ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
July 30, 2020
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ARM Calls for Multi-Stakeholder Pan-European Initiative to Fast-Track Real World Evidence in Support of Patient Access to Advanced Therapies
July 29, 2020 – (ARM) – ARM calls on the European Commission to fast-track the use of RWE for ATMPs by convening a multi-stakeholder forum, including subject matter experts from ARM, manufacturers and patient advocacy groups, to set up a European infrastructure for regular RWE use.
ARM Releases List of Presenting Companies at the 2020 Cell & Gene Meeting on the Mesa
July 29, 2020 – (ARM) – The Meeting on the Mesa features presentations by 115+ leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the regenerative medicine sector. The annual event will be held October 12-16 via a virtual platform, allowing attendees from around the globe to connect with potential partners over the course of the five-day conference while avoiding any risk to their health.
US FDA Approves Kite’s Tecartus, the First CAR-T Treatment for Relapsed or Refractory Mantle Cell Lymphoma
July 24, 2020 – (Kite, a Gilead Company) – The approval of this one-time therapy follows a priority review and FDA Breakthrough Therapy Designation and is based on results of ZUMA-2, a single-arm, open-label study in which 87 percent of patients responded to a single infusion of Tecartus, including 62 percent of patients achieving a complete response.
AlloVir Announces Pricing of Upsized Initial Public Offering
July 30, 2020 – (AlloVir) – The gross proceeds of the offering are expected to be approximately $276M.
Sangamo Announces Global Collaboration With Novartis to Develop Genomic Medicines for Autism and Other Neurodevelopmental Disorders
July 30, 2020 – (Sangamo Therapeutics) – Under the collaboration agreement, Novartis will pay Sangamo a $75M upfront license fee payment within thirty days. In addition, Sangamo is eligible to earn up to $720M in other development and commercial milestone payments.
Atsena Therapeutics Acquires Exclusive Rights to Gene Therapy for GUCY2D-Associated Leber Congenital Amaurosis
July 29, 2020 – (Atsena Therapeutics) – Atsena acquired exclusive rights from Sanofi, which originally licensed it from the University of Florida. Atsena has an ongoing Phase 1/2 clinical trial evaluating this gene therapy in LCA1 patients. The second cohort in the trial is expected to be dosed in the fall of 2020.
Catalent Enters Into Strategic Partnership With Editas Medicine to Support Gene Editing Medicine Pipeline
July 29, 2020 – (Catalent) – Editas Medicine announced that they have entered into a strategic partnership whereby Catalent will provide support for the development, manufacturing, and clinical supply of Editas Medicine’s current and future portfolio of in vivo CRISPR medicines and engineered cell medicines.
Avacta Announces Expansion of Partnership With Daewoong Pharmaceutical to Include COVID-19 Neutralising Affimer Therapy
July 29, 2020 – (Avacta Group) – Avacta Group announced an expansion of its collaboration and license agreement with Daewoong Pharmaceutical and AffyXell Therapeutics, a joint venture established by the two companies, to develop stem cell treatments for the treatment of seriously ill patients with COVID-19.
TCR2 Therapeutics Announces Pricing of Public Offering of Common Stock
July 28, 2020 – (TCR2 Therapeutics) – The gross proceeds from the offering are expected to be $124M. TCR2 intends to use the net proceeds of the offering to advance its clinical and earlier-stage programs and for research and development, working capital and general corporate purposes.
Cynata Completes Phase 1 GvHD Clinical Trial Follow-Up With Positive Results
July 30, 2020 – (Cynata Therapeutics) – The overall survival rate after two years was 60% (9/15 patients), which compares favorably with previously published outcomes. An overall survival rate of just 17% after two years has been reported in patients with steroid-resistant acute GvHD who received standard of care treatment.
Bristol Myers Squibb and bluebird bio Announce Submission of BLA to FDA for Idecabtagene Vicleucel for Adults With Relapsed and Refractory Multiple Myeloma
July 29, 2020 – (bluebird bio) – This submission provides further details on the Chemistry, Manufacturing and Controls module to address the outstanding regulatory requests from the FDA in May 2020 following the original BLA submission from March 2020.
First COVID-19 Induced ARDS Patient Enrolled in Healios ONE-BRIDGE Study
July 29, 2020 – (Healios) – The new cohort will include an additional approximately five pneumonia-induced ARDS patients with COVID19 as the causative disease and investigate the safety of the therapy in these patients.
Krystal Biotech Announces Initiation of Pivotal Phase 3 Study of Beremagene Geperpavec in Patients with Dystrophic Epidermolysis Bullosa
July 28, 2020 – (Krystal Bio) – The top line data from the study and a BLA filing in the US are anticipated in 2021. The EMA is aligned on the pivotal study design, and an MAA is anticipated shortly after the BLA is filed.
4D Molecular Therapeutics Announces First Patient Dosed in Phase 1 Clinical Trial of 4D-110 by Intravitreal Injection for the Treatment of Choroideremia
July 27, 2020 – (4D Molecular Therapeutics) – The study is designed to assess the preliminary safety, tolerability and biological activity of a single intravitreal injection of 4D-110. In addition, the clinical trial will evaluate the effect of 4D-110 on the visual function and retinal degeneration.
