ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
March 13, 2020
Quick Links
ARM Releases 2019 Annual Report & Sector Year in Review
March 5, 2020 – (ARM) – ARM announced the release of its 2019 Annual Report and Sector Year in Review, highlighting the organization’s key priorities and initiatives, as well as offering an in-depth look at trends and metrics for the cell therapy, gene therapy, and tissue engineering sectors.
Update on ARM's 2020 Cell and Gene Meeting on the Mediterranean →
Novartis Sidesteps Europe Travel Ban to Provide CAR-T Drug Kymriah to Patients
March 13, 2020 – (Fierce Pharma) – Novartis in an emailed statement says it has found alternate methods to ship its CAR-T drug Kymriah after the travel ban affected passenger flight services from Europe. It says it is confident its “existing manufacturing supply chain” can handle distribution.
TIKOMED Secures €5M in Funding for the Clinical Development of ILB and IBSOLVMIR
March 13, 2020 – (TIKOMED) – The capital will primarily be used to support the ongoing clinical development program for ILB and IBSOLVMIR and advancing the company’s position within the treatment of acute and degenerative neurological diseases and cell transplantation.
EXUMA Biotech Announces New Round of Financing to Advance Cellular Therapies
March 12, 2020 – (EXUMA Biotech) – EXUMA Biotech announced a $19M Series B round of financing that includes new investments by MSD Partners and F1 BioVentures, as well as conversion of notes held by individual investors.
Lysogene Completes €7.7M Capital Increase in a Placement Led by Leading Healthcare Investor OrbiMed and Lysogene Shareholder and Partner Sarepta
March 12, 2020 – (Lysogene) – Proceeds from the transaction will be used to finance the Phase 1-3 clinical trial of LYS-GM101 for the treatment of GM1 gangliosidosis, the commercial launch preparation in Europe of the LYS-SAF302 in MPSIIIA, and the company's overhead costs and expenses.
Forty Seven and Rocket Pharmaceuticals Announce Research Collaboration for Fanconi Anemia
March 11, 2020 – (Rocket Pharmaceuticals) – Under the terms of the agreement, Rocket will provide its ex vivo LVV HSC gene therapy platform and Forty Seven will contribute its innovative antibody-based conditioning regimen for the collaboration.
InVivo Therapeutics Announces Closing of $7M Public Offering
March 11, 2020 – (InVivo Therapeutics) – The gross proceeds from this offering were approximately $7M, before deducting the placement agent fees and estimated offering expenses payable by InVivo Therapeutics.
J&J Partner Legend Plans IPO to Fund CAR-T Pipeline
March 10, 2020 – (Fierce Biotech) – Genscript disclosed the plan in a filing with the Hong Kong stock exchange that revealed Legend has submitted IPO paperwork to the US SEC. The filing is confidential, for now, meaning key details of how much Legend hopes to raise and why remain under wraps. 
Kuur Therapeutics Launches to Develop and Commercialize “Off-the-Shelf” CAR-NKT Cell Therapies Targeting Hematological and Solid Tumors
March 10, 2020 – (Kuur Therapeutics) – Kuur Therapeutics today announced the launch of its new business in partnership with Baylor College of Medicine and Baylor’s Center for Cell and Gene Therapy. Houston-based Kuur Therapeutics will advance the work of its predecessor, Cell Medica, to develop anti-cancer therapies using its innovative CAR-NKT therapy platform.
Phio Pharmaceuticals and Helmholtz Zentrum München Announce Collaboration and Option Agreement With Medigene
March 10, 2020 – (Medigene) – Under the terms of the new agreement, Medigene will contribute expertise regarding clinical development as well as proprietary research material and has an option to an exclusive license for the clinical and/or commercial exploitation of the potential immune cell enhancers against certain fee payments.
WindMIL Therapeutics and Cognate BioServices Announce Agreement to Expand MILs Manufacturing Capacity
March 10, 2020 – (WindMIL Therapeutics) – The initial contract between the two companies enabled the successful completion of tech transfer and the start of clinical manufacturing in 2019. The expanded agreement will substantially increase WindMIL’s clinical manufacturing footprint at Cognate.
Passage Bio Announces Exercise of Underwriters’ Option to Purchase Additional Shares, Bringing Total Gross Proceeds to $248M
March 9, 2020 – (Passage Bio) – Passage Bio announced that the underwriters of its previously announced IPO of 12,000,000 shares of common stock have exercised their option to purchase additional shares of common stock at the public offering price of $18 per share for gross proceeds of $32.4M and total offering gross proceeds of $248.4M.
BrainStorm Cell Therapeutics Raises $10M in Common Stock Sale
March 6, 2020 – (BrainStorm Cell Therapeutics) – BrainStorm intends to use the proceeds from this transaction to advance the company’s clinical programs; commercial production of the investigational therapeutic NurOwn; regulatory, pre-marketing and commercialization preparation activities of NurOwn for ALS; working capital, and general corporate purposes.
