Clene Nanomedicine to Present Updated Interim Data from Phase 2 Multiple Sclerosis Programs at ACTRIMS Forum 2021
By Dr. Matthew Watson
SALT LAKE CITY, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Clene Inc. (NASDAQ: CLNN) (along with its subsidiaries, “Clene”) and its wholly owned subsidiary Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company, today announced that it will present updated interim data from its REPAIR-MS study and updated blinded interim data from its VISIONARY-MS study in poster presentations at the Americas Committee for Treatment and Research in Multiple Sclerosis’ ACTRIMS Forum 2021 (“ACTRIMS”) taking place virtually from February 25-27, 2021.
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Clene Nanomedicine to Present Updated Interim Data from Phase 2 Multiple Sclerosis Programs at ACTRIMS Forum 2021
Precision Announces 3-Year Pre-clinical Study Results Showing Long-term Durability and Safety of ARCUS In Vivo Gene Editing to Cut LDL Cholesterol…
By Dr. Matthew Watson
Study Led by Gene Therapy Program at the University of Pennsylvania Published in Molecular Therapy Study Led by Gene Therapy Program at the University of Pennsylvania Published in Molecular Therapy
Imara to Present at SVB Leerink 10th Annual Global Healthcare Conference
By Dr. Matthew Watson
BOSTON, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Imara Inc. (Nasdaq: IMRA), a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin, today announced that Rahul Ballal, Ph.D., President and Chief Executive Officer, will participate in a Fireside Chat at the SVB Leerink 10th Annual Global Healthcare Conference on Friday, February 26, from 1:40-2:10 p.m. ET.
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Imara to Present at SVB Leerink 10th Annual Global Healthcare Conference
Pfizer and BioNTech Submit COVID-19 Vaccine Stability Data at Standard Freezer Temperature to the U.S. FDA
By Dr. Matthew Watson
NEW YORK and MAINZ, GERMANY, February 19, 2021 (GLOBE NEWSWIRE) — Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) today announced the submission of new data to the U.S. Food and Drug Administration (FDA) demonstrating the stability of their COVID-19 vaccine when stored at -25°C to -15°C (-13°F to 5°F), temperatures more commonly found in pharmaceutical freezers and refrigerators. The data have been submitted to the FDA to support a proposed update to the U.S. Emergency Use Authorization (EUA) Prescribing Information, which would allow for vaccine vials to be stored at these temperatures for a total of two weeks as an alternative or complement to storage in an ultra-low temperature freezer.
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Pfizer and BioNTech Submit COVID-19 Vaccine Stability Data at Standard Freezer Temperature to the U.S. FDA
BridgeBio Pharma and Affiliate ML Bio Solutions Announce Dosing of First Patient in Phase 2 Trial of BBP-418 in Limb Girdle Muscular Dystrophy Type 2i…
By Dr. Matthew Watson
SAN FRANCISCO, Feb. 19, 2021 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO),?a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers,?and affiliate ML Bio Solutions today announced that the first patient has been dosed in a Phase 2 trial of BBP-418 in patients with LGMD2i. BridgeBio and ML Bio’s BBP-418 is the first-ever oral disease-modifying investigational treatment for LGMD2i. BBP-418 was granted Orphan Drug Designation for LGMD2i by the US Food and Drug Administration (FDA) in 2019, and for LGMD by the European Medicines Agency (EMA) in 2020.
Vaccinex Announces Signing of Two Multi-Project Deals with Leading Pharmaceutical Companies Focused on Leveraging Its ActivMAb® Platform
By Dr. Matthew Watson
Partnerships will use Vaccinex’s ActivMAb® platform for viral display of complex antigens to enable antibody discovery Partnerships will use Vaccinex’s ActivMAb® platform for viral display of complex antigens to enable antibody discovery
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Vaccinex Announces Signing of Two Multi-Project Deals with Leading Pharmaceutical Companies Focused on Leveraging Its ActivMAb® Platform
Mersana Therapeutics to Host Conference Call Announcing Fourth Quarter and Year End 2020 Financial Results and Business Updates
By Dr. Matthew Watson
CAMBRIDGE, Mass., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Mersana Therapeutics, Inc. (NASDAQ:MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, today announced that it will host a conference call and webcast on Friday, February 26, 2021 at 8:00 a.m. ET to report financial results for the fourth quarter and year ended December 31, 2020 and provide business updates.
