ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
April 2, 2020
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ARM Launches COVID-19 Resource Library, a One-Stop Resource for Regenerative Medicine Stakeholders
March 30, 2020 – (ARM) – The Resource Library compiles information from regulatory bodies, nonprofits, media outlets, and other sources worldwide on clinical trial guidance; regulatory news and updates; financial and economic resources for businesses; policy initiatives; shelter in place orders; payer communications; and patient resources. The webpage also provides information on the role regenerative medicine developers may have in developing therapeutic candidates to treat COVID-19 and related complications.
Celularity to Test Natural Killer Cell Therapy for Cancer Against COVID
April 2, 2020 – (Celularity) – The trial, designed to evaluate Celularity drug candidate CYNK-001, will enroll about 100 patients with COVID-19 infection causing pneumonia. Those enrolled will receive infusions of natural killer (NK) cells, a type of white blood cell, which the company anticipates could kill cells infected with the virus and address the resulting inflammation caused by the immune system.
Citius Signs Exclusive Option with Novellus to License Novel Stem-Cell Therapy for Acute Respiratory Distress Syndrome (ARDS) Associated with COVID-19
April 1, 2020 – (Citius Pharmaceuticals) – Novellus's patented process uses its exclusive non-immunogenic synthetic mRNA molecules to create iPSCs that, in turn, generate mesenchymal stem cells with superior immunomodulatory properties.
Coronavirus Emergency Loans: Information for Small Businesses
April 1, 2020 – (US Chamber of Commerce) – The small business emergency loan program, established by the Coronavirus Aid, Relief, and Economic Security (CARES) Act, will go live tomorrow, Friday, April 3. Loans will only be available until funds are exhausted, so the government is advising borrowers to apply as soon as possible.
CRISPR Therapeutics Braces for 'Severe Impact' From COVID-19 Crisis
April 1, 2020 – (Fierce Biotech) – In an SEC filing Tuesday, the biotech said: “We are conducting a number of clinical trials for product candidates in the fields of severe hemoglobinopathies and immuno-oncology in geographies which are affected by the coronavirus pandemic. We believe that the coronavirus pandemic has had, and will likely continue to have, an impact on various aspects of our clinical trials."
GC LabCell to Develop COVID-19 Treatment Using NK Cell Therapy
March 30, 2020 – (Korea BioMedical Review) – Green Cross LabCell said that it has begun research into a COVID-19 medication, and expects to start a clinical trial by the second half of 2020 in Korea and the United States.
Pluristem Treated First Three COVID-19 Patients in Israel Under Compassionate Use
March 30, 2020 – (Pluristem) – Pluristem announced that it has dosed three patients in two different hospitals in Israel under a compassionate use program for the treatment of COVID-19. Pluristem expects to enroll additional patients in Israel in the coming days and anticipates providing updates on clinical outcomes once significant data has been gathered.
Biotech in the Time of the Coronavirus: With China Returning to Work, Preclinical Companies Ramping Back Up
March 27, 2020 – (STAT News) – Two months ago, everybody was worried about slowdowns because of China. Now, it may be that those Chinese CROs help keep the trains going on early-stage, drug discovery programs.
Coronavirus Delays bluebird bio Gene Therapy Zynteglo's EU Launch, US Filing
March 27, 2020 – (Fierce Pharma) – bluebird bio said it’s now expecting to dose the first commercial patient with Zynteglo (LentiGlobin) in Germany in the second half of 2020, rather than in the first half of the year as the company communicated earlier.
CIRM Board Approves $5 Million in Emergency Funding for COVID-19 Research
March 27, 2020 – (CIRM) – Only projects that target the development or testing of a treatment for COVID-19 are eligible. They must also meet other requirements including being ready to start work within 30 days of approval and propose achieving a clear deliverable within six months. The proposed therapy must also involve a stem cell or a drug or antibody targeting stem cells.
For CAR-T, Coronavirus Brings Uneven Impact to Studies, Treatment
March 27, 2020 – (BioPharma Dive) – While Bristol Myers and Servier have announced enrollment halts, Gilead and Novartis, two other leading companies in the field, said their studies are proceeding for now. 
Evotec Expands Its IPSC-Based Cell Therapy Platform Evocells Through Licensing Agreement With PanCELLa
April 2, 2020 – (Evotec) – Under the terms of the agreement, Evotec will receive a non-exclusive license to access panCELLa’s proprietary iPS cell lines "iACT Stealth Cells," which are genetically modified to prevent immune rejection of derived cell therapy products
GPB Scientific Announces $25.5M Financing from Vensana Capital and Amgen Ventures
April 2, 2020 – (GPB Scientific) – GPB Scientific announced a $25.5M funding commitment. The financing is led by Vensana Capital alongside strategic investor Amgen Ventures with ongoing participation by existing investors.
Kite Licenses Antibodies and Establishes Collaboration With Teneobio in Multiple Myeloma
April 2, 2020 – (Gilead) – Kite, a Gilead Company, and Teneobio announced the companies have entered into a license and collaboration agreement through which Kite will receive exclusive rights to certain antibodies directed to B-cell maturation antigen. Kite and Teneobio will also collaborate on the discovery of antibodies directed to four additional targets to be used in CAR-T therapies for multiple myeloma and other cancers.
