means that they became exactly the type of cell needed in the place of injury and they successfully communicated with each other.

According to the scientists, compared to the control group, the mice injected with iPSCs had a significantly better functional recovery. The results of this trial, published in Proceedings of the National Academy of Sciences of the United States of America, are a step forward in the path towards eventually promoting complete functional recovery of spinal cord injury in humans.

Once this method is perfected and made available to the public, doctors will need a cell sample that they can turn into neural cells to treat people who suffer from spinal cord injury. It is important to note that the sooner that sample is taken and preserved, the higher its therapeutic potential will be, not only to treat spinal cord injury but many other conditions like Parkinsons, Alzheimers and macular degeneration.This means that the sooner you take action and have your live cells cryopreserved, the better prepared you will be to take advantage of the revolution of regenerative medicine that is coming. To learn more about the ways you can have your cells banked at Acorn, click here.

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Repairing damage caused by spinal cord injury with stem cells

Spinal Cord Stem Cells Comments Off on Repairing damage caused by spinal cord injury with stem cells | August 12th, 2020

The spinal cord is a bundle of nerves inside the spine that gives your body structure and support. Spinal cord injuries (SCIs) tend to be devastating and most are permanent. Recent research has shown that motor neuron obtained from skin cells could serve as potential treatments for spinal cord injuries, and thus has received considerable research attention. With this, a new door has been opened for treating not only spinal cord injuries, caused by workplace accidents and car crashes, but also Lou Gehrigs disease, known as amyotrophic lateral sclerosis or ALS.

A research team, led by Professor Jeong Beom Kim and his research team in the School of Life Sciences at UNIST has demonstrated that human fibroblasts can be converted into induced motor neurons (iMNs) by sequentially inducing two transcription factors, POU5F1(OCT4) and LHX3. The research team further investigated the therapeutic effects of iMNs for treating traumatic spinal cord injury using rodent spinal cord injury model. Their findings indicate that the sequential induction of two transcription factors is essential for generating self-renewing iMNICs more efficiently. This method not only ensures large-scale production of pure iMNs, but also facilitates the feasibility of iMNs for SCI treatment.

The spinal cord is responsible for transmitting signals from the brain to the rest of the body, and vice versa. Along with motor and sensory deficits, damage to the spinal cord can cause long-term complications, including limited mobility. Although there are many treatment options available for people with SCI, most of them have adverse side effects that impact therapy. And this is why stem cell (SC) therapies to restore functions of damaged tissues are attracting attention, recently. Among those cells constituting the spinal cord, motor neurons that involved in the regulation of muscle function have emerged as a promising candidate for the stem cell-based therapy for SCIs. Despite these encouraging advances, ethical issue of embryonic stem cells (ESCs) and tumorigenic potential of induced pluripotent stem cells (iPSCs) have impeded their translations into clinical trials.

Figure 1. The experimental scheme for the generation of induced motor neurons (iMNs) from human fibroblasts via sequential transduction of two transcription factors.

To overcome these limitations, Professor Kim and his research team established an advanced direct conversion strategy to generate iMNs from human fibroblasts in large-scale with high purity, thereby providing a cell source for the treatment of SCI. These iMNs possessed spinal cord motor neuronal identity and exhibit hallmarks of spinal MNs, such as neuromuscular junction formation capacity and electrophysiological properties in vitro. Importantly, their findings also show that transplantation of iMNs improved locomotor function in rodent SCI model without tumor formation. According to the research team, This proof-of-concept study shows that our functional iMNs can be employed to cell-based therapy as an autologous cell source. Through this, they resolved the problem of immune rejection, and thus reduce the risk of cancer.

In the study, we succeeded in generating iMNs from human fibroblasts by overexpressing POU5F1(OCT4) and LHX3, says Hyunah Lee (Combined MS/Ph.D program of Life Sciences, UNIST), the first author of the study.

Figure 2. Therapeutic effects of iMNs in rat spinal cord injury model in vivo. (A) The position of hindlimbs in control rat and iMN-transplanted rat after 8 weeks of transplantation. (B) C staining analysis of spinal cords after 8 weeks of transplantation (I; Control, J; iMN-transplanted).

The developed motor nerve cell manufacturing method has the advantage of being capable of mass production. A sufficient amount of cells is required for patient clinical treatment, but the existing direct differentiation technique has limited the number of cells that can be obtained. On the other hand, the method developed by the research team is capable of mass production because it undergoes an intermediate cell stage capable of self-renewal. After injecting the produced cells into the spinal cord injury mice, it was confirmed that the lost motor function is restored and the nerves are regenerated in the damaged spinal cord tissue.

Although further investigation on mechanism responsible for cell fate conversion may be needed, our strategy is a safer and simpler methodology that may provide new insights to develop personalized stem cell therapy and drug screening for MN diseases or spinal cord disorders, says Professor Kim. If combined with SuPine Patch, an adhesive hydrogel patches with the purpose of regenerating the damaged spinal cords, its therapeutic effects will be maximized. He adds, As the incidence of spinal cord injury is high due to industrial accidents, synergistic effects with public hospitals specializing in industrial accidents scheduled to be built in Ulsan should be expected.

This study has been jointly carried out with Professor Kims startup company, SuPine Therapeutics Inc. with the support of the Ministry of SMEs and Startups (MSS). The findings of this research have been published in the 2020 June issue of the online edition of eLife, a renowned academic journal of the European Molecular Biology Organizationl (EMBO).

Journal Reference

Hyunah Lee, Hye Yeong Lee, Byeong Eun Lee, et al., Sequentially induced motor neurons from human fibroblasts facilitate locomotor recovery in a rodent spinal cord injury model, eLife, (2020).

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New Study Presents Cell-based Therapy for MN Diseases or Spinal Cord Disorders - Mirage News

Spinal Cord Stem Cells Comments Off on New Study Presents Cell-based Therapy for MN Diseases or Spinal Cord Disorders – Mirage News | August 11th, 2020

Aug. 9, 2020 / PRZen / SUZHOU, China -- Re-Stem Biotech (Re-Stem or the Company), a biotechnology firm engaged in the research and development of cell therapies targeting osteoarthritis, spinal cord injury and various cancers recently funded in part a study titled "Solid-phase inclusion as a mechanism for regulating unfolded proteins in the mitochondrial matrix". Researchers at The Johns Hopkins University School of Medicine, led by Rong Li, Ph.D., published their findings in the journal "Science Advances" (view full article here: https://advances.sciencemag.org/content/6/32/eabc7288) The study may provide mechanistic insights for mitochondrial dysfunction observed in aging and age-related diseases.

About Mitochondrial DysfunctionMitochondrial dysfunction is a hallmark of age-related diseases, such as cardiovascular diseases and neurodegenerative diseases. During aging, damaged/unfolded proteins in mitochondria that are failed to be degraded gradually accumulate. In addition, recent evidence suggests that non-mitochondrial proteins constituting pathological aggregates in neurodegenerative diseases also accumulate in mitochondria and cause mitochondrial dysfunction. However, it remains unclear how excessive damaged proteins are managed within mitochondria when known quality control mechanisms become inadequate, and how they contribute to mitochondrial dysfunction during the aging process.

About the StudyThe researchers discovered that excessive unfolded proteins in the mitochondrial matrix are consolidated into novel structures, which they named Deposits of Unfolded Mitochondrial Proteins (DUMP). DUMP formation is an age-dependent process, while accelerated DUMP formation causes mitochondrial dysfunction and premature aging. They found that DUMP formation was not random, but specific in mitochondria near endoplasmic reticulum (ER), another organelle in cells. The contact sites between mitochondria and ER regulate DUMP formation via transferring lipids between two organelles. Via a series of genetic and live-cell imaging studies, researchers identified key enzymes of mitochondrial lipid metabolism that control DUMP formation. Manipulation of these enzymes modulates DUMP formation, therefore, potentially they could be targets for anti-aging or treating age-related diseases.

About Re-Stem BiotechRe-Stem Biotech (Re-Stem) is a biotechnology firm engaged in the research and development of cell-based therapies and products. Backed by state of the art GMP facilities and an international team of world-leading scientists, doctors and management team, Re-Stem currently has a robust technology platform including four profitable therapies on the market and eight other therapies and products in the pipeline. Incorporated and headquartered in 2012 in Suzhou, China, Re-Stem is focused on the large and aging population of China. It also operates clinics and research and development laboratories in Shenzhen, Beijing, Kunming and Ganzhou. For more information visit Re-Stem Biotech website: https://www.restembio.com/

Forward-Looking StatementsStatements in this press release relating to plans, strategies, trends, specific activities or investments, and other statements that are not descriptions of historical facts and may be forward-looking statements are inherently subject to risks and uncertainties, and actual results could differ materially from those currently anticipated due to a number of factors, which include those regarding our ability to implement objective, plans and strategies for future operations. Forward-looking information may be identified by terms such as "will," "may," "expects," "plans," "intends," "estimates," "potential," or "continue," or similar terms or the negative of these terms. Although Re-Stem believes the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that future results, performance or achievements will be obtained. Re-Stem does not have any obligation to update these forward-looking statements other than as required by law.

Follow the full story here: https://przen.com/pr/33354426

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Re-Stem-Funded Study Targeting Mitochondrial Dysfunction in Aging and Age-Related Diseases Published in Science Advances Journal - Press Release -...

Spinal Cord Stem Cells Comments Off on Re-Stem-Funded Study Targeting Mitochondrial Dysfunction in Aging and Age-Related Diseases Published in Science Advances Journal – Press Release -… | August 10th, 2020

After case studies of COVID-19, researchers from the Max Planck Institute of Experimental Medicine in Gttingen, Germany, believe that erythropoietin (EPO) could be used to treat COVID-19 patients. A go-to for many dopers in endurance sports, EPO stimulates the production of red blood cells, which can help with oxygen supply in a users brain and muscles. The team of Mac Planck researchers are planning a clinical trial to take a more in-depth look at EPO and its effects on the coronavirus, and they have outlined their reasoning in an articleon the institutes website.

The German team of researchers cite a COVID-19 case from Iran which occurred in March. The patient was infected with the coronavirus, but doctors noted that they also had poor blood values. To combat this, the patient was prescribed EPO in addition to the regular care for COVID-19. Just a week after starting the EPO treatment, the patient was well enough to return home.

RELATED: Ottawa Marathon third-place finisher provisionally suspended for EPO

Researchers also point to South American populations that live at high altitudes, where severe illnesses are rarer than in lower regions. The Mac Planck Institute team writes that this may be because people living at higher altitudes form more EPO and are better adapted to oxygen deficiency because they have more red blood cells.

According to the article, experiments on animals have shown that EPO affectsparts of the brain stem and spinal cord that control breathing. As a result, the researchers write, breathing improves when there is an oxygen deficiency. EPO also has an anti-inflammatory effect on immune cells and could thus attenuate the frequently exaggerated immune response in COVID-19 patients.

RELATED: WADA to fund anti-doping project piloting artificial intelligence

Dr. Hannelore Ehrenreich, one of the researchers on the German team, notes the importance of determining whether EPO could be an effective treatment for the coronavirus.

Because COVID-19 can have such severe health-related consequences, we must investigate any evidence of a protective effect of EPO, Ehrenreich says. After all, there is currently neither a vaccine nor a medication for the disease. We are therefore preparing a proof-of-concept study to investigate the effect of EPO on COVID-19 in humans. The clinical trial will see severely ill COVID-19 patients receiving EPO to see if it can alleviate severe disease progression.

RELATED: More than half of Bahrains athletics medals are tainted by doping scandals

Following the release of the Max Planck article, running coach Steve Magness tweeted, Just waiting for some athlete or coach trying to get a TUE for taking EPO for a mild COVID case. TUE stands for Therapeutic Use Exemption, which allows athletes to use banned substances for legitimate medical reasons. Magnesss tweet had the feel of a joke, but theres a ring of truth to it. Dopers will do a lot to get away with cheating, and if using EPO to treat COVID-19 becomes a widespread treatment, some athletes might actually take advantage of it.

