ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
January 30, 2020
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ARM Publishes Recommendations on Enabling Cross-Border and Regional Access to Advanced Therapy Medicinal Products (ATMPs) in Europe
January 27, 2020 – (ARM) – The new position paper aims to stimulate debate and reach consensus among key stakeholders, including marketing authorization holders, payers, and treatment centers, on solutions to ensure all European patients can secure access to ATMPs, irrespective of their country or region of origin.
HemoGenyx Announces Placing and Subscription to Raise Approximately £650,000 ($850,892)
January 30, 2020 – (HemoGenyx) – The funds raised will be used to continue the development and in vivo testing of the company’s CAR programmed T cells against acute myeloid leukemia.
Hitachi Chemical Advanced Therapeutics Solutions Announces Opening of Its New Facility Designed to Manufacture Commercial Cell and Gene Therapies
January 30, 2020 – (Hitachi Chemical) – The new facility is the company’s first to be designed from the ground up to meet the unique needs of commercial cell and gene therapy products and more than doubles HCATS’ existing manufacturing capacity in New Jersey.
AgeX Therapeutics Enters Research Collaboration With Japanese Biopharma Company to Generate Hypoimmunogenic Cells
January 29, 2020 – (Juvenescence) – AgeX’s UniverCyte uses a proprietary, novel, modified form of HLA-G and is intended to permit donor cells to be transplanted into patients without donor-patient tissue matching and without administering immunosuppressant medication.
Decibel Therapeutics Announces Strategic Research Focus on Regenerative Medicine for the Inner Ear
January 29, 2020 – (Decibel Therapeutics) – The company is also announcing a collaboration and option agreement with The Rockefeller University that gives Decibel exclusive access to novel compounds targeting proteins in a critical regenerative pathway. The first program in Decibel’s regeneration portfolio aims to restore balance function using gene therapy (DB-201); the company expects to initiate IND-enabling experiments for this program in the first half of 2020.
Medigene, IRICoR, and Université De Montréal Sign Collaboration and Exclusive Licensing Option on Novel Cancer Antigens
January 29, 2020 – (Medigene) – Under the terms of the agreement, Medigene will evaluate a number of proprietary TSA targets provided by UdeM through IRICoR, and will receive an option to exercise an exclusive and worldwide license to develop and commercialize T cell receptors against up to 5 of these novel cancer antigens. Upfront and near-term payments by Medigene to UdeM and IRICoR could potentially reach mid to high single-digit millions.
New Cell and Gene Therapy Catapult CEO Brings Continuity and Ambition for Further Impact
January 29, 2020 – (CGT Catapult) – The Board of the Cell and Gene Therapy Catapult announced that its new Chief Executive Officer will be Matthew Durdy, who is currently the Chief Business Officer of the organization. The appointment will be effective beginning in April 2020.
Novartis Gene Therapy for Fatal Childhood Disease Delivers Strong Sales During Fourth Quarter
January 29, 2020 – (STAT News) – Novartis reports that approximately 200 patients have been treated with Zolgensma since the June launch, double the figure from the end of the September quarter. The company said reimbursement agreements are now in place with insurers to cover 97% of patients with commercial plans and 50% of patients covered by Medicaid.
Sorrento and Celularity to Initiate Allogeneic Natural Killer Cell Therapy Development for Coronavirus Infection
January 29, 2020 – (Sorrento) – The objective of the collaboration is to expand the therapeutic use of Celularity's CYNK-001, an allogeneic, off-the-shelf, placental-derived Natural Killer cell therapy, to the treatment and prevention of coronavirus infections.
Conatus Pharmaceuticals and Histogen Enter Into Definitive Merger Agreement
January 28, 2020 – (Histogen) – Conatus Pharmaceuticals and Histogen announced the companies have entered into a definitive agreement under which Histogen will merge with a wholly-owned subsidiary of Conatus in an all-stock transaction. The combined company will operate under the name Histogen and will focus on advancement of its patented technology for dermatological and orthopedic indications.
Lonza Supporting Rocket Pharma with Clinical Manufacturing of RP-L201 for the Treatment of Leukocyte Adhesion Deficiency-I
January 28, 2020 – (Lonza) – Lonza announced that a clinical manufacturing agreement is in place with Rocket Pharmaceuticals covering the development and manufacturing of RP-L201, a phase 1/2 candidate for the treatment of Leukocyte Adhesion Deficiency-I.
