COVID 19 to Lead the Sales of Myelofibrosis Treatment to Register Stellar Growth in the Next 10 Years – Cole of Duty
By daniellenierenberg
Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.
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The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.
The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:
As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.
On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.
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Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.
Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.
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COVID 19 to Lead the Sales of Myelofibrosis Treatment to Register Stellar Growth in the Next 10 Years - Cole of Duty
Market Analysis and Technological Opportunities of Cell Therapy Manufacturing Market till 2030 – Medic Insider
By daniellenierenberg
Prophecy Market Insights Cell Therapy Manufacturing market research report provides a comprehensive, 360-degree analysis of the targeted market which helps stakeholders to identify the opportunities as well as challenges. The research report study offers keen competitive landscape analysis including key development trends, accurate quantitative and in-depth commentary insights, market dynamics, and key regional development status forecast 2020-2029. It incorporates market evolution study, involving the current scenario, growth rate, and capacity inflation prospects, based on Porters Five Forces and DROT analyses.
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An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.
Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Cell Therapy Manufacturing market
Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast
Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering
The comprehensive list of Key Market Players along with their market overview, product protocol, key highlights, key financial issues, SWOT analysis, and business strategies. The report dedicatedly offers helpful solutions for players to increase their clients on a global scale and expand their favour significantly over the forecast period. The report also serves strategic decision-making solutions for the clients.
Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.
Segmentation Overview:
Cell Therapy ManufacturingMarket Key Companies:
harmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.
The Cell Therapy Manufacturing research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Cell Therapy Manufacturing market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.
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The study analyses the manufacturing and processing requirements, project funding, project cost, project economics, profit margins, predicted returns on investment, etc. This report is a must-read for investors, entrepreneurs, consultants, researchers, business strategists, and all those who have any kind of stake or are planning to foray into the Cell Therapy Manufacturing industry in any manner.
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Market Analysis and Technological Opportunities of Cell Therapy Manufacturing Market till 2030 - Medic Insider
Aprea Therapeutics Presents Results From French Phase Ib/II Clinical Trial of APR-246 (Eprenetapopt) and Azacitidine in Patients with TP53 Mutant…
By daniellenierenberg
BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc.(Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53, today announced the oral presentation of updated data from its French Phase 1b/2 clinical trial at the 25th European Hematology Association Annual Meeting (EHA). The trial is evaluating the safety and efficacy of APR-246 (eprenetapopt) in combination with azacitidine (AZA) for the treatment of TP53 mutant myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). The clinical trial is sponsored by the Groupe Francophone des Mylodysplasies (GFM).
As of the April 1, 2020 data cutoff, the overall response rate (ORR) in 28 evaluable MDS patients was 75%, with a 57% complete remission (CR) rate, by International Working Group (IWG) criteria. With a median duration of follow-up of 9.7 months, the median overall survival (OS) for all enrolled patients (n=52) was 12.1 months and in MDS patients (n=34) was 12.1 months. For patients who remained on treatment for 3 or more cycles of treatment the median OS was higher at 13.7 months versus 2.8 months for patients who were on treatment for fewer than 3 cycles. Relative to baseline, mutant TP53 variant allele frequency (VAF) was decreased in responding patients by 3 cycles of treatment, including 20 (51%) patients who achieved mutant TP53 negativity by next-generation sequencing (NGS).
The data from this ongoing trial of eprenetapopt with azacitidine continue to be very encouraging in these most difficult-to-treat TP53 mutant MDS and AML patients, who not only have at least one TP53 mutation but the majority of whom also have high risk cytogenetic abnormalities, said Thomas Cluzeau, M.D., co-lead investigator for the GFM trial. We continue to observe ORR and CR rates in these patients that are substantially higher than the GFMs experience with azacitidine monotherapy. Furthermore, with increased duration of follow-up, we now also see the emergence of highly encouraging overall survival that appears to be better than azacitidine alone or in combination with others agents in this very high-risk molecular group of patients with a TP53 mutation.
Details of the on-demand oral presentation are as follows:
Title: APR-246 Combined with Azacitidine in TP53 Mutated Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia. A Phase 2 Study by the Groupe Francophone des Mylodysplasies (GFM)
Oral Abstract Session: Novel treatments for MDS I
Abstract: S181
About the Clinical Trial
Eligible patients in the Phase Ib/II clinical trial include hypomethylating agent (HMA) nave, TP53 mutated MDS and AML. All enrolled patients were to receive APR-246 as a 4,500 mg fixed dose IV daily for 4 days and AZA over 7 days in 28-day cycles. The primary endpoint of the trial is CR rate.
AboutAprea Therapeutics, Inc.
Aprea Therapeutics, Inc.is a biopharmaceutical company headquartered inBoston, Massachusettswith research facilities inStockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivatemutant tumor suppressor protein, p53. The Companys lead product candidate is APR-246 (eprenetapopt), a small molecule in clinical development for hematologic malignancies, including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). APR-246 has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, and Orphan Drug designation from the European Commission for MDS, AML and ovarian cancer. For more information, please visit the company website atwww.aprea.com.
The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.
About Myelodysplastic Syndromes
Myelodysplastic syndromes (MDS) represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in up to 20% of MDS and AML patients and are associated with poor overall prognosis.
About p53 and APR-246 (eprenetapopt)
The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.
APR-246 (eprenetapopt) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein by restoring wild-type p53 conformation and function and thereby induce programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with APR-246 in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with APR-246 has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.
Forward-Looking StatementCertain information contained in this press release includes forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our clinical trials, regulatory submissions and projected cash position. We may, in some cases use terms such as predicts, believes, potential, continue, anticipates, estimates, expects, plans, intends, targeting, confidence, may, could, might, likely, will, should or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with theU.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.
Source:Aprea Therapeutics, Inc.