Intercept to Present at Upcoming Investor Conferences
By Dr. Matthew Watson
NEW YORK, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that Jerry Durso, President and Chief Executive Officer, and Sandip Kapadia, Chief Financial Officer of Intercept, will present at the SVB Leerink 10th Annual Global Healthcare Conference on Friday, February 26, 2021 at 1:00 p.m. ET, and at the Cowen 41st Annual Healthcare Conference on Tuesday, March 2, 2021 at 10:30 a.m. ET.
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Intercept to Present at Upcoming Investor Conferences
RAPT Therapeutics to Present at the 10th Annual SVB Leerink Global Healthcare Conference
By Dr. Matthew Watson
SOUTH SAN FRANCISCO, Calif., Feb. 19, 2021 (GLOBE NEWSWIRE) -- RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in oncology and inflammatory diseases, today announced that Brian Wong, M.D., Ph.D., President and Chief Executive Officer, will present at the 10th Annual SVB Leerink Global Healthcare Conference on Friday, February 26, 2021 at 12:00 pm Eastern Time.
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RAPT Therapeutics to Present at the 10th Annual SVB Leerink Global Healthcare Conference
Assembly Bio Announces Upcoming Investor Events
By Dr. Matthew Watson
SOUTH SAN FRANCISCO, Calif., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a clinical-stage biotechnology company developing innovative therapeutics targeting hepatitis B virus (HBV), today announced two upcoming investor events:
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Assembly Bio Announces Upcoming Investor Events
Yield10 Bioscience Announces Warrant Exercises for Proceeds of $3.8 million
By Dr. Matthew Watson
WOBURN, Mass., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Yield10 Bioscience, Inc. (Nasdaq:YTEN), an agricultural bioscience company, today announced that it has received proceeds of approximately $3.8 million from the exercise of 472,099 warrants exercised between January 1, 2021 and February 16, 2021, and the Company has issued to investors 472,099 shares of common stock in exchange for the exercised warrants. The warrants were issued to investors pursuant to a public offering that closed on November 19, 2019. Since November 19, 2019, a total of 679,395 warrants issued in the public offering have been exercised for total proceeds of $5.4 million. The Series A and Series B warrants issued in the November 2019 public offering are exercisable at $8.00 per share and have expiration dates of May 19, 2022 and May 19, 2027, respectively.
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Yield10 Bioscience Announces Warrant Exercises for Proceeds of $3.8 million
Atea Pharmaceuticals Announces Chugai In-License of AT-527 from Roche for the Treatment of COVID-19 in Japan
By Dr. Matthew Watson
Chugai obtains exclusive development and marketing rights for AT-527 in Japan from Roche, who has ex-US rights for the treatment of COVID-19 Chugai obtains exclusive development and marketing rights for AT-527 in Japan from Roche, who has ex-US rights for the treatment of COVID-19
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Atea Pharmaceuticals Announces Chugai In-License of AT-527 from Roche for the Treatment of COVID-19 in Japan
CEO of Longeveron Inc. to Present at the Intercontinental Summit on Aging & Gerontology
By Dr. Matthew Watson
MIAMI, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and life-threatening conditions, today announced that Geoff Green, Chief Executive Officer of Longeveron, has been invited to give a presentation titled “A Regenerative Medicine Approach to Aging Frailty” at the Intercontinental Summit on Aging & Gerontology being held virtually on March 8, 2021. He will discuss Longeveron’s ongoing clinical research program in Aging Frailty and Alzheimer’s disease, in addition to the regenerative medicine investigational clinical research field in general for diseases and conditions associated with aging.
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CEO of Longeveron Inc. to Present at the Intercontinental Summit on Aging & Gerontology
Agile Therapeutics to Host Fourth Quarter and Full Year 2020 Financial Results Conference Call and Provide Corporate and Twirla Commercial Updates on…
By Dr. Matthew Watson
Live Conference Call and Webcast at 4:30 p.m. ET
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Agile Therapeutics to Host Fourth Quarter and Full Year 2020 Financial Results Conference Call and Provide Corporate and Twirla Commercial Updates on...
Information expressed in the public domain by the Repharm Group is deceptive and contradictory
By Dr. Matthew Watson
Today, on February 19, information has arrived in the public domain that the company from Repharm Group will acquire the shares of JSC “Olainfarm” directly and indirectly owned by Anna Emilija Maligina.
