ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
May 14, 2020
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Join ARM on May 21 for Our Next Webinar: Cell Therapy Treatments for COVID-19
May 14, 2020 – (ARM) – On Thursday, May 21 at 11am ET, representatives from Athersys and Pluristem will join ARM to discuss cell therapy treatments for acute respiratory distress syndrome in COVID-19 patients. Following a brief overview from ARM, the speakers will present on their treatment approaches and plans for near term clinical trials. Click here to register and to access information and recordings from ARM's webinar series.
FDA Approves COVID-19 Trial After Successful Emergency Use of Umbilical Stem Cells From RESTEM
May 14, 2020 – (RESTEM) – RESTEM announced it has received approval from the FDA for a 60-patient Phase 1/2a study using umbilical cord mesenchymal stem cells to treat patients with severe cases of COVID-19.
Pluristem Provides 28-Day Follow Up for Ventilator- Dependent COVID-19 Patients Under Compassionate Use Program in Israel and the US
May 14, 2020 – (Pluristem) – The survival rate of the eight patients treated with PLX cells was 87.5%, with 75% off any mechanical ventilation. Nearly two-thirds of patients treated with PLX cells were discharged alive from the hospital, compared to a reported 3.3% in data from the NY area March-April 2020 for patients requiring mechanical ventilation.
Mesoblast Successfully Completes Financing to Scale-Up Manufacturing of Remestemcel-L for COVID-19 ARDS
May 13, 2020 – (Mesoblast) – Mesoblast announced that it has successfully completed a capital raising of $90M USD ($138M AUD). A significant portion of the net proceeds will be used to scale-up manufacturing of the company’s lead product candidate remestemcel-L for the treatment of critically ill patients suffering with diseases caused by cytokine release syndromes associated with high mortality.
Orgenesis Announces Cell-Based Vaccine Platform Targeting COVID-19 and Other Existing and Emerging Viral Diseases
May 13, 2020 – (Orgenesis) – Orgenesis announced the launch of its new cell-based vaccine platform targeting severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the virus that causes COVID-19, as well as other viral diseases such as Zika, West Nile virus, yellow fever, dengue fever, MERS, HCV and cytomegalovirus infection.
FDA Issues Two Guidances to Accelerate COVID-19 Treatments
May 12, 2020 – (Regulatory Focus) – The FDA issued two guidances intended to accelerate the development of products to treat or prevent COVID-19, laying out recommendations to help companies get to the IND stage and clinical trial design considerations for later-stage studies. As of Monday, FDA said there are 144 active clinical trials for COVID-19 and that it is aware of more than 450 development programs that are still in planning stages.
A Treatment for This Toddler’s Rare Genetic Condition Was in Sight. Then the Pandemic Hit
May 11, 2020 – (Vox) – Even though Amber Freed was racing against time, she was optimistic that her son Maxwell would be treated this year, as experiments on the genetically modified mice were about to begin. But COVID-19 brought most non-coronavirus medical research to a halt, and closed labs.
More Than 1,000 Clinical Trials Were Stopped by the Pandemic – Including Dozens in Phase 3
May 8, 2020 – (Endpoints News) – The vast majority of these studies – 97% – were suspended, a temporary halt that could allow them to resume once the new virus has been tamed and sites reopened. But it may not be that simple.
FDA Emphasizes Clinical Trial Flexibility During Coronavirus Pandemic
May 5, 2020 – (Pharma Intelligence) – Senior FDA representatives from the Office of Tissue and Advanced Therapies, discussed several important considerations for sponsors conducting gene and cell therapy trials during a webcast hosted by ARM. FDA officials have described “around the clock” working hours dedicated to helping trial sponsors navigate a clinical landscape pockmarked with procedural challenges.
CiRA Foundation and the Cell and Gene Therapy Catapult Set to Launch New Stem Cell Collaborative Research
May 12, 2020 – (Cell and Gene Therapy Catapult) – By combining their expertise, the companies will explore novel methods of evaluating cell differentiation and aim to establish reliable tests to predict the potential of iPS cell to differentiation bias, a capability that would help to advance the use of iPS cells for regenerative medicine products.
Japan to Set Price of SMA Gene Therapy Zolgensma at ¥167M
May 12, 2020 – (Nippon) – Japan's health ministry is planning to set the price of Novartis's gene therapy for spinal muscular atrophy at about ¥167M, or approximately $1.55M USD, under the country's health insurance system, informed sources said Tuesday. The treatment is expected to be administered to around 25 patients annually in Japan.
Kriya Therapeutics Announces $80M Series A Financing to Advance Gene Therapies for Highly Prevalent Serious Diseases
May 12, 2020 – (Kriya Therapeutics) – Kriya’s pipeline today includes multiple AAV-based gene therapies for the treatment of type 1 and type 2 diabetes, severe obesity, and other indications.