Exploring the Therapeutic Potential of ST266 Against Numerous Diseases Including COVID-19 – Technology Networks
By daniellenierenberg
Noveome Biotherapeutics is a clinical-stage company focused on developing therapies for the regenerative repair of tissues. Their product ST266, a first-of-its-kind, multi-targeted, non-cellular platform biologic comprised of a complex mixture of biomolecules, is currently being evaluated as a potential treatment for the severe inflammatory response observed in the lungs of some COVID-19 patients.Technology Networks recently spoke with William J. Golden, Noveome Biotherapeutics Founder, Chairman and CEO, who explains the underlying basis for investigating ST266s potential against COVID-19. Golden also elaborates on many of the other indications for which ST266 is being developed to treat.Laura Lansdowne (LL): Could you provide our readers with a brief overview of Noveome Biotherapeutics?William J. Golden (WJG): Noveome is a clinical-stage biotherapeutics company located in Pittsburgh, PA. The company was founded in 2000 by Bill Golden and Lancet Capital. The group was interested in exploring non-embryonic stem cells and identified a technology at the University of Pittsburgh that was using cells derived from human amnion, a membrane that closely covers the fetus during development. The company, named Kytaron Technologies, Inc. at the time, licensed that amnion cell technology but, ultimately, Noveome scientists were able to discover, develop and patent their own unique population of cells, called Amnion-derived Multipotential Progenitor (AMP) cells, using a proprietary culture method that follows current Good Manufacturing Practice (cGMP) regulations. These novel cells were used to produce our product, ST266.LL: What is ST266? Could you elaborate on its mechanism of action in relation to the healing process?WJG: Noveomes product, ST266, is the secretome produced by the AMP cells. It is a completely cell-free solution and is comprised of hundreds of biologically active molecules, including cytokines and growth factors. Interestingly, these cytokines and growth factors exist at very low physiological levels ranging from pg/mL ng/mL concentrations.1 The fact that such low concentrations of these molecules are biologically active is quite remarkable when you consider that traditional protein-based therapies are usually administered at concentrations that are orders of magnitude greater than the concentrations found in ST266.Because the composition of ST266 is so complex, its multiple mechanisms of action have only been partially elucidated. Clinical and preclinical studies have shown ST266 to be anti-inflammatory,2,3 promote wound healing,4,5 reduce apoptosis, reduce vascular permeability (manuscript in preparation), and restore cellular homeostasis.3 Preclinical studies have also shown ST266 to be neuroprotective. In a traumatic brain injury model, ST266 significantly protected against reactive gliosis, suggesting potent anti-inflammatory activity, and resulted in significant recovery of rotarod motor function.6,7 In another study, ST266 was tested in the experimental autoimmune encephalopathy (EAE) mouse model of multiple sclerosis (MS). In this model, the mice develop optic neuritis, which is among the presenting symptoms of MS in humans. ST266 was administered to the nares of mice 15 or 22 days after disease induction. ST266 is absorbed via capillary action along the olfactory nerves which bypasses the blood-brain barrier. This unique route of administration allows for the delivery of high molecular weight biologics to the optic nerve of the eye and the central nervous system. ST266 attenuated visual dysfunction, prevented retinal ganglion cell (RGC) loss, reduced inflammation, and decreased the rate of demyelination of the optic nerve in EAE mice.3Mechanistically, ST266 simultaneously acts on multiple cell receptor-activated and intracellular signaling pathways. For example, in the EAE MS model, neuroprotective effects involved oxidative stress reduction, SIRT1-mediated mitochondrial function promotion, and pAKT signaling.3 In a Phase 2 UV light burn study, ST266 reduced erythema and DNA damage and increased the expression of XPA DNA repair proteins.2Importantly, ST266 has a proven clinical safety profile. It has been administered to 243 patients by various routes of administration (topical skin, topical ocular, topical oral, targeted intranasal), and no drug-related serious adverse events have been reported. Preclinical studies of systemically administered ST266 have also yielded no drug-related safety concerns.LL: For what indications is ST266 currently being evaluated as a treatment?WJG: We refer to ST266 as a platform biologic. By this, we mean that ST266 is one product that has the potential to treat numerous and varied diseases. In the clinic, we have shown anti-inflammatory activity when ST266 is applied topically to UV light-burned the skin2 and topical application to the gums of patients with gingivitis and periodontitis showed a reduction in proinflammatory cytokines in the patients crevicular fluid (manuscript in preparation). We are currently conducting a Phase 2 open label trial of ST266 to treat persistent corneal epithelial defects (PEDs) when applied topically to the eye. Results from this trial will be published soon. We are currently planning a Phase 2b multi-center, randomized, double-masked trial to further evaluate the safety and efficacy of ST266 in this indication. Finally, we are conducting a Phase 1 study in patients at risk for developing glaucoma. This study is using the intranasal route of delivery described above in combination with a novel delivery device. The goal is to deliver ST266 directly to the optic nerve, where it can protect the RGCs that are damaged in glaucoma. We envision this route of delivery will be applicable to central nervous system and other back-of-the eye indications.We also have several ongoing preclinical programs that are evaluating systemically administered ST266 for more generalized inflammatory conditions. These data are not yet published but combined with the data we have compiled in preclinical and clinical studies of topical skin, topical oral and topical ocular administration, we believe ST266 has the potential to be an effective therapy for numerous systemic inflammatory conditions.LL: Could you elaborate on the underlying basis for your evaluation of ST266 as a potential treatment for COVID-19?WJG: As you know, a major complication of COVID-19 is the severe inflammatory response seen in the lungs of some patients. This response is called cytokine storm or cytokine release syndrome. As the pandemic continues and more data have become available, it is now known that the cytokine storm does not just affect the lungs. Multi-organ damage occurs in many of these patients. We believe that systemic delivery of ST266 and its anti-inflammatory activity has the potential to calm the storm. Our as-yet-unpublished preclinical studies with intravenous ST266 support this hypothesis and we are moving rapidly to initiate intravenous ST266 in a Phase 1 study. Once safety in humans is established by this route of administration, we will commence Phase 2 studies in COVID-19 patients.William J. Golden was speaking to Laura Elizabeth Lansdowne, Senior Science Writer for Technology Networks.References
1. Steed, DL, C Trumpower, D Duffy, C Smith, V Marshall, R Rupp, and M Robson. (2008). Amnion-Derived Cellular Cytokine Solution: A Physiological Combination of Cytokines for Wound Healing. Eplasty 8: 15765.
2. Guan, Linna, Amanda Suggs, Emily Galan, Minh Lam, and Elma D. Baron. (2017). Topical Application of ST266 Reduces UV-Induced Skin Damage. Clinical, Cosmetic and Investigational Dermatology. DOI: https://doi.org/10.2147/CCID.S147112.
3. Khan, Reas S, Kimberly Dine, Bailey Bauman, Michael Lorentsen, Lisa Lin, Helayna Brown, Leah R Hanson, et al. (2017). Intranasal Delivery of A Novel Amnion Cell Secretome Prevents Neuronal Damage and Preserves Function In A Mouse Multiple Sclerosis Model. Scientific Reports. DOI: https://doi.org/10.1038/srep41768.
4. Bergmann, Juri, Florian Hackl, Taro Koyama, Pejman Aflaki, Charlotte a Smith, Martin C Robson, and Elof Eriksson. (2009). The Effect of Amnion-Derived Cellular Cytokine Solution on the Epithelialization of Partial-Thickness Donor Site Wounds in Normal and Streptozotocin-Induced Diabetic Swine. Eplasty 9: e49.
5. Franz, Michael G, Wyatt G Payne, Liyu Xing, D K Naidu, R E Salas, Vivienne S Marshall, C J Trumpower, Charlotte A Smith, David L Steed, and M C Robson. (2008). The Use of Amnion-Derived Cellular Cytokine Solution to Improve Healing in Acute and Chronic Wound Models. Eplasty 8: e21.
6. Deng-Bryant, Ying, Zhiyong Chen, Christopher van der Merwe, Zhilin Liao, Jitendra R Dave, Randall Rupp, Deborah a Shear, and Frank C Tortella. (2012). Long-Term Administration of Amnion-Derived Cellular Cytokine Suspension Promotes Functional Recovery in a Model of Penetrating Ballistic-like Brain Injury. The Journal of Trauma and Acute Care Surgery DOI: https://doi.org/10.1097/TA.0b013e3182625f5f.
7. Deng-Bryant, Ying, Ryan D. Readnower, Lai Yee Leung, Tracy L. Cunningham, Deborah A. Shear, and Frank C. Tortella. (2015). Treatment with Amnion-Derived Cellular Cytokine Solution (ACCS) Induces Persistent Motor Improvement and Ameliorates Neuroinflammation in a Rat Model of Penetrating Ballistic-like Brain Injury. Restorative Neurology and Neuroscience. DOI: https://doi.org/10.3233/RNN-140455.
‘I found these 4 affordable winter skin saviours in the chemist beauty aisles.’ – Mamamia
By daniellenierenberg
The light, non-greasy, dermatologically tested formula contains Vitamin B3 to help hydrate and support the skin barrier, and SPF30 for added sun protection. It alsosits beautifully under your regular SPF you'll have on, and makeup.
It won't clog your pores and doesn't include common irritants like fragrance or colour. The no-fuss formula also won't mess with the rest of your skincare, making it the easy to slot into your existing routine. Done!
(*Side note:Always read the label. Follow the directions for use. Avoid prolonged sun exposure and wear protective clothing, hats and eyewear to further reduce risk. Frequent re-application is required.)