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Information expressed in the public domain by the Repharm Group is deceptive and contradictory
Y-mAbs to Announce 2020 Financial and Operating Results on February 25, 2021
By Dr. Matthew Watson
NEW YORK, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB) today announced that it will report its financial results for the fiscal year ended December 31, 2020 on Thursday, February 25, 2021, after the close of the U.S. financial markets. The announcement will be followed by a conference call and webcast with the investment community on Friday, February 26, 2021, at 9 a.m. ET. Participating on the call from Y-mAbs will be Thomas Gad, founder, Chairman and President; Dr. Claus Moller, Chief Executive Officer; and Bo Kruse, Chief Financial Officer.
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Y-mAbs to Announce 2020 Financial and Operating Results on February 25, 2021
Ayala Pharmaceuticals Announces $25 Million Strategic Financing
By Dr. Matthew Watson
Funding Extends Cash Runway through Multiple Expected Value Drivers Into 2023
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Ayala Pharmaceuticals Announces $25 Million Strategic Financing
Stem Cells Market to Inspire a Growth up to US$ 18289.9 Million at a 9.1% CAGR by 2027 – PharmiWeb.com
By daniellenierenberg
Stem cells are undifferentiated cells which are capable of differentiating into any type of cell that make-up the human body and thus, are capable of producing non-regenerative cells such as neural and myocardial cells.
Statistics:
The global stem cells market is estimated to account forUS$ 9,941.2 Mnin terms of value in2020and is expected to reachUS$ 18,289.9 Mnby the end of2027.
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GlobalStem CellsMarket: Drivers
Approval and launch of new products is expected to propel growth of the global stem cells market over the forecast period. For instance, in December 2019, BioRestorative Therapies, Inc. received a Notice of Allowance on its patent application for a method of generating brown fat stem cells from Israeli Patent Office.
Moreover, increasing number of stem cell banking resource centers is also expected to aid in growth of the market. For instance, in March 2020, Stemlife Berhad, a cord blood bank in Malaysia, started a Stem Cell Banking Resource Center in Jerudong Park Medical Center, Brunei.
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Statistics:
Adult stem cells held dominant position in the global stem cells market in 2019, accounting for81.2%share in terms of value, followed by Human Embryonic Stem Cells and Induced Pluripotent Stem Cells, respectively
Figure 1. GlobalStem CellsMarket Share (%), by Value, by Cell Type, 2019.
GlobalStem CellsMarket: Restraints
High cost of stem cell therapy is expected to hinder growth of the global stem cells market. For instance, Bioinformant a research firm engaged in stem cell research, reported that the cost of stem cell therapy ranges between US$ 5,000-8,000 per patient and in some cases it may rise as much as US$ 25,000 or more depending on the complexity of the procedure.
Moreover, restrictions on research activities related to stem cells had hampered the growth of embryonic stem cells historically and resulted in its meager share in the total market in spite of its advantages over adult stem cells.
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GlobalStem CellsMarket: Opportunities
R&D in stem cell donation is expected to offer lucrative growth opportunities for players in the global stem cells market. For instance, in March 2020, researchers from Dankook University and Catholic University, South Korea, reported investigation of the types and degrees of physical and psychological discomfort experienced by hematopoietic stem cell donors before, during, and after the donation process.
Moreover, adoption of online distribution channel is also expected to aid in growth of the global stem cells market. For instance, The US Direct-to-Consumer Marketplace for Autologous Stem Cell Interventions, published in the journal Perspectives in Biology and Medicine, in 2018, the number of new stem cell businesses with websites doubled on average every year between 2009 and 2014, in the U.S.
The global stem cells market was valued atUS$ 9,112.0 Mnin2019and is forecast to reach a value ofUS$ 18,289.9 Mnby2027at aCAGR of 9.1%between2020 and 2027.
Figure 2. GlobalStem CellsMarket Value (US$ Mn), and Y-o-Y Growth (%), 2019-2027
Market Trends/Key Takeaways
Adoption of stem cells for the treatment of various diseases is expected to propel growth of the global stem cells market. For instance, in January 2020, researchers at University of Houston developed biologic cardiac pacemaker-like cells by taking fat stem cells and reprogramming them as an alternative treatment for heart conditions such as conduction system disorders and heart attacks.
