ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
August 7, 2020
Quick Links
New Report: Regenerative Medicine & Advanced Therapies Sector Thriving Despite COVID-19
August 6, 2020 – (ARM) – This week, ARM announced the publication of its H1 2020 Global Sector Report, “Innovation in the Time of COVID-19.” The report provides an in-depth look at trends and metrics in the gene, cell, and tissue-based therapeutic sector in the midst of the pandemic. With more than 1,000 developers active worldwide, the industry is poised to break fundraising records.
Freeline Announces Pricing of Initial Public Offering
August 7, 2020 – (Freeline Therapeutics) – Gross proceeds from the financing are expected to be approximately $159M. In addition, Freeline has granted the underwriters a 30-day option to purchase up to an additional 1,323,529 ADSs at the initial public offering price.
Catalent Gene Therapy Facility Receives FDA Approval as an Additional Manufacturing Site for AveXis’ Gene Therapy
August 6, 2020 – (Catalent) – Catalent announced that it was approved by the FDA to produce commercial drug substance intermediate for AveXis’ spinal muscular atrophy gene therapy at its manufacturing facility located in Harmans, Maryland.
Editas Medicine Regains Full Global Rights to Ocular Medicines
August 6, 2020 – (Editas Medicine) – Editas Medicine announced it has regained full global rights to its ocular medicines, which were previously shared within a strategic research and development alliance with Allergan, which has since been acquired by AbbVie. Editas Medicine and AbbVie have terminated the original agreement and entered into a new agreement.
Immatics Extends Cell Therapy Manufacturing Collaboration With UTHealth
August 6, 2020 – (Immatics) – Under the agreement, UTHealth will provide Immatics with exclusive access to three cGMP suites and support areas for the manufacturing of various adoptive cell therapy products.
T-knife Completes €66M Series A Financing to Develop Next-Generation T-Cell Therapies
August 6, 2020 – (T-knife) – Proceeds from the Series A round will be allocated to advancing at least four programs into the clinic, ramping-up preclinical work for additional selected proprietary pipeline candidates and discovering TCRs against novel targets.  
DCVC Announces Close of $350M Early-Stage Fund Focused on Deep Tech-Enabled Life Sciences
August 5, 2020 – (DCVC) – Since launching in 2018, DCVC has invested in companies delivering individualized cell therapy treatments for cancer, building new living medicines to tackle chronic diseases, discovering genome-based therapeutic treatments for diseases and revolutionizing antibody discovery processes.
GentiBio Launches With $20M Seed Funding to Deliver Immune Tolerizing Therapies for Autoimmune and Inflammatory Diseases
August 5, 2020 – (GentiBio) – GentiBio also announced it has entered into exclusive licensing partnerships with Seattle Children's Research Institute, Benaroya Research Institute at Virginia Mason, and MIGAL Galilee Research Institute to advance the company's unique immune tolerance platform.
Taysha Gene Therapies Announces $95M Series B Financing to Bolster Initial Clinical Studies and Advance Pipeline of Gene Therapies for Monogenic CNS Disease
August 5, 2020 – (Taysha Gene Therapies) – TSHA-101 is expected to initiate clinical studies later this year for the treatment of GM2 Gangliosidosis, followed by TSHA-102 for the treatment of Rett syndrome, TSHA-103 for the treatment of SLC6A1 haploinsufficiency disorder and TSHA-104 for the treatment of SURF1 deficiency.
Versant Ventures Launches Matterhorn Biosciences
August 5, 2020 – (Matterhorn Biosciences) – Versant Ventures announced the debut of a new biotechnology company developing T cell receptor therapies based on the recent discovery of MR1T cells that recognize and kill a wide range of tumors of various tissue origins. Versant has made a $30M commitment to Matterhorn Biosciences.
ViaCyte and Gore Enter Clinical Phase Agreement Based on Novel Membrane Technology for PEC-Encap Product Candidate
August 5, 2020 – (ViaCyte) – The Encaptra System has the potential to negate the need for immunosuppression and is an important component of the PEC-Encap product candidate that ViaCyte is developing as a potentially transformative therapy for patients with type 1 diabetes. 
Appetite for US Biopharma IPOs Remains Hot in July
August 4, 2020 – (BioWorld) – The biopharmaceutical sector welcomed a further 11 new IPO graduates in July, bringing the number to 39 that have started to trade on the US markets this year. The companies going public last month collectively raised more than $2.5B.
Sernova Enters into Exclusive Worldwide License Agreement with University of Miami for Therapeutic Cell Immune Protection Technologies
August 4, 2020 – (Sernova Corp) – This exclusive worldwide license agreement broadens the technology scope of Sernova’s immune protection conformal coating technologies and related intellectual property.
Cytovia Therapeutics Acquires Worldwide Rights to CytoImmune Therapeutics’ EGFR Dual-Targeting CAR for NK Cell Treatment of Glioblastoma & Other Solid Tumors
August 3, 2020 – (Cytovia Therapeutics) – Cytovia will conduct and finance all future development and will apply the EGFR Dual-targeting CAR to its iPSC CAR NK technology. CytoImmune will receive an upfront equity grant in Cytovia, future development milestones, and royalties.
