To keep you healthy from day to day, your body has its own in-built, germ-fighting network.

This network is also known as the lymphatic system, made up of different vessels and organs in your body, from the lymph nodes to the bone marrow. Lymphoma is the name of the cancer that affects this system.

There are many types of lymphoma but Diffuse Large B-Cell Lymphoma (DLBCL) is the most common aggressive type of non-Hodgkin lymphoma, affecting the B-lymphocytes that produce antibodies which help your body fight infections.

According to the World Health Organizations guidance on classifying tumours, DLBCL accounts for 30 to 40 per cent of newly diagnosed cases of non-Hodgkin lymphoma globally.1

In Singapore, Dr Daryl Tan Chen Lung, who specialises in haematology and practises at Mount Elizabeth Hospitals, estimates that lymphoma is the fifth most common cancer in Singapore.2

Unlike other cancer patients, DLBCL patients hardly have any contributing factors to the development of the condition not even hereditary ones though people with existing HIV infections are more prone to getting DLBCL.

As a result of this, and also because lymphoma is less common than other cancers like breast or lung cancer, regular medical check-ups do not test for its genetic indicators. Instead, patients seek medical help only when they spot early symptoms, says Dr Tan. A growing awareness of lymphoma World Lymphoma Awareness Day takes place on Sept 15 each year and better diagnostics measures have led to much earlier detection of the cancer today, compared to a decade ago.

Dr Tan says that early symptoms of DLBCL include lumps in the neck or groin. Sometimes, other organs like the spleen and bone marrow can be affected. However, some patients show no obvious symptoms beyond abdominal pain, fever and loss of weight. It is thus especially important to seek medical attention should you display any of the common symptoms of lymphoma including swollen lymph nodes in the neck, armpit or groin, a persistent fever, excessive night sweats and unexplained weight loss.

Dr Tan adds that the median age of DLBCL patients in Singapore is 60 to 65, though a very small number of patients in their 20s are diagnosed with a subtype of the cancer known as primary mediastinal B-cell lymphoma.

While DLBCL is an aggressive cancer, the good news is that it is highly treatable in the early stages.

Current therapies for treating first-line DLBCL include chemotherapy, chemo-immunotherapy a combination use of chemotherapy and immunotherapy and stem cell transplant in certain groups of patients. About six in 10 of these patients will respond to their treatment3 and not suffer a relapse within the next five years.

This has a huge impact on younger patients who are then able to resume their daily life and go back to work, for example.

Dr Tan remembers a female patient in her early 20s whose career was just taking off. She was admitted with fever and breathlessness. After performing scans on her, it was discovered that there was fluid surrounding her heart and lungs, and she had a tumour in her chest the size of a rugby ball.

Fortunately, because the cure rate for DLBCL is high, the patients condition has since gone into remission.

A combination of chemo-immunotherapy drugs is commonly recommended as a first-line treatment in DLBCL. Radiation therapy or stem cell transplant can also be included as part of the treatment.

However, about four in 10 patients may not eventually respond to these therapies or suffer from a relapse.3 When this happens, treatment options are limited. Some patients can undergo a stem cell transplant, but about half of them are not eligible.4 This group includes older patients with compromised immune systems.

While affected patients can consider continuing with chemotherapy and chemo-immunotherapy, there are new promising treatment options on the horizon, including targeted therapies such as CAR T-cell therapy. These targeted therapies involve using the patients own immune cells also known as T cells or molecules that bind a chemotherapy agent to an antibody to fight cancerous blood cells.

Beyond that, there are also treatment options known as combination therapy. Previously, patients suffering a relapse have had to rely on existing, first-line treatment, which may or may not be effective in combating the cancer. Combination therapy uses the collective effect of different medications to target different areas of cancerous B-cells and destroy them.

Says Dr Tan: [The medical community is] seeing progress in DLBCL immunotherapy. The current cure rate is about 70 per cent. Hopefully, we can increase the number to 80 per cent or more. But we mustnt rest on our laurels as we hope to focus on the 20 per cent of patients who still dont respond to any treatments.

For more information on lymphoma and treatment options, please speak to your healthcare specialist.

1. Lyon, France. World Health Organization Classification of Tumors of Haematopoietic and Lymphoid Tissues. IARC Press; 2008.2. SingHealth. (n.d). Lymphoma. Retrieved on 30 Sept 2020. (https://www.singhealth.com.sg/patient-care/conditions-treatments/lymphoma)3. Maurer, MJ et al. Event-free survival at 24 months is a robust end point for disease-related outcome in diffuse large B-cell lymphoma treated with immunochemotherapy. J Clin Oncol. 2014; 32: 1066-73.4. Gisselbrecht C, Van Den Neste E. How I manage patients with relapsed/refractory diffuse large B cell lymphoma. Br J Haematol. 2018;182:633643.

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AUSTIN, Texas, Oct. 13, 2020 /PRNewswire/ --Direct Biologics, LLC, announced today that the FDA has granted expanded access for ExoFlo in the treatment of patients with COVID-19 associated acute respiratory distress syndrome (ARDS).

While Direct Biologics is already enrolling patients in EXIT COVID-19, its national multi-center, Phase II, placebo controlled, randomized clinical trial, the new expanded access protocol will make ExoFlo available to a broader group of patients with severe COVID-19, many of whom would not meet acceptance criteria for EXIT COVID-19, often under conditions of "compassionate use."

Co-Founder and Chief Executive Officer, Mark Adams, states, "The FDA's approval of expanded access for ExoFlo signifies a critical milestone in the development of advanced treatment for COVID-19 associated illnesses including ARDS. We are excited to be able to provide our product to patients with ARDS associated with COVID-19 in critical need of treatment."

"Amid a potential surge in new COVID-19 cases moving into the fall and winter seasons, this approval could not have come at a better time," notes Joe Schmidt, Co-Founder and President. "Our team is working hard to advance our Phase II EXIT COVID-19 trial to offer additional treatment options."

"Approval of our expanded access protocol offers an option for doctors to administer ExoFlo as a treatment to reverse disease progression, extending hope to COVID-19 patients who are not responding favorably to standard of care," states Chief Medical Officer, Vik Sengupta, MD. "We at Direct Biologics are grateful for every opportunity to help these additional patients in need."

Also in attendance was Congressman Michael McCaul who commented, "I am thrilled to hear a local company from Austin, Texas, has been approved for Expanded Access IND by the FDA. Their product, ExoFlo, will help treat COVID-19 patients who are at risk of severe respiratory infection, which often leads to life-threatening circumstances. Direct Biologics, with support of the FDA, will bring high-class treatments and services to Americans who need them the most."

This approval comes on the heels of multiple approvals for single patient Emergency Investigational New Drug (eIND) applications granted in September and October. Emergency INDs are a mechanism by which physicians can obtain rapid approval to administer medication to a single patient through a direct appeal to the FDA.

About ExoFlo ExoFlo is an investigational new drug that has not been approved or licensed by FDA. It is an extracellular vesicle product isolated from human bone marrow mesenchymal stem or stromal cells (MSCs). ExoFlo provides natural bioactive signals that have been shown to modulate inflammation and direct cellular communication.

About Direct Biologics Direct Biologics, LLC, is headquartered in Austin, Texas, with a recently expanded R&D facility located at the University of California, and an Operations and Order Fulfillment Center located in St. Louis, Missouri. Direct Biologics is a market-leading innovator and cGMP manufacturer of regenerative medical products, including a robust line of extracellular vesicle-based biological products. The Company was created to expand the science of regenerative healing by delivering cutting-edge biologic technologies. Direct Biologics' management team holds extensive collective experience in biologics research, development, and commercialization, making the Company a leader in the evolving, next generation segment of the biotherapeutics industry. Direct Biologics is dedicated to pursuing additional clinical applications of its extracellular vesicle biologic products through the FDA's investigational new drug application process. For more information visit http://www.directbiologics.com.

Phone:1-800-791-1021Email:[emailprotected]

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Using a brush or comb, in the shower, passing your hand through your hair In a 2015 IFOP survey, three out of four French people (76%) without baldness declared that they lost their hair. In addition, after 65 years, three in ten men suffer from androgenetic alopecia (or baldness). In general, therefore, we often see a loss of density with age, both in men and in women. However, science has long looked into the phenomenon of age-related hair loss, both to understand its origin and to treat it. Focusing on stem cells seems to offer the best hope for a cure, based on numerous studies.

The hair transplant procedure is indeed a sometimes expensive and invasive procedure. As for drugs, finasteride used for androgenetic alopecia in men can induce side effects such as loss of libido and erectile dysfunction while minoxidil can cause hypertrichosis. Today, researchers therefore seem to be looking more at alternative solutions, and in particular by focusing on stem cells.

Stem cells, directly linked to hair lossFor decades, researchers have always focused on keratinocytes, cells that make up the epidermis and integuments (body hair, hair, etc.). However, as part of a study conducted by the University of Calgary (Canada), the team of researchers focused on a small cell group present in hair follicles and in skin stem cells. : fibroblasts. And according to them, these fibroblasts are the main cause of age-related hair loss.

By studying the sparse coat of elderly mice, they noticed that fibroblast stem cells had lost their regenerative function or were malfunctioning. There werent enough of them to regenerate fibroblasts. As a result, the fibroblasts and hair follicles began to miniaturize and were no longer able to produce hair, explains Biernaskie, head of the research team.

Remember that fibroblasts are important because they send messages to keratinocytes to force them to divide, and in so doing, orchestrate the growth cycles of hair follicles allowing the production of new hairs. When the fibroblasts become scarce, the signal then becomes too weak to reach the keratinocytes and maintain the process of capillary growth. For Jeff Biernaskie: if we want to one day succeed in preventing hair loss or re-grow those that are already falling, we must work to preserve the function of these stem cells which are found in the hair follicles.

Against hair loss, but not only!This finding may help guide future research on hair loss more precisely. Scientists at the University of Calgary are particularly hoping to find a way to prevent this degeneration by blocking certain genetic mutations that occur directly in stem cells in fibroblasts.

They also believe that this will have wider implications. Indeed, Wisoo Shin, lead author of the study, points out that similar fibroblasts are found in most of our organs, maintaining their integrity and promoting tissue regeneration. Finding a way to promote self-renewal to produce new functional fibroblasts into old age therefore also offers the hope of being able to treat certain injuries and help the skin to regenerate.

Lamia spent a couple of years interning at an organization that offered medical consultation before joining the editorial team at FLWL News. An enthusiastic fitness freak in the room, she offers the best amounts of insights and craft-based writing style to keep us up to date about the medicine industry, health and science.

Email:lamia@flwl.orgPhone: +1 512-845-8162

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Electric organoid: Neurons derived from people with 22q11.2 syndrome are hyperexcitable and show calcium-signaling deficits.

Courtesy of Pasca lab

Neurons derived from people with 22q11.2 deletion syndrome, a genetic condition linked to autism, show deficits in electrical activity and calcium signaling, according to a new study1. A single gene appears to be largely responsible for these defects, the study shows.

Up to 20 percent of people who lack part or all of the chromosomal region 22q11.2 have autism. Individuals with the deletion may also have schizophrenia, seizures, heart defects, immune dysfunction or learning problems.

The new findings uncover factors that may contribute to the development of psychiatric conditions associated with 22q11.2 deletion syndrome. They could also help researchers identify new therapeutic targets, says lead author Sergiu Pasca, associate professor of psychiatry and behavioral sciences at Stanford University in California.

The syndrome is relatively common, occurring in up to 1 in 4,000 newborns, Pasca says. But researchers do not fully understand how genes in the 22q11.2 region contribute to autism or other conditions, he adds.

