ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
June 26, 2020
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ARM Announces ARM Connect, A Virtual Networking Event for Members and Investors
June 26, 2020 – (ARM) – From July 13-24, registrants will have the ability to access a messaging system to connect with one another for the purposes of communication, collaboration, and innovation. ARM Connect is available exclusively and free of charge to ARM members and investors. For more information, click here.
Emer Cooke Nominated as New EMA Executive Director
June 25, 2020 – (European Medicines Agency) – The EMA Management Board has nominated Emer Cooke as the new Executive Director of the Agency. Ms. Cooke has 30 years’ experience in international regulatory affairs, 18 years of which were in leadership roles.
Artiva Biotherapeutics Launches with $78M Series A Financing to Advance NK Cell Therapies
June 26, 2020 – (Artiva Biotherapeutics) – Artiva will use the proceeds of this financing to develop off-the-shelf universal NK cells for use in combination with monoclonal antibody therapies and tumor targeting CAR-NK cell therapies. Artiva has exclusive ex-Asia-Pacific license agreements with GC LabCell covering their core manufacturing and CAR technology and initial product candidates.
CRISPR Therapeutics Announces the Build-Out of New Cell Therapy Manufacturing Facility
June 25, 2020 – (CRISPR Therapeutics) – CRISPR Therapeutics announced that it is building a new cell therapy manufacturing facility in Framingham, Massachusetts for clinical and commercial production of the company’s investigational cell therapy product candidates. As the facility becomes fully operational, CRISPR expects to hire up to approximately 100 full-time employees.
Japan's Gene Therapy Sector Progresses in Post-Zolgensma World
June 25, 2020 – (Scrip – Subscription Only) – The pricing and launch of Zolgensma in Japan in May marked a watershed moment. Other product candidates in Japan continue to move forward, buoyed by this new precedent for approval and reimbursement.
Poseida Therapeutics Raises $110M in Series D Financing
June 25, 2020 – (Poseida Therapeutics) – Poseida’s portfolio includes allogeneic and autologous CAR-T product candidates in both hematological and solid tumor oncology indications, as well as liver-directed gene therapy programs in orphan genetic diseases.
RoslinCT Announces the Construction of a New Cell and Gene Therapy Manufacturing Facility
June 25, 2020 – (RoslinCT) – The new 1,600 square meter facility is located within the Edinburgh BioQuarter, a leading global destination for healthcare delivery, ground-breaking medical research, and life sciences innovation and entrepreneurship.
Ziopharm Oncology Taps Aldevron for Rapid Manufacturing of Personalized Therapeutics
June 25, 2020 – (Aldevron) – Ziopharm will receive neoGMP plasmid DNA for T cell therapeutics targeting solid tumors. The innovative approach is achieved by expressing T cell receptors using the Sleeping Beauty non-viral gene transfer system.
Atara Biotherapeutics Announces Full Exercise of Underwriters’ Option to Purchase Additional Shares
June 24, 2020 – (Atara Biotherapeutics) – The aggregate net proceeds to Atara from the offering, including the shares sold pursuant to the underwriters’ option, were approximately $189M.
BioGeneration Ventures Closes BGV IV Fund at €105M
June 24, 2020 – (BioGeneration Ventures) – BGV is one of the largest VC funds in Europe focused on early investments in new biotech companies, with over €220M in assets under management. The company’s approach is to partner with scientists from major European institutions and entrepreneurs to build new companies.
Kevin Ballinger to Assume CEO Role at Aldevron
June 24, 2020 – (Aldevron) – Ballinger joins Aldevron from Boston Scientific and brings 25 years of operating experience. As one of Boston Scientific’s most tenured executives, Ballinger helped advance the company’s market leadership, growing the IC division into a $3B global business.
Magenta Therapeutics Announces Pricing of Public Offering
June 24, 2020 – (Magenta Therapeutics) – The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, are expected to be $60M.
uniQure Announces License Agreement With CSL Behring to Commercialize Hemophilia B Gene Therapy
June 24, 2020 – (uniQure) – Under the terms of the agreement, uniQure will receive a $450M upfront cash payment and be eligible to receive up to $1.6B in payments based on regulatory and commercial milestones.