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Could EPO be used to treat COVID-19? - Canadian Running Magazine

Spinal Cord Stem Cells Comments Off on Could EPO be used to treat COVID-19? – Canadian Running Magazine | July 8th, 2020

Central nervous system comprises brain and spinal cord, and is responsible for integration of sensory information. Brain is the largest and one of the most complex organs in the human body. It is made up of 100 billion nerves that communicate with 100 trillion synapses. It is responsible for the thought and movement produced by the body. Spinal cord is connected to a section of brain known as brain stem and runs through the spinal canal. The brain processes and interprets sensory information sent from the spinal cord. Brain and spinal cord serve as the primary processing centers for the entire nervous system, and control the working of the body. Neuroprosthetics improves or replaces the function of the central nervous system. Neuroprosthetics, also known as neural prosthetics, are devices implanted in the body that stimulate the function of an organ or organ system that has failed due to disease or injury. It is a brain-computer interface device used to detect and translate neural activity into command sequences for prostheses. Its primary aim is to restore functionality in patients suffering from loss of motor control such as spinal cord injury, multiple sclerosis, amyotrophic lateral sclerosis, and stroke. The major types of neuroprosthetics include sensory implants, motor prosthetics, and cognitive prosthetics. Motor prosthetics support the autonomous system and assist in the regulation or stimulation of affected motor functions.

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Similarly, cognitive prosthetics restore the function of brain tissue loss in conditions such as paralysis, Parkinsons disease, traumatic brain injury, and speech deficit. Sensory implants pass information into the bodys sensory areas such as sight or hearing, and it is further classified as auditory (cochlear implant), visual, and spinal cord stimulator. Some key functions of neuroprosthetics include providing hearing, seeing, feeling abilities, pain relief, and restoring damaged brain cells. Cochlear implant is among the most popular neuroprosthetics. In addition, auditory brain stem implant is also a neuroprosthetic meant to improve hearing damage.

North America dominates the global market for neuroprosthetics due to the rising incidence of neurological diseases and growth in geriatric population in the region. Asia is expected to display a high growth rate in the next five years in the global neuroprosthetics market, with China and India being the fastest growing markets in the Asia-Pacific region. Among the key driving forces for the neuroprosthetics market in developing countries are the large pool of patients, increasing awareness about the disease, improving healthcare infrastructure, and rising government funding in the region.

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Increasing prevalence of neurological diseases such as traumatic brain injury, stroke and Parkinsons disease, rise in geriatric population, increase in healthcare expenditure, growing awareness about healthcare, rapid progression of technology, and increasing number of initiatives by various governments and government associations are some key factors driving growth of the global neuroprosthetics market. However, factors such as high cost of devices, reimbursement issues, and adverse effects pose a major restraint to the growth of the global neuroprosthetics market.

Innovative self-charging neural implants that eliminate the need for high risk and costly surgery to replace the discharge battery and controlling machinery with thoughts would help to develop opportunities for the growth of the global neuroprosthetics market. The major companies operating in the global neuroprosthetics market are Boston Scientific Corporation, Cochlear Limited, Medtronic, Inc., Cyberonics, Inc., NDI Medical LLC, NeuroPace, Inc., Nervo Corp., Retina Implant AG, St. Jude Medical, and Sonova Group.

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Growing population and economies in the developing countries such as India and China are expected to drive growth of the prosthetic heart valves market in Asia. In addition, introduction of innovative products with technological advancements, increasing demand for minimally-invasive devices, and rise in incidences of cardiac valve disorders are expected to create opportunities for the global prosthetic heart valves market. Increasing number of mergers and acquisitions, rise in the number of collaborations and partnerships, and product launches are some of the latest trends in the global prosthetic heart valves market.Some of the major companies operating in the global prosthetic heart valves market are Edwards Lifesciences, Medtronic, Abbott Laboratories,ON-X LIFE TECHNOLOGIES, INC.,and St. Jude Medical.In addition, some of the other companies operating in the global prosthetic heart valves market are Sorin Group, CryoLife, LepuMedical, and Braile Biomedica, Ltda.

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Revenue from the Sales of Neuroprosthetics Market to Increase Exponentially During 2015 2021 - 3rd Watch News

Spinal Cord Stem Cells Comments Off on Revenue from the Sales of Neuroprosthetics Market to Increase Exponentially During 2015 2021 – 3rd Watch News | July 7th, 2020

Spinal Fusion Market 2020: Latest Analysis

Chicago, United States:-TheSpinal Fusion market report5 Years Forecast [2020-2025]focuses on theCOVID19 Outbreak Impact analysis of key points influencing the growth of the market. The research report on the Spinal Fusion Market is a deep analysis of the market. This is a latest report, covering the current COVID-19 impact on the Spinal Fusion market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. Experts have studied the historical data and compared it with the changing market situations. The report covers all the necessary information required by new entrants as well as the existing players to gain deeper insight.

Furthermore, the statistical survey in the report focuses on product specifications, costs, production capacities, marketing channels, and market players. Upstream raw materials, downstream demand analysis, and a list of end-user industries have been studied systematically, along with the suppliers in this market. The product flow and distribution channel have also been presented in this research report.

Top Players of Spinal Fusion Market are studied:CharterWorthington IndustriesCesca TherapeuticsShengjie Cryogenic EquipmentSichuan mountain verticalQingdao Beol

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Note: Covid-19 pandemic affects most industries in the globe. Here at acquire market research we offer you comprehensive data of related industry which will help and support your business in all possible ways.Due to the pandemic of COVID-19 businesses have seen a decrease in their profits. While our intention is to help businesses regain their profits we also provide information regarding the COVID-19 virus to help our customers stay safe during the pandemic

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The analysis includes market size, upstream situation, market segmentation, market segmentation, price & cost and industry environment. In addition, the report outlines the factors driving industry growth and the description of market channels.The report begins from overview of industrial chain structure, and describes the upstream. Besides, the report analyses market size and forecast in different geographies, type and end-use segment, in addition, the report introduces market competition overview among the major companies and companies profiles, besides, market price and channel features are covered in the report.

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Our exploration specialists acutely ascertain the significant aspects of the global Spinal Fusion market report. It also provides an in-depth valuation in regards to the future advancements relying on the past data and present circumstance of Spinal Fusion market situation. In this Spinal Fusion report, we have investigated the principals, players in the market, geological regions, product type, and market end-client applications. The global Spinal Fusion report comprises of primary and secondary data which is exemplified in the form of pie outlines, Spinal Fusion tables, analytical figures, and reference diagrams. The Spinal Fusion report is presented in an efficient way that involves basic dialect, basic Spinal Fusion outline, agreements, and certain facts as per solace and comprehension.

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Report Overview:It includes major players of the globalkeywordmarket covered in the research study, research scope, and market segments by type, market segments by application, years considered for the research study, and objectives of the report.

Global Growth Trends:This section focuses on industry trends where market drivers and top market trends are shed light upon. It also provides growth rates of key producers operating in the globalkeywordmarket. Furthermore, it offers production and capacity analysis where marketing pricing trends, capacity, production, and production value of the globalkeywordmarket are discussed.

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Market Size by Type:This section concentrates on product type segments where production value market share, price, and production market share by product type are discussed.

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Production by Region:Here, the production value growth rate, production growth rate, import and export, and key players of each regional market are provided.

Consumption by Region:This section provides information on the consumption in each regional market studied in the report. The consumption is discussed on the basis of country, application, and product type.

Company Profiles:Almost all leading players of the globalkeywordmarket are profiled in this section. The analysts have provided information about their recent developments in the globalkeywordmarket, products, revenue, production, business, and company.

Market Forecast by Production:The production and production value forecasts included in this section are for the globalkeywordmarket as well as for key regional markets.

Market Forecast by Consumption:The consumption and consumption value forecasts included in this section are for the globalkeywordmarket as well as for key regional markets.

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COVID-19 impact on Spinal Fusion Market Growth Analysis, Demand by Regions and Global Forecasts To 2025 | Charter, Worthington Industries, Cesca...

Spinal Cord Stem Cells Comments Off on COVID-19 impact on Spinal Fusion Market Growth Analysis, Demand by Regions and Global Forecasts To 2025 | Charter, Worthington Industries, Cesca… | July 7th, 2020

Lineage Cell Therapeutics (LCTX) is a small biotech developing 3 different off-the-shelf cell therapy products in dry AMD, spinal cord injuries, and non-small cell lung cancer. These are large unmet medical needs and have attracted interest from third-party groups that have provided some funding to advance Lineages programs. Currently valued at just under $125 million, Lineage could clearly have a huge upside if these programs make it to market. This article provides my assessment of why Lineage is worth the risk at present.

Lineages main pipeline asset is OpRegen which is being developed to treat dry age-related macular degeneration. Dry AMD is a huge market opportunity. One market estimate Ive seen is that the dry AMD market was worth approximately $2.09 billion in 2017 and that the overall growth rate of the AMD market (both dry and wet AMD) as a whole is expected to be around 7.8% in the coming years. It's likely this growth rate understates dry AMD growth potential in particular given the relative lack of treatment options for dry AMD as compared to wet AMD.

According to Lineage, this estimate is likely way under what the true market size could be, too. Lineage says the wet AMD market is greater than $10 billion yet dry AMD represents about 90% of the total cases.

Figure 1: Lineages Dry AMD Competition (source: corporate presentation)

Apellis (APLS) has a Phase 3 candidate for dry AMD that, if successful, will likely hit the market before OpRegen. In addition, there are a couple other potential therapies that are more early-stage products. As you can see from Figure 1 though, OpRegen should stack up pretty well if Lineage can continue to successfully develop the product.

OpRegen utilizes Lineages technology platform of off-the-shelf pluripotent stem cells.

Figure 2: Lineages Technology Platform (source: corporate presentation)

In the case of OpRegen, Lineage is using these pluripotent stem cells to create retinal pigment epithelium cells that are injected into the eye to replace those lost due to disease. This should support photoreceptors that otherwise would have lost function due to disease progression, causing decreased levels of vision and eventually blindness over time in severe cases.

Figure 3: OpRegen Mechanism of Action (source: corporate presentation)

Lineage has an ongoing Phase 1/2a trial that is close to full enrollment, lacking just 3 patients now. Enrollment was temporarily paused due to the COVID-19 pandemic, but the company has re-initiated patient enrollment and hopes to have complete enrollment later this quarter (Q3). Lineage also recently reported that 1 of the 18 patients enrolled to date has already shown signs of retinal tissue regeneration, a first-in-human clinical result.

Although very early, this type of progress is encouraging, and its clear that the market potential is huge if Lineage can make it to the finish line. Investors should be aware though that a major setback in a lead program like OpRegen could be devastating to Lineage and its shareholders.

Lineages Other 2 Pipeline Programs Have Received Third-Party Funding and Similarly Address Large Market Needs

In addition to OpRegen, Lineage has 2 other potentially lucrative products in its pipeline.

Figure 4: Lineages Pipeline (source: Lineages website)

The one that is furthest along after OpRegen is OPC1 for spinal cord injury. Spinal cord trauma was a $2.27 billion market in 2017 and is expected to grow at 3.7% per year. Unfortunately for patients affected, there are no current treatments on the market that affect disease progression or reversal of the traumatic damage, but by contrast, OPC1 is intended to actually improve the function of a patients upper extremities.

Lineage uses its pluripotent stem cell technology to create oligodendrocyte progenitor cells and injects them into the area of the spinal injury. This hopefully allows for re-myelination at the injury site and increased nerve and blood vessel growth in the area. A Phase 1 and a Phase 1/2a trial have already been completed with promising results. Although obviously just one anecdotal case, Lineage has shared the story of 1 clinical trial patient going from complete paralysis to throwing out the first pitch at a baseball game.

Figure 5: Results of 1 OPC1 Trial Participant (source: Lineages website)

Lineage is in the process of evaluating this trial data and plans as a next step to have a meeting with the FDA about proceeding with a Phase 2/3 trial.

Lineages last pipeline asset is VAC2, which is a potential dendritic cell-based cancer vaccine that has received support from Cancer Research UK. The underlying VAC platform could potentially be used for both cancers and infectious diseases based on what type of antigen is loaded into the cells before injection into the body. The first targeted indication is in non-small cell lung cancer, for which there is already an ongoing Phase 1 trial. Interim Phase 1 data should be available in Q4. Upon receiving positive results, Lineage intends to try using VAC2 in conjunction with an immune checkpoint inhibitor in a Phase 2 trial.