Oxford Biomedica Signs Development, Manufacture & License Agreement with Beam Therapeutics for LentiVector Platform for Next-Generation CAR-T Therapeutics
August 3, 2020 – (Oxford Biomedica) – Under the terms of the DMLA, Oxford Biomedica will receive an undisclosed upfront payment, as well as payments related to the development and manufacturing of lentiviral vectors for use in clinical trials; certain development and regulatory milestones; and undisclosed royalties.
UCL Technology Fund Announces First Close of £100M Fund 2
August 3, 2020 – (UCL Business) – The second fund has an immediate pipeline of opportunities with world-renowned UCL academic researchers. In the life sciences, these include innovative preclinical and clinical projects and spinouts. Based on pioneering gene therapy, cell therapy, and drug discovery approaches, they are targeting a variety of indications with a global unmet need.
Legend Biotech Announces Chief Executive Officer Transition
August 2, 2020 – (Legend Biotech) – Legend Biotech Corporation announced that its Board of Directors has appointed Dr. Frank Zhang to serve as Chief Executive Officer. Dr. Zhang has concurrently stepped down as Chief Executive Officer of GenScript, Legend Biotech’s majority shareholder, in order to focus his attention on Legend Biotech.
Thermo Fisher Scientific and Lyell Immunopharma Form Cell Therapy Manufacturing Partnership
July 28, 2020 – (Thermo Fisher Scientific) – The companies will work together to improve the fitness of T cells, a challenge in manufacturing cell therapies, and support Lyell with its development of an integrated yet flexible cGMP-compliant platform including reagents, consumables and instrumentation.
Tessa Therapeutics Announces Results From Two Independent Phase 1/2 Trials of Autologous CD30 CAR-T Cell Therapy in Patients with Relapsed or Refractory Hodgkin Lymphoma
August 6, 2020 – (Tessa Therapeutics) – The overall response rate in the 32 patients with active disease who received fludarabine-based lymphodepletion was 72%, including 19 patients (59%) with complete response.
University of Minnesota Expands Clinical Investigation of Engineered iPSC-Derived Natural Killer Cells, Opening US Clinical Trial for the Treatment of COVID-19
August 6, 2020 – (University of Minnesota) – The first patient has received treatment in a new clinical trial that has opened at the University of Minnesota to test whether a novel cell therapy currently under clinical investigation as a treatment for acute myeloid leukemia and lymphoma can be effective as a treatment for COVID-19.
China's NMPA Has Recommended Breakthrough Therapy Designation for Ciltacabtagene Autoleucel, an Investigational BCMA CAR-T Cell Therapy
August 5, 2020 – (Legend Biotech) – The BTD process is designed to expedite the development and review of therapies that are intended for the treatment of serious diseases for which there is no existing treatment and where preliminary evidence indicates the advantages of the therapy over available treatment options. Cilta-cel is the first product that has been recommended for BTD in China.
Novartis Announces Kymriah Meets Primary Endpoint at Interim Analysis of Pivotal Study in Follicular Lymphoma
August 4, 2020 – (Novartis) – At the interim analysis, the global study met its primary endpoint of complete response rate, as assessed by an independent review committee.
REGENXBIO Announces Additional Positive Interim Phase 1/2a Trial Update and Program Updates for RGX-314 for the Treatment of Wet AMD
August 4, 2020 – (REGENXBIO) – Patients in Cohort 4 and Cohort 5 at one year after administration of RGX-314 demonstrated stable visual acuity with a mean BCVA change of +4 letters and -2 letters from baseline, respectively, as well as decreased retinal thickness.
Kadimastem Reports Positive Interim Results of Cohort B of Its AstroRx Phase 1/2a Clinical Trial in ALS
August 3, 2020 – (Kadimastem) – The rate of ALS disease progression in Cohort B patients, as assessed by the ALSFRS-R slope difference between pre-treatment and post-treatment periods, was significantly reduced.
Implanted Neural Stem Cell Grafts Show Functionality in Spinal Cord Injuries
August 5, 2020 – (University of California San Diego) – Researchers discovered that even in the absence of a specific stimulus, graft neurons fired spontaneously in distinct clusters of neurons with highly correlated activity, much like in the neural networks of the normal spinal cord.
New Hope as Dementia Therapy Reverses Memory Loss
August 30, 2020 – (Macquarie University) – Researchers used gene therapy to activate a naturally protective enzyme in the brain called p38gamma, which prevents the toxic effects of memory loss. In mice, the therapy was found to not only halt memory loss but completely reversed the loss already there.
Coordinator, Membership & Public Affairs
Alliance for Regenerative Medicine – Washington, DC
Senior Director – Cell & Gene Therapy Sales
Be The Match BioTherapies – Boston, MA
Director, CMC Regulatory Affairs
Century Therapeutics – Greater Philadelphia, PA
Business Development Manager, Strategy & Technology
Corning – Remote (Boston, MA; Mid-Atlantic; or San Francisco, CA)
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 360+ 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
Alliance for Regenerative Medicine
Alliance for Regenerative Medicine
Subscribe to Newsletter
Become a Member
Event Calendar
Copyright © 2020 AllianceRM, All rights reserved.

Want to change how you receive these emails?
You can update your preferences or unsubscribe.