Moreover, increasing investment in stem cell therapies is also expected to aid in growth of the market. For instance, in July 2018, the Emory Orthopaedics & Spine Center, in collaboration with Sanford Health, Duke University, Andrews Institute, and Georgia Institute of Technology, received US$ 13 million grant from the Marcus Foundation for a multicenter clinical trial studying stem cell options for treating osteoarthritis. The Phase 3 trial was initiated in March 2019, and is expected to complete by December 2021.
GlobalStem CellsMarket: Competitive Landscape
Major players operating in the global stem cells market include Advanced Cell Technology, Inc., Angel Biotechnology Holdings PLC, Bioheart Inc., Lineage Cell Therapeutics., BrainStorm Cell Therapeutics, Inc., California Stem Cell Inc., Celgene Corporation, Takara Bio Europe AB, Cellular Engineering Technologies, Cytori Therapeutics Inc., Osiris Therapeutics, and STEMCELL Technologies Inc.
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GlobalStem CellsMarket: Key Developments
Major players in the market are focused on adopting collaboration and partnership strategies to expand their product portfolio. For instance, in September 2018, STEMCELL Technologies signed an exclusive license agreement with Brigham and Womens Hospital for rights to commercialize technologies for the generation of human pluripotent stem cell-derived kidney organoids.
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Stem Cells Market to Inspire a Growth up to US$ 18289.9 Million at a 9.1% CAGR by 2027 - PharmiWeb.com
The Untapped Potential of Cell and Gene Therapy – AJMC.com Managed Markets Network
By daniellenierenberg
We can absolutely cut the number of cancer deaths down so that one day in our lifetimes it can be a rare thing for people to die of cancer, said Patrick Hwu, MD, president and CEO of Moffitt Cancer Center in Florida and among gene therapys pioneers. It still may happen here and there, but itll be kind of like people dying of pneumonia. Its like, He died of pneumonia? Thats kind of weird. I think cancer can be the same way.
The excitement returned in spades in 2017 when the FDA signed off on a gene-therapy drug for the first time, approving the chimeric antigen receptor (CAR) T-cell treatment tisagenlecleucel (Kymriah; Novartis) for the treatment of B-cell precursor acute lymphoblastic leukemia. At last, scientists had devised a way to reprogram a persons own T cells to attack tumor cells.
Were entering a new frontier, said Scott Gottlieb, MD, then the FDA Commissioner, in announcing the groundbreaking approval.
Gottlieb wasnt exaggerating. The growth in CAR T-cell treatments is exploding. Although only a handful of cell and gene therapies are on the market, FDA officials predicted in 2019 that the agency will receive more than 200 investigational new drug applications per year for cell and gene therapies, and that by 2025, it expects to have accelerated to 10 to 20 cell and gene therapy approvals per year.1
Essentially, you can kill any cancer cell that has an antigen that is recognized by the immune cell, Hwu said. The key to curing every single cancer, which is our goal, is to have receptors that can recognize the tumor but dont recognize the normal cells. Receptors recognizing and then attacking normal cells is what can cause toxicity.
Cell therapy involves cultivating or modifying immune cells outside the body before injecting them into the patient. Cells may be autologous (self-provided) or allogeneic (donor-provided); they include hematopoietic stem cells and adult and embryonic stem cells. Gene therapy modifies or manipulates cell expression. There is considerable overlap between the 2 disciplines.
Juliette Hordeaux, PhD, senior director of translational research for the University of Pennsylvanias gene therapy program, is cautious about the FDAs predictions, saying shed be thrilled with 5 cell and/or gene therapy approvals annually.
For monogenic diseases, there are only a certain number of mutations, and then well plateau until we reach a stage where we can go after more common diseases, Hordeaux said.
Safety has been the main brake around adeno-associated virus vector (AAV) gene therapy, added Hordeaux, whose hospitals program has the institutional memory of both Jesse Gelsingers tragic death during a 1999 gene therapy trial as well as breakthroughs by Carl June, MD, and others in CAR T-cell therapy.
Sometimes there are unexpected toxicity [events] in trials.I think figuring out ways to make gene therapy safer is going to be the next goal for the field before we can even envision many more drugs approved.