A couple years back, the 20th drug on the list of best-selling anti-cancer agents still brought in $960 million and the 1st brought in $6.7 billion. That market is already larger now and will be even more so by the time Lineages therapy might hit the market because the estimated CAGR is around 7.6%. Non-small cell lung cancer in particular is estimated to be a $10.9 billion market next year and is notoriously hard to treat.

Also noteworthy is that Lineage has said it is investigating using its VAC platform for a potential COVID-19 vaccine. Lineage is reportedly seeking grant funding to continue this program. It seems like a long-shot for Lineage to get meaningfully involved in this effort well after many other better-funded companies, but I am glad to see that Lineage intends to use grant funding to pursue this opportunity rather than its own resources if there is funding to be had.

Lineage reported having $9.8 million in cash and $15.9 million in marketable securities as of the end of Q1. These marketable securities include stakes in OncoCyte (OCX), AgeX (AGE), and Hadasit. Lineage also holds a $24 million promissory note from Juvenescence that matures this August, but this note automatically converts to Juvenescence securities in the event of an IPO by Juvenescence before then.

Lineage has no long-term debt and reported an $8.4 million loss in Q1. This level of cash burn implies that Lineage can only make it to around the end of this year on its cash and securities alone. Lineage should get liquidity on its promissory note from Juvenescence later this year whether through repayment or an IPO. This will buy Lineage until late next year at current levels of spending.

I think its also worth noting that there is certainly a risk that the value of Lineages marketable securities will decline resulting in Lineage getting less cash from an eventual sale. For example, just this week OncoCyte reported that its lung nodule liquid biopsy failed and the stock tanked about 40%. Lineages remaining Oncocyte holdings are now worth only about $8.2 million rather than the $11.3 million estimated value reported at the end of Q1. Lineage may find it has far less cash available than expected if similar things keep happening.

Regardless, a company like Lineage will have to sell a large amount of its own stock to raise cash before its therapies will ever even have a chance of hitting the market. Lineages current stock price would make this means of funding inefficient and highly dilutive. Lineage investors need to be aware that major setbacks to its pipeline at this stage could result in the loss of most, if not all, invested capital.

I first analyzed future revenue and earnings estimates to assess Lineages value proposition. There were only 2 analyst estimates posted for most years over the next decade this is less than I would like to see but still a good starting point since these 2 analysts estimates are reasonably similar.

Figure 6: Lineage Sales and Earnings Estimates (source: Seeking Alpha)

As you can see from Figure 6, once Lineage is estimated to be cash-flow positive in 2023, its sales and earnings ratios become extremely low. Im not sure I can recall analyzing a company that was trading at less than .2x estimated future earnings when 15x is about average. I then discounted these estimates to see how the ratios changed.

Figure 7: Lineage Present Value Estimates (source: sales and earnings estimates from Seeking Alpha and my calculations based on them)

Even at an extremely high 30% discount rate to factor in the risky, early-stage nature of Lineage's pipeline, Lineage shares have roughly 2.5x upside to the present value estimate I calculated at an average 5 P/S and about 4.5x upside to my present value estimate calculated at an average 15 P/E. These are strongly suggestive that Lineage is undervalued. Despite this, given how early-stage and unproven as Lineages therapies are, I wanted to conduct a discounted cash flow analysis to see if it would confirm that Lineage shares provide a good risk/reward balance.

I estimated SG&A expenses as 34% of revenue, marketing expense at 5% of revenue, and cost of goods sold at 10%, at the point where the company has substantial cash flow in a few years, and I scale the numbers up fairly evenly from present values for the years in between. I also adjusted for future cash flow needs based on continuing cash burn for the foreseeable future. I used the market size estimates as described above for each of Lineages potential dry AMD and spinal cord trauma therapies, and for the potential cancer therapy, I modeled it as having peak sales equivalent to what would put it just barely in the top 20 of best-selling cancer drugs, albeit scaled up for expected overall market growth.

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Figure 8: Lineage Discounted Cash Flow Analysis (source: Lineages 10-Q and my calculations based on it)

As you can see in Figure 8, my model shows a potential fair value of $1.69 per share, which is over 80% above where Lineage is currently trading. In my view, this analysis, in combination with the sales and earnings estimates in Figure 7, shows that Lineage does present a relatively good risk/reward opportunity for long-term investors at current price levels. I recently added a small speculative position in Lineage stock to my portfolio, and I intend to hold on to most of the position long term, absent a substantial change to the companys outlook.

Lineage is targeting disease indications that are big, unmet market needs, but each pipeline therapy is still very early. Any potential investor should note that an investment in Lineage comes with a very real possibility of the loss of the entire investment if Lineages technology proves unsuccessful. That being said, I view the risk/reward as a decent bet at current price levels and have initiated a small position myself.

Disclosure: I am/we are long LCTX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: Im not a registered investment advisor. Despite that I strive to provide the most accurate information, I neither guarantee the accuracy nor the timeliness. Past performance does NOT guarantee future results. I reserve the right to make any investment decision for myself without notification. The thesis that I presented may change anytime due to the changing nature of information itself. Investment in stocks and options can result in a loss of capital. The information presented should NOT be construed as a recommendation to buy or sell any form of security. My articles are best utilized as educational and informational materials to assist investors in your own due diligence process. You are expected to perform your own due diligence and take responsibility for your actions. You should also consult with your own financial advisor for specific guidance as financial circumstances are individualized.

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Lineage Cell Therapeutics Is A High-Risk But High-Reward Opportunity To Consider - Seeking Alpha

Spinal Cord Stem Cells Comments Off on Lineage Cell Therapeutics Is A High-Risk But High-Reward Opportunity To Consider – Seeking Alpha | July 3rd, 2020

The doping drug EPO seems to ease severe corona cases. This is what researchers at the Max Planck Institute for Experimental Medicine in Gttingen, Germany, have now discovered. The drug that was originally intended as a cure for anemia might also be able to protect patients from neurological side effects once the SARS Cov-2 virus attacks the brain. The initial case studies were already very positive. The researchers are now starting a randomized clinical trial to systematically investigate the effects of EPO treatment in COVID-19 patients.

A patient with serious symptoms of COVID-19 was admitted to an Iranian hospital at the end of March. As he also had bad blood values, the doctors prescribed EPO in his case too. Another indication that EPO plays a protective role in COVID-19 comes from South America. There, serious diseases are rarer in high altitude areas than in the low-lying regions. Possibly because people living at higher altitudes produce more EPO themselves. In other words, they have more red blood cells and are better adapted to oxygen deficiencies. Could EPO have contributed to the rapid healing of the Iranian patient? And also explain the variances in the frequency of the disease in South America?

Hannelore Ehrenreich believes that this might indeed be possible. The scientist at the Max Planck Institute of Experimental Medicine suspects a correlation between the administration of EPO and a mild progression of the disease. We have found, for example, that dialysis patients tolerate COVID-19 remarkably well. It is precisely these patients who regularly receive erythropoietin (EPO) as part of their dialysis treatment, says Ehrenreich. EPO is released in our bodies as a natural reaction to reduced oxygen levels. The molecule stimulates the formation of red blood cells and thus improves the oxygen supply to the brain and muscles. Athletes who use synthetically produced EPO as a doping drug also benefit from this effect. However, EPO not only stimulates the blood cells but also a lot of other tissues in the body.

Ehrenreich and her colleagues have now summarized the studies on the effects of EPO that are already available. Including animal studies suggesting that EPO acts on the sections of the brain stem and spinal cord that regulate respiration. This improves respiration whenever there is a lack of oxygen. EPO also has an anti-inflammatory effect on immune cells, which may help to reduce the often exaggerated immune response occurring in COVID-19 patients. EPO can also protect against neurological symptoms and side effects of the disease, such as headaches, dizziness, loss of taste and smell, and epileptic seizures.

The protective effects of EPO have been demonstrated both in animals and in a large number of studies on people who have various brain disorders. However, pharmaceutical companies have only limited interest in funding any further required studies on approved active substances such as erythropoietin, (of which the patent protection has since expired). COVID-19 can have such serious consequences for health that we need to examine all evidence of the protective effect that EPO might have. After all, there is currently no vaccine or drug available at present. This is why we are in the process of preparing a clinical trial with people to examine the effect EPO has on COVID-19; a so-called proof-of-concept study, Ehrenreich explains. In this clinical trial, critically ill Covid-19 patients will be given additional amounts of EPO.

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EPO doping drug appears to ease severe cases of COVID-19 - Innovation Origins

Spinal Cord Stem Cells Comments Off on EPO doping drug appears to ease severe cases of COVID-19 – Innovation Origins | July 3rd, 2020

No matter how many molecules he threw at them, Paul Tesar couldnt get the brain cells to survive. Or he got them to survive, but then to everyones bafflement they still couldnt do what they were supposed to.

Tesar, a professor of innovative therapeutics at Case Western University, had spent years building stem cell models for multiple sclerosis, growing brain organoids in dishes and then seeing what small molecules restored myelin production. Now he was trying to do the same for other myelin diseases, particularly an ultra-rare genetic condition called Pelizaeus-Merzbacher disease, where a single mutation leads to the death of the myelin-producing neurons, called oligodendrocytes, and can kill patients in infancy.

Weve screened many thousands of small molecule compounds, Tesar toldEndpoints News. But we could not get them to restore function.

Then Tesar got an email from Ionis, the California biotech that had just used an RNA-modifying technology called antisense to build Spinraza, the first FDA-approved drug for the genetic neurological disorder spinal muscular atrophy.

Now, in a study published inNature,Tesar and Ionis have shown they can use a single dose of drug built from that technology to keep those neurons both alive and well-functioning and treat the disease at least in mice. The publication isnt groundbreaking, antisense researchers say, but it shows for the first time that antisense can be used to effectively target oligodendrocytes, an insight its authors hope will open up other rare myelin disorders to therapy.

Its not that its different than everything thats been done before, but it goes further than everything thats gone before, Jon Watts, a professor at the RNA Therapeutics Institute at UMass Medical School who is not affiliated with Ionis or the paper, told Endpoints, both in terms of duration of effect after a single dose, and the real focus in getting the biology, the therapeutic effect in oligodendrocytes.

The applicability to the most famous and common of myelin disorders, multiple sclerosis, is limited, researchers say, both because the therapy relied on having a specific gene to target and because the paper doesnt prove you can get an effect on the peripheral nervous system. Still, Berit Powers, an assistant director at Ioniss neurology research department and a co-author, pointed to several other genetic myelin disorders, known as leukodystrophies. That includes an Ionis program on Alexander disease, a rare childhood condition with Parkinsons-like symptoms.

Were certainly exploring the potential of ASOs in non-monogenic conditions like MS, Powers told Endpoints, using a shorthand for antisense oligonucleotides. But that work is very new.

This is hardly Tesars first foray into biotech. In 2015, he showed in Naturehow certain small molecules could regenerate myelin the holy grail for an MS therapy and founded Convelo Therapeutics around that work. Last year, they partnered with Genentech for an undisclosed sum and an exclusive option to acquire the company.

Myelin is a fatty substance that coats neurons, insulating them and helping electric currents pass through. Tesars lab was broadly interested in the question of why myelin fails, both in MS and rare diseases, and about 7 years ago he got a grant to work from the PMD Foundation.

First, Tesar built stem cell models of the disease, figuring out how different mutations in a single gene, called PLP1, lead oligodendrocyte progenitor cells (the stem cell-like cells that will become oligodendrocytes) to create a toxic RNA and a mutated protein that kills them soon after they differentiate. Then, he tried to suppress that gene with different chemicals, eventually testing over 3,000 different compounds.

He was able to eventually get the oligodendrocytes to survive, but to his surprise, they didnt produce myelin as they should. The surviving cells still couldnt properly function, revealing, he wrote in a 2018 Cell paper a second phase of pathology. A hypothetical treatment, he argued, would have to both keep progenitor cells alive and then treat the survivors in a way that induces myelination.