To solve this molecular puzzle, Pasca and his team reprogrammed skin cells from 15 people with the deletion and 15 controls into induced pluripotent stem cells. Using a technique they developed in 2015, they coaxed these cells to turn into neurons, which self-organize in a dish into spherical clusters called organoids. The organoids show some key features of the developing cerebral cortex, a brain region implicated in autism.

The neurons derived from people with 22q11.2 syndrome spontaneously fire four times as frequently as neurons derived from controls, the researchers found. And the electrical activity of the 22q cells does not set off the usual spike in calcium levels, which is crucial for neurons to exchange messages.

In some other syndromes tied to schizophrenia and autism, calcium-channel genes are mutated. But the number of channels and the speed at which they work in 22q neurons is the same as in control neurons. Instead, the 22q cells show an unusually low voltage difference across the cell membrane when they arent firing, causing the signaling defects and hyperexcitability, the researchers found.

The researchers suspected that a gene called DGCR8 might be responsible for the neuronal deficits in the organoids because it lies within 22q11.2 and is linked to abnormal electrical activity in the neurons of mice2. DGCR8 is essential for the synthesis of short RNA fragments, called microRNAs, that regulate gene expression.

Lowering DGCR8s expression levels in control neurons reproduced the abnormalities seen in 22q neurons. In contrast, boosting the genes activity in 22q neurons or treating them with antipsychotic drugs prevented them from being overly excitable and reversed their calcium-signaling defects. The study was published 28 September in Nature Medicine.

Previous studies have analyzed lab-grown neurons derived from people with schizophrenia or autism-related disorders such as Rett and fragile X syndromes. But most used only a few human-derived cell lines, says Guo-li Ming, professor of neuroscience at the University of Pennsylvania in Philadelphia. The new study, Ming says, has a total of 30 human lines thats a huge effort.

By studying brain organoids derived from so many people, the researchers were able to identify the gene that might be involved in the psychiatric conditions associated with 22q11.2 syndrome, says Sally Temple, scientific director of the Neural Stem Cell Institute in Rensselaer, New York. Whenever we have a light shining ahead, saying, This is what you should really be looking at, it means that were making progress, she says.

The study participants with 22q11.2 syndrome vary in their psychiatric diagnoses, and yet all the brain organoids derived from their cells show the same neuronal abnormalities. Thats somewhat surprising, because we know there are a lot of differences in the genetic background of different people, Ming says.

The deletion might conspire with other factors to ultimately determine which psychiatric conditions a person has, Pasca says. It could be that the deletion causes cellular defects, and once there is a stressor such as social stress, disease develops. Its also unclear whether these cellular defects are related to the high prevalence of seizures in people with 22q11.2 syndrome, he says.

The hallmarks of most neuropsychiatric conditions can change over time, says Giuseppe Testa, director of the stem cell epigenetics unit at the European Institute of Oncology in Milan, Italy. Studies that look at a larger number of people with 22q11.2 deletion syndrome or other neurodevelopmental conditions could help to elucidate the relationship between the neuronal defects observed in the lab and the psychiatric manifestations of the conditions, Testa says. The new study, however, is a great resource for understanding how the 22q11.2 deletion contributes to schizophrenia and autism, he says.

Pascas team is trying to pinpoint molecules that could open new therapeutic avenues for 22q11.2 deletion syndrome. The antipsychotics they tested restore the unusual voltage differences in the 22q neurons, but they dont address the core mechanisms responsible for psychiatric conditions linked to the syndrome, Pasca says.

Whats more, antipsychotics have many side effects, and not all individuals respond to them, he says. We need better therapies we need to identify what the key molecular players are and target those.

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When thinking about donating bone marrow, most will break out in a cold sweat.

The thought of needles, prodding and poking is enough to put anyone off from becoming a donor but Ndinae Muligwe, Sustainability and Donor Recruitment Coordinator for the South African Bone Marrow Registry (SABMR) explained that it is a less complicated and relatively painless process.

The SABMR was established in 1991 and is a non-profit organisation that conducts searches to find matching bone marrow donors for critically ill children and adults in South Africa who cannot find a match in their own families.

Bone marrow transplants help to treat and even sometimes cure illnesses like leukaemia, Non-Hodgkin lymphoma, bone marrow failure, and some genetic blood and immune-system disorders.

Ndinae explained that the likelihood of a donor finding a match is about one in 100 000. What is more concerning is that there are currently only around 74 000 local donors on the South African Bone Marrow Registry.

Although they do form part of the World Marrow Donor Association that represents about 38 million donors, there are not enough donors for the South African demographic.

Ethnicity plays a role when it comes to who is able to donate, and at the moment the numbers do not match the ethnic groups represented in South Africa. You are more likely to find a match within your own ethnic group.

But how do you become a donor and what is the process involved?

Ndinae said it is as easy as registering on the website. Of course there are some questionnaires to fill in and you will have to meet the criteria and be healthy.

The donating age has recently been lowered from 18 to 16 years of age, and applicants must be between 16 and 45 to register as a potential donor.

If you are eligible you will then be contacted by the SABMR to do a cheek swab free of charge.

Peripheral blood stem cell (PBSC) collection is the most likely way of collecting stem cells. These cells are found in your bone marrow and also the blood stream. A five-day course of growth factor or Granulocyte-Colony Stimulating Factors is given prior to the donation to encourage the stem cells to move from your marrow to your blood.

At the time of donation a needle is placed in one arm. The blood is then passed through a machine that collects the stem cells, and the remaining blood is returned to your body similar to donating blood platelets.

You do not have to pay for anything to make a tissue or blood donation of your bone marrow stem cells, the SABMR covers the cost of testing and collection.

Visitwww.sabmr.co.zafor more information.

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Embryonic stem cell research has been always disputed by the 2020 Republican party. In 2019, Trumps administration paused funding for government scientists to work on studies involving embryonic stem cells, affecting about $31m in research, according to Science Magazine.

Regeneron, on the other hand, doesnt consider these cells fetal tissue because the HEK-293T line of cells has been immortalized and they divide and regenerate themselves in the laboratory.

The investigational drug has been in clinical trials since June. Even though early results from a trial with around 300 non-hospitalised COVID patients showed the drug was safe and could reduce viral levels and improve symptoms, the data is yet to be peer-reviewed.

According to CNN, the treatment is not yet approved for any use from the US FDA. The company, however, is in talks for an emergency approval. Regeneron has also confirmed that it had provided the drug under a compassionate use request for President Trump from the doctors.

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COVID Drug Given to Trump Developed From Aborted Fetus Cells - Quint Fit

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Patients with cystic fibrosis experience recurrent lung infections that eventually destroy their airways, shortening their average life expectancy to 50 years in Canada. Current drug treatments, which target a malfunctioning pathway in cells that causes the infections, are costly and have varying effectiveness.

Now, with funding from Medicine by Design, a researcher at the Hospital for Sick Children (SickKids) is combining stem cells, gene editing and computational modelling to try to hijack an alternative cell pathway in the hopes of restoring lung function in these patients.

If successful, our study will be the first to provide proof-of-concept that this alternative approach to treating cystic fibrosis is effective, saysAmy Wong, a scientist working in developmental and stem cell biology at SickKids who is also an assistant professor in the department of laboratory medicine and pathobiology in the University of Torontos Temerty Faculty of Medicine.

Wongs project is one of seven across U of T and its affiliated hospitals that have been awarded 2020New Ideas AwardsandSeed Fundawards from Medicine by Design. Through a $1 million investment, Medicine by Design is supporting research aimed at advancing new concepts expected to be important to regenerative medicine in the coming years. The funded projects will have potential impacts in diseases and conditions such as vision loss, amyotrophic lateral sclerosis (ALS), intestinal disease in premature babies and more.

Supporting novel strategies and approaches is crucial to moving regenerative medicine into the future, saysMichael Sefton, executive director of Medicine by Designand a University Professor at U of Ts Institute of Biomedical Engineeringand thedepartment of chemical engineering & applied chemistry in the Faculty of Applied Science & Engineering.

Our 2020 New Ideas project portfolio integrates mathematical modelling, physics and computational biology with stem cell biology and biomedical engineering, and strengthens engagement with clinicians who are key to translating our research into patient impact. We are particularly delighted this year to support so many outstanding early-career researchers, who will ensure Toronto remains a global leader in regenerative medicine for years to come.

Wong is one of three investigators to receive a 2020 New Ideas Award, which is valued at $100,000 per year for up to two years. Four additional projects were selected for Seed Fund Awards of $100,000 each for one year to further develop their potential.

Medicine by Design selected the funded projects from among 36 short-listed proposals, which were evaluated and ranked through an external peer review process. Applications were submitted by clinicians and researchers at U of T and its affiliated hospitals from a wide range of disciplines including biochemistry, biomedical engineering, developmental and stem cell biology, immunology, neuroscience and surgery.

Medicine by Design builds on decades of made-in-Canada excellence in regenerative medicine dating back to the discovery of stem cells in the early 1960s by Toronto researchers James Till and Ernest McCulloch. Regenerative medicine uses stem cells to replace diseased tissues and organs, creating therapies in which cells are the biological product. It can also mean triggering stem cells that are already present in the human body to repair damaged tissues or to modulate immune responses. Increasingly, regenerative medicine researchers are using a stem cell lens to identify critical interactions or defects that prepare the ground for disease, paving the way for new approaches to preventing disease before it starts. Medicine by Design is made possible thanks in part to a $114-million grant from theCanada First Research Excellence Fund.

Current cystic fibrosis drug treatments target a genetic mutation that causes epithelial cells, which line the airway and act as a barrier against viruses, to function improperly. The mutation affects the function of an important ion channel in cells, called CFTR, which helps to maintain the right balance of fluid in the airways. Poor function causes mucosal obstructions in the airways and prevents clearance of foreign pathogens, which leads to chronic infections and ultimately destroys airway tissue.

In her project, Wong will explore an alternative ion channel in the epithelial cells to determine if it can be hijacked and used to compensate for the lack of function caused by the mutant CFTR. The research will be conducted using a combination of stem cell-derived lung models, gene editing and computational modelling.

Wongs project builds on decades of cystic fibrosis research at SickKids, where the cystic fibrosis gene was first identified 30 years ago.

To date, more than 2,000 mutations in the cystic fibrosis gene have been identified, says Wong. SickKids scientists and U of T researchers have become the epicentre of incredible cystic fibrosis research to understand how this disease works at the genetic and molecular level.

Wong says that, while the idea of targeting an alternative pathway is not necessarily ground-breaking on its own, its the array of tools now available that makes the idea a potential game changer.

We have access to an incredible resource of primary cells and stem cells from more than 100 individuals with cystic fibrosis harbouring various mutations. Wong says.Our lab has developed human lung models from stem cells that can be used to model lung disease such as cystic fibrosis. And with new advanced tools in single-cell genomics and gene-editing, coupled with key collaborations for computational modelling, we are poised to find new therapeutic targets for cystic fibrosis.

Leo Chou, an assistant professor at the Institute of Biomedical Engineering, andHyun Kate Lee, an assistant professor in the department of biochemistry in the Temerty Faculty of Medicineboth Medicine by Design New Investigators are also leading 2020 New Ideas projects.

Chou, along with co-investigatorsJulie Lefebvre, a scientist at SickKids and U of T assistant professor of molecular genetics, andValerie Wallace, a senior scientist at the Krembil Research Institute, University Health Network and a U of T professor of laboratory medicine and pathobiology and ophthalmology, will focus on cell transplantation in the retina, a process that has demonstrated encouraging pre-clinical results such as partial vision restoration in several animal disease models.