With Cell and Gene Therapy Boom Coming, Experts at Novartis, Kite Trumpet Need for Capacity
June 24, 2020 – (Fierce Pharma) – During a virtual roundtable, manufacturing experts from Novartis, Gilead's Kite unit, BioMarin, and Astellas' Audentes said building capacity and figuring out how to scale production would make the difference in whether the industry will be ready to handle a slate of expected approvals.
Zelluna Immunotherapy Announces Collaboration With Lion TCR to Develop Allogeneic TCR-Based Natural Killer Cell Therapies for the Treatment of Virally Induced Cancers
June 24, 2020 – (Zelluna Immunotherapy) – The collaboration will initially focus on the development of TCR-NK products targeting hepatitis B virus, a leading cause of liver cancer with a high unmet need for efficient treatment options.
Century Therapeutics Announces Acquisition of Empirica Therapeutics
June 23, 2020 – (Century Therapeutics) – Century Therapeutics announced its acquisition of Empirica Therapeutics to leverage its iPSC-derived allogeneic cell therapies against glioblastoma.
China Inc. Eyes Gene Therapy in Post-Coronavirus Growth Trajectory
June 23, 2020 – (Scrip) – Hailed as a potential one-time cure for devastating genetic conditions, gene therapy provides much medical hope, but very few companies in China have taken the leap into the area due to murky regulations and a lack of policy incentives and reimbursement.
Editas Medicine Announces Pricing of Offering of Common Stock
June 23, 2020 – (Editas Medicine) – Gross proceeds from the offering are expected to be approximately $187.5M.
Sana Biotechnology Announces Completion of Initial $700M Financing
June 23, 2020 – (Sana Biotechnology) – Proceeds are expected to support IND-enabling and initial clinical studies for multiple therapeutic candidates, buildout of manufacturing capabilities, and expansion of the company’s portfolio of enabling technologies. They will also support the continued addition of top talent to Sana’s team. 
Cell and Gene Therapy 2.0 Dominates Latest Round of Biotech IPOs in Scramble for a $300M+ Nasdaq Windfall
June 22, 2020 – (Endpoints News) – The market is running red hot for all things drug-development related – and last Friday, Nasdaq loaded up a new round of biotech IPOs to sell to investors.
Epidarex Capital Closes New £102M UK Venture Fund to Build Successful Life Science Companies
June 22, 2020 – (Epidarex Capital) – Epidarex is a transatlantic venture firm that creates and invests in early stage, high-potential life science companies in emerging research hubs. Epidarex partners at an early stage with top scientists and entrepreneurs in the UK and the US to develop highly innovative products addressing major unmet needs in the global healthcare market.
IVERIC bio Announces Closing of Public Offering and Concurrent Private Placement with $160M in Gross Proceeds
June 22, 2020 – (IVERIC bio) – The company received net proceeds of approximately $117M from the public offering and approximately $33M from the concurrent private placement.
Minerva Biotechnologies Announces License Agreement with Memorial Sloan Kettering Cancer Center for 1XX Technology
June 22, 2020 – (Minerva Biotechnologies) – Minerva Biotechnologies announced that it has licensed from Memorial Sloan Kettering Cancer Center “1XX” technology for use with Minerva’s proprietary anti-MUC1* antibodies to increase CAR T cell persistence in patients.
Rexgenero Acquires the Key Technology and Program Assets of
June 22, 2020 – (Rexgenero) – Rexgenero, a regenerative medicine company developing advanced cell therapies to treat chronic limb-threatening ischaemia, announced the acquisition of all the key assets of, a preclinical-stage immunotherapy biotech company.
Sarepta Therapeutics and Codiak BioSciences Collaborate to Research and Develop Exosome-Based Therapeutics for Rare Diseases
June 22, 2020 – (Sarepta Therapeutics) – The two-year agreement includes up to five neuromuscular targets. Codiak is eligible to receive up to $72.5M in upfront and near-term license payments plus research funding. Sarepta is granted an option to any of the candidates developed pursuant to the research alliance.