Noga Therapeutics and Lonza Announce Collaboration to Develop Autologous Lentiviral Gene Therapy on the Cocoon Platform
May 12, 2020 – (Noga Therapeutics) – This collaboration, aimed at the development of an autologous hematopoietic stem cell-based gene therapy for a primary immune deficiency, will leverage Lonza's expertise in process development and the use of its proprietary platform.
bluebird bio Announces Amended BCMA CAR-T Collaboration Agreement With Bristol Myers Squibb
May 11, 2020 – (bluebird bio) – Under the terms of the amended agreement, BMS will buy out its obligations to pay bluebird bio future ex-US milestone and royalty payments for ide-cel and bb21217, the companies’ second BCMA-directed CAR-T immunotherapy, for a one-time upfront payment of $200M.
Dyno Therapeutics Announces Collaborations With Sarepta, Novartis to Develop New Gene Therapy Vectors
May 11, 2020 – (Dyno Therapeutics) – Sarepta Therapeutics and Dyno Therapeutics announced an agreement to develop next-generation AAV vectors for muscle diseases. Dyno could receive more than $40M in upfront, option, and license payments during the research phase of the collaboration. Dyno also announced an ocular collaboration with Novartis to develop improved gene therapies with AAV vectors based on AI technology.
Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma Enter Into Strategic Clinical and Commercial Manufacturing Agreements with bluebird bio
May 11, 2020 – (apceth Biopharma) – The agreement includes late stage drug product manufacturing in Germany and the US for bluebird bio’s LentiGlobin for the treatment of patients with sickle cell disease; expanded commercial drug product manufacturing capacity in Europe for Zyteglo; and expanded clinical and commercial manufacturing capacity for bluebird’s investigational Lenti-D for cerebral adrenoleukodystrophy in Europe.
Thermo Fisher Scientific to Double its Capacity of Viral Vector Manufacturing
May 11, 2020 – (Thermo Fisher) – The $180M project will help to support increasing demand for the development and manufacture of gene therapies and vaccines. Construction of the new 290,000-square-foot facility is expected to be completed in 2022.
Santen Inks $252M License to jCyte Ocular Cell Therapy
May 8, 2020 – (jCyte) – Santen Pharmaceutical has negotiated a $252M ex-US licensing deal for jCyte's jCell, a human retinal progenitor cell therapy initially aimed at treating retinitis pigmentosa. The agreement includes $50M in upfront cash for jCyte plus potential milestone payments and royalties.
Krystal Biotech Announces Positive Interim Results From Phase 1/2 Clinical Trial of KB105 in Patients With TGM1-Related Autosomal Recessive Congenital Ichthyosis
May 14, 2020 – (Krystal Biotech) – KB105-treated areas showed clearly detectable TGM-1 expression in all treated sites following initial and repeat administration. KB105-expressed TGM1 was correctly localized in the epidermis, co-localizing with loricrin, and was functionally active.
Moffitt Cancer Center Study Suggests More Could Benefit From CAR-T Therapy
May 14, 2020 – (Moffitt Cancer Center) – The consortium pooled retrospective data on 298 patients who completed apheresis with the intent of having Yescarta manufactured and administered. The analysis found that the overall response rate of 82%, and estimated 12 month durable response rate of 47%, for the pooled group of patients compared favorably to the ZUMA-1 trial results.
FDA Clears AgenTus IND for Allogeneic iNKT Cell Therapy
May 13, 2020 – (Agenus) – Agenus announced that the FDA accepted their IND application for an allogeneic iNKT therapy, agenT-797, submitted by its subsidiary, AgenTus Therapeutics. AgenTus has also submitted a separate IND for the treatment of COVID-19, which is expected to clear soon.
American Society of Clinical Oncology Abstract Reports Initial ALLO-501 Alpha Phase 1 Data in Relapsed/Refractory Non-Hodgkin Lymphoma
May 13, 2020 – (Allogene) – The ASCO abstract includes preliminary data on the first nine patients treated with escalating doses of ALLO-501 and lower dose ALLO-647. The overall response rate was 78%, with three complete responses and four partial responses.
AVROBIO Reports Updated Clinical Data From Investigational Gene Therapy Programs for Fabry Disease and Cystinosis
May 13, 2020 – (AVROBIO) – Four patients have been dosed in the Phase 2 trial of AVR-RD-01, AVROBIO’s investigational lentiviral gene therapy for Fabry disease. The first patient continued to show increased leukocyte and plasma AGA enzyme activity, now up to 22 months post-treatment. In the Phase 1/2 trial of AVR-RD-04 for cystinosis, the first patient showed evidence of improved kidney function and remains off cysteamine.
Bristol Myers Squibb and bluebird bio Provide Regulatory Update on Idecabtagene Vicleucel (ide-cel, bb2121) for the Treatment of Patients With Multiple Myeloma
May 13, 2020 – (bluebird bio) – Upon preliminary review, the FDA determined that the CMC module of the BLA requires further detail to complete the review. No additional clinical or non-clinical data have been requested or are required. Bristol Myers Squibb is planning to resubmit the BLA no later than the end of July 2020.