Recent research had demonstrated that this restoration is a result of the transfer of proteins complex molecules required for the structure, function and regulation of the bodys tissues between host tissue and donor cells. But the scope of that transfer process is not well understood. Chous project will develop an imaging approach to detect the transfer of mRNA molecules between host and donor cells. The outcomes from this project will inform the future design of cell transplantation therapies and lead to novel methods to deliver therapeutics. This project could improve therapies for retinal diseases and visual impairments, and inform strategies for other degenerative disorders.

Lee and co-investigatorPenney Gilbert,an associate professor at the Institute of Biomedical Engineering, will look at a common but not well-understood structure called the neuromuscular junction (NMJ), which mediates communication between neurons and muscles throughout the body. Defects in NMJ integrity and function underlie fatal diseases such as ALS. NMJ diseases, which affect more than 500,000 people globally, lack effective treatments. This project will use stem cells derived from reprogrammed skin cells of healthy people to develop NMJs in culture. Through high-resolution imaging, the healthy human NMJs will be studied both on their own and along with NMJs built from ALS patient cells. Through this work, the research team aims to identify genes to target to improve the health of NMJs, which could eventually help prevent or delay NMJ degeneration and even promote regeneration.

Michael Garton, an assistant professor at the Institute of Biomedical Engineering, has received a Seed Fund award to tackle the challenge of translating the genetic tools of synthetic biology an area of research that aims to create or redesign biological components using engineering methods into effective medical therapies against a number of diseases.

But they are difficult to translate into human therapies, Garton says, because the bodys T-cells immune cells that detect and destroy cells containing foreign material will identify these tools as foreign and destroy them.

Instead of switching off the T-cells, Gartons goal is to use computational modelling and high-throughput screening to selectively turn off the bodys foreign antigen display system so the immune system will still respond to foreign invaders when necessary, but allow cells containing synthetic tools to survive. If successful, this approach could enable a new generation of synthetic biology-enhanced cell therapies for a range of diseases.

Medicine by Design funding will help to facilitate the integration of synthetic biology and regenerative medicine and aid the development of cell-based therapies that perform better than nature, says Garton.

Other Seed Fund projects will encompass research in repairing the heart after paediatric cardiac surgery, treating an intestinal emergency in premature babies and creating a database for cell lineage paths.

John Parkinson, a senior scientist at SickKids and a U of T professor of biochemistry and molecular genetics, along with co-investigatorsJason Maynes, Wasser Chair in Anesthesia and Pain Medicine at SickKids and a U of Tassociate professor of anesthesiology and biochemistry, andWilliam Navarre, an associate professor in the department of molecular genetics, will investigate manipulating the microbiome, or community of microorganisms in the gut, to improve cardiac repair in post-operative treatment of a congenital heart disorder. Through a process that will identify prebiotics in breast milk that help enhance the production of molecules that research has shown can aid cardiac repair, the team will organize both observational (how disease alters the microbiome) and interventional (how the microbiome alters the disease) multi-site trials, which will provide the opportunity to immediately translate findings into changes in patient care regimens and improve outcomes.

CliniciansAgostino Pierro, a surgeon at the Division of General and Thoracic Surgery at SickKids and a U of T professor of surgery and physiology, and Philip Sherman, a senior scientist and gastroenterologist at the Division of Gastroenterology, Hepatology and Nutrition at SickKids and U of T professor of dentistry, pediatrics and laboratory medicine and pathobiology, have proposed a novel way of enhancing gut repair for a common intestinal emergency in premature babies, called necrotizing enterocolitis (NEC). A leading cause of death for these infants, NEC causes complications such as blindness, intellectual disability, repeat hospitalizations and gut damage even in those that survive. This project will look at whether intestinal organoids organ-like structures grown in the laboratory from stem cells that mimic some of the functions of native intestines can potentially stimulate repair of the gut and recovery from NEC. The project will define how to best transplant organoids, identify how the organoids protect the intestine from injury and assess if organoid transplantation is a valid new treatment for NEC.

Lincoln Stein, who is head of adaptive oncology at the Ontario Institute for Cancer Research and a professor in the department of molecular genetics at U of T, has received seed funding to build a database called Cytomics Reactome, which will be freely available to Canadian and international researchers. The database will build on recent technologies that open the door to the possibility of deciphering cell lineage paths the series of steps that lead a young, undifferentiated cell into a specialized one at single-cell resolution. To accelerate the path from basic research to clinical application, the database will systematically organize pre-existing knowledge of cell lineage paths into a comprehensive, interactive and easily accessible map that can serve as a framework for interpretation and integration of the latest experimental findings.

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U of T's Medicine by Design invests $1 million to advance new ideas in regenerative medicine - News@UofT

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IRVINE, Calif., Oct. 9, 2020 /PRNewswire/ --AIVITA Biomedical, Inc., a private biotechnology company developing personalized vaccines for the treatment of cancer and COVID-19, announced today the publication of the peer-reviewed manuscript, "Retina organoid transplants develop photoreceptors and improve visual function in RCS rats with RPE dysfunction,"in the journal Investigative Ophthalmology & Visual Science. The study, led by researchers at AIVITA Biomedical and the Sue & Bill Gross Stem Cell Research Center of the University of California, Irvine, used 3D-retina organoids generated from human stem cells developed by AIVITA to provide insight into the potential use of transplanted retina organoids as a therapeutic option for blinding diseases.

In the study, transplanted retina organoid sheets were examined to determine if human stem cell-derived photoreceptors coulddevelop, survive and function in vivo without the support of healthy retina pigment epithelium (RPE). Visual function was examined through a variety of tests, including optokinetic testing (OKT), electroretinogram (ERG), and superior colliculus (SC) brain recording. These tests concluded that retina organoid transplantations demonstrated significant improvement in visual function compared to non-surgery and sham surgery controls, supporting the application of AIVITA's stem cell technologies in visual disease therapeutics.

"Leveraging our expertise in stem cell growth and differentiation, I'm excited to see the promise of our technology platform in potential therapeutics for vision loss," said Hans Keirstead, Ph.D., chief executive officer of AIVITA and a contributing author to the paper. "To our knowledge, this study is the first to show that it's possible for photoreceptors derived from stem cells to survive and function after transplantation when a host has a dysfunctional RPE."

This work is supported by funding from the California Institute for Regenerative Medicine (CIRM) and National Institutes of Health (NIH).

About AIVITA Biomedical AIVITA Biomedical is a privately held company engaged in the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. Founded in 2016 by pioneers in the stem cell industry, AIVITA Biomedical utilizes its expertise in stem cell growth and directed, high-purity differentiation to enable safe, efficient and economical manufacturing systems which support its therapeutic pipeline and commercial line of skin care products. All proceeds from the sale of AIVITA's skin care products support the treatment of people with cancer.

SOURCE AIVITA Biomedical, Inc.

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AIVITA Biomedical's Stem Cell Therapeutic in Vision Loss Published in Investigative Ophthalmology & Vision Science - PRNewswire

Skin Stem Cells Comments Off on AIVITA Biomedical’s Stem Cell Therapeutic in Vision Loss Published in Investigative Ophthalmology & Vision Science – PRNewswire | October 10th, 2020

Two years ago I received an Augustinus Bader cream in a goody bag. I hadnt heard of it at the time, so paid little attention, although it looked nice enough a sleek, big blue bottle with rose gold accents. Shortly after, having just run out of my current moisturiser, I dug it out to give it a go. A few days later, forensically inspecting my skin in the mirror as I am wont to do in my more idle moments, I thought this stuff is actually really great. This was swiftly followed by another thought oh dear when I discovered it costs a whopping 205 a pop (for 50ml, you can get 15ml for 65). But it was too late, I was hooked. And that is how I became a woman who drops 205 on a moisturiser.

I am probably the least glamorous and definitely least well-known member of the Augustinus Bader fan club, which has swiftly reached legendary status in the beauty world and beyond. Kim Kardashian West, Naomi Campbell, Kate Bosworth, Priyanka Chopra and Diane Kruger all love the cult brand. Victoria Beckham invited Professor Bader to sit front row at her AW20 show; she also tapped his talents to collaborate with her on her debut skincare line (from seeing VB up close I can testify that this is a woman who knows good skincare, and the Cell Rejuvenating Power Serum is indeed excellent).

Professor Augustinus Bader and Victoria Beckham Getty

The latest addition to the Bader fan club is Emma Corrin. Prepping the new Diana for The Crowns Zoom press junket this week, her makeup artist Florrie White revealed she used The Cream and The Face Oil on the young actress. I use The Cream on everyone; me and my clients, White tells me. It instantly plumps and smooths the skin with three perfect pumps and creates a calm and luxurious base for every make-up look. Everyone always comments on how lovely it feels on their skin.

Today Augustinus Bader is a cult phenomenon but the founder is a man who seems to be more at home in a laboratory than on a red carpet. A leader in stem cell biology, and head of stem cell research at the University of Leipzig, Professor Bader has spent over 30 years focusing on how reawakening these cells can aid the healing process in particular embarking on a mission to help burns victims. In 2008, Bader formulated a groundbreaking wound gel that could help heal third-degree burns without the aid of surgery or skin grafts.

It was this breakthrough that led to the founding of the skincare line. After all, if it could have this effect on serious injuries, imagine what the technology could do for those of us lucky enough to have run-of-the-mill skin niggles? The brand launched with two hero products: The Cream and The Rich Cream which, according to the brand literature, contain patented TFC8 (Trigger Factor Complex) technology, which supports the skins natural processes, leaving all skin types mature, dry, oily, or sensitive looking restored, renewed and regenerated.

I was useless at science at school and all this technical talk is pure gobbledygook to me. What I can tell you is what its done for me. My skin is super sensitive, I have eczema, and have found that Augustinus Bader creams genuinely help to soothe it when it is aggravated. I have found that since using it, my complexion appears clearer, more even and the Holy Grail of beauty dewy (despite drinking gallons of water a day, I have never achieved that before). My mum, who observes me with the scrutiny that only mothers do, confirms that my skin looks great (I am privvy only to this information because she thought it was a result of finally listening to her by stopping smoking and making the hours before midnight count). Is it expensive? Well, yes, theres no way around that. However, in much the same way that I think that an impeccably cut designer jacket earns its value back in cost per wear, I have found that since using The Cream and The Rich Cream my skincare regime has simplified.

Sure, theres an element of hype involved. I still find it thrilling to be In The Know about something. But, for me and my skin, Augustinus Bader lives up to it. Believe me, I wish it wasnt true (I am not snobby with beauty products, and would happily wax lyrical about a 5 wonder find if Id discovered one), but thats the 205 price I have to pay and Id say its worth it.

READ MORE: 'I Just Love The Way A Polished Lipstick Completes A Look': Victoria Beckham's New Posh Lipsticks Explained In Her Own Words

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Discover The Augustinus Bader Wonder Cream Loved By The A List - Grazia

Skin Stem Cells Comments Off on Discover The Augustinus Bader Wonder Cream Loved By The A List – Grazia | October 10th, 2020

The biotech industry has been soaring this year, with the SPDR S&P Biotech ETF (XBI) up 26.6% year to date so far. The race for a COVID vaccine has primarily driven this performance, but what if there was another segment of the biotech industry that top hedge fund managers are all going after right now? While many managers are known to take risks, its certainly worth looking into if there is a consensus between them.

The AlphaClone Alternative Alpha ETF (ALFA) tracks an index of equity securities that hedge funds have significant exposure to. The ETFs top three holdings are all biotech firms working on cancer drugs. While many investors have their attention on biotech and pharmaceutical companies working on a COVID vaccine, big money has been focused on the next big thing in biotech, the future oncology drug boom.