Carisma Therapeutics Announces FDA Clearance of IND Application for First-Ever Engineered Macrophage Immunotherapy
July 27, 2020 – (Carisma Therapeutics) – Under this IND, CARISMA intends to initiate its Phase 1, first-in-human, multi-center study in patients with recurrent or metastatic HER2 overexpressing solid tumors after failure of approved HER2 targeted agents.
jCyte Announces Promising Phase 2b Results of jCell Therapy in Retinitis Pigmentosa
July 27, 2020 – (jCyte) – The study demonstrated mean improvements in best-corrected visual acuity (BCVA) of +7.43 letters (in the higher dose cohort) and +2.96 letters (lower dose cohort) in treated eyes, compared to an improvement of +2.81 letters in sham-treated eyes.
Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative Disease
July 27, 2020 – (Ocugen) – OCU400 has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of NR2E3, expression of which within the retina may help reset retinal homeostasis.
Phase 1/2A Trial of Helixmith's Flagship Gene Therapy to Treat Charcot-Marie Tooth Launches in South Korea
July 27, 2020 – (Helixmith) – In the trial, Engensis will be administered to patients who suffer from muscle loss caused by peripheral nerve damage from CMT disease. The primary efficacy endpoint will compare the change in average daily pain scored on regular intervals in participants treated with Engensis versus placebo.
CAR-T Cell Therapy Shows Promising Results Against Hodgkin Lymphoma
July 24, 2020 – (Baylor College of Medicine) – In the new study, which included 41 patients treated at Baylor and UNC, researchers used an anti-CD30 CAR-T therapy following a preparative regimen in which patients’ existing lymphocytes were greatly depleted with chemotherapy drugs prior to the addition of the CAR-T cells. Researchers found that lymphodepletion seems to produce a more favorable environment for the CAR-T cells.
Sarepta Therapeutics Receives Fast Track Designation for SRP-9001 Micro-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
July 24, 2020 – (Sarepta Therapeutics) – Safety and tolerability data at one year from four clinical trial participants who received SRP-9001 in Study 101 were recently published in JAMA Neurology, and Study 102, a randomized, double-blind, placebo-controlled study of SRP-9001, is ongoing with results expected in early 2021.
Adaptimmune Granted Access to PRIority MEdicines (PRIME) Regulatory Support by the European Medicines Agency for ADP-A2M4 for the Treatment of Synovial Sarcoma
July 23, 2020 – (Adaptimmune) – Access to the PRIME initiative for ADP-A2M4 was granted based on clinical data from the Phase 1 trial demonstrating compelling efficacy and early promising durability, with tolerable safety in patients with synovial sarcoma.
UW Researchers Devise Approach to Treat Rare, Incurable Form of Blindness
July 29, 2020 – (University of Wisconsin-Madison) – The researchers were able to correct the disease in stem cells from patients with BEST1 mutations by overwhelming broken copies of the gene with many functional copies of BEST1.
Getting Gene Therapy to the Brain
July 28, 2020 – (Penn Medicine) – Researchers used an animal model with a brain more similar to humans, cats, to assess the effectiveness of a gene-correcting therapy using a specific vector, which was found to be capable of crossing the blood-brain barrier in mice. They were then able to find evidence that the corrected gene had distributed to various parts of the brain.
A New Cell & Gene Therapy Approach to Treat Common Bleeding Disorder
July 28, 2020 – (Wake Forest) – Researchers demonstrated that human placental cells constitutively secrete low levels of Factor 8, suggesting they may be ideally suited as a cellular platform for delivering and producing Factor 8 to correct hemophilia A.
“Self-Eating” Process of Stem Cells May be the Key to New Regenerative Therapies
July 23, 2020 – (University of Pennsylvania) – Minimal CMA activity allows stem cells to maintain high levels of alpha-ketoglutarate, a metabolite that is crucial to reinforce a cell’s pluripotent state, the researchers found. When it’s time for differentiation, the cells begin to upregulate CMA.
3D Printing and iPS Cells Regenerate Nerves in Rat
July 22, 2020 – (Center for iPSC Research & Application) – Conduits made of mesenchymal stem cells derived from iPS cells regenerated damaged peripheral nerves in rats when grafted at the damaged site.
House Approves $3.2 Billion FDA Budget
July 28, 2020 – (Endpoints) – The House of Representatives passed a four-bill appropriations minibus for fiscal year 2021 that includes an amendment giving the FDA the authority to recall drugs. The agency’s proposed budget also includes targeted boosts to spending for medical product and food safety activities and influenza vaccine manufacturing technologies.
Coordinator, Membership & Public Affairs
Alliance for Regenerative Medicine – Washington, DC
Senior Director – Cell & Gene Therapy Sales
Be The Match BioTherapies – Boston, MA
Director, CMC Regulatory Affairs
Century Therapeutics – Greater Philadelphia, PA
Business Development Manager, Strategy & Technology
Corning – Remote (Boston, MA; Mid-Atlantic; or San Francisco, CA)
Executive Director
Standard Coordinating Body for Regenerative Medicine – Gaithersburg, MD
About ARM

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory and reimbursement initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 11-year history, ARM has become the voice of the sector, representing the interests of 360+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit
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