In total, 3 CAR T-cell therapies are now on the market, all targeting the CD19 antigen. Tisagenlecleucel was the first. Gilead Sciences received approval in October 2017 for axicabtagene ciloleucel (axi-cel; Yescarta), a CAR T-cell therapy for adults with large B-cell non-Hodgkin lymphoma. Kite Pharma, a subsidiary of Gilead, received an accelerated approval in July 2020 for brexucabtagene autoleucel (Tecartus) for adults with relapsed or refractory mantle cell lymphoma.
On February 5, 2021, the FDA approved another CD19-directed therapy for relapsed/refractory large B-cell lymphoma, lisocabtagene maraleucel (liso-cel; JCAR017; Bristol Myers Squibb). The original approval date was missed due to a delay in inspecting a manufacturing facility (see related article).
Idecabtagene vicleucel (ide-cel; bb2121; Bristol Myers Squibb) is under priority FDA review, with a decision expected by March 31, 2021. The biologics license application seeks approval for ide-cel, a B-cell maturation antigendirected CAR therapy, to treat adult patients with multiple myeloma who have received at least 3 prior therapies.2
The number of clinical trials evaluating CAR T-cell therapies has risen sharply since 2015, when investigators counted a total of 78 studies registered on the ClinicalTrials.gov website. In June 2020, the site listed 671 trials, including 357 registered in China, 256 in the United States, and 58 in other countries.3
Natural killer (NK) cells are the research focus of Dean Lee, MD, PhD, a physician in the Division of Hematology and Oncology at Nationwide Childrens Hospital. He developed a method for consistent, robust expansion of highly active clinical-grade NK cells that enables repeated delivery of large cell doses for improved efficacy. This finding led to several first-in-human clinical trials evaluating adoptive immunotherapy with expanded NK cells under an FDA Investigational New Drug application. He is developing both genetic and nongenetic methods to improve tumor targeting and tissue homing of NK cells. His eff orts are geared toward pediatric sarcomas.
The biggest emphasis over the past 20 to 25 years has been cell therapy for cancer, talking about trying to transfer a specific part of the immune system for cells, said Lee, who is also director of the Cellular Therapy and Cancer Immunology Program at Nationwide Childrens Hospital, at The Ohio State University Comprehensive Cancer Center Arthur G. James Cancer Hospital, and at the Richard J. Solove Research Institute.
The Pivot Toward Treating COVID-19 and Other Diseases
However, Lee said, NKs have wider potential. This is kind of a natural swing back. Now that we know we can grow them, we can reengineer them against infectious disease targets and use them in that [space], he said.
Lee is part of a coronavirus disease 2019 (COVID-19) clinical trial, partnering with Kiadis, for off-the-shelf K-NK cells using Kiadis proprietary platforms. Such treatment would be a postexposure preemptive therapy for treating COVID-19. Lee said the pivot toward treating COVID-19 with cell therapy was because some of the very early reports on immune responses to coronavirus, both original [SARS-CoV-2] and the new [mutation], seem to implicate that those who did poorly [overall] had poorly functioning NK cells.
The revolutionary gene editing tool CRISPR is making its initial impact in clinical trials outside the cancer area. Its developers, Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD, won the Nobel Prize in Chemistry 2020.
For patients with sickle cell disease (SCD), CRISPR was used to reengineer bone marrow cells to produce fetal hemoglobin, with the hope that the protein would turn deformed red blood cells into healthy ones. National Public Radio did a story on one patient who, so far, thanks to CRISPR, has been liberated from the attacks of SCD that typically have sent her to the hospital, as well from the need for blood transfusions.4
Its a miracle, you know? the patient, Victoria Gray of Forest, Mississippi, told NPR.
She was among 10 patients with SCD or transfusion-dependent beta-thalassemia treated with promising results, as reported by the New England Journal of Medicine.5 Two different groups, one based in Nashville, which treated Gray,5 and another based at Dana-Farber Cancer Institute in Boston,6 have reported on this technology.
Stephen Gottschalk, MD, chair of the department of bone marrow transplantation and cellular therapy at St Jude Childrens Research Hospital, said, Theres a lot of activity to really explore these therapies with diseases that are much more common than cancer.
Animal models use T cells to reverse cardiac fibrosis, for instance, Gottschalk said. Using T cells to reverse pathologies associated with senescence, such as conditions associated with inflammatory clots, are also being studied.
