ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
July 16, 2020
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Promethera to Form Joint Venture to Develop Cell-Based and Biological Therapies for the Treatment of Liver Diseases in China and Southeast Asia With Hao Tian Development
July 16, 2020 – (Promethera) – Promethera will manufacture and supply the therapeutic products, and provide ongoing technical support during development and commercialization. The Joint Venture Company will seek opportunities for cooperation with Itochu Corporation.
Orchard Therapeutics Announces Global License Agreements for Stable Cell Line Technology From GSK
July 15, 2020 – (Orchard Therapeutics) – Orchard Therapeutics has entered into two worldwide license agreements with GSK for use of their proprietary lentiviral stable cell line technology for Orchard’s investigational gene therapies for Wiskott Aldrich syndrome (OTL-103) and transfusion-dependent beta thalassemia (OTL-300). Orchard plans to submit a BLA and MAA for OTL-103 for the treatment of WAS in the US and EU, respectively, in 2021.
Acepodia and JW Therapeutics Enter Into Option and License Agreement for Off-the-Shelf NK Cell Therapies
July 14, 2020 – (Acepodia) – Acepodia and JW Therapeutics have announced a collaboration to develop and commercialize ACE1702 and ACE1655, two of Acepodia’s cell therapy candidates for the treatment of solid tumors and hematological malignancies, respectively, in Mainland China, Hong Kong, and Macau. Acepodia will receive upfront and milestone payments in addition to royalties on sales.
Fate Therapeutics Announces Exclusive License Agreement With Baylor College of Medicine for Rejection-Resistant iPSC-Derived Cellular Therapies
July 14, 2020 – (Fate Therapeutics) – Preclinical studies demonstrate that allogeneic cells engineered with a novel alloimmune defense receptor (ADR) are protected from both T- and NK-cell mediated rejection, and provide proof-of-concept that ADR-expressing allogeneic cell therapies can durably persist in immunocompetent recipients.
Nkarta Announces Closing of Upsized Initial Public Offering and Exercise in Full of Underwriters' Option to Purchase Additional Shares
July 14, 2020 – (Nkarta) – The aggregate gross proceeds from the offering were approximately $290M, before deducting underwriting discounts and commissions and other offering expenses payable by Nkarta. 
ACell Plans $75M Initial Public Offering
July 13, 2020 – (ACell) – ACell is offering 5,000,000 shares of its common stock at an expected initial public offering price between $14 and $16 per share.
Akron Biotech Submits eCTD Drug Master Files With FDA for Its Suite of cGMP Immunotherapy Cytokines
July 13, 2020 – (Akron Biotech) – The submission of these eCTD DMFs will enable a thorough assessment by regulators of the quality and consistency of the cGMP cytokines, assuring developers that the materials embedded in their manufacturing processes are sufficiently robust for further clinical development, and eventually for marketing approval.
Single-Cell Company Berkeley Lights Sets Terms for $153M IPO
July 13, 2020 – (Genetic Engineering & Biotechnology News) – Berkeley Lights estimated that the net proceeds from the offer would be $114M. However, the net proceeds could rise to as much as $131.5M should underwriters of the IPO exercise in full their 30-day option to purchase up to 1.1 million additional shares at the mid-point of the range
Fosun Kite Secures $20M to Accelerate First CAR-T Therapy Launch in China
July 9, 2020 – (BioWorld) – Fosun Kite Biotechnology secured a $20M investment from Shanghai Fosun Pharmaceutical and Kite Pharma, which each contributed $10M. The fund will support further clinical trials and manufacturing of its CAR-T candidates, including FKC-876 (Yescarta), which looks likely to become the first CAR-T therapy approved in China.
Poseida Therapeutics Announces Pricing of Initial Public Offering
July 9, 2020 – (Poseida Therapeutics) – The gross proceeds to Poseida from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, are expected to be $224M.
Celyad Oncology Announces FDA Clearance of IND Application for CYAD-211, First shRNA-Based, Non-Gene Edited Allogeneic CAR-T Therapy
July 14, 2020 – (Celyad Oncology) – Celyad plans to initiate the clinical trial for patients with multiple myeloma by the end of 2020, and accelerate the development of additional shRNA-based allogeneic candidates towards clinical trials.