Mogrify Awarded $1.1M Additional Funding From SBRI Healthcare
January 28, 2020 – (Mogrify) – The funding will be used to assess its regenerative cartilage therapy, for the treatment of cartilage defects, osteoarthritis, and other musculoskeletal conditions, before entering clinical trials.
Passage Bio Appoints Bruce Goldsmith, Ph.D. as CEO
January 28, 2020 – (Passage Bio) – Dr. Bruce Goldsmith joins Passage Bio from Deerfield Management where he was a venture partner responsible for early-stage investments and served as interim CEO of Civetta Therapeutics since April 2019. Previously, Dr. Goldsmith was chief operating officer at Lycera, a company developing first-in-class small molecules for immuno-oncology and autoimmune disease.
Autolus Announces Closing of Public Offering
January 27, 2020 – (Autolus) – The gross proceeds to Autolus are approximately $80M. The net proceeds to Autolus from the offering are approximately $74M, after deducting the underwriting discount and other offering expenses payable by Autolus.
Cognate BioServices Closes Series B and Completes Acquisition of Cobra Biologics
January 27, 2020 – (Cognate BioServices) – Cognate BioServices announced that it has completed the acquisition of Cobra Biologics, a leading CDMO specialized in providing development and manufacturing services for plasmid DNA and viral vectors.
Adaptimmune Therapeutics Announces Closing of Public Offering of American Depositary Shares
January 24, 2020 – (Adaptimmune) – The net proceeds to Adaptimmune from the offering are approximately $78.1M, after deducting the underwriting discount and other offering expenses payable by Adaptimmune. Adaptimmune intends to use the net proceeds from this offering to advance the development of its immunotherapies into and through clinical trials as well as for other general corporate purposes.
Ervaxx and Cardiff University Enter Collaboration to Develop Novel T-Cell and T-Cell Receptor-Based Immunotherapeutics Targeting Dark Antigens
January 24, 2020 – (Ervaxx) – The new collaboration will support a multi-year research program focusing on the discovery and characterization of T-cells and TCRs reactive to cancer-specific antigens and ligands, including Ervaxx’s proprietary Dark Antigens. Ervaxx will fund the program.
Krystal Biotech Breaks Ground On Second Commercial Gene Therapy Manufacturing Facility
January 24, 2020 – (Krystal Biotech) – The ASTRAfacility will initially be used as a commercial back up facility for B-VEC, which is being developed for the treatment of dystrophic epidermolysis bullosa, a rare and devastating skin disorder, and expand to produce investigational and commercial material for other pipeline products.
SanBio and Jutendo University Announce Joint Research Agreement to Develop Regenerative Medicine Product for Type 1 Diabetes
January 24, 2020 – (SanBio) – The two parties aim to collect the data necessary to proceed to clinical trials by combining the SanBio’s expertise in regenerative medicine accumulated in the process of SB623 development with Juntendo University’s discovery of direct reprogramming of somatic cells, and evaluating efficacy and safety in an animal model for type 1 diabetes.
Autolus Therapeutics Presents Encouraging Additional Data Showcasing Clinical Progress of Programmed T Cell Therapy Pipeline in Blood Cancers
January 30, 2020 – (Autolus Therapeutics) – In the cohorts dosed at 450 x 106 AUTO3 cells plus pembrolizumab, five out of seven patients (ORR=71%) achieved a response (complete response + partial response) and four out of seven patients (CRR=57%) achieved a complete response.
Rubius Therapeutics Announces First Patient Dosed in Phase 1b Trial of RTX-134 for the Treatment of Patients with Phenylketonuria
January 30, 2020 – (Rubius Therapeutics) – RTX-134 is a an-the-shelf red cell therapeutic product candidate that is genetically engineered to express the enzyme PAL inside the cell. RTX-134 is designed to circulate in the bloodstream and degrade toxic levels of phenylalanine that accumulate due to a deficiency in the phenylalanine hydroxylase enzyme.
SQZ Biotech Announces First Patient Dosed in Phase 1 Trial of SQZ-PBMC-HPV for HPV+ Tumors
January 30, 2020 – (SQZ Biotech) – SQZ-PBMC-HPV is an autologous cell therapy candidate comprised of SQZ-engineered antigen presenting cells designed to induce CD8 T cell responses against HPV16.