MolMed Signs a Multi-Year Agreement and Announces the Beginning of a New Collaboration in the Field of T Cell Therapies
March 6, 2020 – (MolMed) – Under the multi-year agreement with Autolus Therapeutics, MolMed will develop and supply viral vectors for the manufacture of certain Autolus’s CAR-T programs for use in clinical trials and potentially for commercial sale.
Amicus Opens New Global Research and Gene Therapy Center of Excellence in Philadelphia
March 5, 2020 – (Amicus Therapeutics) – The 75,000 sq. ft. center consists of office and state-of-the-art laboratories. It will ultimately house approximately 200 researchers and drug developers focused exclusively on gene therapies. 
Akouos Closes $105M Series B Financing
March 3, 2020 – (Akouos) – Proceeds from the Series B will be used to advance Akouos’s lead program, AK-OTOF, to first-in-human clinical studies subject to IND filing and acceptance; establish the company’s in-house GMP manufacturing capabilities; accelerate the development of multiple pipeline programs; and expand its team across research, clinical development and manufacturing.
Avectas and CCRM Announce Collaboration to Accelerate Development of Novel Technology for Cell Therapies
March 3, 2020 – (Avectas) – Avectas and CCRM announced they have entered into a collaboration to accelerate the translation of Avectas's non-viral cell engineering platform, Solupore, into the clinic. Solupore is designed for use with mRNA, DNA, and proteins, including gene editing tools such as CRISPR.
Fujifilm Cellular Dynamics Begins Operations for Advanced Cell Therapies at Its New Facility
March 2, 2020 – (Fujifilm Cellular Dynamics) – The new facility, i-FACT, will house development, and manufacture of iPSCs for therapeutic applications. The i-FACT will enable FCDI to accelerate the development of its own pipeline of regenerative medicine therapies using iPSCs.
Marker Therapeutics and Aspire Capital Enter Into a Common Stock Purchase Agreement for up to $30M
March 2, 2020 – (Marker Therapeutics) – Under the terms of the Agreement, Aspire has committed to purchase up to $30M of the company’s common stock at Marker’s discretion. The transaction will further support the advancement of the company's clinical programs including the first Marker-sponsored clinical trial this year investigating the company's MultiTAA cell therapy.
Biogen and Sangamo Announce Global Collaboration to Develop Gene Regulation Therapies for Alzheimer’s, Parkinson’s, Neuromuscular, and Other Neurological Diseases
February 27, 2020 – (Sangamo Therapeutics) – Biogen and Sangamo Therapeutics announced that they have executed a global licensing collaboration agreement to develop and commercialize ST-501 for tauopathies including Alzheimer’s disease, ST-502 for synucleinopathies including Parkinson’s disease, a third undisclosed neuromuscular disease target, and up to nine additional undisclosed neurological disease targets.
Arcellx Announces FDA Orphan Drug Designation for CART-ddBCMA for the Treatment of Multiple Myeloma
March 12, 2020 – (Arcellx) – Arcellx announced that the US FDA has granted Orphan Drug Designation to CART-ddBCMA, its engineered T cell therapy utilizing a novel binding domain, for the treatment of patients with multiple myeloma. Arcellx continues to enroll patients in its Phase 1 trial of CART-ddBCMA therapy for the treatment of relapsed and refractory multiple myeloma.
Freeline Receives Orphan Drug Designation From the European Commission for FLT190 for the Treatment of Fabry Disease
March 10, 2020 – (Freeline Therapeutics) – Preliminary data showed a 3 to 4-fold increase in plasma αGLA activity was achieved by week 4 and sustained through the data cutoff at week 20.
AVROBIO Receives Orphan Drug Designation from US FDA for AVR‑RD‑04 for Cystinosis
March 9, 2020 – (AVROBIO) – A Phase 1/2 clinical trial to evaluate the safety and efficacy of the investigational gene therapy in patients with cystinosis is ongoing.
PsiOxus Therapeutics Announces Clinical Trial with Third Cancer Gene Therapy Treatment
March 5, 2020 – (PsiOxus) – The Phase 1 STAR study is being conducted at multiple cancer centers in the United States and will assess the safety, tolerability and preliminary anti-tumor activity of NG-641 in subjects with solid tumors.
Allergan and Editas Medicine Announce Dosing of First Patient in Landmark Phase 1/2 Clinical Trial of CRISPR Medicine AGN-151587 (EDIT-101) for the Treatment of LCA10
March 4, 2020 – (Editas) – AGN-151587 (EDIT-101) is an experimental medicine under development for the treatment of Leber congenital amaurosis 10. The BRILLIANCE clinical trial is a Phase 1/2 study and is the world’s first human study of an in vivo CRISPR genome editing medicine. 