Cancer is the second leading cause of death in the U.S. behind heart disease. Almost everyone knows someone that has been affected by one of the many vicious types of cancer. There are now numerous companies focused on ways to treat and cure the various forms. While COVID is at the forefront, cancer is a long-term play. There is even an ETF that covers the cancer industry, the Loncar Cancer Immunotherapy ETF (CNCR), which is up over 39% over the last six months.

As the oncology drug market is expected to reach $394 billion by 2027, here are the three top cancer stocks based on a consensus of hedge fund managers: Seattle Genetics (SGEN), Fate Therapeutics (FATE), and Blueprint Medicines (BPMC).

Seattle Genetics (SGEN)

SGEN is a biotech firm focused on developing antibody-drug conjugates. Its lead lymphoma drug, Adcetris, has been performing quite well since it launched, and it is the primary growth driver for the company. The drugs label was also expanded, providing more revenue for the company. SGEN has been collaborating with Takeda (OTCMKTS:TKPHF), a Japanese pharmaceutical company, for the global development and commercialization of Adcetris.

In addition to Adcetris, the company has a promising pipeline of drug candidates for its antibody-drug conjugate (ADC) technology. In December, the FDA granted accelerated approval to Padcev to treat patients with metastatic bladder cancer, who were previously treated with a checkpoint inhibitor and platinum-based chemotherapy. This drug was created in collaboration with Astellas Pharma (OTCMKTS:ALPMF), another pharmaceutical company.

In April, the FDA approved Tukysa for the treatment of metastatic HER2-positive breast cancer. Investors should also be happy with the news Merck (MRK) plans to buy a 2.9% stake in SGEN. The companies are co-developing and selling SGENs breast cancer therapy, ladiratuzumab vedotin.

The company is rated a Strong Buy in our POWR Ratings system, with a grade of A in Trade Grade, Buy & Hold Grade, and Peer Grade. Those are three out of the four components that make up the POWR Ratings. The stock is also ranked #2 out of 377 Biotech stocks.

Fate Therapeutics (FATE)

FATE is a clinical-stage biopharmaceutical company engaged in the development of programmed cellular immunotherapies for cancer and immune disorders. The company has been building up its pipeline of immuno-oncology product candidates. These treatments are designed to elicit an immune response in patients with cancer.

The companys progress with FT596 is encouraging. FT596 is cell cancer immunotherapy derived from its iPSC line. The induced pluripotent stem cell (iPSC) platform provides a competitive advantage for the company as iPSC cells are stem cells that can become almost any cell type. They are grown from the same cell instead of a patients donated cells. This means that one engineered cell line can be manufactured for many patients, creating what is known as off the shelf immunotherapy.

If the development of this type of therapy is successful, this would reduce the cost of manufacturing and provide a potential cash cow for the company. FATE has entered into collaborations with other companies for fund and research expertise. It is currently working with Ono Pharmaceutical (OTCMKTS:OPHLY) for two off-the-shelf iPSC-derived CAR T-cell product candidates, and Janssen Biotech develop iPSC-derived CAR NK and CAR T-cell product candidates.

FATE is rated a Strong Buy in our POWR Ratings system. It holds a grade of A in Trade Grade, Buy & Hold Grade, and Peer Grade. It is also ranked #24 out of 377 stocks in the Biotech industry. The stock is up a whopping 145.3% after finishing the day up 6.7%.

Blueprint Medicines (BPMC)

BPMC is a biopharmaceutical company focused on improving patients lives with diseases driven by abnormal kinase activation. The company has developed a small molecule drug pipeline in cancer. The firms lead product, Ayvakit, was approved by the FDA in January to treat metastatic gastrointestinal stromal tumor. The drug generated $5.7 million in the second quarter, so its off to a good start.

The company is also looking to expand its label as it is being studied for advanced and smoldering forms of systemic mastocytosis, a condition where certain immune cells, called mast cells, build up under the skin and, or in the bones, intestines, and other organs. If approved for other labels, that should help drive further growth.

Last month, the FDA approved the companys second drug, Gavreto, for the treatment of RET fusion-positive NSCLC or non-small lung cancer. BPMC worked on the drug with Roche (OTCMKTS:RHHBY). Lung cancer is responsible for more cancer deaths than any other in men and women. If Gavreto can become a standard treatment, it could become a goldmine for the company. The drug can also treat medullary thyroid cancers.

BPMC is rated a Strong Buy in our POWR Ratings system. It has grades of A in Trade Grade, Buy & Hold Grade, and Peer Grade. It is also the #9 ranked stock in the Biotech industry. The stock is up over 27% for the past three months and 8.5% over the past week.

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SGEN shares were unchanged in after-hours trading Friday. Year-to-date, SGEN has gained 75.72%, versus a 9.31% rise in the benchmark S&P 500 index during the same period.

David Cohne has 20 years of experience as an investment analyst and writer. Prior to StockNews, David spent eleven years as a Consultant providing outsourced investment research and content to financial services companies, hedge funds, and online publications. David enjoys researching and writing about stocks and the markets. He takes a fundamental quantitative approach in evaluating stocks for readers. More...

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SGEN: The 3 Top Biotech Stocks That Hedge Fund Managers LOVE - StockNews.com

Skin Stem Cells Comments Off on SGEN: The 3 Top Biotech Stocks That Hedge Fund Managers LOVE – StockNews.com | October 10th, 2020

Find out what the pros had to say.

You never forget your first. Grey hair, that is. Maybe you pluck it? Heck, its just oneout of sight, out of mind, right? Yeah, we know, you pluck one and five more grow back in its place, and then those five turn into tenyou get the idea. Nothing that a good dye job cant fix. That is, until a pandemic forces you into quarantine with no access to your colorist, and it becomes brutally apparent just how grey your hair actually is these days. This standoff between my stealthy foe and I got me thinking: We know we cant reverse grey hair, but can we slow its progression or delay its initial onset? I called on the pros to find out, but first, some basic hair biology.

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A protein called melanin is responsible for the color of our hair, and the cells in the hair follicles that make melanin are called melanocytes. Melanocytes are made by stem cells in the root of the hair, says Dr. Morgan Rabach, board-certified dermatologist, assistant professor at Mount Sinai School of Medicine, and co-founder of LM Medical NYC, and over time the stem cells die out and stop making melanocytes, leaving us with no cells to color the hair.

The age at which we start to go grey varies and depends on many factors. Dr. Caroline Robinson, MD, FAAD, and dermatologist and founder of Tone Dermatology, believes there is likely a genetic tendency at play in most cases of greying hair, but, like many genetic tendencies, there are environmental factors that influence how these changes show up in each of us. Dr. Rabach concurs, adding that grey hair is a combination of genetics and lifestyle.

Premature greying is generally considered less than 20 years old and it is thought to be an inherited predisposition. However, premature greying can also be attributed to certain illnesses and deficiencies. Dr. Robinson notes the importance of annual physicals and doctor visits when it comes to premature greying, as it could be an early sign of metabolic abnormalities in a select population.

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Search results for grey hair yield a slew of information centered around the notion that going grey is a result of an accumulation of hydrogen peroxide in the follicle, and that it could be eradicated by a topical pseudocatalase cream. This claim seems to have originated from a segment of research on vitiligo, which then appeared to have been manipulated for headlines. Dr. Robinson weighed in with her thoughts on the claim, saying, There are no double-blinded, controlled clinical studies to support the use of topical pseudocatalase-based creams for loss of pigmentation in hair.

Similarly, some products have appeared on the market claiming to re-pigment the hair, but the consensus among experts is that the claims are unfounded. I havent seen any convincing evidence for products that claim to reverse or prevent greying of the hair, says Holden.

We know all too well the havoc that stress wreaks on our overall health, but the type of stress were referring to here is oxidative stressthe imbalance of free radicals and antioxidants in the body, which leads to cell and tissue damage. In oxidative stress, free radicals damage our cells and tissues when our body doesnt have enough antioxidants to combat them, and in our hair, this oxidative stress can damage the cells that produce melanin, says Kate Holden, consultant trichologist.

The pros agree that oxidative stress plays a key role in the loss of hair pigmentation. From recent research we know that oxidative stress, the same type of stress that our skin faces in response to UV rays and pollution, can be an important factor in the loss of hair pigmentation, says Dr. Robinson. While oxidative stress occurs naturally in our bodies, environmental factors can increase its effects, such as alcohol, smoking, sugar and processed foods, cortisol levels, etc.

In addition, a recent study conducted by a group of Harvard researchers looked at the impact of stress (like, say, the kind you feel during a worldwide pandemic) on pigment-producing cells and found that the hyper-activation of the sympathetic nerves caused the depletion of melanocyte stem cells. Trichologist Leata A. Williams explains, When we are under stress, our bodies signal the fight-or-flight response, and it is the nerves that send the response to our hair follicles, causing the hair to grey.

Just think of how many world leaders have gone grey while in officethats the sympathetic nervous system depleting their hair follicles of melanocytes. And although most of us will never feel the stress of running a country, were still susceptible to the same greying effect from our everyday lives.

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Unfortunately for us, the pros agree that there is no real evidence to suggest that we can turn back the clock and re-pigment or slow the greying of hair in any tangible way. What we can do, however, is engage in healthy lifestyle behaviors that promote our overall health, and increase our intake of antioxidants. Increasing antioxidant intake through foods in our diet (leafy veggies, fruit) has not directly been shown to impact greying hair, but it can help to decrease overall oxidative stress levels in the body, says Dr. Robinson, which is something she discusses with her patients.

Dr. Rabach agrees and adds that she believes that a stress-free and healthy lifestyle makes your whole body healthier, and this would be reflected in the youthfulness of your hair. While we may not be able to reverse or halt grey hairs from popping up, what is encouraging is Dr. Rabachs belief that through good hair health, nutritious diet, and stress management, we might be able to delay their initial appearance. Increasing your antioxidant intake, whether it be from your diet, a multivitamin, or even a product formulated for hair health, will be beneficial in reducing oxidative stress and may help delay the initial onset of grey hair, says Dr. Rabach, who also encourages the use of hair products with antioxidants.

According to Dr. Robinson, there are some reports that Platelet Rich Plasma (PRP) therapy an in-office procedure that involves scalp injection of a processed form of ones own bloodcan promote hair re-pigmentation or slow greying because of its ability to deliver growth factors to the the hair follicle. While it sounds promising, more data needs to be gathered to determine its efficacy as a hair treatment.

Bottom line: Dont waste your money on products that claim to turn back the clock on grey hair, and focus instead on increasing your antioxidant intake and mitigating the effects of oxidative stress. Here are a couple products to get you started.

$17

Dr. Rabach promotes the use of antioxidant-rich hair products to help protect against the effects of oxidative stress. Look for one that is formulated with powerful superfruits like goji, acai, and pomegranate, and includes biotin for the added hair-nurturing benefits.

Buy

$65

This ingestible powder is chock-full of antioxidant-rich superfoods, adaptogens, and a probiotic blend to help your body resist stress, support detoxification, and support immunity. Add a teaspoon to water or a cold beverage of your choice, and reap the benefits of this all-natural, whole-food blend and help fend off the effects of oxidative stress.

Buy

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Gray Hair: What You Need to Know about Causes and Possible Prevention - Coveteur

Skin Stem Cells Comments Off on Gray Hair: What You Need to Know about Causes and Possible Prevention – Coveteur | October 9th, 2020

A Yes vote authorizes the state to sell $5.5 billion in general obligation bonds primarily for stem cell research and the development of new medical treatments in California. A No vote would mean the state's stem cell research agency will probably shut down by 2023.