Anyone else get dry lips in winter? This lip balm is brilliant for a few reasons.
A) It's super affordable and accessible - you'll find it at most pharmacies.
B) It has SPF50+ broad spectrum protection.
C) The texture isn't too thick or too thin, it's just right.
And D) You can wear the clear formula over the top of alip stain or lip liner.
Just don't leave it in the car, OK?
(And yes, you know the drill:Always read the label. Follow the directions for use. Avoid prolonged sun exposure and wear protective clothing, hats and eyewear to further reduce risk. Frequent re-application is required.)
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'I found these 4 affordable winter skin saviours in the chemist beauty aisles.' - Mamamia
Bone Marrow Processing Systems Market : Industry Trends and Developments 2018 2025 – Cole of Duty
By daniellenierenberg
Bone marrow aspirationand trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.
The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.
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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.
In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.
Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific.
Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.
Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.
Covid 19 Impact [emailprotected]https://www.trendsmarketresearch.com/report/covid-19-analysis/3374
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Bone Marrow Processing Systems Market : Industry Trends and Developments 2018 2025 - Cole of Duty
EHA25Virtual: Combined Irradiation and Chemotherapy Better Prepares Children for Stem Cell Transplantation than Chemotherapy Alone – PR Newswire UK
By daniellenierenberg
THE HAGUE, Netherlands, June 12, 2020 /PRNewswire/ -- Treatment of childhood cancer is a success story, particularly for acute lymphoblastic leukemia (ALL). More than 90% of ALL patients below 18 years of age are rescued with contemporary chemotherapy. However, the remaining 10% have resistant or reoccurring leukemia and require alternative treatment regimens. One of the most powerful leukemia therapies is hematopoietic stem cell transplantation from a donor (allogeneic HSCT). Approximately 50-80% of pediatric ALL patients that receive allogeneic HSCT are cured, 20% experience leukemic reoccurrence (relapse), and 10% die from complications.
Allogeneic HSCT is a multistep procedure:
For high-risk leukemia, the gold standard conditioning procedure is a combination of total body irradiation (TBI) and high dose chemotherapy. This approach is very effective in controlling leukemia in the conditioning step, but patients may experience highly negative consequences of this procedure later in life: sterility, growth retardation, lung problems, and secondary cancer.
Therefore, a large consortium of pediatric transplant experts initiated a global study to investigate whether chemotherapy-based conditioning could substitute TBI. The study is called FORUM (For Omitting Radiation Under Majority Age) and had to be stopped because chemotherapy-based conditioning had significantly poorer outcomes (i.e., lower overall survival rates) than the combination of TBI and chemotherapy. The researchers will now perform prospective monitoring to better define the advantages and limitations of various conditioning approaches.
Presenter:Dr Christina PetersAffiliation:Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, AustriaAbstract:#S102 TBI OR CHEMOTHERAPY BASED CONDITIONING FOR CHILDREN AND ADOLESCENTS WITH ALL: A PROSPECTIVE RANDOMIZED MULTICENTER-STUDY "FORUM" ON BEHALF OF THE AIEOP-BFM-ALL-SG, IBFM-SG, INTREALL-SG AND EBMT-PD-WP
Embargo: Please note that our embargo policy applies to all selected abstracts in the Press Briefings. For more information click here.
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SOURCE European Hematology Association (EHA)
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EHA25Virtual: Combined Irradiation and Chemotherapy Better Prepares Children for Stem Cell Transplantation than Chemotherapy Alone - PR Newswire UK
BrainStorm to Present at the Raymond James Human Health Innovations Conference – Yahoo Finance
By daniellenierenberg
Management to Host One-on-One Investment Meetings
NEW YORK, June 11, 2020 /PRNewswire/ --BrainStorm Cell Therapeutics Inc.(NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced Chaim Lebovits, CEO and Ralph Kern, MD, MHSc, President and Chief Medical Officer, will present a corporate overview on Thursday, June 18 at 9:00 am EST, during theRaymond James Human Health Innovations Conference, a virtual event connecting institutional investors with company management teams that will be held June 15-18, 2020.
Mr. Lebovits and Dr. Kern will update conference participants on the Company's investigational therapeutic, NurOwn, that is currently in a fully enrolled phase 3 study for the treatment of ALS and a phase 2 study for the treatment of progressive multiple sclerosis. Additionally, they will present an overview of the Company's financial position and pipeline. After the presentation, the management team will participate in a question and answer session with institutional investors.
Mr. Lebovits and Dr. Kern will be joined by David Setboun, PhD, MBA, Chief Operating Officer, Stacy Lindborg, PhD, Head of Global Clinical Research, and Preetam Shah, PhD, MBA, Chief Financial Officer, for a series of one-on-one meetings, with select institutional investors arranged by Raymond James.
Participants can view the presentation via the event link and those unable to join will have access to an archived link on the Company's Events and Presentation webpage after the conclusion of the conference.
EVENT: Raymond James Human Health Innovations Conference
PRESENTATION: Thursday, June 18th at 9:00 am EST
LINK: https://bit.ly/2YmZf8u
About NurOwn
NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently receivedU.S.FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began inMarch 2019.
AboutBrainStorm Cell Therapeutics Inc.
BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at sixU.S.sites supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a filing forU.S.FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently receivedU.S.FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment inMarch 2019.
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Safe-Harbor Statement
Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.
CONTACTS
Investor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: +1-862-397-1860pshah@brainstorm-cell.com
Media:
Sean LeousWestwicke/ICR PRPhone: +1-646-677-1839sean.leous@icrinc.com
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SOURCE Brainstorm Cell Therapeutics Inc
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BrainStorm to Present at the Raymond James Human Health Innovations Conference - Yahoo Finance
Avalon GloboCare strikes three-way material transfer agreement with Weill Cornell Medicine and Arbele Limited – Proactive Investors USA & Canada
By daniellenierenberg
The company said the move was aimed at the development of next generation cellular immunotherapy FLASH-CAR technology
(), a clinical-stage developer of cell-based technologies and therapeutics, announced Thursday that it has struck a three-way material transfer agreement (MTA) with Weill Cornell Medicine in New York City and the companys strategic partner, Arbele Limited.
With this agreement, Avalon GloboCare and Arbele Limited intend to collaborate with Weill Cornell Medicine and co-develop the standardized laboratory steps necessary to generate clinical-grade CAR-T and CAR-natural killer (NK) cells for use in future human clinical trials with Avalons first FLASH-CAR platform candidate, AVA-011.Similar to T-cells, NK cells are a type of white blood cell, also able to attack cancer cells, but utilize different mechanisms.
The company said this process development step will provide the bridge between Avalons benchtop research and the bio-manufacturing processes to potentially deliver the clinical-grade cellular immunotherapy product to patients.
READ:Avalon GloboCare advancing immune cell therapy to treat blood cancers using FLASH-CAR technology
We are excited about this agreement to translate our cellular therapy candidates into standardized, clinical-grade cell products that could be used in future clinical trials, Avalon GloboCare CEO David Jin said in a statement.
This step reflects our dedication to establishing an infrastructure to develop our cellular immunotherapy candidates and to maintain the highest possible standards for generating clinical-grade cells for human cancer trials, he added.
AVA-011 is a next generation cellular immunotherapy candidate using Avalons FLASH-CAR technology that targets both CD19 and CD22 tumor antigens on cancer cells. Avalon has already successfully completed pre-clinical research on AVA-011, including tumor cytotoxicity studies.
Avalon expects to begin a first-in-human clinical trial with AVA-011 for the treatment of relapsed or refractory B-cell lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma in the first quarter of 2021. The goal is to use AVA-011 as a bridge to bone marrow stem cell transplant therapy, currently the only curative approach for patients with these blood cancers.
