ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
January 9, 2020
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ARM's 10th Annual Cell & Gene Therapy State of the Industry Briefing Takes Place Next Monday, January 13, 8:00-9:50am PT
January 9, 2020 – (ARM) – The State of the Industry Briefing – which is free and open to the public – is the largest cell and gene therapy-focused event taking place during JP Morgan week. If you'll be in San Francisco, it's not too late to register! For those of you who can't make it in person, a live webcast will be available here. You can also follow along on Twitter – we'll be live-tweeting at #SOTI20.
Aspect Biosystems Announces $20M Series A Financing to Advance Its Leading Platform for 3D Bioprinting of Human Tissue
January 9, 2020 – (Aspect Biosystems) – Aspect will use this capital to advance multiple tissue therapeutic programs, expand its technology platform capabilities, and grow its world-leading team.
Genethon Strengthens Its Collaboration With Sarepta Therapeutics for the Development of the Gene Therapy Product GNT0004 in Duchenne Muscular Dystrophy
January 9, 2020 – (Genethon) – Following an initial research and development collaboration on an innovative gene therapy product using micro-dystrophin, Genethon and Sarepta Therapeutics have now started a co-development clinical program for Duchenne muscular dystrophy.
Ori Biotech Announces a $9.4M Seed Round to Advance Innovation in Cell and Gene Therapy Manufacturing
January 9, 2020 – (Ori Biotech) – Ori Biotech announced that they successfully closed a $9.4M (£7M) seed round which will be used to bring their innovative manufacturing platform to market. The Ori platform will deliver scalable solutions to flexibly address the critical clinical and commercial manufacturing needs of CGT developers.
Tacitus Therapeutics Launches in Collaboration with Mount Sinai to Develop Stem Cell Therapies for Life-Threatening Diseases
January 9, 2020 – (Tacitus Therapeutics) –Their first therapy, HSC100, is currently being investigated in a Phase 1 clinical trial for treatment of hematological malignancies.
Eyevensys Closes $30M Series B Financing
January 8, 2020 – (Eyevensys) – The company will use the funds to continue the development of its clinical lead candidate EYS606 for the treatment of chronic non-infectious uveitis, including the launch of its Electro Study.
Aegle Therapeutics Announces $4M Financing to Fund Groundbreaking Stem Cell Exosome Clinical Trial to Treat Dystrophic Epidermolysis Bullosa
January 7, 2020 – (Aegle Therapeutics) – Aegle's IND for the treatment of DEB patients has been cleared by the FDA. The company plans to begin clinical trials in DEB in the first half of 2020.
Bristol-Myers Unloads Old-School Plant as It Readies for CAR-T Future
January 7, 2020 – (Fierce Pharma) – Tidying up business left from last year, Bristol-Myers Squibb has completed the sale of an old-school operation in Italy even as it prepares to move into a whole new manufacturing arena after its $74B buyout of Celgene. The New York-based drugmaker said that the 208,000-square-foot facility in Anagni, Italy now belongs to Catalent.
Cytovia Therapeutics and the University of California, San Francisco Enter Into a Partnership to Develop Precision Gene-Edited CAR-NK Cell Therapy
January 6, 2020 – (Cytovia Therapeutics) – Cytovia Therapeutics and UCSF announced that they have entered a 3-year research partnership to develop novel precision gene editing to improve the performance, safety, and persistence of NK cells and CAR-NK cells.
RootPath Raises $11M Series A to Accelerate Clinical Translation of its Proprietary Synthetic Immunology Platform
January 6, 2020 – (RootPath) – The company will use the Series A proceeds to validate its tumor-reactive T-cell receptor discovery workflow, select a lead therapeutic candidate, demonstrate its safety and efficacy in preclinical models, and begin IND-enabling development activities.
Charles River Laboratories Completes the Acquisition of HemaCare Corporation
January 3, 2020 – (Charles River Laboratories) – Charles River Laboratories International announced that it has completed the previously announced acquisition of HemaCare Corporation for approximately $380M. Acquiring HemaCare expands Charles River’s scientific capabilities in the emerging, high-growth cell therapy sector.
Acepodia Announces FDA Clearance of IND for Its NK Cell Therapy Drug Candidate ACE1702 to Treat Patients with HER2-expressing Solid Tumors
January 9, 2020 – (Acepodia) – ACE1702 is a potential off-the-shelf cell therapy developed using Acepodia’s Antibody-Cell Conjugation technology. The ACC platform is able to link any antibody, including those that have already proven effective in targeting tumors, to Acepodia's natural killer cell line without the need for genetic engineering.