With antisense, he and Powers Ionis team were able to do both. Antisense oligonucelotides consist of strands of RNA that are a mirror image of the RNA you want to target. The mirror binds to and silences, or turns off, that gene. In the study, the researchers confirmed that PLP1 was disease-causing by knocking out the gene in cell lines with CRISPR. Then they injected mice with antisense strands through the spinal cord, the same way Spinraza is delivered. (You cant use CRISPR to treat the disease in humans, because theres no good way yet of delivering it.)

Powers and Tesar were unsure if they would be able to target oligodendrocytes and progenitor cells. What they found, though, was complete restoration of oligodendrocytes and a profound rescue of neurological function. Myelin, too, was finally restored. Mice that died after 3 weeks now lived for over 200 days.

Ionis hasnt licensed the drug and its unclear yet the implications for other diseases, but researchers say the results could translate into humans quickly, at least by drug development standards.

I do think its very rapidly translatable, Watts said. Based on the data theyre showing here, and based on the unmet need, this appears to be something that could be translated pretty quickly into a Phase I trial.

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Ionis, leading MS researcher throw antisense at a new type of brain cells - Endpoints News

Spinal Cord Stem Cells Comments Off on Ionis, leading MS researcher throw antisense at a new type of brain cells – Endpoints News | July 3rd, 2020

New York City, United States The effect of the coronavirus pandemic and the lockdown it activated is unmistakably obvious in budgetary markets. Yet, there is still no clearness on the more profound effect that it is having across organizations and modern areas. In view of evaluations made by various examiners and industry body Ficci, here is an effect investigation in human services area.

Spinal Cord Trauma Treatment Market: Global Industry Analysis 2012 2016 and Forecast 2017 2025is the recent report of Persistence Market Research that throws light on the overall market scenario during the period of eight years, i.e. 2017-2025. According to this report, Globalspinal cord trauma treatment marketis expected to witness significant growth during the forecast period.

This growth is expected to be primarily driven by increasing incidence of spinal cord trauma, and increasing government support to reduce the burden of spinal cord injuries. Additionally, development of nerve cells growth therapy is expected to boost the market in near future.

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The global market for spinal cord trauma treatment is is estimated to be valued at US$ 2,276.3 Mn in terms of value by the end of 2017. The global spinal cord trauma treatment market is expected to expand at a CAGR of 3.7% over the forecast period to reach a value of US$ 3,036.2 Mn by 2025end.

Global Spinal Cord Trauma Treatment Market: Trends

Global Spinal Cord Trauma Treatment Market: Forecast by End User

On the basis of end user, the global spinal cord trauma treatment market is segmented into hospitals and trauma centers. Hospitals segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period.

Hospitals and trauma centers segments are expected to approximately similar attractive index. Hospitals segment accounted for 53.2% value share in 2017 and is projected to account for 52.5% share by 2025 end.

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Global Spinal Cord Trauma Treatment Market: Forecast by Injury Type

On the basis of injury type, the global spinal cord trauma treatment market is segmented into complete spinal cord injuries and partial spinal cord injuries.

Partial spinal cord trauma treatment segment is expected to show better growth than the completed spinal cord treatment segment due to higher growth in the incidence rate of partial spinal cord trauma than the complete spinal cord trauma. With US$ 1,870.3 Mn market value in 2025, this segment is likely to expand at CAGR 3.8% throughout the projected period.

Global Spinal Cord Trauma Treatment Market: Forecast by Treatment Type

On the basis of treatment type, the global spinal cord trauma treatment market is segmented into corticosteroid, surgery, and spinal traction segments.

Surgery segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period. Surgery segment is the most attractive segment, with attractiveness index of 2.6 over the forecast period.

Global Spinal Cord Trauma Treatment Market: Forecast by Region

This market is segmented into five regions such as North America, Latin America, Europe, APAC and MEA. Asia-Pacific account for the largest market share in the global spinal cord trauma treatment market.

Large patient population due to the high rate of road accidents and crime is making the Asia Pacific region most attractive market for spinal cord trauma treatment. On the other hand, MEA and Latin America is expected to be the least attractive market for spinal cord trauma treatment, with attractiveness index of 0.3 and 0.5 respectively over the forecast period.

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Spinal Cord Trauma Treatment Market Size : Technological Advancement and Growth Analysis with Forecast to 2025 - 3rd Watch News

Spinal Cord Stem Cells Comments Off on Spinal Cord Trauma Treatment Market Size : Technological Advancement and Growth Analysis with Forecast to 2025 – 3rd Watch News | June 29th, 2020

On February 67, 2020, the Simons Foundation Autism Research Initiative (SFARI) convened a two-day workshop to explore the possibility of gene therapies for autism spectrum disorder (ASD), a neurodevelopmental condition associated with changes in over 100 genes. Inspired by the recent, stunning successes of gene therapy for the fatal neuromuscular disorder spinal muscular atrophy (SMA)1, and by the accumulation of genes confidently associated with ASD2, SFARI welcomed a diverse collection of researchers to begin to think about whether a similar approach could be taken for ASD. Because gene therapy attempts to fix what is broken at the level of a causative gene, it would offer a more direct and imminent strategy than mitigation of the many and as yet mostly unclear downstream effects of a damaged gene.

The workshop was organized in 20 talks and several discussion panels, which tackled many outstanding issues, including how to choose candidate target genes and predict outcomes; how to optimize vectors for gene delivery; how to decide when to intervene; which animal models to develop; how to find appropriate endpoints for clinical trials and understand the available regulatory pathways. SFARI also raised the question of how its funding might best propel gene therapy efforts amid the emerging, complex ecosystem of academic laboratories, biotech companies, and pharmaceutical industries.

Even the opportunity to have this discussion is very rewarding, said SFARI Investigator Matthew State of the University of California, San Francisco (UCSF), one of the investigators who directed teams of geneticists to analyze the Simons Simplex Collection (SSC).

These efforts have offered up multiple potentially feasible therapeutic targets. Though rare, de novo disruptive mutations in the highest confidence ASD genes often result in severe impairment characterized not only by social difficulties, but also by intellectual disability and seizures. The combination of a single gene mutation of large effect coupled with particularly severe outcomes that include ASD are likely to offer the most immediate targets for gene therapy. For now, this leaves out a large number of individuals with autism for whom genetic causes are not yet known and are likely the result of a combination of many small effect alleles across a large number of genes.

Highlights from talks and discussion panel, chaired by Rick Lifton of Rockefeller University

In the first talk of the workshop, State brought the group up to speed on ASD genomics. The most recent tally from exome-sequencing in simplex cases of ASD highlighted 102 genes in which rare mutations confer individually large risks2. In contrast, the task of identifying common variants carrying very small risks remains quite challenging, with less than a half dozen alleles so far identified with confidence3. The rare, disruptive mutations that result in loss of function of one gene copy are an attractive focus for gene therapy because of the tractability of targeting a single spot in the genome per individual and because, in the vast majority of cases, there remains a single unchanged allele. This points to ways to boost gene and/or protein expression back toward the normal state by leveraging the unaffected copy. But both the limited number of cases known so far combined with the possibility that different mutations to the same gene may have different effects complicate thinking about how to prioritize targets for gene therapy.

State made several points that were continually touched on throughout the workshop. Many ASD genes are highly expressed during midfetal development in the cortex, and additional experiments will need to determine whether and how long a window of opportunity may be present for successful gene therapy postnatally. Given the relatively small number of people with these conditions, new clinical trial designs are needed that dont rely on comparisons between large control and intervention groups (see also Bryan Kings talk below).

Beyond the gene-crippling mutations found in the exome, disruptions to transcription may also dramatically raise risk for autism and may be corrected with a type of gene therapy using ASOs. SFARI Investigator Stephan Sanders of UCSF focused on the role of splicing, the process by which an initial transcript is turned into messenger RNA by removal of introns and joining together of exons. Splicing is disrupted in at least 1.5 percent of individuals with ASD4, and possibly many more, as suggested by transcript irregularities found in postmortem autism brain5. Sanders described Illuminas Splice AI project in which machine-learning helps predict noncoding variants that can alter splicing, including those beyond typical splice sites found near a gene6. As a result of incorporating sequence information around and between splice sites, this computational tool detected more mutations with predicted splice-altering consequences in people with ASD and intellectual disability than in those without the condition.

An ASO designed to bind specific portions of RNA could conceivably correct errors in transcription. ASOs have already been approved for use in other disorders in order to skip exons, retain exons or to degrade mRNA. Unlike other forms of gene therapy, ASOs do not permanently alter the genome, making it a kind of gene therapy lite. This reversibility has both disadvantages (having to re-infuse the ASO every few months) and advantages (multiple opportunities to optimize the dose and target; serious adverse effects are not permanent).

Jonathan Weissman of UCSF discussed the available toolbox for controlling gene expression developed by many different laboratories. To turn genes on or off, he has developed a method to combine CRISPR with an enzymatically inactive (dead) Cas9, which can then be coupled with a transcriptional activator (CRISPRa) or repressor (CRISPRi)7 (Figure 2). In the case of loss-of-function mutations, Weissman outlined strategies to make the remaining good allele work harder: increase transcription via CRISPRa, decrease mRNA turnover, increase translation of a good transcript via modification of upstream open reading frames (uORFs) or increase a proteins stability, possibly through small molecules acting on the ubiquitin system8. That said, the effects on a cell may be complicated. Using Perturb-Seq screens, Weissman described genetic interaction manifolds that show nonlinear mapping between genotype and single cell transcriptional phenotypes9. Additionally, Weissman summarized recent work from his laboratory that has identified large numbers of uORFs that result in polypeptides, some of which affect cellular function.

SFARI Investigator Michael Wigler of Cold Spring Harbor Laboratories echoed the idea of a gene-therapy strategy that increases expression of the remaining good copy of a gene, especially given that in his estimate, 45 percent of simplex cases of autism carried a de novo, likely disrupting variant. He also called attention to the uterine environment, especially the challenge posed by expression of paternally derived antigens in the fetus and the impact of a potential maternal immune response, and the need to understand how it interacts with de novo genetic events.

Highlights from talks and discussion panel, chaired by Arnon Rosenthal of Alector

The discussion turned to finding ways of getting genes into the central nervous system. The AAV is the darling of gene therapy, given that it does not replicate and is not known to cause disease in humans. A version that can cross the blood-brain barrier (AAV9) was used to deliver a gene replacement to children with SMA intravenously; though this effectively delivered the genetic cargo to ailing motor neurons in the spinal cord, it does not work that well at delivering genes throughout the brain.

Ben Deverman of the Stanley Center at the Broad Institute of MIT and Harvard detailed his efforts to optimize AAV for efficient transduction of brain cells through a targeted evolution process: his team engineers millions of variants in the capsid of the virus, then screens them for entry into the nervous system and transduction of neurons and glia. This has yielded versions (called AAV-PHP.B and AAV-PHP.eB) that more efficiently enter the brain10,11. One successfully delivered the MECP2 gene to the brain of a Rett syndrome mouse model, resulting in ameliorated symptoms and an extended lifespan12. Unfortunately, these viruses dont work in human cells or in all mouse strains. A quick mouse genome-wide association study (GWAS) revealed that the Ly6a gene mediates efficient blood-brain barrier crossing of AAV-PHP.B and AAV-PHP.eB13. Now his group has identified Ly6a-independent capsids that may translate better to humans. He also noted that the PHP.B vectors have tissue specificity for brain and liver.

With an estimated 87 percent of autism-associated genes raising risk through haploinsufficiency (having only one functional gene copy out of the two), SFARI Investigator Nadav Ahituv of UCSF made the case for approaches that boost expression of the remaining good copy of a gene through endogenous mechanisms a strategy he called cis-regulation therapy. This method also provides a way to work around the small four kb payload of AAV, which strains to contain cDNA of many autism genes. A recent study by his group used CRISPRa targeted at an enhancer or promoter of SIM1 and promoter of MC4R, both obesity genes, in mice. Using one AAV vector for a dCas9 joined to a transcription activator, and another AAV vector having a guide RNA targeting either a promoter or an enhancer, and a guide RNA targeting a promoter, the researchers injected the vectors together into the hypothalamus, which resulted in increased SIM1 or MC4R transcription and reversed the obesity phenotype brought on by loss of these genes14. Targeting regulatory elements had the added benefit of tissue specificity, and there seemed to be a ceiling effect for SIM1 expression, which suggested an endogenous safeguard against overexpression at work. He is now collaborating with SFARI Investigator Kevin Bender, also at UCSF, to apply this approach to the autism gene SCN2A.