Adverum Biotechnologies Announces First Patient Randomized in Phase 2 INFINITY Trial of ADVM-022 Single Injection Gene Therapy for Diabetic Macular Edema
July 13, 2020 – (Adverum Biotechnologies) – The INFINITY trial will enroll approximately 33 patients in a Phase 2, multi-center, randomized clinical trial. ADVM-022 has received the US FDA's Fast Track designation.
Freeline Presents New Hemophilia B Data at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Meeting
July 13, 2020 – (Freeline Therapeutics) – Long term durability up to 2 years was seen in the 2 patients in the lowest dose cohort with an average FIX level activity at 52 and 104 weeks, both of 38%, which are levels commonly associated with mild hemophilia B. To date no bleeds have required supplemental FIX in any of the patients studied.
Rocket Pharmaceuticals Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency
July 13, 2020 – (Rocket Pharmaceuticals) – The global Phase 1 open-label, single-arm clinical trial is expected to enroll six adult and pediatric transfusion-dependent PKD patients in the US and Europe. 
Spark Therapeutics Announces Updated Data on SPK-8011 From Phase 1/2 Clinical Trial in Hemophilia A at ISTH 2020 Virtual Congress
July 12, 2020 – (Spark Therapeutics) – Results from twelve participants across three dose cohorts show an acceptable safety profile, 91% reduction in annualized bleed rate, 96% reduction in FVIII infusions and stable, and durable factor FVIII expression after between two and 3.3 years of follow-up.
Ziopharm Oncology Announces Initiation of Phase 1 Trial of CAR-T Technology in Patients with Relapsed CD19+ Leukemias and Lymphomas
July 9, 2020 – (Ziopharm Oncology) – Ziopharm Oncology announced the initiation of a Phase 1 clinical trial to evaluate CD19-specific CAR-T, using its Rapid Personalized Manufacturing technology, as an investigational treatment for patients with relapsed CD19+ leukemias and lymphomas. The trial is now open for enrollment at The University of Texas MD Anderson Cancer Center.
Researchers 3D Print a Working Heart Pump With Real Human Cells
July 15, 2020 – (University of Minnesota) – Researchers found that they could achieve the goal of high cell density within less than a month to allow the cells to beat together, just like a human heart.
Metabolic Engineering Generates a Transgene-Free Safety Switch for Cell Therapy
July 13, 2020 – (Auxolytic) – Auxolytic, in collaboration with researchers at Stanford University School of Medicine, demonstrates that it has developed a novel safety “off” switch for various types of cell therapies, potentially enabling a physician to mitigate serious side-effects when they are observed. The switch works by knocking out a specific gene in the cell, creating a dependency on a particular nutrient for its survival.
Update on the RMAT Designation
July 15, 2020 – (Regulatory Focus) – ARM CEO Janet Lambert and Caladrius Biosciences' VP of Global Regulatory Affairs William Sietsema provide an update on the RMAT designation four years after its rollout. There are currently 52 product candidates with the designation, including 43 that have been announced publicly. For a list of those therapies which have been announced as having RMAT designation, click here.
Stakeholders Urge CMS to Finalize New CAR-T Therapy Payment Rule
July 10, 2020 – (Healthcare Finance) – Patient advocacy organizations, including American Cancer Society Cancer Action Network, BMT Infonet, Cancer Support Community, CLL Society, CrowdCare Foundation/Myeloma Crowd, International Myeloma Foundation, and the Leukemia & Lymphoma Society, among others, signed a letter urging CMS to finalize a proposed rule that would create a new DRG for CAR-T therapies.
Mitochondrial Genome Editing: Another Win for Curiosity-Driven Research
July 15, 2020 – (Nature) – A promising biomedical tool began life as part of efforts to answer a different question. Mutations in mitochondrial DNA can result in devastating disorders, but current gene-editing methods can’t make precise changes to mitochondrial genomes, because crucial components can’t get into membrane-bound mitochondria.
Director, CMC Regulatory Affairs
Century Therapeutics – Greater Philadelphia, PA
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 350+ 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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