ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
July 9, 2020
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Alliance for Regenerative Medicine Responds to EU Pharmaceutical Roadmap
July 7, 2020 – (ARM) – ARM's feedback to the European Commission's Roadmap on the Pharmaceutical Strategy focuses on three key priorities: ensuring regulatory procedures are robust, transparent, evidence-based, and harmonized across Europe; streamlining regulatory requirements for clinical trials with ATMPs across different member states; and ensuring timely patient access to ATMPs.
Juno Submits New Drug Application in China CAR-T Race
July 7, 2020 – (STAT News) – Last week, JW Therapeutics submitted a new drug application for its CAR-T cell therapy, JWCAR029, records from China’s Center for Drug Evaluation showed. The therapy targets CD19, a biomarker present in many types of B cell cancers.
Editas Medicine and Azzur Group Announce a Multi-Year Agreement for EDIT-301 and EDIT-201 Manufacturing
July 9, 2020 – (Editas Medicine) – Editas Medicine will utilize the space and Azzur’s services to execute preclinical and early-phase clinical manufacturing activities for its cell medicines, including EDIT-301, in development for the treatment of sickle cell disease and beta thalassemia and EDIT-201, a healthy donor natural killer cell medicine in development for solid tumor cancers.
Orchard Therapeutics and MolMed Announce Extension of Gene Therapy Manufacturing Collaboration
July 9, 2020 – (Orchard Therapeutics) – With the extension of the collaboration, MolMed will continue to support activities related to the development and manufacturing of vectors and drug products for several of Orchard’s investigational ex vivo gene therapies in the upcoming years, including OTL-200 for metachromatic leukodystrophy (MLD). OTL-200 for MLD is currently under review by the EMA with a decision expected later this year.
APEIRON Biologics and MaxCyte Enter Into Clinical and Commercial Licensing Agreement for APN401
July 8, 2020 – (MaxCyte) – APEIRON Biologics will obtain non-exclusive clinical and commercial rights to use MaxCyte’s Flow Electroporation technology and ExPERT platform for the advancement of APN401, a siRNA-based cell therapy currently in clinical development for various solid tumors. In return, MaxCyte will receive undisclosed development and approval milestones and sales-based payments in addition to other licensing fees.
Kiadis Licenses Preclinical K-NK-Cell Programs to Sanofi, With Total Potential Deal Value of €875M
July 8, 2020 – (Kiadis Pharma) – The agreement covers Kiadis’ proprietary CD38 knock out (CD38KO) K-NK therapeutic for combination with anti-CD38 monoclonal antibodies, including Sarclisa, Sanofi’s recently approved therapy for patients with multiple myeloma. Additionally, Sanofi has obtained exclusive rights to use Kiadis’ K-NK platform for two undisclosed preclinical programs. Kiadis will receive a €17.5M upfront payment
Tessera Therapeutics Emerges From Three Years of Stealth Operations to Pioneer Gene Writing as a New Genome Engineering Technology
July 8, 2020 – (Flagship Pioneering) – The company's Gene Writing platform can alter the genome by efficiently inserting genes and exons, introducing small insertions and deletions, or changing single or multiple DNA base pairs.
AlloVir Guns for $100M IPO as Public Funding Window Stays Wide Open
July 7, 2020 – (Fierce Biotech) – AlloVir is looking for $100M for its T-cell work, which includes an early-stage attempt at a T-cell therapy against the SARS-CoV-02 outbreak partnered with the Baylor College of Medicine.
BioLife Solutions Announces Closing of $86M Public Offering of Common Stock
July 7, 2020 – (BioLife Solutions) – BioLife Solutions announced the closing of its public offering of 5,951,250 shares of common stock for $14.50 per share.
Cabaletta Bio and Artisan Bio Announce Gene Editing Research and Collaboration Agreement to Develop Next-Generation CAAR T Cell Therapies
July 7, 2020 – (Cabaletta Bio) – Cabaletta Bio and Artisan Bio announced they have signed a research and collaboration agreement to accelerate the development of next-generation CAAR T cell therapies for patients with B cell-mediated autoimmune diseases. The financial terms of the agreement were not disclosed.
