ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
July 2, 2020
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ARM's 2020 Cell & Gene Meeting on the Mesa Goes Virtual
July 2, 2020 – (ARM) – The conference, which will now take place over five days, includes a virtual form of the meeting's signature partnering system, expected to facilitate more than 3,000 one-to-one meetings between industry leaders. The program will include 15+ digital panels and workshops featuring key industry leaders and 80+ presentations from leading public and private companies in the space. Early bird rates are available through July 24. Register today!
Join ARM, the ARM Foundation, and ISCT for a Webinar on Potential COVID-19 Treatments Using Cell Therapies
July 2, 2020 – (ARM) – On Tuesday, July 7 at 11:30 am ET, join leading experts in the cell and gene therapy sector for a webinar bringing together science, clinical information, and business ethics surrounding the use of mesenchymal stem cells to treat COVID-19. This 75-minute webinar will cover the science and potential therapeutic benefit of MSCs to treat COVID-19, as well as what patients and patient groups should understand about these therapies. See the full agenda here.
Vericel Announces Submission of Biologics License Application to the FDA for NexoBrid for the Treatment of Severe Thermal Burns
June 30, 2020 – (Vericel) – The BLA submission for NexoBrid is based on multiple preclinical and clinical studies, including the pivotal Phase 3 US clinical study of NexoBrid in adult patients with deep partial-thickness and full-thickness thermal burns up to 30% of total body surface area. The study met its primary endpoint of complete eschar removal as well as all secondary endpoints.
Clinical Trial of Pioneering T Cell Therapy for COVID-19 Patients
July 1, 2020 – (TC BioPharm) – The company has submitted documents to UK regulators to start safety trials on an experimental therapy designed to kill COVID-19 infected cells. The initial clinical trial site is anticipated to be the Edinburgh Royal Infirmary.
Biopharma Tackles COVID-19, HIV and Other Viruses With Gene and Cell Therapies
June 29, 2020 – (Fierce Biotech) – Biopharma companies and academic researchers have created engineered immune cells, CRISPR-edited gene products and more to combat not just COVID-19 but also influenza and HIV, the virus that causes AIDS. Though many challenges have to be overcome before these therapies are ready for widespread use against viruses, early signals suggest they could offer a promising new strategy.
BioLife Solutions Announces Pricing of $75M Public Offering of Common Stock
July 2, 2020 – (BioLife Solutions) – BioLife anticipates using net proceeds from the offering for general corporate purposes, which includes potentially investing in or acquiring companies that are synergistic with or complementary to the company's technologies, and working capital.
Immatics Announces Completion of Business Combination With Arya Sciences
July 2, 2020 – (Immatics) – Proceeds from this transaction were approximately $253M, which included funds held in Arya’s trust account and the common stock private investment in public equity financing contributed by a group of leading US healthcare institutional investors.
Massachusetts Eye and Ear Enters Licensing Agreement with Biogen to Develop Treatment for Inherited Retinal Disorder
July 2, 2020 – (Massachusetts Eye and Ear) – Massachusetts Eye and Ear has entered into an exclusive licensing agreement with Biogen to develop a potential treatment for inherited retinal degeneration due to mutations in the PRPF31 gene, which are among the most common causes for autosomal dominant retinitis pigmentosa.
MiMedx Announces Concurrent $15M Private Equity and Debt Financings
July 2, 2020 – (MiMedx) – The additional resources are intended to strengthen areas critical to the business, prioritize investments that enhance research & development, manufacturing, and commercial operations, and pursue the growth opportunities afforded by the company's amniotic tissue products and know-how.
Sarepta Therapeutics Signs Agreement With Hansa Biopharma for Imlifidase
July 2, 2020 – (Sarepta Therapeutics) – Under the terms of the agreement, Sarepta obtains an exclusive, worldwide license to develop and promote imlifidase as a pre-treatment to enable Sarepta gene therapy administration in Duchenne muscular dystrophy and limb-girdle muscular dystrophy, for patients who may otherwise not be eligible for treatment.
Lysogene Announces a Research Collaboration With the Weizmann Institute of Science
July 1, 2020 – (Lysogene) – The agreement involves collaboration between Lysogene and Yeda Research and Development, the commercial arm of the Weizmann Institute of Science, with the aim of developing a novel AAV gene therapy approach for neuronopathic Gaucher disease, Parkinson disease, and other diseases associated with mutations of the GBA1 gene.
Akouos Announces Closing of Initial Public Offering
June 30, 2020 – (Akouos) – The gross proceeds of the offering were approximately $244M. In addition, Akouos has granted the underwriters an option for a period of 30 days to purchase up to 1.8M additional shares of common stock at the initial public offering price.
Carmine Therapeutics and Takeda Collaborate to Discover and Develop Rare Disease Gene Therapies Using Novel Red Blood Cell Extracellular Vesicles Platform
June 30, 2020 – (Carmine Therapeutics) – Under the terms of the agreement, Carmine will receive an upfront payment, research funding support, and is eligible for over $900M in total milestone payments plus tiered royalties.
CRISPR Therapeutics Announces Pricing of Public Offering of Common Shares
June 30, 2020 – (CRISPR Therapeutics) – CRISPR Therapeutics anticipates its gross proceeds from the offering to be approximately $450M.
Freeline Closes $120M Series C Financing Round
June 30, 2020 – (Freeline Therapeutics) – Freeline expects to use the proceeds from the financing to bring its lead program in hemophilia B into a pivotal trial, as well as to continue its Phase 1/2 clinical program for Fabry disease and further progress its pipeline programs for Gaucher disease and hemophilia A.