DiscGenics Announces Completion of Enrollment in U.S. Phase 1/2 Clinical Trial of Discogenic Cell Therapy for Degenerative Disc Disease
March 4, 2020 – (DiscGenics) – This prospective, randomized, double-blinded, vehicle- and placebo-controlled, multicenter clinical study is designed to evaluate the safety and efficacy of IDCT in patients with symptomatic, single-level, mild to moderate lumbar DDD.
PDC*Line Pharma Announces the Administration of a First Patient With Its Therapeutic Cancer Vaccine Candidate (PDC*lung01) Targeting Non-Small Cell Lung Cancer
March 3, 2020 – (PDC*Line Pharma) – A total of 62 evaluable HLA-A*02:01 positive NSCLC patients are expected in three clinical centers in Belgium and six in France.
MeiraGTx Announces Priority Medicines (PRIME) Granted by the European Medicines Agency to AAV-RPGR Gene Therapy for the Treatment of X-Linked Retinitis Pigmentosa
March 2, 2020 – (MeiraGTx) – MeiraGTx and Janssen Pharmaceuticals are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.
Prevail Therapeutics Announces IND Application Active for PR006 for the Treatment of Frontotemporal Dementia with GRN Mutation
March 2, 2020 – (Prevail Therapeutics) – Prevail Therapeutics announced that the FDA has accepted the company’s IND application for its experimental gene therapy program, PR006, for the treatment of frontotemporal dementia patients with GRN mutation. The company plans to begin dosing for the trial this year.
Safer Way of Editing Gene Mutations
March 13, 2020 – (Center for iPSC Research and Applications) – Researchers have developed a new molecular delivery system, NanoMEDIC, to insert genome editing protein tools into cells to correct gene mutations. In initial tests, the rate of untargeted gene editing was much lower than other gene editing delivery methods, suggesting a major increase in safety.
New Method to Grow Human Blood Vessels
March 11, 2020 – (University of Minnesota) – Researchers proved the ability to grow human-derived blood vessels in a pig – a novel approach that has the potential for providing unlimited human vessels for transplant purposes. Because these vessels were made with patient-derived skin cells, they are less likely to be rejected by the recipient. 
Gene Therapy Reverses Heart Failure in Mouse Model of Barth Syndrome
March 9, 2020 – (Boston Children's Hospital) – Tests showed that TAZ gene therapy provided durable treatment of the animals' cardiomyocytes and skeletal muscle cells, but only when at least 70 percent of heart muscle cells had taken up the gene.
'Primitive' Stem Cells Shown to Regenerate Blood Vessels in the Eye
March 9, 2020 – (Johns Hopkins) – Scientists injected cells called vascular progenitors into the eyes of mice bred to have a form of diabetic retinopathy. They found that the naive vascular progenitors migrated into the retina’s innermost tissue layer that encircles the eye, with higher efficiencies than have been reported with vascular cells made from conventional stem cell approaches. 
From Scorpion to Immunotherapy: City of Hope Scientists Repurpose Nature’s Toxin for First-Of-Its Kind CAR T Cell Therapy to Treat Brain Tumors
March 4, 2020 – (City of Hope) – The CLTX-CAR uses a 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain. Scientists found that CLTX bound to a greater proportion of patient tumors, and cells within these tumors.
Danish Regulator Eyes Sanctions – Including Prison Time – Against Trial Sponsors That Fail to Post Results
March 11, 2020 – (STAT News) – Under the current legal framework in Denmark, clinical trial sponsors that do not report results on time can be fined or given a prison sentence of up to four months. Although some drug makers and universities responded to a reminder warning issued last fall, most trial results have still not been posted as required and so the agency will approach the public prosecutor about penalties.
With Second Patient Cured of HIV, Doctors Seek Clues to a Future AIDS Treatment
March 10, 2020 – (CBS News) – Two patients with HIV have been functionally cured through the use of a bone marrow transplant from donors with CCR5, a genetic mutation that provides resistance to HIV, and a third patient also appears be HIV-free. While these patients received the transplants to treat hematological malignancies, it points to the possibility that a gene therapy to treat HIV could provide a similar effect.
Vice President, Global Policy and Advocacy
Alliance for Regenerative Medicine – Washington, DC
Manager, Clinical Quality
Abeona Therapeutics – Cleveland, OH
Director, Regulatory Affairs
Cardinal Health – Remote
Senior Research Scientist
Kytopen – Cambridge, MA
Head of Clinical Pharmacology
Magenta Therapeutics – Cambridge, MA
Medical Director / Senior Medical Director
Magenta Therapeutics – Cambridge, MA
Senior Research Associate, Immunology / Stem Cell Biology 
Magenta Therapeutics – Cambridge, MA
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
Regenerative Medicine Forum
Alliance for Regenerative Medicine
Alliance for Regenerative Medicine
Subscribe to Newsletter
Become a Member
Event Calendar
Copyright © 2020 AllianceRM, All rights reserved.

Want to change how you receive these emails?
You can update your preferences or unsubscribe.