Botond Roska of the Institute of Molecular and Clinical Ophthalmology in Basel, Switzerland pointed out that getting genes to the cells where they are needed is crucial when treating eye diseases. Off-target effects there can induce degeneration of healthy cells. For this reason, Roska and his group have created AAVs that target specific cell types in the retina by developing synthetic promoters that efficiently promote expression of the viruss cargo15. The promoters they designed were educated guesses based on four approaches: likely regulatory elements close to genes expressed with cell-type specificity in the retina, conserved elements close to cell typespecific genes, binding sites for cell typespecific transcription factors and open chromatin close to cell typespecific genes. Screening a library of these in mouse, macaque and human retina revealed some with high cell-type specificity (Figure 3). Importantly, macaque data predicted success in human retina much better than did mouse data. In preliminary experiments, and more relevant to gene therapy for ASD, these cell-specific vectors also had some success in mouse cortex, for example lighting up parvalbumin neurons or an apparently new type of astrocyte.

Roska also described new methods for delivery, in which nanoparticles are coated with AAV, then drawn into the brain using magnets16. This magnetophoresis technique allows a library of experimental AAVs to be tested at the same time in one monkey. Steering nanoparticles with magnets gives more control of vector placement and gene delivery. He argued that these in the future could access even deep structures of the brain.

Highlights from talks and discussion panel, chaired by Steven Hyman of the Broad Institute at MIT and Harvard

Kathy High of Spark Therapeutics reviewed the story of gene therapy for spinal muscular atrophy (SMA) type 1. Though she was not directly involved in that research, she is well aware of the regulatory atmosphere surrounding gene therapy, given that Spark Therapeutics developed the first approved AAV-delivered gene for a form of retinal dystrophy. The SMA story is a useful case study in that an ASO-based therapy (nusinersen, marketed as Spinraza), approved in 2016, set the stage for a gene-replacement therapy, marketed as Zolgensma (onasemnogene abeparvovec). Ultimately, the amount of data supporting Zolgensmas approval was modest: a Phase one dose study of 15 infants1, and an ongoing Phase three trial of 21 infants and safety data from 44 individuals. Yet the approval was helped by the dramatic results and clear endpoints: those receiving a single intravenous infusion of an AAV9 vector containing a replacement gene all remained alive at 20 months of age, whereas only 8 percent survived to that age in the natural history data, which compiles the diseases untreated course. High mentioned that maintaining product quality for gene therapeutics may prove trickier than for typical medications.

The attractive, highly customizable nature of gene therapy might have a regulatory downside in that different vector payloads, even when designed to do the same thing, could invite separate approval processes. Though not knowing how regulatory agencies would view this, High said that their perspectives are bound to evolve as more gene therapy trials are completed.

Getting to ASD-related syndromes, Bender talked about SCN2A, which encodes the sodium channel Nav1.2. SCN2A mutations in humans can be gain of function or loss of function; gain-of-function mutations are associated with early onset epilepsy, and loss-of-function mutations with intellectual disability and ASD. In a mouse model missing one copy of SCN2A, Bender and his group have discovered a role for SCN2A in action potential generation in the first week after birth, and in synaptic function and maturation afterward through regulation of dendritic excitability18 (Figure 4). Using AAV containing CRISPRa constructs developed with the Ahituv lab, the researchers successfully increased SCN2A expression, and recovered synapse function and maturity, even when done several weeks postnatally. Getting the appropriate dosage is critical since gain-of-function mutations are linked to epilepsy. However, Bender reported even when SCN2A expression increased to double normal levels, no hints of hyperexcitability appeared. We might be able to overdrive this channel as much as we want and actually may not have risk of producing an epileptic insult, he said. Next steps are to figure out the developmental windows for intervention, evaluate changes in seizure sensitivity and extend this kind of cis-regulatory approach to other ASD genes.

Angelman syndrome is another condition that attracts interest for gene therapy, in part because neurons already harbor an appropriate replacement gene. Angelman syndrome stems from mutations to the maternally inherited UBE3A gene, which is particularly damaging to neurons because they only express the maternal allele, while the paternal allele is silenced by an antisense transcript. SFARI Investigator Mark Zylka of the University of North Carolina and colleagues showed in 2011 that this paternal allele could be unsilenced with a cancer drug in a mouse model of Angelman syndrome19. Since then, three companies have built ASOs to do the same thing, and these are going into clinical trials. To get a more permanent therapeutic, Zylka has been developing CRISPR/Cas9 systems to reactivate paternal UBE3A, and preliminary experiments show that injecting this construct into the brains of embryonic mice, and then again at birth, results in brain-wide expression of paternal UBE3A and is long-lasting (at least 17 months). Zylka is now making human versions of these constructs. He later noted rare cases of mosaicism for the Angelman syndrome mutation people with 10 percent normal cells in blood have a milder phenotype20, which suggests that even inefficient transduction of a gene vector could help.

Zylka also made a case for prenatal interventions in Angelman syndrome: studies of mouse models indicate that early reinstatement of UBE3A expression in mouse embryos rescues multiple Angelman syndrome-related phenotypes, whereas later postnatal interventions rescue fewer of these21; for humans, a diagnostic, cell-based, noninvasive prenatal test will be available soon22; ultrasound-guided injections into fetal brain of nonhuman primates have been developed23; prenatal surgeries are now standard of care for spinal bifida; and intervening prenatally decreases the risk of an immunogenic response to an AAV vector or its cargo. During the discussion, it was noted that another benefit of acting early was that less AAV would be needed to transduce a much smaller brain; however, a drawback is the lack of data on Angelman syndrome development from birth to one year of age. This natural history would be necessary for understanding whether a prenatal therapy is more effective than treatment of neonates.

SFARI Investigator Guoping Feng of the Massachusetts Institute of Technology has been investigating SHANK3, a high-confidence autism risk gene linked to a severe neurodevelopmental condition called Phelan-McDermid syndrome, which is marked by intellectual disability, speech impairments, as well as ASD. SHANK3 is a scaffold protein important for organizing post-synaptic machinery in neurons. Mouse studies by Feng have shown that SHANK3 re-expression in adult mice that have developed without it can remedy some, but not all, of their phenotypes, including dendritic spine densities, neural function in the striatum and social interaction24. Furthermore, early postnatal re-expression rescued most phenotypes. This makes SHANK3 a potential candidate for gene therapy; however, it is a very large gene 5.2kb as a cDNA that is difficult to fit into a viral vector. To get around this, Fengs group has designed a smaller SHANK3 mini-gene as a substitute for the full-sized version. Preliminary experiments show that AAV delivery of the mini-gene can rescue phenotypes like anxiety, social behavior and corticostriatal synapse function in SHANK3 knockout mice. Feng also discussed his success in editing the genome in marmosets and macaques using CRISPR/Cas9 technology and showed data from a macaque model of SHANK3 dysfunction25. These models may help test gene therapy approaches and identify biomarkers of brain development closely related to the human disorder.

For people with rare conditions brought on by even rarer mutations, individualized gene therapies can provide a pathway for treatment. SFARI Investigator Timothy Yu of Boston Childrens Hospital/Harvard described his N-of-1 study in treating a girl with Batten disease, a recessive disorder in which a child progressively loses vision, speech and motor control while developing seizures. In a little over a year, an ASO that targeted her unusual splice-site mutation in the CLN7 gene was designed, developed and given intrathecally to the girl26. The lift was in negotiating with the FDA and working with private organizations, not just in the science, Yu said. After a year of treatment with the ASO (dubbed milasen after the girl, Mila), there were no serious adverse events; seizure frequency and duration had decreased (Figure 5); and possibly her decline had slowed. Though she remains blind, without intelligible speech and unable to walk on her own, she was still attentive and could respond happily to her familys voices. The highly personalized framework for this drugs approval is completely different from how medications meant for populations are approved, and it opens a regulatory can of worms, Yu said, though he added that the regulators were willing to countenance drug approval for an individuals clinical benefit.

Rett syndrome is a neurodevelopmental condition caused by mutations to the MECP2 gene that has a substantial research base in mouse models. Over 10 years ago, mouse models highlighted the possibility for therapeutics in this condition when Rett-associated phenotypes were rescued by adding back MECP2, even in adulthood27. This reversibility has spurred interest in gene therapy for Rett syndrome, but getting the MECP2 dose right is critical, said Stuart Cobb of the University of Edinburgh and Neurogene: just as too little MECP2 leads to Rett syndrome, too much also results in severe phenotypes. For this reason, it would be nice to package a replacement MECP2 gene with other regulatory elements to control its expression, but this results in constructs that do not fit into viral vectors. To make more room, Cobb and his colleagues have been able to chop away two-thirds of the MECP2, reserving two domains that interact to make a complex on DNA (Figure 6). Mice with this mini-gene are viable and have near normal phenotypes; likewise, injecting this mini-gene into MECP2-deficient mice extended their survival28. Doubling the dose, however, substantially lowered survival. Putting in safety valves to prevent overexpression is going to be quite important, he said. One idea is to add back a construct containing only the last two exons of MECP2, which is where most Rett mutations land. These would then be spliced into native transcripts (called trans-splicing), and thus their expression controlled by endogenous regulatory elements.

Underscoring the double-edged sword of MECP2 dosage, Yingyao Shao from Huda Zoghbis lab at Baylor described an MECP2 duplication syndrome (MDS) in humans, which features hypotonia, intellectual disability, epilepsy and autism. Experiments in an MDS mouse model, which carries one mouse version and one human version of MECP2, recapitulates some of the phenotypes of the human condition and can be rescued by an ASO targeting the human allele29. Shao described work to optimize the ASO for translation into humans, which involved developing a more humanized MDS model that carries two human MECP2 alleles. An acute injection of the ASO was able to knock down MECP2 expression in a dose-dependent manner in these mice, and RNA levels dropped a week after injection, with protein levels falling a week later. MECP2 target genes also normalized their expression level, and one maintained this for at least 16 weeks post-injection. The ASO also rescued behavioral phenotypes of motor coordination and fear conditioning, but not of anxiety; these corrections followed the molecular effects, and these timelines would be important to keep in mind while designing clinical trials. Shao also noted that overtreatment with the ASO resulted in Rett-associated phenotypes, but that this was reversible, which suggests that some fine-tuning of dosing in humans might be possible.

To avoid overtreatment and toxicity of any MDS-directed therapy, Mirjana Maletic-Savatic, also at Baylor, is leaving no stone unturned in a hunt for MDS biomarkers that can predict, in each individual, the safety of a particular dose and regimen. Such biomarkers would also help monitor individuals during treatment, give information about target engagement and identify candidates for a particular treatment. Anything found to be sensitive to expression levels of MECP2 could also be useful for Rett, though she noted that MECP2 levels measured in blood do not track linearly with gene copy number. Thus, because of interindividual variability, her approach is to collect a kitchen sink of data deriving composite biomarkers that accurately reflect the stage and severity of disease in a given case. She and her colleagues are collecting clinical, genetic, neurocircuitry (such as EEG and sleep waves), immunology and molecular data detected in blood, urine and CSF. These measures are also being explored in induced neurons derived from skin samples of people with MDS. She highlighted two interrelated potential biomarkers in the blood of those with this condition; both measures are downstream targets of MECP2 and are responsive to ASO treatment.

Highlights from Early detection and clinical trial issues talks and panel discussion, chaired by Paul Wang of SFARI

Coming up with objective measures of a persons status either their eligibility for a treatment, or whether the treatment has engaged with its target or even whether the treatment is effective is a real necessity in autism-related conditions, which comprise multiple interrelated behaviors. Eye-tracking methodology may provide such a marker, argued SFARI Investigator Ami Klin of Emory University. Focusing on the core social challenges of autism, Klin, Warren Jones and colleagues have been studying children as they view naturalistic social scenes to quantify their social attention patterns. This has revealed how remarkably early in development social visual learning begins and that this process is disrupted in infants later diagnosed with ASD prior to features associated with the condition appearing. By missing social cues, autism in many ways creates itself, moment by moment, Klin said. In considering gene therapy, it may be useful to know that eye looking (how much a subject looks at a persons eyes, an index of social visual engagement) in particular and social visual engagement in general are under genetic control30; that eye-tracking differences emerge as early as 26 months of age; and that homologies in social visual engagement exist between human babies and nonhuman infant primates.