Avalons next generation immune cell therapy using FLASH-CAR technology is being co-developed with the companys strategic partner Arbele Limited. The adaptable FLASH-CAR platform can be used to create personalized cell therapy from a patients own cells, as well as off-the-shelf cell therapy from a universal donor, expanding the reach of cancer patients that can be treated.
Avalon, based in Freehold, New Jersey, specializes in developing cell-based technologies and is involved in the management of stem-cell banks and clinical laboratories.
Contact the author Uttara Choudhury at [emailprotected]
Follow her on Twitter: @UttaraProactive
CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001 in Severe Hemoglobinopathies at the 25th…
By daniellenierenberg
-Beta thalassemia: Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia-
-Sickle cell disease: Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion-
-Five patients with beta thalassemia and two patients with sickle cell disease have been treated to date with CTX001 and all have successfully engrafted-
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), and highlighted recent progress in the CTX001 development program. These data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress by Dr. Selim Corbacioglu, Professor of Pediatrics and the Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Regensburg University Hospital, Regensburg, Germany.
CLIMB-111 Trial in Transfusion-Dependent Beta Thalassemia Updated ResultsData presented today at EHA demonstrate clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated. CRISPR Therapeutics and Vertex announced initial data for the first TDT patient in November of 2019.
Patient 1 with TDT has the 0/IVS-I-110 genotype, which is associated with a severe phenotype similar to 0/0, and had a transfusion requirement of 34 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. After CTX001 infusion, two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia, and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. New data presented today show that at 15 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 100.0%. Bone marrow allelic editing was 78.1% at 6 months and 76.1% at one year.
Patient 2 with TDT has the 0/IVS-II-745 genotype and had a transfusion requirement of 61 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. The patient achieved neutrophil engraftment 36 days after CTX001 infusion and platelet engraftment 34 days after infusion. After CTX001 infusion, two SAEs occurred, neither of which the PI considered related to CTX001: pneumonia and an upper respiratory tract infection; both subsequently resolved. At 5 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.4%.
Hemoglobin data over time are presented for Patient 1 and Patient 2 below.
Figure 1accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/35581299-d683-44b0-a75e-7a1a9b9fe9eb
CLIMB-121 Trial in Severe Sickle Cell Disease Updated Results Data presented today at EHA reflect longer-duration follow-up data for the first patient with SCD treated with CTX001. CRISPR Therapeutics and Vertex announced initial data for this first SCD patient in November of 2019.
Patient 1 with SCD experienced seven vaso-occlusive crises (VOCs) and five packed red blood cell transfusions per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. After CTX001 infusion, three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis and abdominal pain; all subsequently resolved. New data presented today show that at 9 months after CTX001 infusion, the patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%. Bone marrow allelic editing was 81.4% at 6 months. Figure 2 presents the hemoglobin data over time for this patient.
Figure 2 accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7610c5bd-25c8-4f5b-be86-8bc16ed57eb1
With these new data, we are beginning to see early evidence of the potential durability of benefit from treatment with CTX001, as well as consistency of the therapeutic effect across patients, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. These highly encouraging early data represent one more step toward delivering on the promise and potential of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases.
The data announced today are remarkable, including the demonstration of clinical proof-of-concept in TDT, said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. While these are still early days, these data mark another important milestone for this program and for the field of gene editing. The results presented at this medical conference add to results previously shared demonstrating that CRISPR/Cas9 gene editing has the potential to be a curative therapy for severe genetic diseases like sickle cell and beta thalassemia.
In my 25 years of caring for children and young adults facing both sickle cell disease and beta thalassemia, I have seen how these diseases can adversely affect patients lives in very significant ways, said Dr. Haydar Frangoul, Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center and senior author of the abstract presented at the EHA virtual congress. I am encouraged by the preliminary results, which demonstrate, in essence, a functional cure for patients with beta thalassemia and sickle cell disease.
Recent Progress in the Phase 1/2 Clinical TrialsCLIMB-111 for TDT has dosed a total of 5 patients, and all patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of the first two patients. Additionally, CLIMB-111 has been expanded to allow enrollment of 0/0 patients and is in the process of being expanded to allow enrollment of pediatric patients ages 12 years or older.
CLIMB-121 for SCD has dosed a total of 2 patients and both patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of these first two patients.
The initial safety profile in these trials appears to be consistent with myeloablative busulfan conditioning and an autologous hematopoietic stem cell transplant.
In March 2020, clinical trial sites in the U.S. and Europe temporarily paused their elective hematopoietic stem cell transplant programs due to the COVID-19 pandemic, and as a result, CRISPR and Vertex temporarily paused conditioning and dosing in these trials. Enrollment, mobilization and drug product manufacturing in each trial remains ongoing. The companies are now in the process of re-initiating dosing with CTX001 at certain clinical trial sites. The CLIMB-111 and CLIMB-121 clinical trials are ongoing, and patients will be followed for 2 years following CTX001 infusion. The companies expect to provide additional data in the second half of 2020.
About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.
Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT) from the U.S. FDA, Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), and Fast Track Designation from the FDA for both SCD and TDT.
CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. CTX001 is the most advanced gene-editing approach in development for TDT and SCD.
About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.
About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.
About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.
About the CRISPR-Vertex Collaboration CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.
About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.
CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases and activities at clinical trial sites) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that is being presented at the European Hematology Associations virtual congress; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.
About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, and statements regarding our plans and expectations for our clinical trials and clinical trial sites, and our expectations regarding future data announcements. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com
CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167reides@wcgworld.com
Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187
Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275
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CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001 in Severe Hemoglobinopathies at the 25th...
Impact of Corona on Spinal Fusion Market Statistics, Investment Trends, Key Players and Forecast 2020-2026 | Cotton, Rayon, Blended – Cole of Duty
By daniellenierenberg
Spinal Fusion Market 2020: Latest Analysis
Chicago, United States:- The report titled Global Spinal Fusion Market is one of the most comprehensive and important additions to Report Hive Research archive of market research studies. It offers detailed research and analysis of key aspects of the global Spinal Fusion market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Spinal Fusion market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Spinal Fusion market is carefully analyzed and researched about by the market analysts.
Top Players of Spinal Fusion Market are Studied: Market by Materials, Cotton, Rayon, Blended
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Segmentation by Application: Cord Blood Stem Cells CryopreservationOther Stem Cells Cryopreservation
Segmentation by Type: Liquid phaseVapor phase
NOTE:Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Spinal Fusion Market which would mention How the Covid-19 is Affecting the Spinal Fusion Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Spinal Fusion Players to Combat Covid-19 Impact.
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* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size
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Table of Contents
Market Overview: This is the first section of the report that includes an overview of the scope of products offered in the global Spinal Fusion market, segments by product and application, and market size.
Market Competition by Player: Here, the report shows how the competition in the global Spinal Fusion market is growing or decreasing based on deep analysis of market concentrate rate, competitive situations and trends, expansions, merger and acquisition deals, and other subjects. It also shows how different companies are progressing in the global Spinal Fusion market in terms of revenue, production, sales, and market share.
Company Profiles and Sales Data: This part of the report is very important as it gives statistical as well as other types of analysis of leading manufacturers in the global Spinal Fusion market. It assesses each and every player studied in the report on the basis of main business, gross margin, revenue, sales, price, competitors, manufacturing base, product specification, product application, and product category.
Market Status and Outlook by Region: The report studies the status and outlook of different regional markets such as Europe, North America, the MEA, Asia Pacific, and South America. All of the regional markets researched about in the report are examined based on price, gross margin, revenue, production, and sales. Here, the size and CAGR of the regional markets are also provided.