Capricor Announces Positive Top-Line Final Results From HOPE-2 Study in Patients with Duchenne Muscular Dystrophy Treated With Lead Candidate CAP-1002
May 13, 2020 – (Capricor) – With the exception of steroids, preservation of function in DMD is uncommon. In the trial, the placebo patients declined consistent with natural history, but in the treated group, most patients were stable or improved throughout the one-year treatment period.
Janssen’s BCMA CAR-T Therapy JNJ-4528 Showed Early, Deep, and Durable Responses in Heavily Pretreated Patients With Multiple Myeloma
May 13, 2020 – (Janssen) – Longer-term follow-up data from Phase 1b/2 CARTITUDE-1 study demonstrate 100% overall response rate, 86% stringent complete response rate at a median of 11.5 months and 86% progression-free survival at 9 months.
Ultragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1/2 Study of DTX301 Gene Therapy in OTC Deficiency
May 13, 2020 – (Ultragenyx) – Six of nine patients in the study have responded to the gene therapy (three female, three male), including all three patients in Cohort 3 who are now confirmed responders. The three previously disclosed complete responders, who have discontinued all ammonia scavengers and liberalized their diet, remain clinically and metabolically stable.
Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies
May 12, 2020 – (Gamida Cell) – Gamida Cell announced positive topline results from its Phase 3 study evaluating the safety and efficacy of omidubicel, a potential treatment option for patients in need of bone marrow transplant. The median time to neutrophil engraftment was 12 days for patients treated with omidubicel compared to 22 days for the control group.
Rocket Pharmaceuticals Presents Positive Updates on FA and LAD-I Gene Therapy Programs at the 23rd Annual Meeting of ASGCT
March 12, 2020 – (Rocket Pharmaceuticals) – Additional results from the first patient treated with RP-L201 for LAD-I continue to demonstrate evidence of safety and potential efficacy. The patient demonstrated peripheral blood VCN levels of 1.3 and CD18-expression of 47%, which is sustained from the 45% expression observed three months post treatment.
CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA RMAT Designation Granted to CTX001 for the Treatment of Severe Hemoglobinopathies
May 11, 2020 – (CRISPR Therapeutics) – CRISPR Therapeutics and Vertex Pharmaceuticals announced that the FDA granted RMAT designation to CTX001, an investigational autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease and transfusion-dependent beta thalassemia.
TolerogenixX Reports Positive Phase 1b Data of Its Lead Product to Combat Transplant Rejection
May 11, 2020 – (TolerogenixX) – In the study, patients showed excellent graft function without rejection or de novo human leukocyte antigen antibodies and retained full immune competence against bacteria and viruses.
In a Medical First, Doctors Treat Parkinson’s With a Novel Brain Cell Transplant
May 12, 2020 – (STAT News) – Since the 2018 surgery, Lopez has received regular brain scans, which suggest that the transplanted cells are churning out dopamine, and have apparently “engrafted,” or made long-term connections with other neurons – including, crucially, in motor regions.
Magnetic Nanopropellers Deliver Genetic Material to Cells
May 8, 2020 – (Max Planck Institute) – Major challenges to using magnetic nanoparticles in biomedicine include unacceptably high toxicity, difficulty of fabrication, low chemical stability, or weak magnetic moments. However, researchers have developed magnetic nanopropellers that can deliver genetic material to cells using a material that outperforms the strongest known micromagnets, yet is chemically stable, non-toxic and biologically compatible.
Gene Therapy in Mice Builds Muscle, Reduces Fat
May 8, 2020 – (Washington University in St. Louis) – The researchers found that gene therapy helped build significant muscle mass quickly and reduced the severity of osteoarthritis in the mice, even though they didn’t exercise more. The therapy also staved off obesity, even when the mice ate an extremely high-fat diet.
CMS Releases FY2021 Medicare Hospital Inpatient Prospective Payment System and Long Term Acute Care Hospital Proposed Rule
May 11, 2020 – (Centers for Medicare and Medicaid Services) – CMS is proposing to create a new MS-DRG specifically for cases involving CAR T-cell therapies. The new payment group would help to predictably compensate hospitals for their costs in delivering necessary care to Medicare beneficiaries and provide payment flexibility for the future as new CAR-T therapies become available.
Deluge of Genome Editing Therapies End Research Drought for Sickle Cell Disease
May 12, 2020 – (STAT News) – As scientists now evaluate multiple genetic strategies, “it’s a great problem to have, especially compared to the [R&D] desert we had,” said sickle cell expert Alexis Thompson of Children’s Hospital of Chicago. The new approaches use CRISPR editing, in different forms and with different targets, but all in hematopoietic stem cells.
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
Business Manager (US)
Ovizio Imaging Systems – US East Coast
Senior Application Scientist – Cell Therapy
Treefrog – Pessac, France
About ARM

The Alliance for Regenerative Medicine (ARM) is the preeminent global advocate for regenerative and advanced therapies. ARM fosters research, development, investment and commercialization of transformational treatments and cures for patients worldwide. By leveraging the expertise of its membership, ARM empowers multiple stakeholders to promote legislative, regulatory and public understanding of, and support for, this expanding field. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit
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