In the ramp-up to the 2004 election, a California TV viewer may have come across the popular actor Michael J. Fox urging her to vote Yes on a state proposition. His voice slurred faintly by Parkinsons disease, he still sounded wry, boyish and familiar.

My most important role lately is as an advocate for patients and for finding new cures for diseases, said Fox, eyes level with the camera. Californias Stem Cell Research Initiative 71 will support research to find cures for diseases that affect millions of people, including cancer, diabetes, Alzheimer's and Parkinson's.

Within that 30-second spot, Fox, diagnosed at age 29 with a neurodegenerative disorder that typically does not strike until after 60, used the word "cures" three times.

Proposition 71, which passed with 59% of the vote, authorized the sale of $3 billion in bonds to create an agency that funded stem cell research. The successful campaign grew out of a time, in the early 2000s, when the promise of stem cell and regenerative medicine excited both scientists and the public.

Whether the project has lived up to that promise is a matter of opinion. How voters view the record of the agency may go a long way in their decision whether or not to replenish the fund, which is fast running out of money, with an additional $5.5 billion to be raised with new bonds authorized by Proposition 14, now on the ballot.

President Bush A Demon to Attack

Scientists since the1800s have known about stem cells, which are not yet dedicated to any particular anatomical function and have the potential to become nerve cells, blood cells, skin cells or any other type. They are found in blastocysts, which are human embryos four to five days after fertilization, and in a few areas, such as bone marrow and gonads, in adults.

In the late 1990s, researchers developed ways to steer the development of these cells, and the possibilities for improving medicine seemed endless. If malfunctioning cells were at the root of a particular disease, could new healthy cells tailored to the job fix what was wrong? Scientists and many members of the public were eager to find out.

Anti-abortion groups, however, a key constituency of President George W. Bush, opposed the research, and in 2001 he limited federal funding to a few existing lines of embryonic stem cells, severely curtailing research.

Some in the state of California wanted to get around Bushs restrictions, and Proposition 71 was born.

"(T)hey had this demon they could attack in the campaign the Bush administration," said David Jensen, author of "California's Great Stem Cell Experiment," who also writes the blog California Stem Cell Report. "They could say, 'This is a great opportunity, and the only way we're going to get it done is to do it here in California.'"

The measure created the California Institute for Regenerative Medicine. The stem cell research agency is unique in the U.S.

"No other state has done this kind of level of funding and focus on this kind of thing, said Jensen. It's a really cutting-edge area of science."

A Few Successes

The pace of innovation has been slower than many hoped. As it turned out, grand discoveries were not around the corner, and to date there is no widespread stem cell treatment approved for the public. To date, CIRM has funded more than 64 trials directly and aided in 31 more. Not all have or will result in treatments.

But despite the lack of a marquee cure like one for Alzheimers or Parkinsons, the agency has seen some notable triumphs.

"Probably one of the most spectacular successes they have certainly so far," said Jensen, "is clinical trials that have saved the lives of what they say are 40 children."

Those children were born with severe combined immunodeficiency (SCID), commonly known as "bubble baby syndrome," a rare, generally fatal condition in which a child is born without a working immune system. An FDA-approved gene therapy that grew out of CIRM-funded research can now cure the disease by taking a patients own blood stem cells and modifying them to correct the SCID mutation. The altered cells generate new, healthy blood cells and repair the immune system.

The FDA has also approved two drugs for rare blood cancers that were developed with CIRM funds.

Sandra Dillon, a graphic designer in San Diego, credits one of the drugs with saving her life. She was diagnosed when she was just 28, in 2006. Her doctors told her they would try to manage her symptoms, but that she was going to get progressively sicker.

"Even just the idea of a cure or getting better wasn't even on the table back then," said Dillon, who is featured in ads for the Yes on 14 campaign.

"I remember just praying and begging into the universe, please, someone just look at my disease, please someone help, who is going to look at this thing.

By 2010, Dillon was extremely ill. She connected with a doctor at UC San Diego who received early-stage funding from CIRM and told her she could take part in clinical trials.

"For the first time, there was this moment of, 'Oh, my gosh! There are researchers doing something. And it could help me and I can get access to it.' It was amazing."

The drug received FDA approval in 2019, and today Dillons cancer has retreated to the point where she can live a normal life.

"I love that I am not tethered to a hospital anymore. I can go out on long backpacking trips and hiking and surfing," she said. "I am a completely different person with this drug. And I have a whole future ahead of me."

The original funding raised by Proposition 71 is running out. Proposition 14 would authorize the sale of a new bond to refill the agency piggy bank. Gov. Gavin Newsom, the UC Board of Regents, and scores of patient advocacy groups also support the measure.

Many newspaper editorial boards, however, oppose the proposition, including the San Francisco Chronicle, Mercury News and Los Angeles Times.

Right now the state still owes about $1 billion toward the debt created by Proposition 71. If Proposition 14 passes, the yearly price tag to pay off the new bond would be about $260 million per year for about 30 years.

One of the selling points of the original proposition was the potential for the state to earn big money in royalties from the treatments it helped develop, says Jeff Sheehy, an HIV patient advocate and the only CIRM board member to oppose Proposition 14.

"The promises were made that this would pay for itself. We would be able to pay back the bonds with the money we would get from royalties, etc., etc.

That has not worked out as envisioned: CIRM estimates it has received less than $500,000 in royalties. Early this year, Forty Seven, a company whose therapies were heavily funded by CIRM, sold to Gilead for $4.9 billion. While millions went to various researchers, neither CIRM nor the state of California received anything.

One of the flaws in the original measure is that we [the agency] cannot hold stock in the products that we develop," says Sheehy. "And that's because the California Constitution says that the state of California cannot, as a government entity, hold equity.

Proposition 14 makes it impossible for the state to use profits from its investment on, say, schools or other funding priorities. Instead, any royalties earned must be fed back into programs to make CIRM-funded treatments more affordable.

"What it does is it basically takes all of our returns that we get from this and gives it back to the pharmaceutical and biotech companies," said Sheehy. "It becomes just a blatant giveaway to these companies when we should be requiring access and requiring fair pricing."

Sheehy says he supports medical research, but doesn't like the state going into more debt to pay for it. The greater the state's obligations in bond money, which has to be paid back with interest, the less there is in the general fund, and Sheehy says the state has more pressing needs than stem cell research things like housing, education and transportation.

"The biggest and perhaps the most compelling reason why I feel so strongly that this is not a good idea is that we simply cannot afford it, he said. "If we think this is so important," asks Sheehy, "why don't we just don't pay for [this research] out of the general fund? It would be cheaper.

Opponents of Proposition 14 also point to longstanding complaints of conflicts of interest among the agency board. Most of the $3 billion distributed by the agency has gone to institutions with connections to board members. Critics say the structural conflicts of interest between the board and agency are not addressed in the new measure. Proposition 14 would balloon an already huge board of 29 members to 35.

Funding needs for stem cell research also are not as acute as they were back in 2004. The federal National Institutes of Health now funds some basic stem cell research, spending about $2 billion a year, with $321 million of that going toward human embryonic stem cell research. And private ventures, like nonprofits started by tech billionaires, are pouring more money into biotech.

The problem with assuming that, says Melissa King, executive director of Americans for Cures, the stem cell advocacy group behind the Yes on 14 campaign, is that CIRM fills a neglected funding need.

The NIH does not fund clinical trials at nearly the rate that CIRM can and has been, King said.

She says that's important because of what she calls the "Valley of Death," where promising early-stage research frequently fails to translate into promising treatments that can be tested in clinical-stage research. (What works well in a test tube often does not work well in an organism.) This weeding-out process is costly but necessary. And its where CIRM focused a lot of its effort.

The first- and maybe even second-phase clinical trials, its very difficult to get those funded, King said. It is too much of a risk for business to take on on its own. Venture [capital] isnt going there. Angel [funding] isnt going there.

What voters have to ask themselves, says writer Jensen, is whether stem cell funding is "a high priority for the state of California? Different people make different judgments about that."

CIRM supporters say if Prop. 14 doesn't pass, critical research will stall. Others say federal and private funding will step in and fill the gap.

Absent new funding, the institute expects it will wind down operations leading to a complete sundown in 2023.

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Proposition 14: With Just Handful of Cures, California Stem Cell Agency's Fate Is In Hands of Voters - KQED

Bone Marrow Stem Cells Comments Off on Proposition 14: With Just Handful of Cures, California Stem Cell Agency’s Fate Is In Hands of Voters – KQED | October 9th, 2020

DetailsCategory: DNA RNA and CellsPublished on Thursday, 08 October 2020 15:15Hits: 339

NEW YORK, NY, USA and PARIS, France I October 08, 2020 I Cytovia Therapeutics ("Cytovia"), an emerging biopharmaceutical company, announces today that it has entered a research and licensing agreement with Inserm to develop NK engager bi-specific antibodies and iPSC CAR NK cell therapy targeting CD38, a key marker of multiple myeloma. The licensing agreement has been negotiated and signed by Inserm Transfert, the private subsidiary of Inserm, on behalf of Inserm (the French National Institute of Health and Medical Research) and its academic partners. Cytovia is licensing Inserm's CD38 antibody and Chimeric Antigen Receptor (CAR) patent and applying its proprietary NK engager bispecific antibody and iPSC CAR NK technology platforms. The research agreement will include evaluation of the therapeutic candidates at Hpital Saint-Louis Research Institute (Inserm Unit 976) under the leadership of Professors Armand Bensussan and Jean-Christophe Bories.

Dr Daniel Teper, Cytovia's Chairman and CEO commented: "We are delighted to partner with one of the top centers of excellence in the world for research and treatment in hematology. CD38 is a validated target and Natural Killer cells have significant cytotoxicity to Myeloma cells. We are looking forward to bringing promising new options to address the unmet needs of patients with Multiple Myeloma and aim for a cure."

Professor Armand Bensussan, Director of The Immuno-Oncology Research Institute at Hpital Saint-Louis added: "We have demonstrated the selectivity of our novel CD38 antibody in killing myeloma cells but not normal cells such as NK, T, and B cells. The activation of NK cells through NKp46 may enhance the efficacy of the bispecific antibody in patients not responsive to CD38 monoclonal antibody therapy. CD38 CAR NK is a promising approach forrelapsed/refractory patients and an alternative to CAR T therapies."

About Multiple MyelomaMultiple Myeloma is a currently incurable cancer, affecting a type of white blood cell known as plasma cells. It leads to an accumulation of tumor cells in the bone marrow, rapidly outnumbering healthy blood cells. Instead of producing beneficial antibodies, cancerous cells release abnormal proteins causing several complications. While symptoms are not always present, the majority of patients are diagnosed due to symptoms such as bone pain or fracture, low red blood cell counts, fatigue, high calcium levels, kidney problems, and infections. According to the World Cancer Research Fund, Multiple Myeloma is the second most common blood cancer, with nearly 160,000 new annual cases worldwide, including close to 50,000 in Europe. 32,000 in the US, and 30,000 in Eastern Asia. Over 95% of cases are diagnosed late, with a 5-year survival rate of 51%. Initial treatment comprises of a combination of different therapies, including biological and targeted therapies, corticosteroids, and chemotherapy, with the option for bone marrow transplants for eligible patients. Immunotherapy and cell therapy are the most promising new treatment option for Multiple Myeloma, with the potential for long term cancer remission.