AGTC Reports Positive Six-Month Data from Its Ongoing Phase 1/2 Clinical Trial in X-Linked Retinitis Pigmentosa
January 9, 2020 – (AGTC) – The results show that all nine patients treated centrally had stable or improving visual acuity, with 78% seeing a 5 letter or more improvement, a result not reported in other XLRP trials.
Aruvant Receives FDA Rare Pediatric Disease Designation for ARU-1801 for the Treatment of Sickle Cell Disease
January 8, 2020 – (Roivant) – ARU-1801 has been designed to deliver a highly potent gene payload with enhanced anti-sickling properties, allowing for a Reduced Intensity Conditioning regimen.
Promethera Announces Initiation of Phase 2b DHELIVER Study of HepaStem in Patients With Acute-on-Chronic Liver Failure (ACLF)
January 8, 2020 – (Promethera) – The trial is open for recruitment and aims to include 363 patients with ACLF at 110 study sites across 22 countries in Europe. Topline results are expected to be released at a medical conference at the end of 2023.
Gracell Initiates Investigational Study of TruUCAR Therapy for Relapsed or Refractory T-cell Malignancies
January 7, 2020 – (Gracell) – Gracell's proprietary TruUCAR technology does not require co-administration of anti-CD52, a cytotoxic agent for ablating cancerous cells while inducing long term immune depletion in the patient. Instead, GC027 utilizes CRISPR genome editing strategy that is expected to avoid graft-versus-host disease as well as graft rejection.
Japanese Clinical Study of DiscGenics' Cell Therapy for Disc Degeneration Passes Initial Safety Review
January 7, 2020 – (DiscGenics) – The DSMC reported there were no safety issues and recommended that the study continue with no changes to the protocol.
Gene Therapy Shown to Offer Long-Term Benefits for People With Hemophilia A
January 6, 2020 – (Queen Mary University of London) – Researchers confirmed that all the patients involved in the trial were still benefiting from a substantial fall in the rates of bleeding three years after receiving the treatment. None of the 13 patients had required regular factor VIII to prevent bleeding during that period.
Precigen Receives FDA Orphan Drug Designation for PRGN-3006 UltraCAR-T in Patients with Acute Myeloid Leukemia
January 6, 2020 – (Precigen) – Precigen announced in Q3 2019 that it had completed enrollment for the first cohort of this clinical trial and expects an initial data readout in the 2H 2020.
Virus Surfaces Help Engineers Study Vaccine and Gene Therapy Applications
January 8, 2020 – (Michigan Technological University) – To improve manufacturing for vaccines and gene therapy, a team of researchers at Michigan Tech used surface charge to determine the isoelectric point of different viruses.
Nanoparticles Deliver ‘Suicide Gene’ Therapy to Pediatric Brain Tumors Growing in Mice
January 8, 2020 – (Johns Hopkins) – Researchers found that a combination of the suicide gene and ganciclovir delivered by intraperitoneal injection to mice killed more than 65% of the two types of pediatric brain tumor cells.
Quest to Use CRISPR Against Disease Gains Ground
January 6, 2020 – (Nature) – Medical applications of CRISPR–Cas9 had a banner year in 2019. In the coming years, researchers are looking ahead to more sophisticated applications of CRISPR genome editing that could lay the foundation for treating an array of diseases, from blood disorders to hereditary blindness.
New Technology Allows Control of Gene Therapy Doses
December 26, 2020 – (Scripps Research) – Researchers demonstrated that they could suppress expression of a gene to very low levels with a special embedded molecule, and could then increase the gene’s expression, over a wide dynamic range, using injected control molecules called morpholinos that the FDA has found to be safe for other applications. 
5 Trends to Watch at the FDA in 2020
January 9, 2020 – (BioPharma Dive) – Can FDA continue to deliver, while preventing therapies of more dubious value from reaching market? And can it do so while being asked to implement new initiatives like the White House's drug importation plans? And what mark will new Commissioner Stephen Hahn make on the agency?
A New Biotechnology and Pharmaceutical Industry Commitment to Patients and the Public
January 8, 2020 – (STAT News) – Signed by more than 200 biopharma CEOs and industry leaders, the statement includes a commitment to patient access by pricing therapies to reflect innovation and value to patients; ensuring price increases are reasonable and necessary to promote innovation; and supporting generic and biosimilar approvals of safe and effective medicines after legitimate patent and regulatory protections expire.
Director, Clinical & Regulatory Affairs
DiscGenics – Salt Lake City, UT
Marketing and Communications Manager
International Society for Cell & Gene Therapy – Vancouver, Canada
Translational Research Director
Project 8 – Remote
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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