ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
June 11, 2020
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ARM Issues Statement, Convenes Members on Racial Inequity
ARM released the following statement through social media channels on June 5: "ARM believes inclusion and diversity are fundamental to the amazing, life-changing innovation we represent. We oppose racism. We support equal justice for all. We admire and stand with the millions of peaceful protestors demanding needed change."
  • On June 9, ARM convened a call of its CEO Forum to give ARM member leaders an opportunity to discuss their own and ARM’s actions regarding racial injustice and the global protests following the death of George Floyd. The group launched a task force to explore ARM-led sector wide responses.
ARM Resource Guide for Creating an Anti-Racist Workplace
ARM has compiled a resource guide for individuals and organizations interested in understanding the current moment and taking action to create an anti-racist workplace.
Across the Globe, Scientists Are Striking for Black Lives
June 10, 2020 – (Wired) – The Shut Down STEM to Strike for Black Lives, which took place Wednesday, was intended as a call for researchers to stop their work and spend the day learning about how systemic racism functions in their fields and at their institutions, and then to draft plans for eradicating those inequalities.
NIH Director Dr. Francis Collins Shares Statement on Racism, Health Disparities
June 10, 2020 – (Twitter) – On Twitter, Dr. Collins called for the NIH community to “foster a culture of inclusion, equity, and respect for one another,” and asked stakeholders to “consider what you might do to help end the scourge of racism and health disparities.” Beyond Molecules and Microbes: Biopharma's Responsibility to Racial Justice in and Outside Its Own Walls
June 5, 2020 – (BioCentury) – The same patterns of thought and action that biopharma companies use to turn scientific insights into medicines must to applied to achieving justice, both in societies and in individual companies.
Across the Globe, Scientists Are Striking for Black Lives
June 3, 2020 – (Fierce Pharma) – Even those not on the ground as protests play out nationwide are speaking out, sometimes for the first time. Many of the biopharma industry's leaders are reaching out to their employees and the public to get their views on record.
Measuring Diversity in the Biotech Industry: Building an Inclusive Workforce
January 30, 2020 – (Biotechnology Innovation Organization) – Released this past January, the report looks at where biotech companies stand on representation in terms of gender, race, and ethnicity and assesses the steps they are taking to improve diversity and inclusion.
Join ARM for Our Upcoming Webinar: The Next Wave of Commercial Development
June 11, 2020 – (ARM) – The next wave of cell and gene therapies is poised to enter the market. How are developers shaping their commercial plans? Panelists will address how the landscape has changed since the first products were introduced, lessons from the first wave of products, how payors and regulators are evolving in their approach to new therapies, and how COVID-19 is affecting commercial strategies. Register today!
Pluristem Announces Activation of Clinical Sites and Commencement of Patient Enrollment in US FDA Phase 2 COVID-19 ARDS Trial
June 11, 2020 – (Pluristem) – The randomized, double-blind, placebo-controlled, multicenter, parallel-group 140-patient study is expected to include up to 25 sites.
Cell and Gene Therapies: FDA Official on COVID-19 Impact
June 8, 2020 – (Regulatory Focus) – While the pandemic has not resulted in an increase in applications for gene therapies, Wilson Bryan, director of the Office of Tissues and Advanced Therapies, said his office has been busy with an increase in interest in using cell therapies to treat COVID-19. However, even with the pressing need for new treatments for COVID-19, Bryan stressed that well-designed clinical trials are essential in the long run.
Scientists Aim Gene-Targeting Breakthrough Against COVID-19
June 4, 2020 – (Stanford University) – When packaged with coronavirus-targeting PAC-MAN (Prophylactic Antiviral CRISPR in human cells), the researchers' approach reduced the amount of synthetic SARS-CoV-2 in a solution by more than 90%.
Accellix Raises $9.5M in Series D Round
June 11, 2020 – (Accellix) – This latest round of funding will be used to further expand the market reach of the Accellix system for Cell Therapy QC, enabling its customers greater access to the expertise, scale, resources, and technology needed to effectively assess the quality of life saving immunotherapies.