Market by Product: This section carefully analyzes all product segments of the global Spinal Fusion market.
Market by Application: Here, various application segments of the global Spinal Fusion market are taken into account for research study.
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Marketing Strategy Analysis, Distributors: Here, the research study digs deep into behavior and other factors of downstream customers, distributors, development trends of marketing channels, and marketing channels such as indirect marketing and direct marketing.
Research Findings and Conclusion: This section is solely dedicated to the conclusion and findings of the research study on the global Spinal Fusion market.
Appendix: This is the last section of the report that focuses on data sources, viz. primary and secondary sources, market breakdown and data triangulation, market size estimation, research programs and design, research approach and methodology, and the publishers disclaimer.
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COVID-19 impact on Spinal Fusion Market Share, Size, Revenue, Gross Margin and Growth Rate Analysis 2020-2026
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Impact of Corona on Spinal Fusion Market Statistics, Investment Trends, Key Players and Forecast 2020-2026 | Cotton, Rayon, Blended - Cole of Duty
Canine Stem Cell Therapy Market to Expand with Significant CAGR – WorldsTrend
By daniellenierenberg
Health care stakeholders need to invest in value-based care, innovative care delivery models, advanced digital technologies. XploreMR will help you to know declarative, procedural, contextual, and somatic information about the Canine Stem Cell Therapy Market. It also provides a critical assessment of the performance of emerging and mature markets in a new publication titled Global Market Study on Canine Stem Cell Therapy: Ongoing Clinical Trials and Focus on Advancements to Push Adoption in Veterinary Clinics.
A synopsis of the global canine stem cell therapy market with reference to the global healthcare pharmaceutical industry
Despite the economic and political uncertainty in the recent past, the global healthcare industry has been receiving positive nudges from reformative and technological disruptions in medical devices, pharmaceuticals and biotech, in-vitro diagnostics, and medical imaging. Key markets across the world are facing a massive rise in demand for critical care services that are pushing global healthcare spending levels to unimaginable limits.
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A rapidly multiplying geriatric population; increasing prevalence of chronic ailments such as cancer and cardiac disease; growing awareness among patients; and heavy investments in clinical innovation are just some of the factors that are impacting the performance of the global healthcare industry. Proactive measures such as healthcare cost containment, primary care delivery, innovation in medical procedures (3-D printing, blockchain, and robotic surgery to name a few), safe and effective drug delivery, and well-defined healthcare regulatory compliance models are targeted at placing the sector on a high growth trajectory across key regional markets.
Parent Indicators Healthcare
Research Methodology
XploreMR utilizes a triangulation methodology that is primarily based on experimental techniques such as patient-level data, to obtain precise market estimations and insights on Molecule and Drug Classes, API Formulations and preferred modes of administration. Bottom-up approach is always used to obtain insightful data for the specific country/regions. The country specific data is again analysed to derive data at a global level. This methodology ensures high quality and accuracy of information.
Secondary research is used at the initial phase to identify the age specific disease epidemiology, diagnosis rate and treatment pattern, as per disease indications. Each piece of information is eventually analysed during the entire research project which builds a strong base for the primary research information.
Primary research participants include demand-side users such as key opinion leaders, physicians, surgeons, nursing managers, clinical specialists who provide valuable insights on trends and clinical application of the drugs, key treatment patterns, adoption rate, and compliance rate.
Quantitative and qualitative assessment of basic factors driving demand, economic factors/cycles and growth rates and strategies utilized by key players in the market is analysed in detail while forecasting, in order to project Year-on-Year growth rates. These Y-o-Y growth projections are checked and aligned as per industry/product lifecycle and further utilized to develop market numbers at a holistic level.
On the other hand, we also analyse various companies annual reports, investor presentations, SEC filings, 10k reports and press release operating in this market segment to fetch substantial information about the market size, trends, opportunity, drivers, restraints and to analyse key players and their market shares. Key companies are segmented at Tier level based on their revenues, product portfolio and presence.
Please note that these are the partial steps that are being followed while developing the market size. Besides this, forecasting will be done based on our internal proprietary model which also uses different macro-economic factors such as per capita healthcare expenditure, disposable income, industry based demand driving factors impacting the market and its forecast trends apart from disease related factors.
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Canine Stem Cell Therapy Market to Expand with Significant CAGR - WorldsTrend
New Results From Landmark NURTURE Study Show That Pre-Symptomatic SMA Patients Treated With SPINRAZA (nusinersen) Continue to Demonstrate Sustained…
By daniellenierenberg
DetailsCategory: DNA RNA and CellsPublished on Wednesday, 10 June 2020 16:58Hits: 131
CAMBRIDGE, MA, USA I June 10, 2020 I Biogen Inc.(Nasdaq: BIIB) today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA) that is transforming expectations of early treatment with SPINRAZA (nusinersen). In infants genetically diagnosed with SMA, new data demonstrate that early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented survival. Patients continued to maintain and make progressive gains in motor function compared to the natural course of the disease. These results are being presented at the virtual Cure SMA Research & Clinical Care Meeting taking place June 10-12, 2020.
The new data include nearly a year of additional follow-up for NURTURE study participants. As of February 2020, all patients treated (n=25; median age of 3.8 years old) were alive and remained free of permanent ventilation. In the absence of treatment, the majority of children with SMA Type 1 would, on average, not reach their second birthday. Additionally, all children who achieved the motor milestone of being able to walk independently (many within a normal timeframe) have maintained that ability from the first occurrence until the last visit.
The impact of early and sustained SPINRAZA treatment on these infants and their families is remarkable. Ive had the privilege to watch them grow into active young children, many of whom have experienced progress in motor function consistent with children their age who do not have SMA, said Kathryn Swoboda, M.D., the Katherine B. Sims, M.D., Endowed Chair in Neurogenetics and Director of the Neurogenetics Program, Massachusetts General Hospital. The new results from NURTURE continue to bolster the substantial benefit of both prompt diagnosis and early and longer-term treatment with SPINRAZA.
NURTURE is an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA before 6 weeks old. The study has been extended by an additional three years, enabling Biogen to evaluate the longer-term efficacy and safety of SPINRAZA through 8 years of age and further understand the impact of early treatment. More information on the NURTURE study (NCT02386553) is available onclinicaltrials.gov.
Additional results from the updated interim analysis as of February 2020 show:
About SPINRAZA (nusinersen)2-4 SPINRAZA is the first therapy approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is approved in more than 50 countries. As of March 31, 2020, more than 10,000 individuals have been treated with SPINRAZA. It is the only SMA treatment to combine unsurpassed real-world experience with a robust level of clinical evidence across a broad spectrum of patient populations.
SMA is a rare, genetic, neuromuscular disease that is characterized by a loss of motor neurons in the spinal cord and lower brain stem that can result in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA, and people of all ages are impacted by the disease. It is a leading genetic cause of infant mortality.
SPINRAZA, a foundation of care in SMA, is an antisense oligonucleotide (ASO), developed using Ionis Pharmaceuticals proprietary technology that is designed to target a root cause of SMA by increasing the amount of full-length survival motor neuron (SMN) protein, which is critical to maintaining motor neurons. It is administered by intrathecal injection into the fluid surrounding the spinal cord where motor neurons reside to deliver the treatment where the disease starts.