About CAR NK cellsChimeric Antigen Receptors (CAR) are fusion proteins that combine an extracellular antigen recognition domain with an intracellular co-stimulatory signaling domain. Natural Killer (NK) cells are modified genetically to allow insertion of a CAR. CAR-NK cell therapy has demonstrated initial clinical relevance without the limitations of CAR-T, such as Cytokine Release Syndrome, neurotoxicity or Graft vs Host Disease (GVHD). Induced Pluripotent Stem Cells (iPSC) - derived CAR-NKs are naturally allogeneic, available off-the-shelf and may be able to be administered on an outpatient basis. Recent innovative developments with the iPSC, an innovative technology, allow large quantities of homogeneous genetically modified CAR NK cells to be produced from a master cell bank, and thus hold promise to expand access to cell therapy for many patients.

About CytoviaCytovia Therapeutics Inc is an emerging biotechnology company that aims to accelerate patient access to transformational immunotherapies, addressing several of the most challenging unmet medical needs in cancer and severe acute infectious diseases. Cytovia focuses on Natural Killer (NK) cell biology and is leveraging multiple advanced patented technologies, including an induced pluripotent stem cell (iPSC) platform for CAR (Chimeric Antigen Receptors) NK cell therapy, next-generation precision gene-editing to enhance targeting of NK cells, and NK engager multi-functional antibodies. Our initial product portfolio focuses on both hematological malignancies such as multiple myeloma and solid tumors including hepatocellular carcinoma and glioblastoma. The company partners with the University of California San Francisco (UCSF), the New York Stem Cell Foundation (NYSCF), the Hebrew University of Jerusalem, and CytoImmune Therapeutics. Learn more at http://www.cytoviatx.com

About InsermFounded in 1964, the French National Institute of Health and Medical Research (Inserm) is a public science and technology institute, jointly supervised by the French Ministry of National Education, Higher Education and Research, and the Ministry of Social Affairs, Health and Womens Rights. Inserm is the only French public research institute to focus entirely on human health and position itself on the pathway from the research laboratory to the patients bedside. The mission of its scientists is to study all diseases, from the most common to the rarest. With an initial 2020 budget of 927.28 million, Inserm supports nearly 350 laboratories throughout France, with a team of nearly 14,000 researchers, engineers, technicians, and post-doctoral students. http://www.inserm.fr

SOURCE: Cytovia Therapeutics

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Cytovia Therapeutics partners with Inserm to develop selective CD38 NK therapeutics and offer new treatment options for Multiple Myeloma patients |...

Bone Marrow Stem Cells Comments Off on Cytovia Therapeutics partners with Inserm to develop selective CD38 NK therapeutics and offer new treatment options for Multiple Myeloma patients |… | October 9th, 2020

There are only a few specialised childhood cancer centres in Southern Africa.

Leukaemia and lymphoma are two of the most prevalent cancers in children in South Africa with between 800 and 1000 children diagnosed annually. Tragically, it is estimated that half of the children with cancer in this country are never diagnosed.

Dr Marion Morkel, Chief Medical Officer at Sanlam, believes that we all need to educate ourselves so we can recognise the symptoms of cancer.

Below, Dr Morkel explains what can be done in the fight against leukaemia and lymphoma.

Knowledge is key

You must be aware of the symptoms related to leukaemia and lymphoma so that you can notify your health professional should you see these symptoms in your child.

Leukaemia

Leukaemia is the most common childhood cancer accounting for 25% of all cases in South Africa.

Symptoms include:

Lymphoma

Lymphoma primarily originates from the lymph nodes and can often appear like any other illness that triggers an inflammatory response.

Symptoms to look out for include:

While other childhood illnesses can present in the same manner as leukaemia and lymphoma, health professionals have been trained to look out for symptoms that persist after routine treatment and will conduct tests to rule out the possibility of these childhood blood-related cancers.

Parents are encouraged to consult their doctor if there are any concerns about their childs health.

ALSO READ|Should I be worried if my child has pain in his tummy?

Register to become a blood stem cell (bone marrow) donor

The Sunflower Fund is a non-profit organisation that fights blood diseases through a blood stem cell transplant which replaces a persons defective stem cells with healthy ones and can be a potentially life-saving treatment for more than 70 different diseases.

Kim Webster, Head of Communications at The Sunflower Fund advises that finding a matching donor for a stem cell transplant is not as easy as finding a blood type match.

There is only a 1:100 000 chance of a patient finding their life-saving match with siblings only having a 25% chance of a match.

You can register to become a donor online via http://www.sunflowerfund.org.

Submitted to Parent24 by Atmosphere

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Knowledge is key: What you need to know about the most common childhood cancer in SA - News24

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The study methodologies used to examine Hematopoietic Stem Cell Transplantation (HSCT) Market for the forecast period, 2020 - 2027 further classifies the industry by type, geography, end-use and end-user to add more precision and bring to light factors responsible for augmenting business development.

This press release was orginally distributed by SBWire

New York, NY -- (SBWIRE) -- 10/08/2020 -- Reports and Data has recently published a research report on the Global Hematopoietic Stem Cell Transplantation (HSCT) Market, expanding its extensive database. The research study offers a detailed assessment of the current and emerging market trends and offers a holistic overview of the sector considering product portfolio, types, segmentation, applications, and supply chain analysis. It also provides a comprehensive analysis of the international market, growth trends, development patterns, competitive landscape, demand and supply dynamics, and gross margins.

Additionally, the report examines the impact of the COVID-19 crisis on the Hematopoietic Stem Cell Transplantation (HSCT) market and its key segments. The report assesses the changing market dynamics, demands, and trends of the Hematopoietic Stem Cell Transplantation (HSCT) industry with regards to the COVID-19 pandemic. The report furthermore presents a present and future impact analysis of the crisis on the Hematopoietic Stem Cell Transplantation (HSCT) market and offers a futuristic outlook with regards to trends and demands patterns in a post-COVID-19 scenario.

Get a Sample of the Report @ https://www.reportsanddata.com/sample-enquiry-form/3627

The Global Hematopoietic Stem Cell Transplantation (HSCT) Market research report provides a complete outlook on the challenges existing in the industry and also discusses the emerging threats, constraints, and limitations. The report is an investigative study that offers an extensive breakdown of the market dynamics such as drivers, growth prospects, product portfolio, technological advancements, and an extensive analysis of the key competitors of the market. The global Hematopoietic Stem Cell Transplantation (HSCT) market is further segmented into types, applications, technology, end-users, industry verticals, and key geographies across the world. Furthermore, the report offers a forecast estimation of the valuation of the Hematopoietic Stem Cell Transplantation (HSCT) market.

Leading companies profiled in the report are China Cord Blood Corp, Pluristem Therapeutics Inc., CBR Systems Inc CellGenix Technologie Transfer GmbH, Cryo-Save AG Kite Pharma Inc., Regen Biopharma Inc., ViaCord Inc., BiolineRx, Cynata Therapeutics, Cesca Therapeutics Inc, Lonza Group Ltd, TiGenix N.V., Bluebird Bio, Cellular Dynamics International, and Escape Therapeutics Inc., among others.

Type Outlook (Revenue in USD Million; 2017-2027)

AllogeneicAutologous

Indication Outlook (Revenue in USD Million; 2017-2027)

LeukemiaLymphatic disorderMyelomaOther non-malignant disorders

Application Outlook (Revenue in USD Million; 2017-2027)

Peripheral blood cellsBone marrowUmbilical cord blood

The report presents a detailed study of the Hematopoietic Stem Cell Transplantation (HSCT) industry through data gathered by thorough primary and secondary research. The data formulated is verified and validated by industry experts and professionals. Additionally, the report utilizes advanced analytical tools such as SWOT analysis, Porter's Five Forces analysis along with feasibility analysis and investment return analysis. The report provides a comprehensive analysis of the prominent players of the market with a detailed analysis of their company overview, product portfolio, production and manufacturing capacity, technological and product developments, and revenue estimations. The report further examines key statistical data and facts pertaining to the Global Hematopoietic Stem Cell Transplantation (HSCT) market. The report further aims to provide a competitive advantage to the readers, clients, consumers, and market professionals engaged in the industry.

Browse Full Report @ https://www.reportsanddata.com/report-detail/hematopoietic-stem-cell-transplantation-hsct-market

Key Geographies Mapped in the Report are:

North America (U.S, Canada, and Rest of North America)Europe (Germany, France, Italy, and Rest of Europe)Asia Pacific (China, Japan, India, South Korea, and Rest of Asia-Pacific)Latin America (Brazil, Argentina, and Rest of Latin America)Middle East & Africa (South Africa, Saudi Arabia, U.A.E, and Rest of MEA

The report offers:

An in-depth overview of the Hematopoietic Stem Cell Transplantation (HSCT) market landscapeAssessment of the global industry trends for the historical period of 2017-2018, the current year 2019-2020, and a forecast estimation for the period 2020-2027Overview of the company profiles and product portfoliosR&D advancements and technological developments in the Hematopoietic Stem Cell Transplantation (HSCT) industry.Market dynamics, trends, opportunities, and risksStudy of the market in terms of revenue and product consumption patterns.

Additionally, the report offers historical analysis and forecast analysis for the global Hematopoietic Stem Cell Transplantation (HSCT) market.

Historical Years: 2017-2018

Base Year: 2019

Estimated Year: 2020

Forecast Years: 2020-2027

The report answers radical questions about the global Hematopoietic Stem Cell Transplantation (HSCT) market. It aims to offer a competitive edge to the reader by providing insightful data about strategic alliances such as mergers and acquisitions, joint ventures, collaborations, partnerships, agreements, government deals, and product launches.

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Thank you for reading our report. For further inquiries or queries regarding customization, kindly connect with us.

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Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis of Key Vendors with Their Size, Share and Year-over-Year Growth 2027 - Press Release -...

Bone Marrow Stem Cells Comments Off on Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis of Key Vendors with Their Size, Share and Year-over-Year Growth 2027 – Press Release -… | October 9th, 2020

Grady Smith, 10, and Southfield resident Jessica Carroll were able to meet for the first time via Zoom in September at the DKMS Gala. Carroll was the bone marrow donor that helped save the boys life.

Photo provided by the Smith family

SOUTHFIELD If you were to take one look at 10-year-old Grady Smith, youd see a young boy who enjoys sports and school.

But the young Salem, New Hampshire, boy has been through more in 10 years than some people have in 50.

Grady was diagnosed with adrenoleukodystrophy, or ALD, back in 2018.

According to Boston Childrens Hospital, ALD is a rare genetic condition that causes the buildup of very long chain fatty acids in the brain. When the fatty acids accumulate, they destroy the protective myelin sheath around nerve cells, responsible for brain function. Without the myelin sheath, the nerves can no longer relay information to and from the brain.

Every single thing I read said, terminal, slow deterioration to death, one to five years, horrible, horrible death, Jillian Smith said. We just died. I havent been the same person since that day. It just changes you for the rest of your life.

With a diagnosis, Grady and his parents looked for options on how to help. Grady had a lesion with a Loes score which is a way of rating severity of 10. Scores range from 0-34.

His parents werent sure Grady would qualify for a bone marrow transplant because they usually only perform transplants for boys with scores of 9 and under. Grady was in luck, however, as Boston Childrens Hospital decided to move forward anyway.

The next move was to find a match for the boy, but that process could take weeks, months or even years. In Gradys case, it took just a few weeks.

Southfield resident Jessica Carroll registered as a potential bone marrow/blood stem cell donor with DKMS, a German bone marrow donor file, in 2014, but she didnt think much would come of it.

Four years later she got a call from the nonprofit organization letting her know that she was a match for a young boy. After some research, Carroll was totally on board with donating.