Magenta Therapeutics Announces Collaboration With the National Marrow Donor Program / Be the Match to Advance Development of MGTA-145
June 11, 2020 – (Magenta Therapeutics) – Under the collaboration, Magenta and NMDP/Be The Match will run a Phase 2 clinical trial of MGTA-145 to mobilize and collect hematopoietic stem cells from donors which will then be transplanted into patients with blood cancers in need of a stem cell transplant.
Verve Therapeutics Raises $63M in Series A2 Financing to Advance Gene-Editing Therapies for Heart Disease
June 11, 2020 – (Verve Therapeutics) – The company will use the financing to advance its lead program through IND-enabling studies and fuel follow-on pipeline programs. Verve is developing one-time gene-editing therapies to safely edit targeted genes in the liver to permanently lower LDL cholesterol and triglycerides.
Zelluna Immunotherapy Raises Funds to Fully Focus on Developing Allogeneic “Off-The-Shelf” TCR-Based Natural Killer Cell Therapies
June 11, 2020 – (Zelluna Immunotherapy) – A total of approximately €7.5M has been raised in equity financing and public grants. In support of this strategy, Zelluna has recently announced the formation of partnerships with world-renowned NK organizations, providing access to NK cells, manufacturing facilities, and additional competency and expertise.
Mustang Bio Announces Pricing of Public Offering of Common Stock
June 11, 2020 – (Mustang Bio) – Assuming the full exercise of the underwriter’s option, gross proceeds would be $40.3M. Mustang intends to use the proceeds for the continued development of its product candidates; the potential in-license, acquisition, development and commercialization of other pharmaceutical products; and for general corporate purposes.
JW Therapeutics Raises $100M to Support CAR-T Portfolio
June 9, 2020 – (JW Therapeutics) – JW Therapeutics plans to use the proceeds to further advance its lead product JWCAR029, a CAR-T cell product targeting CD19, currently in Phase 2 development; further build-out the company's pipeline; and establish commercialization capabilities to support product launch.
CANbridge Pharmaceuticals Enters Into Rare Disease Gene Therapy Research Agreement With UMass Medical School
June 9, 2020 – (CANbridge Pharmaceuticals) – CANbridge Pharmaceuticals announced that it has initiated a research program with the Horae Gene Therapy Center at the UMass Medical School for gene therapy research in rare genetic diseases, with a focus on neuromuscular conditions.
Fate Therapeutics Announces Pricing of Public Offering of Common Stock
June 9, 2020 – (Fate Therapeutics) – Fate Therapeutics intends to use the net proceeds from the $164M offering to fund clinical trials and nonclinical studies, the manufacture of its clinical product candidates, the expansion of its cGMP compliant manufacturing operations, the conduct of preclinical research and development, and for general corporate purposes.
Oxford BioMedica Provides Update on Ophthalmology Programs Out-Licensed to Sanofi
June 9, 2020 – (Oxford BioMedica) – Sanofi intends to return to Oxford BioMedica the rights to two gene therapy programs, SAR422459 for Stargardt disease and SAR421869 for Usher’s syndrome type 1b.
Achilles Therapeutics to Set Up Manufacturing Operations at the CGT Catapult Manufacturing Center
June 8, 2020 – (CGT Catapult) – The collaboration will see Achilles Therapeutics expand their manufacturing footprint at the CGT Catapult manufacturing center to supply clinical trials of their novel T cell therapy products. Onboarding has begun into one of the newly operational modules at the state-of-the-art facility.
Akouos Aims for an IPO to Advance Hearing Loss Gene Therapy to Clinic
June 8, 2020 – (Xconomy) – In paperwork filed with regulators late Friday, Boston-based Akouos set a preliminary $100M goal for its IPO. Paperwork seeking FDA permission to start a clinical trial is on track to be submitted in 2021, Akouos says in its filing.