SPINRAZA currently maintains a robust clinical data set in SMA based on data from approximately 300 patients across a broad range of SMA populations demonstrating a favorable benefit:risk profile. SPINRAZA was evaluated in two randomized, double-blind, sham-controlled studies of infantile and later-onset SMA (ENDEAR and CHERISH, respectively) and supported by open-label studies that include pre-symptomatic infants (NURTURE), individuals with later-onset SMA (CS2/CS12) and an extension study of individuals who previously participated in the clinical development program (SHINE). The most common adverse events observed were respiratory infection, fever, constipation, headache, vomiting and back pain. Hypersensitivity, meningitis and hydrocephalus have been observed in the post-marketing setting. Renal toxicity and coagulation abnormalities, including acute severe low platelet counts, have been observed after administration of some ASOs. Laboratory tests can monitor for these signs.
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in five years.
About BiogenAt Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the worlds first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics and is focused on advancing research programs in multiple sclerosis and neuroimmunology, Alzheimers disease and dementia, neuromuscular disorders, movement disorders, ophthalmology, immunology, neurocognitive disorders, acute neurology and pain.
We routinely post information that may be important to investors on our website at http://www.biogen.com. To learn more, please visit http://www.biogen.com and follow us on social media Twitter, LinkedIn, Facebook, YouTube.
References:
SOURCE: Biogen
Molecular alterations in the extracellular matrix in the brains of newborns with congenital Zika syndrome – Science
By daniellenierenberg
How Zika affects the extracellular matrix
In some cases, Zika virus (ZIKV) infection during pregnancy leads to a series of severe defects in the fetus collectively known as congenital Zika syndrome (CZS). These include microcephaly, defective neuronal migration, and impaired cortical development. Aguiar et al. combined genomic, transcriptomic, and proteomic analyses of blood and postmortem brains and demonstrated that ZIKV-infected neonates showed a reduction in collagen expression and an increase in adhesion factor expression, alterations in the extracellular matrix consistent with the brain defects seen in CZS. Together, these datasets form a useful resource for those investigating the molecular mechanisms underlying CZS in humans.
Zika virus (ZIKV) infection during pregnancy can cause a set of severe abnormalities in the fetus known as congenital Zika syndrome (CZS). Experiments with animal models and in vitro systems have substantially contributed to our understanding of the pathophysiology of ZIKV infection. Here, to investigate the molecular basis of CZS in humans, we used a systems biology approach to integrate transcriptomic, proteomic, and genomic data from the postmortem brains of neonates with CZS. We observed that collagens were greatly reduced in expression in CZS brains at both the RNA and protein levels and that neonates with CZS had several single-nucleotide polymorphisms in collagen-encoding genes that are associated with osteogenesis imperfecta and arthrogryposis. These findings were validated by immunohistochemistry and comparative analysis of collagen abundance in ZIKV-infected and uninfected samples. In addition, we showed a ZIKV-dependent increase in the expression of cell adhesion factors that are essential for neurite outgrowth and axon guidance, findings that are consistent with the neuronal migration defects observed in CZS. Together, these findings provide insights into the underlying molecular alterations in the ZIKV-infected brain and reveal host genes associated with CZS susceptibility.
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Molecular alterations in the extracellular matrix in the brains of newborns with congenital Zika syndrome - Science
Augmenting Demand for Stem Cell Characterization and Analysis Tools to Bolster Global Market Revenue Growth During the Crisis Period of COVID 19 – The…
By daniellenierenberg
Stem cell characterization is the study of tissue-specific differentiation. Thera are various type of stem cell such as embryonic stem cell, epithelial stem cell and others. Further, various techniques are used to characterized stem cells such as immunological techniques, used for depiction of different population of stem cells. These techniques are generally based on immunochemistry using staining technique or florescent microscopy. Besides, stem cells characterization and analysis tools are used against target chronic diseases. In 2014, the San Diego (UCSD) Health System and Sanford Stem Cell Clinical Center at the University of California announced the launch of a clinical trial, in order to assess the safety of neural stem cellbased therapy in patients with chronic spinal cord injury.
The factors driving the growth of stem cell characterization and analysis tools market due to increasing chronic disorders such as cancer, a diabetes and others. In addition, increasing awareness about among people about the therapeutic potency of stem cells characterization in the management of effective diseases is anticipated to increase the demand for stem cell characterization and analysis tools. Further, there are various technologies such as flow cytometry which is used to characterize the cell surface profiling of human-bone marrow and other related purposes are expected to increase the growth of stem cell characterization and analysis tools market. In addition, increasing investment by private and public organization for research activities are likely to supplement the market growth in near future.
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On the other hand, the unclear guidelines and the technical limitation for the development of the product are expected to hamper the growth of stem cell characterization and analysis tools market.
Rapid increase in corona virus all around the world is expected to hamper the growth of stem cell characterization and analysis tools market. The virus outburst has become one of the threats to the global economy and financial markets. The impact has made immense decrease in revenue generation in the field of all healthcare industry growth for the market in terms of compatibility and it has led in huge financial losses and human life which has hit very hard to the core of developing as well as emerging economies in healthcare sector. It further anticipated that such gloomy epidemiological pandemic environment is going to remain in next for at least some months, and this is going to also affect the life-science market which also include the market of stem cell characterization and analysis tools market.
Based on the Products and Service Type, stem cell characterization and analysis tools market are segmented into:
Based on the Technology, stem cell characterization and analysis tools market are segmented into:
Based on the Applications, stem cell characterization and analysis tools market are segmented into:
Based on the End User, stem cell characterization and analysis tools market are segmented into:
Based on the segmentation, human embryonic stem cell is expected to dominate the market due to their indefinite life span and higher totipotency as compared to other stem cells. Further, on the basis of technology segmentations, cell production is anticipated to increase the demand for stem cell characterization and analysis tools due to their emerging applications for stem cells in drug testing in the management of the effective diseases. Furthermore, on the basis of application segmentations, oncology is expected to show significant growth rate due to increase in the number of pipelines products for the treatment of cancers or tumors. Based on the end user, pharmaceutical and biotechnology companies are expected to dominate the market due to rising global awareness about the therapeutics research activities.
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Geographically, the global stem cell characterization and analysis tools market is segmented into regions such as Latin America, Europe, North America, South Asia, East Asia Middle East & Africa and Oceania. North America is projected to emerge as prominent market in the global stem cell characterization and analysis tools market due to growing cases of target chronic diseases and increasing investments for research activities. Europe is the second leading region to dominate the market due to technological advancement and also surge in therapeutic activities, funded by government across the world. Asia-pacific is likely to witness maximum growth in near future due to increasing disposable income and with the development of infrastructure.
Some of the major key players competing in the global stem cell characterization and analysis tools market are Osiris Therapeutics, Inc., Caladrius Biosciences, Inc., U.S. Stem Cell, Inc., Astellas Pharma Inc., TEMCELL Technologies Inc., BioTime Inc., Cellular Engineering Technologies Inc., Cytori Therapeutics, Inc., and BrainStorm Cell Therapeutics Inc.
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Augmenting Demand for Stem Cell Characterization and Analysis Tools to Bolster Global Market Revenue Growth During the Crisis Period of COVID 19 - The...
Autologous Bone Marrow Transplantation and Metformin, a Hope for the Cure of Multiple Sclerosis – Gilmore Health News
By daniellenierenberg
On Friday, June 5, a few days after World MS Day on May 30, there was a day of online conferences and workshops to learn more about multiple sclerosis. It was an opportunity to shed light on autologous bone marrow transplantation, a little known treatment that could cure multiple sclerosis.
Neuron
Multiple sclerosis (MS) is a neurodegenerative autoimmune disease that causes stiffness, pain, and fatigue. It is the main cause of disability, exclusion from the labor market, and social exclusion among young people, as it occurs mainly among people between 25 and 35 years old. According to the National MS Society, approximately 1 million people in the United States suffer from MS.
Currently, there is no treatment to cure MS, but there is hope: Autologous bone marrow transplantation or autologous hematopoietic stem cell transplantation. This treatment allows patients to go from the more common forms of multiple sclerosis into remission. If carried out early enough, it enables at least partial recovery from the disability.