Aspen Neuroscience Announces $70M Series A Financing to Advance Development of the First Autologous Neuron Replacement Therapy to Treat Parkinson Disease
April 1, 2020 – (Aspen Neuroscience) – Aspen's lead product, ANPD001, is currently undergoing investigational new drug (IND)-enabling studies for the treatment of sporadic forms of Parkinson disease. Aspen's second product, ANPD002, combines gene correction and autologous neuron therapy for the treatment of genetic forms of Parkinson disease.
Affinia Therapeutics Raises $60M in Series A Financing to Advance Rational Design AAV Platform and Transformative Gene Therapies
March 31, 2020 – (Affinia Therapeutics) – The proceeds will be used to advance the company’s platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need. The company’s technology was licensed from Lonza and Massachusetts Eye and Ear.
REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV Technology Platform for the Treatment of Rare Metabolic Disorder
March 31, 2020 – (Ultragenyx) – Under the terms of the agreement, REGENXBIO has granted Ultragenyx an exclusive, worldwide license to REGENXBIO's NAV AAV8 and AAV9 vectors for the development and commercialization of gene therapy for a rare metabolic disorder. REGENXBIO will receive an upfront payment of $7M, ongoing fees, milestone payments, and royalties.
Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology
March 31, 2020 – (Ultragenyx) – Under the terms of the agreements, Daiichi Sankyo will make an upfront payment of $125M and will purchase $75M of Ultragenyx common stock. Daiichi Sankyo will pay an additional $25M upon completion of the technology transfer of the HeLa PCL and HEK293 platforms as well as single-digit royalties on net sales of products manufactured in either system. 
ElevateBio Closes $170M Series B Financing
March 30, 2020 – (ElevateBio) – The Series B proceeds will enable ElevateBio BaseCamp to be fully operational with cGMP manufacturing; the advancement of clinical development with at least six cell and gene therapies from across its portfolio companies to enter clinical studies this year and into 2021; key milestones and continued development of ElevateBio’s enabling technology platforms.
No Sanctions for Novartis as FDA Ends Review of Gene Therapy Violations
March 30, 2020 – (BioPharma Dive) – The FDA has closed out an investigation of data violations tied to Novartis' gene therapy Zolgensma. Based on its review of the information available, FDA continues to find Zolgensma to be safe and effective for its intended use.
FDA Accepts Mesoblast’s Biologics License Application for Ryoncil and Agrees to Priority Review
April 1, 2020 – (Mesoblast) – Mesoblast announced that the US FDA has accepted for priority review the company’s BLA filing for Ryoncil (remestemcel-L), its allogeneic cell therapy for the treatment of children with steroid-refractory acute graft versus host disease. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2020, and if approved, Mesoblast will make Ryoncil immediately available in the US.
FDA Accepts IND for CRISPR-Based Sickle Cell Disease Therapeutic Candidate Developed Under Collaboration with Intellia Therapeutics
March 31, 2020 – (Intellia Therapeutics) – Intellia Therapeutics announced that the US FDA has accepted the IND application submitted by its collaborator, Novartis, for a CRISPR/Cas9-based engineered cell therapy for the treatment of sickle cell disease. Novartis’ IND application triggered a milestone payment to Intellia, and the company is eligible to receive additional downstream success-based milestones and royalties.
Bristol Myers Squibb and bluebird bio Announce Submission of BLA for Anti-BCMA CAR-T Therapy Ide-cel (bb2121) to FDA
March 31, 2020 – (bluebird bio) – Ide-cel was granted Breakthrough Therapy Designation by the FDA and PRIME designation by the European Medicines Agency for relapsed and refractory multiple myeloma.
Daiichi Sankyo Submits Application for CAR T Therapy Axicabtagene Ciloleucel (Yescarta) for Treatment of Patients with Certain Relapsed/Refractory B-Cell Lymphomas in Japan
March 30, 2020 – (Daiichi Sankyo) – The NDA submission is based on previous pivotal trial data and a Phase 2 study in Japan in patients with certain relapsed/refractory B-cell lymphomas. Daiichi Sankyo has exclusive rights to axicabtagene ciloleucel in Japan, where it has received Orphan Drug Designation.
Could CAR-T Version 2.0 Be on the Horizon?
March 31, 2020 – (Children's Hospital Los Angeles) – Researchers engineered a T-cell that targets three antigens on cancer cells instead of one. Their study shows that these new CAR-T cells could be the answer to resistant leukemia.
New In Vivo Priming Strategy to Train Stem Cells Can Enhance Cardiac Repair Effectiveness
March 27, 2020 – (City University of Hong Kong) – Researchers found that the hMSCs genetically engineered to have human hepatocyte growth factor protein had a higher survival rate compared with unprimed ones in the patches attached to the failing hearts.
OPINION: Potential Inequities in New Medical Technologies
March 28, 2020 – (Scientific American) – How can we develop and use these tools to build a more equitable future and make them available to all, not just to the most privileged? The first step is to work with those who have been abused, neglected, and left behind by the health care system to build trust in new science like CRISPR and gene therapies and the treatments they ultimately may yield.
OPINION: How Medicare Is Limiting Cancer Care for Seniors
March 27, 2020 – (Morning Consult) – Every day, medical advancements inch us closer to beating this devastating disease. However, policymakers must ensure that no patient is cut off from today’s best treatments or tomorrow’s future innovations — especially some of our most vulnerable seniors.
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
Manager, Clinical Quality
Abeona Therapeutics – Cleveland, OH
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
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