ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
July 23, 2020
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Pharmaceutical Strategy Could Save European ATMP Development
July 22, 2020 – (Pink Sheet – Subscription Only) – “We really want to ensure that Europe remains a leader in ATMPs [...] We think the new pharma strategy is really an opportunity to get in and get this infrastructure right.” ARM's Paige Bischoff, SVP, Public Affairs, discusses the organization's response to the EU Pharmaceutical Roadmap, which could accelerate ATMP development in the EU if it addresses existing obstacles.
Encoded Therapeutics Announces $135M Series D Financing to Advance Pipeline of Gene Therapies for Debilitating Neurological Disorders
July 22, 2020 – (Encoded Therapeutics) – The company also announced that its lead asset, ETX101, was granted Orphan Drug designation and Rare Pediatric Disease designation by the FDA for the treatment of SCN1A+ Dravet Syndrome. Encoded plans to initiate Phase 1 clinical trials of ETX101 in 2021.
Vesigen Therapeutics Launches With $28.5M Series A Investment Led by Leaps by Bayer and Morningside Ventures
July 22, 2020 – (Vesigen Therapeutics) – Vesigen’s ARMMs technology is intended to enable translation of new modalities such as RNAs and gene-editing complexes into novel treatments for multiple diseases. Vesigen will use the capital raised to build out the ARMMs platform as well as to advance numerous therapeutic agents into preclinical and clinical development.
Forge Biologics Launches With $40M Series A Financing to Manufacture and Develop Gene Therapies
July 21, 2020 – (Forge Biologics) – Forge will use the proceeds of this financing to expand AAV manufacturing CDMO capabilities in 2020, with cGMP production capacity available by mid-2021, and for the development of a novel gene therapy pipeline.
UCLB and Apollo Therapeutics Secure First Out-Licensing Deal to Deerfield, a Leading US Healthcare Investment Firm
July 21, 2020 – (Apollo Therapeutics) – UCL Business and Apollo Therapeutics, along with Deerfield Management Company, have completed the licensing of the first Apollo-supported project, a novel gene therapy program developed at University College London.
Annual Review 2020 Highlights Impact on Growth and Productivity in the UK Cell and Gene Therapy Industry
July 20, 2020 – (CGT Catapult) – Among many milestones achieved over the year is the construction of new modules at the CGT Catapult manufacturing center in Stevenage which is now fully operational, doubling capacity for collaboration at the facility.
Freeline Files Registration Statement for Proposed Initial Public Offering in the United States
July 20, 2020 – (Freeline Therapeutics) – Freeline Therapeutics filed with the SEC to raise up to $100M in an initial public offering.
GEMoaB and Intellia Therapeutics Enter Into Agreement for Next-Generation Cellular Immunotherapy Based on Proprietary RevCAR and CRISPR Gene Editing Platforms
July 20, 2020 – (GEMoaB) – Under the terms of the agreement, GEMoaB will receive payments for each product based on target reservation and selection; achievement of regulatory, clinical, and commercial milestones; as well as tiered royalties based on net sales. Intellia will lead the research collaboration, and be responsible for clinical development and commercialization.
JW Therapeutics Acquires Syracuse Biopharma and License to Eureka Therapeutics’ Solid Tumor Technology in China
July 20, 2020 – (JW Therapeutics) – JW Therapeutics announced the acquisition of Syracuse Biopharma, which includes the license for Eureka Therapeutics’ ARTEMIS antibody TCR and solid tumor technology for exclusive use in China and the ASEAN countries.
Axogen RECON Clinical Study Completes Target Enrollment of 220 Subjects
July 22, 2020 – (Axogen) – The last patient is expected to complete the study no later than October of 2021. The company anticipates it will provide a preliminary report of trial data in the second quarter of 2022 and expects to file the BLA in 2023.
XyloCor Therapeutics Doses Patients in Phase 1/2 Trial Evaluating Novel Gene Therapy XC001 in Refractory Angina
July 21, 2020 – (XyloCor Therapeutics) – The novel gene therapy, designed to stimulate the formation of new coronary blood vessels, is being studied in patients with treatment-resistant angina with no remaining treatment options.
Orchard Therapeutics Announces Orphan Drug and Rare Pediatric Disease Designations for OTL-203 for the Treatment of MPS-I
July 20, 2020 – (Orchard Therapeutics) – Orchard recently announced new interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203. Improved motor skills compared to baseline, stable cognitive scores, and normal growth was seen in the first two patients with at least one year of follow-up.
European Medicines Agency Validates Bristol Myers Squibb’s Application for CAR-T Cell Therapy Lisocabtagene Maraleucel
July 17, 2020 – (Bristol Myers Squibb) – Validation of the application confirms the submission is complete and begins the EMA’s centralized review process.
MeiraGTx Announces Positive Clinical Data Demonstrating Treatment With AAV-RPGR Investigational Gene Therapy Improves Vision in X-Linked Retinitis Pigmentosa Patients
July 17, 2020 – (MeiraGTx) – At six months, significant improvement in retinal sensitivity was demonstrated in patients treated with low and intermediate dose AAV-RPGR. Improvement was evident at first post-treatment perimetry assessments at three months, with improvements generally sustained or increased at six months.
New State-of-the-Art Center to Scale Up COVID-19 Vaccine and Gene Therapy Manufacturing Gets £100M Investment
July 23, 2020 – (CGT Catapult) – As well as addressing the immediate need to produce a COVID-19 vaccine, the new Cell and Gene Therapy Center, developed with Innovate UK and the Cell and Gene Therapy Catapult, will be at the forefront of the growing UK cell and gene therapy industry.
Catalent Biologics to Invest $30M to Create European Clinical Manufacturing Center of Excellence in Limoges, France
July 21, 2020 – (Catalant Biologics) – The project will commence in September 2020, with completion anticipated in 2022. Catalent also plans to gradually hire approximately 80 additional employees to support the new center of excellence and its updated capabilities.
Few CMOs Are Equipped to Manufacture Cell and Gene Therapies
July 21, 2020 – (GlobalData) – With a growing number of cell and gene therapies in development and only 152 CMOs equipped to manufacture them, production bottlenecks are the major obstacle to large-scale commercial manufacture, experts say.
Gene Therapy Genesis: Low-Tech Production of Curative Therapies
July 20, 2020 – (Forbes) – To date, the biotech industry has largely indolently recycled technologies and processes used for traditional protein drug production and applied them to gene therapy manufacturing as a “platform.”  The manufacturing process should be deconstructed and optimized for gene therapies to streamline manufacturing.
Australian Scientists Use Nanotechnologies to Engineer Low-Cost Immunotherapies
July 16, 2020 – (Monash University) – Researchers are using nanotechnologies to simplify the process by completely eliminating what is currently the most expensive and time-consuming step to CAR-T cell manufacturing – the use of a viral vector to genetically encode the T cells.
Researchers Hope Experimental Gene Therapy Is an Answer to a Fatal Genetic Disorder
July 21, 2020 – (NPR) – Conner Curran was 4 when he was diagnosed with Duchenne muscular dystrophy. His muscles were already beginning to waste away. Today, Conner is 9 and not just walking, but running. The reason - an experimental gene therapy.
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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