Read Also: Combo of Diabetes and Hypertension Drugs Causes Cancer Cell Death, Researchers Find
The aim of this treatment is to rebuild a new immune system in patients. This includes intensive chemotherapy followed by reinjection of the patients hematopoietic stem cells. Several studies conducted between 2015 and 2019 on this technique have shown that 83.3 of patients with the relapsing-remitting form had no attack in the four years following auto-transplantation and three years after transplantation 78% of patients with secondary progressive multiple sclerosis and 66% of patients with primary progressive multiple sclerosis experienced no worsening of their disability, Mediapart continues.
One of the main obstacles to this treatment remains the difficulty of access. Many patients testify that their neurologist often finds this method too experimental and too risky. Another factor that discourages the use of autologous bone marrow transplantation is the risk-benefit ratio, which is considered unbalanced. Transplant-related mortality is between 5 and 10%, which justifies doctors preference for a treatment that is considered safer.
Read Also: Diabetes: Metformin Transfers Blood Sugar From the Blood to the Intestines
Another treatment has shown encouraging results in multiple sclerosis. This is a drug for diabetes, metformin, which rejuvenates stem cells to convert them into myelin-producing cells and thus help combat multiple sclerosis. These results have been published in the journal Cell Stem Cell, and it is expected that the tests, which are currently only carried out on mice, will also be carried out on humans within a year. I am very optimistic, study author Professor Robin Franklin told The Guardian newspaper.
References
Metformin Restores CNS Remyelination Capacity by Rejuvenating Aged Stem Cells
Autologous Hematopoietic Cell Transplantation in Multiple Sclerosis: Changing Paradigms in the Era of Novel Agents
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Autologous Bone Marrow Transplantation and Metformin, a Hope for the Cure of Multiple Sclerosis - Gilmore Health News
Hematopoietic Stem Cell Transplantation (HSCT) Market Trends 2020: In-Depth Analysis of Industry Growth & Forecast Up To 2026 – Cole of Duty
By daniellenierenberg
The Hematopoietic Stem Cell Transplantation (HSCT) Market research report enhanced worldwide Coronavirus COVID19 impact analysis on the market size (Value, Production and Consumption), splits the breakdown (Data Status 2014-2020 and 6 Year Forecast From 2020 to 2026), by region, manufacturers, type and End User/application. This Hematopoietic Stem Cell Transplantation (HSCT) market report covers the worldwide top manufacturers like (Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc) which including information such as: Capacity, Production, Price, Sales, Revenue, Shipment, Gross, Gross Profit, Import, Export, Interview Record, Business Distribution etc., these data help the consumer know about the Hematopoietic Stem Cell Transplantation (HSCT) market competitors better. It covers Regional Segment Analysis, Type, Application, Major Manufactures, Hematopoietic Stem Cell Transplantation (HSCT) Industry Chain Analysis, Competitive Insights and Macroeconomic Analysis.
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Hematopoietic Stem Cell Transplantation (HSCT) Market report offers comprehensive assessment of 1) Executive Summary, 2) Market Overview, 3) Key Market Trends, 4) Key Success Factors, 5) Hematopoietic Stem Cell Transplantation (HSCT) Market Demand/Consumption (Value or Size in US$ Mn) Analysis, 6) Hematopoietic Stem Cell Transplantation (HSCT) Market Background, 7) Hematopoietic Stem Cell Transplantation (HSCT) industry Analysis & Forecast 20202026 by Type, Application and Region, 8) Hematopoietic Stem Cell Transplantation (HSCT) Market Structure Analysis, 9) Competition Landscape, 10) Company Share and Company Profiles, 11) Assumptions and Acronyms and, 12) Research Methodology etc.
Scope of Hematopoietic Stem Cell Transplantation (HSCT) Market:In 2019, the market size of Hematopoietic Stem Cell Transplantation (HSCT) is million US$ and it will reach million US$ in 2025, growing at a CAGR of from 2019; while in China, the market size is valued at xx million US$ and will increase to xx million US$ in 2025, with a CAGR of xx% during forecast period.
In this report, 2018 has been considered as the base year and 2019 to 2025 as the forecast period to estimate the market size for Hematopoietic Stem Cell Transplantation (HSCT).
On the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, shipments, revenue (Million USD), price, and market share and growth rate foreach application.
Peripheral Blood Stem Cells Transplant (PBSCT) Bone Marrow Transplant (BMT) Cord Blood Transplant (CBT)
On the basis of product type, this report displays the shipments, revenue (Million USD), price, and market share and growth rate of each type.
Allogeneic Autologous
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Geographically, the report includes the research on production, consumption, revenue, Hematopoietic Stem Cell Transplantation (HSCT) market share and growth rate, and forecast (2020-2026) of the following regions:
Important Hematopoietic Stem Cell Transplantation (HSCT) Market Data Available In This Report:
Strategic Recommendations, Forecast Growth Areasof the Hematopoietic Stem Cell Transplantation (HSCT) Market.
Challengesfor the New Entrants,TrendsMarketDrivers.
Emerging Opportunities,Competitive Landscape,Revenue Shareof Main Manufacturers.
This Report Discusses the Hematopoietic Stem Cell Transplantation (HSCT) MarketSummary; MarketScopeGives A BriefOutlineof theHematopoietic Stem Cell Transplantation (HSCT) Market.
Key Performing Regions (APAC, EMEA, Americas) Along With Their Major Countries Are Detailed In This Report.
Company Profiles, Product Analysis,Marketing Strategies, Emerging Market Segments and Comprehensive Analysis of Hematopoietic Stem Cell Transplantation (HSCT) Market.
Hematopoietic Stem Cell Transplantation (HSCT) Market ShareYear-Over-Year Growthof Key Players in Promising Regions.
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Major skin cancer research study to begin at The Hormel Institute – Austin Daily Herald – Austin Herald
By daniellenierenberg
Dr. Rebecca Morris, leader of the Stem Cells and Cancer lab at The Hormel Institute, received a multi-year grant to study stem cells originating in adult bone marrow and their possible effects on skin diseases, including cancer. The grant, from the Nation Institute of Healths National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), awarded Morris with $373,688 over two years for her research project Identification of Novel Epidermal Progenitors.
Morris said this research is significant because it will contribute new understanding of epithelial biology, and blood and bone marrow in general, provide possible new targets for epithelial cancer prevention and control, validate liquid biopsy of blood as a diagnostic tool, and help her and her team to achieve their goal of preventing and alleviating chronic skin diseases including cancer, psoriasis, and epidermolysis bullosa.
Many years ago, I contributed basic research on identification and isolation of adult tissue stem cells from skin epidermis, and demonstrated their role in skin cancer initiation and promotion, Morris said. Now, I am again thrilled to be on the edge of discovery of a new population of epithelial stem cells and have the opportunity to determine their roles in regeneration and cancer.
Cells in the body that cover surfaces (like the epidermis, or top layer of skin) or line spaces (like ducts in mammary gland or lining of the colon) are called epithelial cells. In adults, most cancers originate from these epithelial cells. However, new research has identified certain bone marrow derived epithelial cells (BMDECs) in normal, healthy human subjects.
Morris and her team do not believe anyone has yet described the features and nature of these cells, or analyzed their function.
The research team has hypothesized that the epithelial cells from the bone marrow are epithelial stem cells. They therefore hope to demonstrate that BMDECs include a novel population of adult tissue stem cells that can be gathered to chronically compromised epithelium, such as skin cancer or psoriasis, and regenerate it.