Vor Biopharma Closes $110M Series B Financing
July 7, 2020 – (Vor Biopharma) – Proceeds will advance the company’s lead candidate VOR33 into clinical trials, deepen its portfolio, and accelerate the validation of additional targets for its scientific platform, which is designed to remove redundant proteins so that transplanted stem cells become invisible to targeted therapies while leaving diseased cells vulnerable.
Biopharma Industry on Pace to Smash Record for Financings
July 6, 2020 – (BioWorld) – Amid a world that has been brought to its knees by the COVID-19 pandemic, the biopharma industry has learned how to quickly adapt under these extreme circumstances.
Poseida Therapeutics Sets Terms for Upcoming IPO, Aims to Raise $150M for Cancer Treatments
July 6, 2020 – (San Diego Union-Tribune) – The company has one therapy advancing to Phase 2 clinical trials for multiple myeloma, a type of cancer that develops in certain cells in the bone marrow. The company expects to file for investigational new drug authorization with the US FDA for its myeloma treatment later this year.
Precision BioSciences Regains Rights to In Vivo Hepatitis B Virus Program
July 6, 2020 – (Precision BioSciences) – Precision BioSciences announced that the company will regain full rights and all data it generated for the in vivo chronic hepatitis B virus program developed under its 2018 collaboration agreement with Gilead Sciences. The company does not anticipate any changes to its cash runway.
Fate Therapeutics Announces FDA Clearance of IND Application for First-Ever iPSC-Derived CAR-T Cell Therapy
July 9, 2020 – (Fate Therapeutics) – The company plans to initiate clinical investigation of FT819 for the treatment of patients with relapsed / refractory B-cell malignancies, including chronic lymphocytic leukemia, acute lymphoblastic leukemia, and non-Hodgkin lymphoma.
Abeona Therapeutics Reinitiates Enrollment in EB-101 Pivotal Phase 3 Study in RDEB After COVID-19 Related Pause
July 8, 2020 – (Abeona Therapeutics) – Enrollment in the VIITAL study at Stanford University Medical Center was paused in March 2020 in order to redirect healthcare resources to COVID-19 patients and to ensure the safety of study participants and site staff.
FDA Approves Talaris Therapeutics’ IND for Its Allogeneic Cell Therapy FCR001 to be Evaluated in Patients With a Severe Form of Scleroderma
July 8, 2020 – (Talaris Therapeutics) – Approval of this IND allows Talaris to initiate a Phase 1/2a trial at sites across the US, including Duke University and the University of Michigan.
REGENXBIO Provides Update on Progress of Clinical Programs for Rare Genetic Neurodegenerative Diseases
July 8, 2020 – (REGENXBIO) – RGX-111 and RGX-121 continue to be well-tolerated in patients with MPS I and MPS II following one-time intracisternal administration. Data from a single-patient investigator-initiated IND of RGX-111 for the treatment of MPS I shows encouraging biomarker activity and continued progression in neurocognitive development.
Ziopharm Oncology Doses First DIPG Patient in Phase 1/2 Trial of Controlled IL-12 for the Treatment of Pediatric Brain Tumors
July 8, 2020 – (Ziopharm Oncology) – Up to 12 patients may be enrolled in Phase 1 of the study, which is being conducted at leading pediatric cancer centers across the US, including the Dana-Farber Cancer Institute in Boston and the University of California in San Francisco.
FDA Grants Orphan Drug Designation to Neurogene’s Gene Therapy for the Treatment of CLN5 Batten Disease
July 7, 2020 – (Neurogene) – The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States.