In getting to a point to test gene therapies, identifying those who need them is essential. Wendy Chung of Columbia University and the Simons Foundation illustrated how diagnosis is yoked closely to therapy. To illustrate this, she described her pilot study of newborn blood spots to screen for SMA; at the start, no treatment was available, but the screen identified newborns for a clinical trial of nusinersin. Notably, the screen only cost an additional 11 cents per baby. In the three years since her pilot screen began, the FDA approved two gene therapies for SMA and the SMA screen was adopted for nationwide newborn screening. Currently she is piloting a screen for Duchenne muscular dystrophy and plans to develop a platform that will allow researchers to add other conditions. In prioritizing genetic conditions for gene therapy, she outlined some ideas for focus, such as genes resulting in phenotypes that would not be identified early without screening, those that are relatively frequent, those that are lethal or neurodegenerative, those with a treatment in clinical trials or with FDA-approved medications, and those conditions that are reversible.

In the meantime, Chung also outlined SFARIs involvement in establishing well-characterized cohorts of individuals with autism, which can help lay a groundwork for gene therapy. People with an ASD diagnosis can join SPARK (Simons Foundation Powering Autism Research for Knowledge), which collects medical, behavioral and genetic information (through analysis of DNA from saliva, at no cost to the participant). If a de novo genetic variant is found in one of ~150 genes, that person is referred to Simons Searchlight, which fosters rare conditions communities and which is also compiling natural history data on people with these mutations.

Bryan King of UCSF discussed how current trial designs for ASD were inadequate for gene therapy trials. As ASD prevalence has grown, parallel design trials with one group receiving an experimental medicine and the other a placebo are the standard, but these wont be possible for the rare conditions that are candidates for gene therapy. Also, change is hard to capture, given the malleable nature of ASD: with no intervention, diagnosis can shift between ASD and pervasive developmental disorder-not otherwise specified (PDD-NOS) in 1284 months (as defined by the DSM-IV). Current scales are subjective and may miss specific items of clinical significance. (Last year, SFARI funded four efforts to develop more sensitive outcome measures.) King outlined other pitfalls in ASD clinical trials, including significant placebo responses, inadequate sample sizes and not being specific enough when asking about adverse effects. King also mentioned improvements that may arise from just enrolling in a study, which could prompt previously housebound families to venture out with their child, which could kick off a cascade of positive effects. He reiterated how, for gene therapy, a natural history comparison group may be more appropriate, combined with solid outcome measures.

SFARI Investigator James McPartland of Yale University then underlined the need for objective biomarkers for clinical trials, for which there are currently none that are FDA qualified for ASD. As the director of the Autism Biomarkers Consortium for Clinical Trials (ABC-CT), he works with other scientists to develop reliable biomarkers that can be scaled for use in large samples across different sites. McPartland noted a biomarker studied in the ABC-CT: an event-related potential (N170) to human faces, which is on average slower in ASD than in typically developing children. He is working on ways to make it easier for people with ASD and intellectual disabilities to participate in biomarker studies and to make them more socially naturalistic. In discussion, he mentioned he thought it would be possible to look for these kinds of biomarkers in younger children.

SFARI Investigator Shafali Jeste of the University of California, Los Angeles recounted her experience in working with children with genetic syndromes associated with neurodevelopmental conditions. Though she is asked to participate in clinical trials for these conditions, she senses the field has some work to do to be ready for these trials, particularly in those with additional challenges such as epilepsy and intellectual disability. Meaningful and measurable clinical endpoints are still insufficient, and there needs to be more ways to improve accessibility of these trials for these rare conditions. This means developing new measures, such as gait-mat technology that senses walking coordination, or EEG measures in waking and sleep, which have been applied to people with chromosome 15q11.2-13.1 duplication (dup15q) syndrome, who have severe intellectual disability and motor impairments. Jeste also emphasized that increasing remote access to some measures can make a big difference for a trial; for example, a trial of a behavioral intervention for tuberous sclerosis complex that required weekly lab visits was disappointingly under-enrolled until researchers revamped it so most of the intervention could be done remotely31.

By grappling with the challenges to gene therapy for ASD, the workshop marked out a faint road map of a way forward. As the scientific questions are answered, the regulatory and clinical trial infrastructure will need to develop apace, and coordination between private, academic and advocacy sectors will be essential. But as gene therapy for diverse human conditions continues to be explored and gene discovery in ASD continues, there is reason to believe that some forms of ASD can eventually benefit from this strategy.This workshop provided a terrific discussion about the challenges in developing targeted gene interventions and their potentially transformative effects as therapies, said John Spiro, Deputy Scientific Director of SFARI. We are grateful to all theparticipants, and SFARI looks forward to translating these discussions into focused funding decisions in the near future.

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SFARI | SFARI workshop explores challenges and opportunities of gene therapies for autism spectrum disorder - SFARI News

Spinal Cord Stem Cells Comments Off on SFARI | SFARI workshop explores challenges and opportunities of gene therapies for autism spectrum disorder – SFARI News | June 25th, 2020

Researchers have known for quite some time that HD causes a progressive loss of neurons. But what if we could find a way to fill their place? In a new report, researchers used an intriguing strategy in living mice to do just that they converted a different type of brain cell into neurons, with very promising results.

In HD research we spend a lot of time talking about neurons. And understandably so! Neurons are the cell type in the brain most affected by HD, and they are the cells that exchange messages to drive our movements, moods, and memories. You can think of neurons like the computer programmers of the brain they convert information into action.

In particular, neurons in an area of the brain called the striatum striatal neurons tend to be most vulnerable to the mutation that causes HD. Right now no one knows exactly why those cells are especially vulnerable. But researchers know that many of the symptoms of HD are related to the loss of neurons in this area of the brain.

But there are lots of different types of cells in the brain. In fact, the most abundant cell type in the brain isnt neurons its a cell type called glia. Glia is a general term that describes several kinds of cells in the brain and spinal cord that provide support, insulation, and protection. You can think of glia like the body guard of the brain they make sure other cell types have the support they need to function.

One type of glia are brain cells called astrocytes. A lot of the nervous system is made up of astrocytes 30% in fact! Because astrocytes are everywhere in the brain, theyre also present in the areas where neurons degenerate due to HD the striatum. And unlike neurons that stop dividing when theyre fully mature, glia continue to divide.

Recently, scientists took advantage of the abundance of glia in the brain and their ability to reproduce. They used an experimental technique in the brains of mice to turn astrocytes into new, functioning neurons. So to stick with our analogy, they encouraged the body guards of the brain to change jobs and become computer programmers.

The work was led by Dr. Gong Chen, a former professor at Penn State University, who is now leading the Institute of CNS Regeneration at Jinan University in China. His team took advantage of a technique to turn cells that arent neurons into neurons something called direct conversion.

This technique allows researchers to coax different cell types, such as astrocytes, into becoming neurons, by adding chemical cocktails to boost the action of genes that influence a cells role. This is a bit like changing the job description of a certain cell type - but this has been done before. Many times in fact. Its old news that scientists can take one cell type grown in a laboratory dish and directly turn it into a neuron.

So what did this report add, and why was it worthy of publication in the prestigious journal Nature Communications? Because these authors did direct conversion inside the brains of living mice! They used a harmless virus to deliver their chemical cocktail that gave a genetic nudge to the astrocytes, encouraging them to change jobs and become neurons. In this way, they were able to turn abundant astrocytes into potentially valuable striatal neurons a very cool accomplishment!

We know what you may be thinking Did you just say virus?! We all get a little weary when we hear that word, especially in the days of COVID-19! But rest assured, this is a very harmless method used frequently in biology.

Its actually just the outside of the virus thats used, without any of the inside bits that typically make viruses so harmful. Similar to a letter in an envelope researchers here are repurposing an envelope and adding something new inside. So the old message is removed, and the envelope is sent with new instructions that body guards should change jobs and become computer programmers!

An important finding from the paper was that the overall number of astrocytes didnt decrease over time. This is related to the point we made above about astrocytes they continue to divide. So even though the researchers turned some of the astrocytes into neurons, the astrocytes that remained produced more astrocytes to replace them. This approach provided a source of new striatal neurons for these HD mice without affecting the astrocyte population! And because these astrocytes are already located in the striatum, the intervention occurs in the exact area of the brain that could use more neurons.

Chen and colleagues also showed that these new neurons in the striatum fired signals just like native neurons. They also connected with other areas of the brain, just like native neurons. Most compellingly, with the addition of these new neurons in the striatum, the HD mice performed better on movement tests and had an extended lifespan. All very exciting and promising results!

The idea of adding back lost neurons in HD isnt new. The big difference is that previous studies have added new cells through surgery, performing whats called cell transplantation. So while direct conversions, like the experiments performed by Chen and his team, are like changing jobs within the same company, cell transplantations are like getting a job at a new company.

Several research groups have experimented with cell transplantation as a therapy for HD, and some of these options are moving toward clinical trials. More recently, cell transplantations have been done with immature cells known as stem cells or neural progenitor cells that havent fully committed to becoming a specific cell type yet. The benefit of using immature cells is that they can obtain cues from the surrounding environment, letting them know what cell type is needed.

Cell transplantations have shown promise, but can come with some risks. Theres no guarantee that the cells will become exactly the type of neuron you want. And theres no guarantee that the cells will survive long-term because thats not their native environment.

Chens group got around these issues by triggering specific biological machinery to convert astrocytes into striatal neurons. The researchers knew exactly what type of neuron they were going to get in the end. And because the astrocytes they targeted were already present in the striatum, they knew the new neurons would be in exactly the right place!

One thing to keep in mind with this approach is that the astrocytes used to make the neurons come from the HD mouse. That means the new striatal neurons also contain the genetic error (mutation) that causes HD. Researchers dont yet know what that means for the lifespan of those neurons.

While the results from this study are very exciting and potentially provide another tool in our belt to combat HD, this study was done as a proof-of-concept and still has a long way to go before it reaches the clinic. But so far, even though the new neurons carry the HD mutation, the direct conversion technique seems to improve HD-related symptoms in the mice.

Follow up studies are likely to try this technique in larger animals or to test it in combination with huntingtin lowering, which will undoubtedly provide interesting results. Well be eagerly waiting!

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Changing jobs: converting other cell types into neurons - HDBuzz

Spinal Cord Stem Cells Comments Off on Changing jobs: converting other cell types into neurons – HDBuzz | June 24th, 2020

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues andtumors, wherein their toxicity, impurity, and other aspects are studied.

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With the growing number of successfulstem cell therapytreatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

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Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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Stem Cell Assay Market In-Depth Analysis and Forecast 2017-2025 - Daily Veterans

Spinal Cord Stem Cells Comments Off on Stem Cell Assay Market In-Depth Analysis and Forecast 2017-2025 – Daily Veterans | June 24th, 2020

Dublin, June 22, 2020 (GLOBE NEWSWIRE) -- The "Global Regenerative Medicine Market: Size & Forecast with Impact Analysis of COVID-19 (2020-2024)" report has been added to ResearchAndMarkets.com's offering.

This report provides an in-depth analysis of the global regenerative medicine market with description of market sizing and growth. The analysis includes market by value, by product, by material and by region. Furthermore, the report also provides detailed product analysis, material analysis and regional analysis.

Moreover, the report also assesses the key opportunities in the market and outlines the factors that are and would be driving the growth of the industry. Growth of the overall global regenerative medicine market has also been forecasted for the years 2020-2024, taking into consideration the previous growth patterns, the growth drivers and the current and future trends.

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Company Coverage:

Regenerative medicines emphasise on the regeneration or replacement of tissues, cells or organs of the human body to cure the problem caused by disease or injury. The treatment fortifies the human cells to heal up or transplant stem cells into the body to regenerate lost tissues or organs or to recover impaired functionality. There are three types of stem cells that can be used in regenerative medicine: somatic stem cells, embryonic stem cells (ES cells) and induced pluripotent stem cells (iPS cells).