Hordeaux said she foresees AAV being used more widely to transmit neurons to attack neurodegenerative diseases.
The neurons are easily transduced by AAV naturally, she said. AAV naturally goes into neurons very efficiently, and neurons are long lived. Once we inject genetic matter, its good for life, because you dont renew neurons.
Logistical Issues
Speed is of the essence, as delays in producing therapies can be the difference between life and death, but the approval process takes time. The process of working out all kinks in manufacturing also remains a challenge. Rapid production is difficult, too, because of the necessary customization of doses and the need to ensure a safe and effective transfer of cells from the patient to the manufacturing center and back into the patient.7
Other factors that can slow down launches include insurance coverage, site certification, staff training, reimbursement, and patient identification. The question of how to reimburse has not been definitively answered; at this point, insurers are being asked to issue 6- or even 7-figure payments for treatments and therapies that may not work.8
CAR T, I think, will become part of the standard of care, Gottschalk said. The question is how to best get that accomplished. To address the tribulations of some autologous products, a lot of groups are working with off -the-shelf products to get around some of the manufacturing bottlenecks. I believe those issues will be solved in the long run.
References
1. Statement from FDA Commissioner Scott Gottlieb, MD, and Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. News release. FDA website. January 15, 2019. https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics. Accessed January 13, 2021.
2. Bristol Myers Squibb provides regulatory update on lisocabtagene maraleucel (liso-cel). News release. Bristol Myers Squibb; November 16, 2020. Accessed January 11, 2021. https://news.bms.com/news/details/2020/Bristol-Myers-Squibb-Provides-Regulatory-Update-on-Lisocabtagene-Maraleucel-liso-cel/default.aspx
3. Wei J, Guo Y, Wang Y. et al. Clinical development of CAR T cell therapy in China: 2020 update. Cell Mol Immunol. Published online September 30, 2020. doi:10.1038/s41423-020-00555-x
4. Stein R. CRISPR for sickle cell diseases shows promise in early test. Public Radio East. November 19, 2019. Accessed January 11, 2021. https://www.publicradioeast.org/post/crisprsickle-cell-disease-shows-promise-early-test
5. Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-Cas9 gene editing for sickle cell disease and -Thalassemia. N Engl J Med. Published online December 5, 2020. DOI: 10.1056/NEJMoa2031054
6. Esrick EB, Lehmann LE, Biffi A, et al. Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease. N Engl J Med. Published online December 5, 2020. doi:10.1056/NEJMoa2029392
7. Yednak C. The gene therapy race. PwC. February 5, 2020. Accessed January 11, 2021. https://www.pwc.com/us/en/industries/healthindustries/library/gene-therapy-race.html
8. Gene therapies require advanced capabilities to succeed after approval. PwC website. Accessed January 11, 2021. https://www.pwc.com/us/en/industries/health-industries/library/commercializing-gene-therapies.html
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The Untapped Potential of Cell and Gene Therapy - AJMC.com Managed Markets Network
Grayson-Jockey Club Research Foundation Board approves record funding for Equine Research – Past The Wire
By daniellenierenberg
LEXINGTON, Ky. Board of directors of Grayson-Jockey Club Research Foundation announced today that it has authorized expenditure of $1,638,434, the most that the foundation has ever allocated in a year, to fund 12 new projects at 12 universities, 12 continuing projects, and two career development awards worth $20,000 each. This marks the seventh straight year that more than $1 million has been approved. The 2021 slate of research brings Grayson-Jockey Club Research Foundations totals since 1983 to more than $30.6million to underwrite 396 projects at 45 universities.
We are heartened by the continued commitment of universities to supporting equine veterinary research throughout these difficult times and that we are able to distribute more funding than ever before, enabling us to help horses of all breeds and disciplines, said Dell Hancock, chair of Grayson.
Despite a challenging year, Grayson-Jockey Club was excited to receive 51 grant applications from a variety of veterinary institutions in North America as well as five other countries, said Dr. Stephen M. Reed, chair of Graysons research advisory committee. The subject matter is diverse and ranges from identifying new methods to treat and prevent infectious disease to development of computational models using big data to investigation of novel imaging techniques to prevent orthopedic injuries.