AVROBIO Announces New Patients Dosed in Gaucher Disease and Cystinosis Clinical Trials
July 6, 2020 – (AVROBIO) – AVROBIO announced that the first patient has been dosed in the company’s Phase 1/2 investigational study evaluating AVR-RD-02 for Gaucher disease type 1. The trial is currently recruiting patients in Australia and Canada, with new clinical sites expected to open in the US and Israel by year-end.
Cellectis Reports Clinical Hold Placed on MELANI-01 Study
July 6, 2020 – (Cellectis) – Cellectis is working closely with the FDA to address the agency’s requests including changes to the MELANI-01 clinical protocol, and expects to submit requested information including an amended protocol in due course.
Expanded Access Protocol Initiated for Compassionate Use of Remestemcel-L in Children With Multisystem Inflammatory Syndrome Associated With COVID-19
July 6, 2020 – (Mesoblast) – MIS-C is a life-threatening complication of COVID-19 in otherwise healthy children and adolescents that includes massive simultaneous inflammation of multiple critical organs and their vasculature. In approximately 50% of cases, this inflammation is associated with significant cardiovascular complications that directly involve heart muscle and may result in decreased cardiac function.
GenSight Biologics Reports Sustained Efficacy and Safety Among LHON Patients Three Years After LUMEVOQ Treatment
July 6, 2020 – (GenSight Biologics) – At two years post-treatment, participants had already experienced an average gain of +18.8 letters equivalent relative to the low point of their visual acuity in their LUMEVOQ-treated eyes and +17.3 letters equivalent in their sham-treated eyes. One year later, the bilateral benefit was maintained, with LUMEVOQ-treated eyes recording a mean improvement of +20.5 letters and sham-treated eyes demonstrating a mean improvement of +19.4 letters.
New Molecular Tool Precisely Edits Mitochondrial DNA
July 8, 2020 – (Broad Institute) – Researchers have discovered a new type of molecular editor that can make precise C to T and G to A nucleotide changes in mitochondrial DNA. One goal for the field now will be to develop editors that can precisely make other types of genetic changes in mitochondrial DNA, which could lead to treatments for mitochondrial-derived diseases.
Salk Scientists Discover Genetic “Dial” to Turn Immune Function Up and Down to Target Cancer, Autoimmune Disease
July 7, 2020 – (Salk Institute) – The researchers used CRISPR to selectively remove the SWI/SNF complex genes from regulatory T cells. The deletion of one gene in the ncBAF complex, Brd9, had a particularly strong effect on the immune cells. In mice with cancer, treatment with the weakened immune cells without Brd9 enabled other immune cells normally blocked by the regulatory T cells to infiltrate and shrink tumors.
Tiny Mineral Particles Are Better Vehicles for Promising Gene Therapy
July 2, 2020 – (University of Wisconsin-Madison) – Researchers used the mineral-coated microparticles (MCMs) – which are 5 to 10 micrometers in diameter, about the size of a human cell – in a series of experiments to deliver mRNA to cells surrounding wounds in diabetic mice. Wounds healed faster in MCM-treated mice, and cells in related experiments showed much more efficient pickup of the mRNA molecules than other delivery methods.
House Proposes $3.2B for FDA in FY 2021
July 7, 2020 – (Regulatory Focus) – This amount is an increase of $40.8M over the prior fiscal year’s approximate $3.2B budget. FDA funding will total $5.99B when user fee revenue is included. Specifically, $70M is appropriated in the bill for further medical product development acceleration according to the provisions of the 21st Century Cures Act.
Korean Regulator Revises Rules for Swift Approval of High-Tech Bio Drugs
July 3, 2020 – (Korea Biomedical Review) – The Korean Ministry of Food and Drug Safety has proposed new rules to screen and approve up-to-date biopharmaceutical products, such as cell therapies and genetic treatments, swiftly and smoothly while minimizing their safety problems. The agency will be accepting comments on the proposed rules through July 23.
‘It Will Consume Your Life’: 4 Families Take On Rare Diseases
July 7, 2020 – (New York Times) – Confronted by illnesses that most scientists overlook, these families are working out their own approaches to find treatments.
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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