The regenerative medicine also has the capability to treat chronic diseases and conditions, including Alzheimer's, diabetes, Parkinson's, heart disease, osteoporosis, renal failure, spinal cord injuries, etc. Regenerative medicines can be bifurcated into different product type i.e., cell therapy, tissue engineering, gene therapy and small molecules and biologics. In addition, on the basis of material regenerative medicine can be segmented into biologically derived material, synthetic material, genetically engineered materials and pharmaceuticals.

The global regenerative medicine market has surged at a progressive rate over the years and the market is further anticipated to augment during the forecasted years 2020 to 2024. The market would propel owing to numerous growth drivers like growth in geriatric population, rising global healthcare expenditure, increasing diabetic population, escalating number of cancer patients, rising prevalence of cardiovascular disease and surging obese population.

Though, the market faces some challenges which are hindering the growth of the market. Some of the major challenges faced by the industry are: legal obligation and high cost of treatment. Whereas, the market growth would be further supported by various market trends like three dimensional bioprinting , artificial intelligence to advance regenerative medicine, etc.

Key Topics Covered:

1. Executive Summary

2. Introduction2.1 Regenerative Medicine: An Overview2.2 Regeneration in Humans: An Overview2.3 Expansion in Peripheral Industries of Regenerative Medicine2.4 Approval System for Regenerative Medicine Products2.5 Regenerative Medicine Segmentation

3. Global Market Analysis3.1 Global Regenerative Medicine Market: An Analysis3.1.1 Global Regenerative Medicine Market by Value3.1.2 Global Regenerative Medicine Market by Products (Cell Therapy, Tissue Engineering, Gene Therapy and Small Molecules and Biologics)3.1.3 Global Regenerative Medicine Market by Material (Biologically Derived Material, Synthetic Material, Genetically Engineered Materials and Pharmaceuticals)3.1.4 Global Regenerative Medicine Market by Region (North America, Europe, Asia Pacific and ROW)

3.2 Global Regenerative Medicine Market: Product Analysis3.2.1 Global Cell Therapy Regenerative Medicine Market by Value3.2.2 Global Tissue Engineering Regenerative Medicine Market by Value3.2.3 Global Gene Therapy Regenerative Medicine Market by Value3.2.4 Global Small Molecules and Biologics Regenerative Medicine Market by Value

3.3 Global Regenerative Medicine Market: Material Analysis3.3.1 Global Biologically Derived Material Market by Value3.3.2 Global Synthetic Material Market by Value3.3.3 Global Genetically Engineered Materials Market by Value3.3.4 Global Regenerative Medicine Pharmaceuticals Market by Value

4. Regional Market Analysis4.1 North America Regenerative Medicine Market: An Analysis4.2 Europe Regenerative Medicine Market: An Analysis4.3 Asia Pacific Regenerative Medicine Market: An Analysis4.4 ROW Regenerative Medicine Market: An Analysis

5. COVID-195.1 Impact of Covid-195.2 Response of Industry to Covid-195.3 Variation in Organic Traffic5.4 Regional Impact of COVID-19

6. Market Dynamics6.1 Growth Drivers6.1.1 Growth in Geriatric Population6.1.2 Rising Global Healthcare Expenditure6.1.3 Increasing Diabetic Population6.1.4 Escalating Number of Cancer Patients6.1.5 Rising Prevalence of Cardiovascular Disease6.1.6 Surging Obese Population6.2 Challenges6.2.1 Legal Obligation6.2.2 High Cost of Treatment6.3 Market Trends6.3.1 3D Bio-Printing6.3.2 Artificial Intelligence to Advance Regenerative Medicine

7. Competitive Landscape7.1 Global Regenerative Medicine Market Players: A Financial Comparison7.2 Global Regenerative Medicine Market Players' by Research & Development Expenditure

8. Company Profiles8.1 Bristol Myers Squibb (Celgene Corporation)8.1.1 Business Overview8.1.2 Financial Overview8.1.3 Business Strategy8.2 Medtronic Plc8.2.1 Business Overview8.2.2 Financial Overview8.2.3 Business Strategy8.3 Smith+Nephew (Osiris Therapeutics, Inc.)8.3.1 Business Overview8.3.2 Financial Overview8.3.3 Business Strategy8.4 Novartis AG8.4.1 Business Overview8.4.2 Financial Overview8.4.3 Business Strategy

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Outlook on the Worldwide Regenerative Medicine Industry to 2024 - Rising Global Healthcare Expenditure Presents Opportunities - GlobeNewswire

Spinal Cord Stem Cells Comments Off on Outlook on the Worldwide Regenerative Medicine Industry to 2024 – Rising Global Healthcare Expenditure Presents Opportunities – GlobeNewswire | June 23rd, 2020

The personalized cell therapies market estimated to expand at a growth rate of 26.5%, and projected to reach US$ 18,000 Mn by 2025. Personalized cell therapies market report gives comprehensive outlook on personalized cell therapies or cytotherapies across the globe with special emphasis on key regions such as North America, Europe, Asia-Pacific, Latin America, and Middle East & Africa (MEA). Research & Development is evolving, various cell types developed into treatments as novel cell therapies due to its potential applications. Bone marrow transplant or hematopoietic stem cell transplant widely used cell therapy due to treatment of blood cancer. Increased prevalence of cancer, rise in research and adoption of advanced technologies are expected attract the demand for personalized cell therapies market.

Major Key Players of the Personalized Cell Therapies Market are:PCT Cell Therapy Services, LLC (U.S), S. Stem Cell, Inc. (U.S), Bellicum Pharmaceuticals, Inc. (U.S), Saneron CCEL Therapeutics, Inc. (U.S), StemGenex (U.S), Vericel Corporation.(U.S), Cytori Therapeutics Inc.(U.S.), MolMed S.p.A. (Italy)

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The rise in prevalence of chronic disorders and unmet needs in current treatment methods are expected to catapult the personalized cell therapies market.

Increasing prevalence of various disorders such as diabetes, cancer, autoimmune disorders, neurovascular disorders is expected to create demand for personalized cell therapies market over the forecast period. Moreover, potential applications of cell therapies include treating autoimmune disease, cancers, infectious disease, urinary problems, spinal cord injuries, rebuilding damaged cartilage in joints, improving a weakened immune system, neurological disorders are expected to create lucrative opportunities for personalized cell therapies market.

Major Types of Personalized Cell Therapies covered are:Hematopoietic Stem CellSkeletal Muscle Stem CellMesenchymal Stem CellsLymphocytesOthers

Major Applications of Personalized Cell Therapies covered are:Cardiovascular DisordersDiabetesNeurological DisordersCancerOthers

Research objectives:-

To study and analyze the global Personalized Cell Therapies consumption (value & volume) by key regions/countries, product type and application, history data. To understand the structure of the Personalized Cell Therapies market by identifying its various sub-segments. Focuses on the key global Personalized Cell Therapies manufacturers, to define, describe and analyze the sales volume, value, market share, market competitive landscape, SWOT analysis, and development plans in the next few years. To analyze the Personalized Cell Therapies with respect to individual growth trends, future prospects, and their contribution to the total market. To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).

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Table of Content

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered1.4 Market Analysis by Type1.5 Market by Application1.6 Study Objectives1.7 Years Considered

2 Global Growth Trends2.1 Personalized Cell Therapies Market Size2.2 Personalized Cell Therapies Growth Trends by Regions2.3 Industry Trends

3 Market Share by Key Players3.1 Personalized Cell Therapies Market Size by Manufacturers3.2 Personalized Cell Therapies Key Players Head office and Area Served3.3 Key Players Personalized Cell Therapies Product/Solution/Service3.4 Date of Enter into Personalized Cell Therapies Market3.5 Mergers & Acquisitions, Expansion Plans

4 Breakdown Data by Product4.1 Global Personalized Cell Therapies Sales by Product4.2 Global Personalized Cell Therapies Revenue by Product4.3 Personalized Cell Therapies Price by Product

5 Breakdown Data by End User5.1 Overview5.2 Global Personalized Cell Therapies Breakdown Data by End User

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In the end, Personalized Cell Therapies industry report specifics the major regions, market scenarios with the product price, volume, supply, revenue, production, market growth rate, demand, forecast and so on. This report also presents SWOT analysis, investment feasibility analysis, and investment return analysis.

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Personalized Cell Therapies Market Growth by 2025 Cytori Therapeutics, MolMed SpA - Cole of Duty

Spinal Cord Stem Cells Comments Off on Personalized Cell Therapies Market Growth by 2025 Cytori Therapeutics, MolMed SpA – Cole of Duty | June 18th, 2020

Induced Pluripotent Stem Cells Forming an Emerging Market

WELLESLEY, Mass.-June 16, 2020- (Newswire.com)

The global market for induced pluripotent stem cells (iPSCs), should reach $3.8 billion, growing 9.2% annually, according to the latest report by BCC Research, Induced Pluripotent Stem Cells: Global Markets.

iPSCs are reprogrammed cells from a patients body and are not harvested from embryos, thus avoiding the ethical debates associated with embryonic stem cells. Like other stem cells, they hold the immense promise of transforming into various types of human tissue cells and potentially representing highly effective treatments for serious illnesses.

Report Highlights

Read the full report here: https://www.bccresearch.com/market-research/biotechnology/induced-pluripotent-stem-cells-report.html

As a breakthrough technology recognized by the Nobel Prize in Physiology or Medicine 2012, iPSCstechnology has brought a revolutionary change to modern medicine, write BCC Research analysts. iPSC-related products are forming an emerging market despite the fact that clinical applications are still at their early stage. The potential scope of the iPSC market is becoming clear.

About BCC ResearchBCC Research publishes market research reports that provide organizations with intelligence to drive smart business decisions. By partnering with industry experts worldwide, BCC Research provides unbiased measurements and assessments of global markets covering current and emerging industrial and technology sectors. For more information about BCC Research, visit bccresearch.com.

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Original Source:Stem Cells Without Ethical Implications Are Ready for the Spotlight

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Stem Cells Without Ethical Implications Are Ready for the Spotlight - Press Release - Digital Journal

Spinal Cord Stem Cells Comments Off on Stem Cells Without Ethical Implications Are Ready for the Spotlight – Press Release – Digital Journal | June 17th, 2020

Stem cell characterization is the study of tissue-specific differentiation. Thera are various type of stem cell such as embryonic stem cell, epithelial stem cell and others. Further, various techniques are used to characterized stem cells such as immunological techniques, used for depiction of different population of stem cells. These techniques are generally based on immunochemistry using staining technique or florescent microscopy. Besides, stem cells characterization and analysis tools are used against target chronic diseases. In 2014, the San Diego (UCSD) Health System and Sanford Stem Cell Clinical Center at the University of California announced the launch of a clinical trial, in order to assess the safety of neural stem cellbased therapy in patients with chronic spinal cord injury.

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The factors driving the growth of stem cell characterization and analysis tools market due to increasing chronic disorders such as cancer, a diabetes and others. In addition, increasing awareness about among people about the therapeutic potency of stem cells characterization in the management of effective diseases is anticipated to increase the demand for stem cell characterization and analysis tools. Further, there are various technologies such as flow cytometry which is used to characterize the cell surface profiling of human-bone marrow and other related purposes are expected to increase the growth of stem cell characterization and analysis tools market. In addition, increasing investment by private and public organization for research activities are likely to supplement the market growth in near future.

On the other hand, the unclear guidelines and the technical limitation for the development of the product are expected to hamper the growth of stem cell characterization and analysis tools market.

Rapid increase in corona virus all around the world is expected to hamper the growth of stem cell characterization and analysis tools market. The virus outburst has become one of the threats to the global economy and financial markets. The impact has made immense decrease in revenue generation in the field of all healthcare industry growth for the market in terms of compatibility and it has led in huge financial losses and human life which has hit very hard to the core of developing as well as emerging economies in healthcare sector. It further anticipated that such gloomy epidemiological pandemic environment is going to remain in next for at least some months, and this is going to also affect the life-science market which also include the market of stem cell characterization and analysis tools market.