Below is an alphabetical list by school of the new projects:
Passive Immunization of Foals with RNA-AB against R Equi
Jeroen Pollet, Baylor College of Medicine
By inhalation therapy, we intend to deliver the genetic code for a protective antibody against Rhodococcus equi into the lung cells of newborn foals, to rapidly protect them against infection.
Hyperthermia and Acidosis in Exertional Muscle Damage
Michael Davis,Oklahoma State University
This project will identify an underlying cause of exercise-associated muscle fatigue and soreness and allow trainers to more precisely condition horses with fewer training days lost to muscle soreness.
Developing an Improved Serological Test for Strangles
Noah Cohen, Texas A&M
We propose to develop a more accurate blood test to identify horses infected with the bacterium that causes strangles to improve control and prevention of strangles.
Mitigation of Equine Recurrent Uveitis through SOCS
Joseph Larkin, University of Florida
We seek to design a topical eye drop, using a natural protein, which helps to prevent pain and blindness associated with equine recurrent uveitis.
Environmental Origins of Equine Antimicrobial Resistance
Brandy Burgess, University of Georgia
This study will elucidate how antimicrobial resistance and virulence determinants are shared among horses and hospital environment, as well as the role antimicrobial exposure plays at this interface.
Treatment of Joint Injury with Mesenchymal Stromal Cells
Thomas Koch, University of Guelph
Evaluation of equine umbilical cord blood-derived mesenchymal stromal cells to treat joint injuries in horses.
Optimizing Bone Growth to Reduce Equine Fracture
Mariana Kersh, University of Illinois UrbanaChampaign
Reduction in distal limb fractures through exercise in young horses would have a significant positive impact on horse welfare and the economics and public perception of the horse industry.
New Generation Equine Influenza Bivalent VLP Vaccine
Thomas Chambers, University of Kentucky
We propose to create a novel, safe and effective vaccine for equine influenza based on the 21st-century technology of noninfectious virus-like particles produced in plants.
Injury Prediction from Stride Derived Racing Load
Chris Whitton, University of Melbourne
By studying patterns in bone fatigue accrual over time in racehorses, we will better, and earlier, identify horses at risk of limb injury, facilitating timely evidence based preventative strategies.
Predicting Exercising Arrhythmias with Resting ECGs
Molly McCue, University of Minnesota
We will use at rest ECGs to identify horses with irregular heart rhythms at exercise that can cause sudden cardiac death (SCD), allowing for increased monitoring and improved understanding of SCD.
Understanding and Preventing Supporting Limb Laminitis
Andrew Van Eps,University of Pennsylvania
We aim to make supporting limb laminitis preventable through analysis of archived model tissues, a multi-center limb motion study of horses at risk, and development of a prototype therapeutic device.
Diagnosis of Incipient Condylar Stress Fracture
Peter Muir,University of Wisconsin-Madison
This study will save the lives of racehorses by establishing screening using fetlock CT for diagnosis of horses with a high risk of imminent serious injury for personalized clinical care.
The Storm Cat Career Development Award, inaugurated in 2006, grants $20,000 to an individual considering a career in equine research.This years recipient is Dr. Callum G. Donelly of the University of California, Davis. Dr. Donelly has completed his residency program and is in a research training position under the mentorship of Dr. Carrie Fino. His project, Proteomic Investigation of Equine Spinal Ataxia, is expected to identify novel protein biomarkers that differentiate normal horses from those with spinal ataxia, with high sensitivity and specificity.
The Elaine and Bertram Klein Career Development Award was first awarded in 2015 and grants $20,000 to a prospective equine researcher. This years recipient is Dr. Aileen Rowland of Texas A&M University. Dr. Rowlands research focuses on the efficacy of xenogeny-free mesenchymal stem cells for osteoarthritis.
We are pleased to continue our funding of two career development awards to support individuals passionate about equine research, said Dr. Johnny Mac Smith, consultant to the research advisory committee. Dr. Donelly and Dr. Rowland are worthy recipients of these grants, and I look forward to seeing how their current and future projects contribute to improving equine health in the future.
Details on the new projects are available at the following link:grayson-jockeyclub.org/default.asp?section=2&area=Research&menu=2.
Grayson-Jockey Club Research Foundation is traditionally the nations leading source of equine research funding. The projects it supports enhance the health and safety of horses of all breeds. Additional information about the foundation is available atgrayson.jockeyclub.org.
Press Release