Skin cancer is by far the most common type of cancer in the United States, with millions of people diagnosed each year. As we enter summer, it is important to remember simple steps like staying out of the sun during the middle of the day, staying in the shade, and wearing sunscreen can help reduce your skin cancer risk.
Next steps for Morriss research include determining how these blood borne epithelial cells are recruited to the skin, the recruiting molecules, how the recruitment can be good or bad, and how to modulate their recruitment to alleviate disease.
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Major skin cancer research study to begin at The Hormel Institute - Austin Daily Herald - Austin Herald
Microneedling therapeutic stem cells into damaged tissues – Science Codex
By daniellenierenberg
(LOS ANGELES) -- Mesenchymal stem cells (MSCs) are multipotent in that they naturally replenish the cell types that build our bone, cartilage and adipose tissues. However, their much broader regenerative potential, based on their capacity to migrate and engraft in injured tissues and secrete factors that enhance the formation of new blood vessels, suppress inflammation and cell death, and promote healing, makes them exquisite candidates for cell-based therapies for diseases as varied as cardiovascular, liver, bone and cartilage diseases, lung and spinal cord injuries, autoimmune diseases and even cancer and skin lesions.
MSCs provoke no or negligible adverse reactions in patients that receive them from healthy donors, and can be easily isolated from human tissues, expanded to clinical scales, biopreserved, and stored for point-of-care delivery. This efficiency in preparing medical grade MSCs contrasts with the relative inefficiency with which they currently can be delivered to target tissues in patients. Clinicians often need to administer massive numbers of MSCs with high precision to reach sufficient numbers of cells that successfully engraft and remain functional over time.
To overcome this bottleneck, researchers have developed materials-based approaches in which MSCs are embedded in biomaterial scaffolds that then can be implanted as "patches" in minimally invasive procedures into damaged tissues. However, those cells are often limited in their ability to migrate, overcome tissue barriers, and successfully engraft in tissue microenvironments where their action is needed most. In principle, injection approaches can introduce MSCs into tissues via hypodermic needles in a more targeted manner, but any direct injection to the tissue is invasive and can cause inadvertent tissue damage and side effects like the formation of scar tissue.
Now, a new study reported in Advanced Functional Materials by a team at the Terasaki Institute for Biomedical Innovation in Los Angeles and the University of California, Los Angeles (UCLA) has developed a minimally invasive approach, which deploys "microneedles" that provide a bioactive depot of MSCs. By embedding comparatively low numbers of MSCs in a gel-like material that prolongs their viability and functionality, and targeting damaged tissues with high spatial precision, the researchers showed their approach to accelerate wound healing in a mouse model with excised skin segments.
"Microneedles have been successfully used in the past to painlessly deliver drugs to target tissues such as skin, blood vessels and eyes. We demonstrate here with 'Detachable Microneedle Depots' that an analogous approach can deploy therapeutic cells at target sites," said co-corresponding author Ali Khademhosseini, the Director and CEO of the Terasaki Institute who was previously Director of the UCLA Center for Minimally Invasive Therapeutics. "To achieve this, we developed an entirely new microneedle patch that supports stem cells' viability, responsiveness to wound stimuli, and ability to accelerate wound healing."
At the beginning of their study, Khademhosseini and his co-workers hypothesized that embedding MSCs in a biocompatible and biodegradable biomaterial matrix could help create a hydrated environment with the mechanical properties that stem cells need in order to remain alive and functioning over a longer time. The researchers started by engineering a matrix of gelatin fibers that are cross-linked to each other into a network that could accommodate MSCs. The biomaterial mimicked the normal extracellular environment of tissues that MSCs normally reside in, and it helped to remodel the specific matrix environment in a way that allowed MSCs to take up nutrients and communicate with damaged tissue via soluble factors that they normally receive and dispatch.
The other part of the challenge was to introduce the literal "needle" quality into the cell-delivering device that would enable it to gently penetrate tissues in order to reach their target sites. To this aim, the researcher encased the softer MSC-containing gelatin matrix with a second, much harder biomaterial known as poly(lactic-co-glycolic)acid, in short PLGA. Once the needles were brought into place in a wound bed, the "PLGA shell", which also is biocompatible and biodegradable, slowly degraded, but during the process kept the MSC-containing gelatin matrix in place, allowing MSCs to release their therapeutic factors through emerging gaps in the shell into the damaged tissue. The team showed that in the composite microneedle 90% of MSCs were kept viable for 24 hours, and that, importantly the cells did not lose their potential as stem cells ("stemness"), which was critical for their healing properties.
Finally, the team set out to investigate their microneedle concept in a mouse skin wound model in which a defined excision is made in the epidermal tissue layers. To be able to strategically place individual microneedles within the wound bed, a simple and effective deployment mechanism was devised by attaching an array of microneedles on a small strip of scotch tape with their pointy ends facing away from the tape. Precisely positioning the tape with its patterned microneedle surface on the wound, allowed the individual microneedles to penetrate into the wound bed. Then, the tape was peeled off, causing the microneedles to detach and remain embedded in the wound tissue. Khademhosseini and his co-workers summarized the device's salient features by naming it: "Detachable Hybrid Microneedle Depot" (d-HMND).
In the mouse model, the MSC-loaded d-HMND device indeed stimulated a number of critical parameters associated with wound healing. Compared to an equal number of MSCs injected directly into wounded skin, and a version of the d-HMND device that did not contain any MSCs (cell-free), the MSC-containing d-HMND accelerated the contraction of the wound and re-growth of the epidermal skin layers (re-epithelialization). The researchers used a panel of histological and molecular markers to confirm over a period of 14 days that the device suppressed inflammation, and stimulated tissue remodeling, the formation of new blood vessels, and re-growth of hair - all vital signs of a robust wound healing response.
"In future scenarios, d-HMNDs could be rapidly fabricated in clinical laboratories shortly before use, applied to treat skin injuries, and explored more broadly as treatments for a variety of other disorders, including melanoma and other dermatological disorders that could benefit from the power of MSC cells," said Khademhosseini. "The concept would even be compatible with using patient-derived cells in more personalized device approaches." Khademhosseini and his colleagues are exploring further uses of this technology as part of the Terasaki Institute's research program.
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Microneedling therapeutic stem cells into damaged tissues - Science Codex
Pressure Tools Boost Collagen – Truth In Aging
By daniellenierenberg
Recently, I have been impressed by the unlikely efficacy of pressure tools such as the Pause Fascia StimulatingTool ($115 in the shop) and the KNESKO Quartz Roller and mask set ($115 in the shop). I say unlikely because these tools have no underlying technology and require only a little pressure as you move them over the skin. They really appear to firm and rejuvenate, but there was no research to back this up. Until now.
I was super excited to come across some research conducted by beauty giant, Shiseido and Jichi University in Japan. Their studies revealed that the application of pressure to the skin stimulates the proliferation of stem cells and ultimately boosts collagen.
The research noted that stem cells are more prolific near the sebaceous glands in the skin and dubbed these stem cell reservoirs. When they applied pressure to the skin, the stem cells in the reservoirs proliferated. And not just by a few, the number of stem cells was increased significantly.
Thats all well and good, but does an increased number of stem cells result in better and/or younger looking skin? So the team then investigated whether the cells proliferated by pressure would function in the dermal layer.
For dermal cells to function properly, they need to connect to each other and reconstruct a network. When the researchers observed the cells in pressurized skin, they did indeed connect to each other and they reconstructed a network.
And the really good news is that cells that have reconstructed a network produce collagen. The the production of collagen allows the dermis to regain its elasticity and firmness.
So now we have it, tools that allow us to apply gentle pressure to our skin are helping us stimulate collagen production.
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Pressure Tools Boost Collagen - Truth In Aging