Based on the Products and Service Type, stem cell characterization and analysis tools market are segmented into:

Based on the Technology, stem cell characterization and analysis tools market are segmented into:

Based on the Applications, stem cell characterization and analysis tools market are segmented into:

Based on the End User, stem cell characterization and analysis tools market are segmented into:

Based on the segmentation, human embryonic stem cell is expected to dominate the market due to their indefinite life span and higher totipotency as compared to other stem cells. Further, on the basis of technology segmentations, cell production is anticipated to increase the demand for stem cell characterization and analysis tools due to their emerging applications for stem cells in drug testing in the management of the effective diseases. Furthermore, on the basis of application segmentations, oncology is expected to show significant growth rate due to increase in the number of pipelines products for the treatment of cancers or tumors. Based on the end user, pharmaceutical and biotechnology companies are expected to dominate the market due to rising global awareness about the therapeutics research activities.

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Geographically, the global stem cell characterization and analysis tools market is segmented into regions such as Latin America, Europe, North America, South Asia, East Asia Middle East & Africa and Oceania. North America is projected to emerge as prominent market in the global stem cell characterization and analysis tools market due to growing cases of target chronic diseases and increasing investments for research activities. Europe is the second leading region to dominate the market due to technological advancement and also surge in therapeutic activities, funded by government across the world. Asia-pacific is likely to witness maximum growth in near future due to increasing disposable income and with the development of infrastructure.

Some of the major key players competing in the global stem cell characterization and analysis tools market are Osiris Therapeutics, Inc., Caladrius Biosciences, Inc., U.S. Stem Cell, Inc., Astellas Pharma Inc., TEMCELL Technologies Inc., BioTime Inc., Cellular Engineering Technologies Inc., Cytori Therapeutics, Inc., and BrainStorm Cell Therapeutics Inc.

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Augmenting Demand for Stem Cell Characterization and Analysis Tools to Bolster Global Market Revenue Growth During the Crisis Period of COVID 19 - 3rd...

Spinal Cord Stem Cells Comments Off on Augmenting Demand for Stem Cell Characterization and Analysis Tools to Bolster Global Market Revenue Growth During the Crisis Period of COVID 19 – 3rd… | June 16th, 2020

Spinal Fusion Market 2020: Latest Analysis

Chicago, United States:- The report titled Global Spinal Fusion Market is one of the most comprehensive and important additions to Report Hive Research archive of market research studies. It offers detailed research and analysis of key aspects of the global Spinal Fusion market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Spinal Fusion market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Spinal Fusion market is carefully analyzed and researched about by the market analysts.

Top Players of Spinal Fusion Market are Studied: Market by Materials, Cotton, Rayon, Blended

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Global Spinal Fusion Market is estimated to reach xxx million USD in 2020 and projected to grow at the CAGR of xx% during 2020-2026. According to the latest report added to the online repository of Report Hive Research the Spinal Fusion market has witnessed an unprecedented growth till 2020. The extrapolated future growth is expected to continue at higher rates by 2026.

Our exploration specialists acutely ascertain the significant aspects of the global Spinal Fusion market report. It also provides an in-depth valuation in regards to the future advancements relying on the past data and present circumstance of Spinal Fusion market situation. In this Spinal Fusion report, we have investigated the principals, players in the market, geological regions, product type, and market end-client applications. The global Spinal Fusion report comprises of primary and secondary data which is exemplified in the form of pie outlines, Spinal Fusion tables, analytical figures, and reference diagrams. The Spinal Fusion report is presented in an efficient way that involves basic dialect, basic Spinal Fusion outline, agreements, and certain facts as per solace and comprehension.

Segmentation by Application: Cord Blood Stem Cells CryopreservationOther Stem Cells Cryopreservation

Segmentation by Type: Liquid phaseVapor phase

NOTE:Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Spinal Fusion Market which would mention How the Covid-19 is Affecting the Spinal Fusion Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Spinal Fusion Players to Combat Covid-19 Impact.

The Essential Content Covered in the GlobalSpinal Fusion Market Report:

* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

The report provides a 6-year forecast (2020-2026) assessed based on how the Spinal Fusion market is predicted to grow in major regions like USA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others.

Key Questions Answered In this Report:

What is the overall market size in 2019? What will be the market growth during the forecast period i.e. 2020-2026?

Which region would have high demand for product in the upcoming years?

What are the factors driving the growth of the market?

Which sub-market will make the most significant contribution to the market?

What are the market opportunities for existing and entry-level players?

What are various long-term and short-term strategies adopted by the market players?

What are the key business strategies being adopted by new entrants in the Spinal Fusion Market?

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Table of Contents

Market Overview: This is the first section of the report that includes an overview of the scope of products offered in the global Spinal Fusion market, segments by product and application, and market size.

Market Competition by Player: Here, the report shows how the competition in the global Spinal Fusion market is growing or decreasing based on deep analysis of market concentrate rate, competitive situations and trends, expansions, merger and acquisition deals, and other subjects. It also shows how different companies are progressing in the global Spinal Fusion market in terms of revenue, production, sales, and market share.

Company Profiles and Sales Data: This part of the report is very important as it gives statistical as well as other types of analysis of leading manufacturers in the global Spinal Fusion market. It assesses each and every player studied in the report on the basis of main business, gross margin, revenue, sales, price, competitors, manufacturing base, product specification, product application, and product category.

Market Status and Outlook by Region: The report studies the status and outlook of different regional markets such as Europe, North America, the MEA, Asia Pacific, and South America. All of the regional markets researched about in the report are examined based on price, gross margin, revenue, production, and sales. Here, the size and CAGR of the regional markets are also provided.

Market by Product: This section carefully analyzes all product segments of the global Spinal Fusion market.

Market by Application: Here, various application segments of the global Spinal Fusion market are taken into account for research study.

Market Forecast: It starts with revenue forecast and then continues with sales, sales growth rate, and revenue growth rate forecasts of the global Spinal Fusion market. The forecasts are also provided taking into consideration product, application, and regional segments of the global Spinal Fusion market.

Upstream Raw Materials: This section includes industrial chain analysis, manufacturing cost structure analysis, and key raw materials analysis of the global Spinal Fusion market.

Marketing Strategy Analysis, Distributors: Here, the research study digs deep into behavior and other factors of downstream customers, distributors, development trends of marketing channels, and marketing channels such as indirect marketing and direct marketing.

Research Findings and Conclusion: This section is solely dedicated to the conclusion and findings of the research study on the global Spinal Fusion market.

Appendix: This is the last section of the report that focuses on data sources, viz. primary and secondary sources, market breakdown and data triangulation, market size estimation, research programs and design, research approach and methodology, and the publishers disclaimer.

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COVID-19 impact on Spinal Fusion Market Share, Size, Revenue, Gross Margin and Growth Rate Analysis 2020-2026

Why Go For Report Hive Research?Report Hive Research delivers strategic market research reports, statistical surveys, industry analysis and forecast data on products and services, markets and companies. Our clientele ranges mix of global business leaders, government organizations, SMEs, individuals and Start-ups, top management consulting firms, universities, etc. Our library of 700,000 + reports targets high growth emerging markets in the USA, Europe Middle East, Africa, Asia Pacific covering industries like IT, Telecom, Semiconductor, Chemical, Healthcare, Pharmaceutical, Energy and Power, Manufacturing, Automotive and Transportation, Food and Beverages, etc. This large collection of insightful reports assists clients to stay ahead of time and competition. We help in business decision-making on aspects such as market entry strategies, market sizing, market share analysis, sales and revenue, technology trends, competitive analysis, product portfolio, and application analysis, etc.

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Impact of Corona on Spinal Fusion Market Statistics, Investment Trends, Key Players and Forecast 2020-2026 | Cotton, Rayon, Blended - Cole of Duty

Spinal Cord Stem Cells Comments Off on Impact of Corona on Spinal Fusion Market Statistics, Investment Trends, Key Players and Forecast 2020-2026 | Cotton, Rayon, Blended – Cole of Duty | June 12th, 2020

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 10 June 2020 16:58Hits: 131

CAMBRIDGE, MA, USA I June 10, 2020 I Biogen Inc.(Nasdaq: BIIB) today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA) that is transforming expectations of early treatment with SPINRAZA (nusinersen). In infants genetically diagnosed with SMA, new data demonstrate that early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented survival. Patients continued to maintain and make progressive gains in motor function compared to the natural course of the disease. These results are being presented at the virtual Cure SMA Research & Clinical Care Meeting taking place June 10-12, 2020.

The new data include nearly a year of additional follow-up for NURTURE study participants. As of February 2020, all patients treated (n=25; median age of 3.8 years old) were alive and remained free of permanent ventilation. In the absence of treatment, the majority of children with SMA Type 1 would, on average, not reach their second birthday. Additionally, all children who achieved the motor milestone of being able to walk independently (many within a normal timeframe) have maintained that ability from the first occurrence until the last visit.

The impact of early and sustained SPINRAZA treatment on these infants and their families is remarkable. Ive had the privilege to watch them grow into active young children, many of whom have experienced progress in motor function consistent with children their age who do not have SMA, said Kathryn Swoboda, M.D., the Katherine B. Sims, M.D., Endowed Chair in Neurogenetics and Director of the Neurogenetics Program, Massachusetts General Hospital. The new results from NURTURE continue to bolster the substantial benefit of both prompt diagnosis and early and longer-term treatment with SPINRAZA.

NURTURE is an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA before 6 weeks old. The study has been extended by an additional three years, enabling Biogen to evaluate the longer-term efficacy and safety of SPINRAZA through 8 years of age and further understand the impact of early treatment. More information on the NURTURE study (NCT02386553) is available onclinicaltrials.gov.

Additional results from the updated interim analysis as of February 2020 show:

About SPINRAZA (nusinersen)2-4 SPINRAZA is the first therapy approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is approved in more than 50 countries. As of March 31, 2020, more than 10,000 individuals have been treated with SPINRAZA. It is the only SMA treatment to combine unsurpassed real-world experience with a robust level of clinical evidence across a broad spectrum of patient populations.

SMA is a rare, genetic, neuromuscular disease that is characterized by a loss of motor neurons in the spinal cord and lower brain stem that can result in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA, and people of all ages are impacted by the disease. It is a leading genetic cause of infant mortality.

SPINRAZA, a foundation of care in SMA, is an antisense oligonucleotide (ASO), developed using Ionis Pharmaceuticals proprietary technology that is designed to target a root cause of SMA by increasing the amount of full-length survival motor neuron (SMN) protein, which is critical to maintaining motor neurons. It is administered by intrathecal injection into the fluid surrounding the spinal cord where motor neurons reside to deliver the treatment where the disease starts.

SPINRAZA currently maintains a robust clinical data set in SMA based on data from approximately 300 patients across a broad range of SMA populations demonstrating a favorable benefit:risk profile. SPINRAZA was evaluated in two randomized, double-blind, sham-controlled studies of infantile and later-onset SMA (ENDEAR and CHERISH, respectively) and supported by open-label studies that include pre-symptomatic infants (NURTURE), individuals with later-onset SMA (CS2/CS12) and an extension study of individuals who previously participated in the clinical development program (SHINE). The most common adverse events observed were respiratory infection, fever, constipation, headache, vomiting and back pain. Hypersensitivity, meningitis and hydrocephalus have been observed in the post-marketing setting. Renal toxicity and coagulation abnormalities, including acute severe low platelet counts, have been observed after administration of some ASOs. Laboratory tests can monitor for these signs.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in five years.

About BiogenAt Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the worlds first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics and is focused on advancing research programs in multiple sclerosis and neuroimmunology, Alzheimers disease and dementia, neuromuscular disorders, movement disorders, ophthalmology, immunology, neurocognitive disorders, acute neurology and pain.

We routinely post information that may be important to investors on our website at http://www.biogen.com. To learn more, please visit http://www.biogen.com and follow us on social media Twitter, LinkedIn, Facebook, YouTube.

References:

SOURCE: Biogen

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New Results From Landmark NURTURE Study Show That Pre-Symptomatic SMA Patients Treated With SPINRAZA (nusinersen) Continue to Demonstrate Sustained...

Spinal Cord Stem Cells Comments Off on New Results From Landmark NURTURE Study Show That Pre-Symptomatic SMA Patients Treated With SPINRAZA (nusinersen) Continue to Demonstrate Sustained… | June 10th, 2020