It was great knowing during that donation that this little bit that I went through was potentially saving somebodys life, Carroll said. Thats all I really cared about, was that I was helping somebody.

Grady was able to get his transplant in 2018.

According to his mother, Grady hasnt had any progression and has even made some recovery. Hes back in line with his academics and is playing sports again.

Grady has auditory processing issues, which make it hard for him to comprehend language and sound. His mother said he relies on reading lips to communicate.

Theres still a lot to it. It stopped the monster thats how we look at it but its not just so cut and dry, Jillian said. Hes a very rare outcome with his Loes score and with just how well hes doing. Hes just a really, really good boy. He works really hard to help bring awareness.

Carroll and the Smiths have talked via text, and they were able to meet virtually for the first time in September at the DKMS Gala.

For the Smiths and Carroll, the meeting was emotional. Grady was finally able to put a face to his donor, and vice versa for Carroll.

It was of course emotional, Carroll said. Being able to hear everything they went through, though, definitely made me so happy that I had chosen to register.

The Smiths and Carroll still talk periodically throughout the year, and Sept. 20 was the two-year anniversary of the transplant.

They are hoping to be able to meet in person soon, and the DKMS team wants to bring them to next years gala to help make that happen. However, they hope it will be sooner.

Throughout this journey with Grady, the Smiths have advocated, learned and spoken more about ALD.

Prior to Gradys birth, Massachusetts wasnt testing for ALD in newborns, but it has since started. New Hampshire wasnt either, but the Smiths got the state to add ALD to the newborn screening panel.

The next goal is to get more states to add the ALD screening. The family has also spoken at conferences to share Gradys story and have become big proponents of what DKMS has been able to do for not only their family, but families around the world.

I think a big thing, too, that we really want to get out there is bone marrow transplant or stem cell transplant, how easy it is, Jillian said. All people need to do is go on DKMSs website, and they can get a packet sent out to them. They just swab their cheeks, send it in and they could be saving anyones life, someone just like Grady.

According to the DKMS website, the organization is dedicated to the fight against blood cancer and blood disorders by creating awareness, recruiting bone marrow donors to provide a second chance at life, raising funds to match donor registration costs and supporting the improvement of blood cancer therapies by our own research.

Those looking for more information or wanting to register can visit dkms.org/en or call (212) 209-6700.

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Southfield woman meets boy she saved with bone marrow donation - C&G Newspapers

Bone Marrow Stem Cells Comments Off on Southfield woman meets boy she saved with bone marrow donation – C&G Newspapers | October 7th, 2020

Emmanuelle Charpentier and Jennifer Doudna have scooped the 2020 Nobel prize in chemistry for the development of a method for genome editing. Specifically, theyve been awarded the prize for their discovery of the CrisprCas9 genome editing technique that allows scientists to make precise alterations to the genetic code of living organisms. CrisprCas9 is a powerful tool that could revolutionise many aspects of our lives, from medical treatments to the way we produce food. Its also seen its fair share of controversy in recent years. Here, we take a deeper look at these genetic scissors and why theyve won the Nobel prize.

Since Charpentier and Doudna began investigating the CrisprCas9 system in 2011, the field has exploded. Due to the relative simplicity and affordability of Crispr systems, researchers around the world have been able to apply the tools to all manner of different problems. Today there are entire journals, conferences and companies dedicated to the technique.

The ability to cut any DNA molecule at a chosen site has huge potential from treating genetic illnesses to creating disease-resistant crops. Trials have even shown how Crispr-delivered genetic modifications can spread through populations of mosquitoes and stop malaria infections such gene-drives offer a way to eliminate the disease altogether. And in the face of the Covid-19 pandemic, researchers have found ways to use Crispr in rapid coronavirus diagnostic tests and have also proposed using it to attack the viruss genome.

As Claes Gustafsson, chair of the Nobel committee for chemistry, said at the award announcement, There is enormous power in this genetic tool, which affects us all.

Crispr technology has even been used to make more delicious beer.

The whole Crispr gene editing tool has been adapted from the immune system of bacteria. The term Crispr comes from clustered regularly interspaced short palindromic repeats, which refers to distinct genetic sequences found in the genomes of bacteria. Each Crispr sequence is transcribed into RNA sequences that will target the DNA of a virus. These sequences also include cas (Crispr-associated) genes that code for DNA-cutting Cas enzymes. Together, the guide RNA and Cas enzyme form a complex that hunts out viral DNA and chops it up.

In Crispr gene editing, scientists repurpose this system by designing a guide RNA sequence of around 20 nucleobases that matches up to a DNA sequence they wish to target in a cells genome. This RNA sequence is paired with the Cas9 enzyme that will cut the DNA strand at the targeted site. The whole DNA sequence coding for both these components of the Crispr-Cas9 tool can be delivered to the target cell via a plasmid.

The tool can therefore be used to edit a cells genome with incredible precision for example, it can cut out a dysfunctional gene associated with a hereditary illness. And if the healthy version of the gene is also delivered to the cell, the cells own repair system will then incorporate the healthy strands at the site where it has been cleaved.

In 2011, when investigating the bacteria Streptococcus pyogenes, Charpentier discovered a molecule called tracrRNA that forms a key part of the CrisprCas system in bacteria.

Meanwhile, Doudna had been investigated the function of the cas genes, and learned that the Cas proteins they code for are involved in cutting up DNA as part of the bacterial immune system against pathogenic viruses.

That year Charpentier teamed up with Doudna to investigate the system further. Together they revealed how the Cas9 protein, CrisprRNA and tracrRNA worked together to snip DNA strands into two parts. They then simplified the system by combining the CrisprRNA and tracrRNA into a single molecule guide RNA making it easier to use, and showed how this could be used to cut any DNA strand at a site of their choosing, opening the door to using the tool in all manner of genome editing experiments.

While previous tools for genetic editing existed before Crispr-Cas9, the new tools are much simpler and cheaper. This has led to the huge expansion of the field by making gene editing accessible for scientists all around the globe.

For years Crispr has been at the centre of a long-running patent dispute. Shortly after Doudna and Charpentiers discovery, Feng Zhangs team the Broad Institute in Cambridge, US, patented a way to use the technique in eukaryotic cells. There have been protracted court battles between Doudnas group at the University of California in Berkeley, US, and the Broad team over who holds the key piece of intellectual property. In the meantime, numerous groups and companies have been granted patents for many new Crispr-related technologies, meaning that as time goes on, the original patents at the centre of the dispute are becoming less relevant.

Another area of controversy surrounds the potential consequences of using genome editing tools at all. As the genome is so complex, we cant always know what will happen when we edit genes. Some genes have multiple and often unknown functions editing them to correct for one problem could end up creating new unforeseen ones. This is particularly important when it comes to editing germline cells (those that can be passed on to an organisms children), because the modified genes can be inherited by future generations.

As a relatively new technique, we also know that Crispr itself isnt perfect. Some studies have shown off-target cuts, where the tool has snipped DNA strands at additional locations to the desired site. This clearly can have harmful consequences, and so many researchers are looking into ways to improve the technique and make it more suitable for medical uses.

With these concerns in mind, scientists worldwide including Doudna and Charpentier have called for a moratorium on editing human germline cells, until we can know more about the consequences. Such calls intensified after the rogue Chinese scientist He Jiankui edited human embryos that were then brought to term in 2018. He is now serving a three year prison sentence for conducting the study.

Several clinical trials have already begun on Crispr-based therapies, with promising reports emerging this year. In February, the first study to look at a cancer treatment using Crispr-edited immune cells reported that the modified cells were safe, with no serious side-effects in the three patients studied. While the efficacy of the treatment on the cancers was minimal, it may help to inform future Crispr-based T-cell treatments.

One month later, a patient with hereditary blindness became the first person ever to have a CrisprCas9 therapy directly administered into their body. And in June, the Swiss gene-editing company Crispr Therapeutics announced that two patients with beta thalassaemia and one with sickle cell disease would no longer require blood transfusions after their bone marrow stem cells were edited using Crispr techniques.

Earlier this week, Doudna launched a new company, Scribe Therapeutics, to begin work on treatments for amyotrophic lateral sclerosis.

Other Crispr-based technologies are coming closer to commercial reality. For example, the US genome engineering company eGenesis is developing ways to use the technique to edit pigs genes so that their organs might be transplanted safely into humans. In the agricultural sector, many companies are working on ways to use Crispr to speed up the selection process for crops with desirable traits such as disease-resistance or improved flavour.

At the fundamental level, researchers are working on ways to improve the system itself. By using alternative Cas proteins, some groups hope to make the tool more effective and easier to use in certain settings. Doudnas group recently reported on a CasX protein that is smaller than Cas9 and potentially easier to introduce into target cells.

Delivering DNA into cells and tissues is an important part of gene therapy, even more so for Crispr-Cas9 approaches because plasmids carrying this system are very large. This research paper describes a non-viral vector for delivering plasmid DNA carrying Crispr-Cas9 into tumour spheroids, which are good in vitro models for tissues but also challenging transfecting targets.

1 S J Zamolo, T Darbre and J-L Reymond, Transfecting tissue models with CRISPR/Cas9 plasmid DNA using peptide dendrimers, Chem. Commun., 2020, DOI: 10.1039/d0cc04750c

Regulating the function of Crispr-Cas9 is on the agenda for many researchers because the ability to restrict it in a spatial and temporal manner opens the door to precisely manipulating genomes and minimising any side effects. By introducing photolabile groups into the system, these researchers have shown how they can regulate Cas9 activity with light

2 Y Wang et al, Photocontrol of CRISPR/Cas9 function by site-specific chemical modification of guide RNA,Chem. Sci., 2020, DOI: 10.1039/d0sc04343e

It seems that Crispr-Cas systems arent just handy for gene editing. This paper describes how the Crispr-Cas system was used to assemble a multi-enzyme cascade containing five distinct enzymes. The team behind the work hope it could be the beginnings of a general method for building complex scaffolded biocatalytic pathways

3 S Lim et al, CRISPR/Cas-directed programmable assembly of multi-enzyme complexes, Chem. Commun., 2020, 56, 4950 (DOI: 10.1039/d0cc01174f)

And to round things off, here are some reviews on how Crispr-Cas9 works, the delivery processes for therapeutic nanoparticles and the physiological obstacles for those process

4 Y Xu, R Liu and Z Dai, Key considerations in designing CRISPR/Cas9-carrying nanoparticles for therapeutic genome editing, Nanoscale, 2020, DOI: 10.1039/d0nr05452f

5 Y Gong et al,Lipid and polymer mediated CRISPR/Cas9 gene editing, J. Mater. Chem. B, 2020,8, 4369 (DOI: 10.1039/d0tb00207k)

Excerpt from:
Explainer: What is Crispr and why did it win the Nobel prize? - Chemistry World

Bone Marrow Stem Cells Comments Off on Explainer: What is Crispr and why did it win the Nobel prize? – Chemistry World | October 7th, 2020

Autologous Stem Cell Based Therapies Market Report Delivering Growth Analysis with Key Trends of Top Companies (2020-2026)

A comprehensive research study on the Autologous Stem Cell Based Therapies Marketwas recently published by Market Report Expert. This is an up-to-date report, covering the current COVID-19 impact on the market. The Coronavirus (COVID-19) has affected every aspect of life globally and thus altering the global market scenario. The changes in the market conditions are drastic. The swiftly changing market scenario and initial and future assessment of the impact on Autologous Stem Cell Based Therapies market is covered in the report.The Autologous Stem Cell Based Therapies Market report is a precise and deep-dive study on the current state that aims at the major drivers, market strategies, and imposing growth of the key players. Worldwide Autologous Stem Cell Based Therapies Industry also offers a granular study of the dynamics, segmentation, revenue, share forecasts, and allows you to make superior business decisions. The report serves imperative statistics on the market stature of the prominent manufacturers and is an important source of guidance and advice for companies and individuals involved in the Autologous Stem Cell Based Therapies industry.