Kiadis Announces First Patient Enrolled in Clinical Study in R/R AML With Off-the-Shelf K-NK Cells From Universal Donors as Part of Its K-NK003 Program
June 25, 2020 – (Kiadis Pharma) – The trial is expected to provide further clinical proof-of-concept of Kiadis’ K-NK003 product. Kiadis is supporting the investigator-sponsored study through a collaborative research agreement with OSUCCC-James.
Achilles Therapeutics Doses First Patient in Phase 1/2 Study in Advanced Non-Small Cell Lung Cancer
June 23, 2020 – (Achilles Therapeutics) – The trial is expected to recruit approximately 40 patients and report interim data in the first half of 2021.
Medigene Enters Phase 1 Clinical Development With Its TCR-T Immunotherapy MDG1021 in Hematological Cancers
June 23, 2020 – (Medigene) – MDG1021 is a TCR-T immunotherapy targeting a specific, immunogenic form of the antigen HA-1. The Phase 1 study will be initiated at the Leiden University Medical Center in the Netherlands, and will enroll patients suffering from relapsed or persistent blood cancers after allogeneic HSCT.
Ziopharm Oncology Completes Enrollment of Phase 2 Trial Evaluating Controlled IL-12 in Combination with Libtayo in Patients With Glioblastoma
June 22, 2020 – (Ziopharm Oncology) – Ziopharm’s Controlled IL-12 platform is an investigational gene therapy designed to induce and control the production of human interleukin 12, a master-regulator of the immune system.
uniQure Announces First Two Patients Treated in Phase 1/2 Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
June 19, 2020 – (uniQure) – The first two patients will be observed for an initial period of 90 days, followed by a meeting of the Data Safety Monitoring Board (DSMB). The DSMB will review the data on the first two patients and make a determination about continued dosing of the next patients.
Cynata Announces Ethics Approval and Expedited Regulatory Pathway for Phase 3 Osteoarthritis Clinical Trial
June 18, 2020 – (Cynata Therapeutics) – The TGA has advised that the trial can be conducted under the Clinical Trial Notification scheme subject to Cynata supplying appropriate GMP compliance documentation to the trial sponsor.
Mallinckrodt Completes Rolling Submission of Biologics License Application for StrataGraft Regenerative Skin Tissue to US FDA
June 9, 2020 – (Mallinckrodt) – The FDA granted StrataGraft skin tissue orphan drug status, and it was among the first products receive Regenerative Medicine Advanced Therapy (RMAT) designation under the provisions of the 21st Century Cures Act.
CRISPR Gene Editing in Human Embryos Wreaks Chromosomal Mayhem
June 25, 2020 – (Nautre) – A suite of experiments that use the gene-editing tool CRISPR–Cas9 to modify human embryos have revealed how the process can make large, unwanted changes to the genome at or near the target site.
One-Time Treatment Generates New Neurons, Eliminates Parkinson’s Disease in Mice
June 24, 2020 – (UC San Diego) – Researchers found that inhibiting or deleting just a single gene, the gene that encodes PTB, transforms several types of mouse cells directly into neurons.
Identifying the Risks of Unproven Regenerative Medicine Therapies
June 17, 2020 – (JAMA) – The commentary piece by FDA Director Dr. Stephen Hahn and CBER Director Dr. Peter Marks asserts, "It is time for unproven and unapproved regenerative medicine products to be identified and recognized for what they frequently are: uncontrolled experimental procedures at a cost to patients, both financially and physically."
  • ARM's statement on appropriate regulatory oversight of regenerative medicine and advanced therapy product candidates is available here.
A Year In, First Patient To Get Gene Editing For Sickle Cell Disease Is Thriving
June 23, 2020 – (NPR) – As the one-year anniversary of her landmark treatment approaches, Victoria Gray has just received good news: the billions of genetically modified cells doctors infused into her body clearly appear to be alleviating virtually all the complications of her sickle cell disease.
Director, Communications
Alliance for Regenerative Medicine – Washington, DC
Senior Director, Sales & Business Development, BioTherapies
Be The Match / National Marrow Donor Program – Boston, MA
Global Medical Affairs Therapeutic Area Leader, Hematology
Spark Therapeutics – Philadelphia, PA
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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