CureDuchenne Launches 2020 Pitch Contest
June 8, 2020 – (CureDuchenne) – Through July 13, 2020, CureDuchenne is seeking applications from innovators who are focused on new therapeutics, therapeutic targets, or technology platforms related to Duchenne muscular dystrophy. Two applicants will receive no-strings-attached $25,000 awards. Click here for more information.
Oxford Biomedica Signs Five-Year Collaboration Agreement With the Vaccines Manufacturing and Innovation Center
June 8, 2020 – (Oxford Biomedica) – This five-year agreement will involve the organizations working collaboratively to enable the manufacture of viral vector-based vaccines, contributing towards a rapid increase in UK domestic capacity for this specialized field of vaccine manufacturing.
NIH Regenerative Medicine Innovation Project Announces New Funding Opportunities
June 8, 2020 – (NIH) – The NIH announced Monday that a new 2020 RMIP Clinical Trial  Funding Opportunity Announcement has been published.
Sernova to Acquire Cellular Local Immune Protection Technology Accelerating Expansion of Its Regenerative Medicine Therapeutics Platform
June 8, 2020 – (Sernova) – Pursuant to an asset purchase agreement with Converge Biotech, Sernova is acquiring all intellectual property associated with Converge’s conformal coating cell encapsulation technology.
TreeFrog Therapeutics Opens Its First Facility Dedicated to iPSC Therapy Scale-Up in Bordeaux, France
June 6, 2020 – (TreeFrog Therapeutics) – TreeFrog Therapeutics has commissioned a 13,000 square foot facility in Bordeaux, France dedicated to the development and scale-up of cell therapy manufacturing processes using human iPS cells.
Legend Biotech Corporation Announces Pricing of Initial Public Offering
June 5, 2020 – (Legend Biotech) – Legend Biotech announced the pricing of its initial public offering of 18,425,000 American depositary shares, for total gross proceeds of approximately $423.8M.
INmune Bio Receives Regulatory Approval From UK MHRA to Initiate Phase 1 Trial of INKmune in Patients With High-Risk MDS
June 11, 2020 – (INmune Bio) – INmune Bio announced that the MHRA approval to initiate a Phase 1 clinical trial of INKmune in patients with high-risk myelodysplastic syndrome. INmune Bio expects to initiate the study in the second half of this year.
Axovant Completes Enrollment of Low-Dose Cohort in Phase 1/2 Study of AXO-AAV-GM1 and Expands Study to Include Type I (Infantile Onset) Patients With GM1 Gangliosidosis
June 8, 2020 – (Axovant Gene Therapies) – Currently, the study is on track to report 6-month data (n=5) from the low-dose cohort of the AXO-AAV-GM1 clinical trial by Q4 2020.
Sarepta Therapeutics Announces Positive Expression and Functional Data From the SRP-9003 Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E
June 8, 2020 – (Sarepta Therapeutics) – In Cohort 1 (low dose), at one year all three participants continued to show improvements from baseline across all functional measures. Preliminary results from Cohort 2 showed a strong dose-dependent increase in transduction and expression when compared with the low-dose cohort.
Precision BioSciences Announces Dosing of First Patient in Phase 1/2a Clinical Trial of PBCAR269A for Multiple Myeloma
June 8, 2020 – (Precision BioSciences) – In preclinical disease models, PBCAR269A, Precision's third allogeneic CAR-T candidate, demonstrated potent in vivo clearance of BCMA+ tumor cells and overall tumor volume reduction, with no evidence of graft-versus-host disease.
TVAX Biomedical Receives Fast Track Designation From the FDA for Brain Cancer
June 4, 2020 – (TVAX Biomedical) – Phase 1 and 2a studies of TVAX Biomedical's vaccine-enhanced adoptive T cell therapy for treatment of multiple cancers, including glioblastoma multiforme, have demonstrated significant benefit to patients.
Director, Communications
Alliance for Regenerative Medicine – Washington, DC
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
Senior Director, Sales & Business Development, BioTherapies
Be The Match / National Marrow Donor Program – Boston, MA
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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