The Global Autologous Stem Cell Based Therapies Market poised to grow from US$ XX million in 2020 to US$ XX million by 2026 at a compound annual growth rate (CAGR) of XX% during the projection period of 2020-2026.

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Regeneus, Mesoblast, Pluristem Therapeutics Inc, U.S. STEM CELL, INC., Brainstorm Cell Therapeutics, Tigenix, Med cell Europe

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Majortype, primarily split into

Embryonic Stem CellResident Cardiac Stem CellsUmbilical Cord Blood Stem Cells

Major applications/end users, including

Neurodegenerative DisordersAutoimmune DiseasesCardiovascular Diseases

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Autologous Stem Cell Based Therapies Market Size, Business Revenue Forecast, Leading Competitors And Growth Trends 2026| Regeneus, Mesoblast,...

Cardiac Stem Cells Comments Off on Autologous Stem Cell Based Therapies Market Size, Business Revenue Forecast, Leading Competitors And Growth Trends 2026| Regeneus, Mesoblast,… | October 7th, 2020

CRANFORD, N.J., Oct. 7, 2020 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company developing and commercializing critical care drug products, announced that it has signed an exclusive agreement with Novellus Therapeutics Limited ("Novellus") to license iPSC-derived mesenchymal stem cells (iMSCs), and has created a new subsidiary, NoveCite, that will be focused on developing cellular therapies.

NoveCite has a worldwide exclusive license from Novellus, an engineered cellular medicines company, to develop and commercialize NoveCite mesenchymal stem cells ("NC-iMSCs") to treat acute respiratory conditions with a near term focus on Acute Respiratory Distress Syndrome ("ARDS") associated with COVID-19. Several cell therapy companies using donor-derived MSC therapies in treating ARDS have demonstrated that MSCs reduce inflammation, enhance clearance of pathogens and stimulate tissue repair in the lungs. Almost all these positive results are from early clinical trials or under the emergency authorization program.

NC-iMSCs are the next generation mesenchymal stem cell therapy. They are believed to be differentiated and superior to donor-derived MSCs. Human donor-derived MSCs are sourced from human bone marrow, adipose tissue, placenta, umbilical tissue, etc. and have significant challenges (e.g., variable donor and tissue sources, limited supply, low potency, inefficient and expensive manufacturing). iMSCs overcome these challenges because they:

Globally, there are 3 million cases of ARDS every year out of which approximately 200,000 cases are in the United States. The COVID-19 pandemic has added significantly to the number of ARDS cases. Once the COVID patients advance to ARDS, they are put on mechanical ventilators. Death rate among patients on ventilators can be as high as 50% depending on associated co-morbidities. There are no approved treatments for ARDS, and the current standard of care only attempts to provide symptomatic relief.

"NoveCite iMSCs have the potential to be a breakthrough in the field of cellular therapy for acute respiratory conditions because of the high potency seen in Novellus' pre-clinical studies, and because iMSCs are iPSC-derived, and therefore overcome the manufacturing challenges associated with donor derived cells," said Myron Holubiak, Chief Executive Officer of Citius.

"We are excited to be part of this effort because of the promise to save lives and reduce long term sequelae in patients with devastating respiratory diseases such as ARDS caused by COVID-19," said Dr. Matthew Angel, Chief Science Officer of Novellus. "Our iMSC technology has multimodal immunomodulatory mechanisms of action that make it potentially promising therapy to treat acute respiratory diseases."

About Citius Pharmaceuticals, Inc.

Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Novellus, Therapeutics, Limited

Novellus is a pre-clinical stage biotechnology company developing engineered cellular medicines using its patented non-immunogenic mRNA high specificity gene editing, mutation-free & footprint-free cell reprogramming and serum insensitive mRNA lipid delivery technologies. Novellus is privately held and is headquartered in Cambridge, MA. For more information, please visit http://www.novellus-inc.com.

About NoveCite iMSC (NC-iMSC)

NoveCite's mesenchymal stem cell therapy product is derived from a human induced pluripotent stem cell (iPSC) line generated using a proprietary mRNA-based (non-viral) reprogramming process. The NC-iMSCs produced from this clonal technique are differentiated from human donor-derived MSCs (bone marrow, placenta, umbilical cord, adipose tissue, or dental pulp) by providing genetic homogeneity. In in-vitro studies, NC-iMSCs exhibit superior potency and high cell viability. NC-iMSCs secrete immunomodulatory proteins that may reduce or prevent pulmonary symptoms associated with acute respiratory distress syndrome (ARDS) in patients with COVID-19. NC-iMSC is an allogeneic (unrelated donor) mesenchymal stem-cell product manufactured by expanding material from a master cell bank.

First generation (human donor-derived) MSCs are isolated from donated tissue followed by "culture expansion". Since only a relatively small number of cells are isolated from each donation, first generation MSCs are increased by growing the cells in culture. Unfortunately, these type of MSCs start to lose potency, and ultimately become senescent. Each donation produces a limited number of MSCs, so a continuous supply of new donors is needed to produce commercial scale. The number and quality of MSCs that can be isolated from different donors can vary substantially.

About Acute Respiratory Distress Syndrome (ARDS)

ARDS is an inflammatory process leading to build-up of fluid in the lungs and respiratory failure. It can occur due to infection, trauma and inhalation of noxious substances. ARDS accounts for approximately 10% of all ICU admissions and almost 25% of patients requiring mechanical ventilation. Survivors of ARDS are often left with severe long-term illness and disability. ARDS is a frequent complication of patients with COVID-19. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

Safe Harbor

This press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: the risks associated with developing the NoveCite technology as a treatment for ARDS; risks associated with developing any of our product candidates, including any licensed from Novellus, Inc., including that preclinical results may not be predictive of clinical results and our ability to file an IND for such candidates; our need for substantial additional funds; the estimated markets for our product candidates, including those for ARDS, and the acceptance thereof by any market; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; the early stage of products under development, including the NoveCite technology; our ability to obtain, perform under and maintain licensing, financing and strategic agreements and relationships; our ability to attract, integrate, and retain key personnel; risks related to our growth strategy; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contact:Andrew ScottVice President, Corporate Development(O) 908-967-6677 x105(M) 646-522-8410ascott@citiuspharma.com

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SOURCE Citius Pharmaceuticals, Inc.

Company Codes: NASDAQ-SMALL:CTXR

Original post:
Citius Pharmaceuticals Signs an Exclusive Worldwide Licensing Agreement with Novellus Therapeutics for Unique iMSC-Therapy for Acute Inflammatory...

Skin Stem Cells Comments Off on Citius Pharmaceuticals Signs an Exclusive Worldwide Licensing Agreement with Novellus Therapeutics for Unique iMSC-Therapy for Acute Inflammatory… | October 7th, 2020

Despite the challenges brought about by Covid-19, leading cosmetic manufacturers, including Mibelle Biochemistry, Cargill Beauty, Givaudan Active Beauty and Lucas Meyer, put forward their most creative innovations for consideration by the in-cosmetics Global Awards jury.

Judged across three categories - Best Active Ingredient, Best Functional Ingredient and Best Green Ingredient - by some of the industrys foremost experts, the awards recognise the cosmetic ingredient manufacturers who are helping to push the boundaries of innovation, while responding to evolving consumer demands.

The winners were announced yesterday during a virtual ceremony from a list of finalists dominated by skin care ingredients.

Scooping both the Gold and Silver awards in the Best Active Ingredient category, Vytrus Biotech was recognised for its Kannabia Sense and Deobiome Noni ingredients.

Kannabia Sense took the top accolade. This probiotic treatment stimulates the skin microbiota to produce an in-situ postbiotic cocktail that promotes the synthesis of cutaneous oxytocin. Recommended for use in sensitive skin treatments, anti-ageing cosmetic products and delicate facial treatments, it is made from plant stem cells of Cannabis sativa.

Deobiome Noni is a prebiotic deodorant treatment that reduces the generation of body odour while allowing the skin to breathe and respecting the skin microbiota.

Closing the category, Mibelle Biochemistry and Clariant Active were announced as this years joint Bronze Award winners.

Alpine Rose Active, Mibelle Biochemistrys novel senolytics concept, was chosen for its ability to eliminate senescent skin cells and protect skin proteins from oxidative stress.

Clariant Active was applauded for Prenylium. Thanks to Clariants Plant Milking technology, Prenylium is extracted from the root of the mulberry tree (Morus Alba) without causing any damage. This innovative and sustainable approach to sourcing helps to stimulate root growth and results in a concentration of prenylated flavonoids 2000% higher than what is typically found in mulberry root extracts.

German cosmetic manufacturer Symrise took the top award in the Best Functional Ingredient category for its Symrise SymEffect Sun. Based entirely on renewable raw materials from responsible sources, the new ingredient combines the advantages of conventional sunscreen products with the increasing sustainability requirements of consumers.

Also demonstrating a sustainable approach to sun protection, Dow Chemical was awarded the Silver award for its SunSpheres BIO SPF Booster, a bio-based and readily biodegradable SPF boost that enables greater SPF efficiency in suncare and daily skincare products.

The category also honoured two bronze award winners: Clariant and Gattefoss. The former was awarded for Velsan Flex which, thanks to its high water solubility and Renewable Carbon Index (RCI) of 93%, offers preservation boosting powers together with broad formulation flexibility. Gattefosss Emulium Dolcea MB a natural origin O/W emulsifier - has the ability to create a wider range of textures, from fluid serum to thick butter in a wide range of applications, such as skincare, suncare, make up and haircare.

As sustainability continues to drive cosmetic manufacturers to assess the ingredients used in their formulations, this years Green Ingredient Award - in partnership with Ecovia Intelligence - recognises the ingredients championing sustainability in the personal care and beauty market.

Henry Lamotte Oils Paradise Nut Oil/Magdalena River Nut Oil scooped the Gold Award for its ability to support the regeneration of the skins lipid film, while improving the water retention capability of the skin, reducing trans-epidermal water loss.

Unveiled as the Silver Award winner, Cargill Beautys FiberDesign Sensation was praised for its approach to sustainability and upcycling. Derived from 100% natural origins, the texturiser and emulsion stabiliser, designed specifically for skincare, is based on citrus peel fibres from the pectin production side stream.

Joining the line-up of award winners, Minasolve SAS scooped the Bronze Award for its A-Leen Aroma-3, a nature-derived perfuming agent that also offers a broad-spectrum antimicrobial effect. It is a 100% natural version of phenylpropanol, it is produced from cassia essential oil.

Cosmetic Ingredients - October 2020

For more information on the latest cosmetic ingredients launches, also read our special issue:

How far will clean beauty go?

Interest in upcycled ingredients expected to rise in the cosmetics industry

Driven by new consumer preferences, demand for cupuau butter is on the rise

in-cosmetics Awards 2020

Ingredient news: Akott, BASF, Berkem, Clariant, Codif, Covestro, DSM, Firmenich, Givaudaun, Grolman, Imerys, Inter Actifs, Lubrizol, Mibelle, Sederma, Seppic, Silab, Symrise

Read online for free or download the pdf version here.

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Who are the winners of the in-cosmetics Global Awards 2020? - Premium beauty

Skin Stem Cells Comments Off on Who are the winners of the in-cosmetics Global Awards 2020? – Premium beauty | October 7th, 2020