Magenta Therapeutics Announces Closing of Public Offering, Including Full Exercise of Underwriters’ Option to Purchase Additional Shares
June 30, 2020 – (Magenta Therapeutics) – The total gross proceeds from the offering are expected to be $69M. Magenta intends to use the net proceeds from this offering to advance its clinical and earlier-stage programs and for research and development, working capital, and general corporate purposes.
Marker Therapeutics Announces New Manufacturing Facility to Support Clinical Development of MultiTAA-Specific T Cell Therapy Product Candidates
June 30, 2020 – (Marker Therapeutics) – The facility, located in Houston, TX, will allow production according to US FDA guidelines and is designed to be scalable using modular processes. The facility is expected to be completed by year-end and operational in 2021.
Public Biopharmaceutical Companies Post Strong Gains in Second Quarter
June 30, 2020 – (BioWorld) – The share prices of blue-chip biopharmaceutical companies closed out the month on a high note to contribute to their stellar collective performance during the second quarter, with the BioWorld Biopharmaceutical index increasing almost 20%. Small- and mid-cap biopharma companies also enjoyed a strong period fueled by positive clinical trial data and new drug approvals.
Immunotech Plans to Raise $142M on HKEX to Advance AAL Therapy for Liver Cancer
June 29, 2020 – (BioWorld) – The HK$1.1B (US$142M) financing is expected to close on July 10. Proceeds will advance the company's therapy for liver cancer.
Editas Medicine Announces Closing of Offering of Common Stock and Full Exercise by Underwriter of Option to Purchase Additional Shares
June 26, 2020 – (Editas Medicine) – Gross proceeds from the offering were approximately $215M. All of the shares in the offering were sold by Editas Medicine.
BrainStorm Announces Completion of All Dosing in NurOwn Phase 3 Clinical Trial in ALS
July 2, 2020 – (BrainStorm Cell Therapeutics) – The trial enrolled approximately 200 participants, randomized to receive three doses of MSC-NTF cells or placebo. The company expects top-line data from the trial to be available in the fourth quarter of 2020.
IVERIC bio Announces First Patient Dosed in Second Zimura Phase 3 Clinical Trial for the Treatment of Geographic Atrophy Secondary to Age-Related Macular Degeneration
June 30, 2020 – (IVERIC bio) – Approximately 400 patients will be randomized to receive either monthly administration of Zimura or sham during the first 12 months of the trial. If the 12 month results are positive, the company plans to file an application with the US FDA and the European Medicines Agency for marketing approval of Zimura.
First Patient Infused in Joint Clinical Study by Humanigen and Kite, A Gilead Company
June 30, 2020 – (Humanigen) – The study is evaluating the potential effects of lenzilumab prior to Yescarta in adults with relapsed or refractory large B-cell lymphoma.
Avalon GloboCare Announces Successful Completion of Phase 1 Clinical Study of CAR-T Cell Therapy Candidate AVA-001
June 29, 2020 – (Avalon GloboCare) – Nine out of ten patients with relapsed/refractory B-cell acute lymphoblastic leukemia achieved complete remission within one month of the administration of AVA-001. All patients who achieved CR successfully proceeded to allogeneic bone marrow transplant with curative intent.
ReNeuron Announces Additional Positive Data From Trial of Cell Therapy for Retinitis Pigmentosa
June 29, 2020 – (ReNeuron) – The company reports that the latest data, which include up to 18 months of follow up, continue to demonstrate the efficacy of the therapy, with a clinically meaningful benefit being observed at all time-points.
Rocket Pharmaceuticals Announces FDA Clearance of IND for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis
June 29, 2020 – (Rocket Pharmaceuticals) – The non-randomized, open-label Phase 1 clinical trial will enroll two pediatric patients, one month of age or older. The trial is designed to assess the safety and tolerability of RP-L401, as well as preliminary efficacy, including potential improvements in bone abnormalities/density, hematologic status, and endocrine abnormalities.
SanBio's Regenerative Cell Medicine SB623 Awarded Orphan Designation in Japan
June 29, 2020 – (SanBio) – The company is making preparations to apply for manufacturing and marketing approval of regenerative cell medicine SB623 for the treatment of chronic effects associated with TBI in Japan within the second half of FY2020.
A Simpler Way to Make Sensory Hearing Cells
June 30, 2020 – (University of Southern California) – To achieve reprogramming, the scientists exposed fibroblasts and supporting cells to a cocktail of four transcription factors. The resulting iHCs resembled naturally occurring hair cells in terms of their structure, electrophysiology, and genetic activity.
Engineered Immune Cells Recognize, Attack Human and Mouse Solid-Tumor Cancer Cells
June 29, 2020 – (University of Illinois) – In a new study, researchers report that they have dramatically broadened the potential targets of CAR-T using antibodies known to interact with a specific type of abnormally formed sugar attached to a protein on solid-tumor cancer cells in mice.
In Its First Tough Test, CRISPR Base Editing Slashes Cholesterol Levels in Monkeys
June 27, 2020 – (STAT News) – When CRISPR base editing was used to knock out two cholesterol-associated genes in monkeys, the animals’ blood levels of heart-disease-causing LDL (“bad”) cholesterol and triglycerides plunged as much as 60% and 65%, respectively.
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
Executive Director
Standard Coordinating Body for Regenerative Medicine – Gaithersburg, MD
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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