FDA Grants DSG3-CAART Orphan Drug Designation for the Treatment of Pemphigus Vulgaris
January 29, 2020 – (Cabaletta Bio) – DSG3-CAART is designed to target the cause of mucosal PV, B cells that express pathogenic autoantibodies directed against the DSG3 protein, while preserving normal B cell immune function.
Orchard Therapeutics Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-Linked Chronic Granulomatous Disease (X-CGD)
January 29, 2020 – (Orchard Therapeutics) – At 12 months post-treatment, six of seven surviving patients, all of whom were adults or late adolescents, exceeded the minimum threshold hypothesized in published literature to demonstrate potential clinical benefit, defined as 10% functioning, oxidase-positive neutrophils in circulation, and have discontinued preventive antibiotics.
Unum Therapeutics Provides Updates to Its Phase 1 Trial of ACTR707 for HER2+ Solid Tumor Cancers
January 29, 2020 – (Unum Therapeutics) – Patient enrollment is complete with five patients in this first cohort in the ATTCK-34-01 Phase 1 trial, a multicenter, open-label, single-arm, dose-escalation trial.
European Medicines Agency Validates Kite’s Marketing Application for Company’s Second CAR T Cell Therapy
January 28, 2020 – (Gilead) – Kite submitted a BLA for KTE-X19 to the US FDA on December 11, 2019 for the treatment of adult patients with relapsed or refractory MCL. KTE-X19 has been granted Breakthrough Therapy Designation by the FDA and PRIME designation by the EMA.
Osaka University Transplants iPS Cell-Based Heart Cells in World's First Clinical Trial
January 27, 2020 – (Japan Times) – The cells on the degradable sheets attached to the surface of the patients’ hearts are expected to grow and secrete a protein that can regenerate blood vessels and improve cardiac function.
Penn Researchers Identify Cancer Cell Defect Driving Resistance to CAR T Cell Therapy
January 30, 2020 – (Penn Medicine) – The team discovered that in ALL cells resisting CAR T attack, there was depletion of genes involved in activating the cell death pathway (FADD, BID, CASP8 and TNFRSF10B) and enrichment of genes required for resisting the cell death pathway (CFLAR, TRAF2 and BIRC2).
Nanoparticle Delivery System Brings a Less Toxic Approach to CAR-T Immunotherapy
January 28, 2020 – (Penn Medicine) – The technique involves ferrying mRNA across the T cell’s membrane via a lipid-based nanoparticle, rather than using a modified HIV virus to rewrite the cell’s DNA. Using the former approach is preferable, as it only confers a temporary change to the patient’s immune system.
New Gene Correction Therapy for Duchenne Muscular Dystrophy
January 27, 2020 – (Technical University of Munich) – A research team has for the first time succeeded in correcting the mutated dystrophin gene in living pigs. In order to cut the defective gene sequence from the DNA of the animals' muscle and heart cells, the researchers modified the CRISPR-Cas9 gene scissors.
FDA Continues Strong Support of Innovation in Development of Gene Therapy Products
January 28, 2020 – ( – The FDA announced the release of a number of important policies: six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.
U.S. Judge Rejects FDA Bid to Shut Down Stem Cell Clinics, Dealing Blow to Regulators
January 28, 2020 – (Los Angeles Times) – The government’s California lawsuit was filed May 9, 2018, simultaneously with a complaint against Florida-based U.S. Stem Cell Clinic. In that case, U.S. Judge Ursula Ungaro of Miami granted the government’s motion for summary judgment last June, effectively shutting down the Florida operation. The two judges’ rulings don’t have any direct legal effect on each other.
Gene Therapy Approvals By US FDA Could Double in 2020
January 26, 2020 – (Pink Sheet – Subscription Only) – The number of gene therapies approved by the US FDA could double in 2020, thanks to a surge in biologics license applications in the waning days of 2019. Four new gene therapies are now under review, and rolling submissions are underway or imminent for at least two more.
Director / Vice President, Finance and Operations
Alliance for Regenerative Medicine – Washington, DC
Vice President, Global Policy and Advocacy
Alliance for Regenerative Medicine – Washington, DC
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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