ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
April 16, 2020
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Lineage CEO Discusses Effects of COVID-19 in Letter to Shareholders
April 16, 2020 – (Lineage Cell Therapeutics) – Lineage reported that patient enrollment in the clinical study of VAC2 would be affected by enrollment restrictions.
Magenta Therapeutics Provides Business Update in Light of COVID-19
April 16, 2020 – (Magenta Therapeutics) – Based on COVID-19-related impacts at Magenta’s clinical sites, enrollment timelines for the Phase 2 trial of the company's MGTA-456 cell therapy in inherited metabolic disorders have been shifted into 2021. The trial remains open with seven of 12 patients enrolled and continued follow-up on these patients will be conducted.
NantKwest and ImmunityBio Announce Therapeutics and Vaccines for Combatting COVID-19; Clinical Trials Anticipated to Begin This Quarter
April 14, 2020 – (NantKwest) – Leveraging ImmunityBio’s expertise in vaccine development and natural killer cell activation and NantKwest’s extensive experience in off-the-shelf, cell-based therapeutics, the companies are combining their resources to design and develop cell-based therapeutics and vaccines for COVID-19.
Analyst-Turned Biotech Exec-Turned Analyst Reads the Biotech Q1 Tea Leaves
April 13, 2020 – (Endpoints) – The coronavirus pandemic is in full swing. Biopharma R&D and regulatory timelines have been disrupted, supply chains interrupted and what the future holds is anybody’s guess. Now with the first quarter having come to a close, what can we expect in earnings reports?
Biotech in the Time of Coronavirus: The Return of Mergers, Acquisitions, and Deals
April 13, 2020 – (STAT News) – Assuming biotech deal-making does come roaring back, it will be interesting to watch how, if at all, disrupted clinical trials, grounded commercial operations, and other lingering effects of the shutdown impact the valuations of target companies.
FDA Authorizes Athersys to Initiate a Pivotal Clinical Trial Evaluating MultiStem Cell Therapy in Patients With COVID-19 Induced Acute Respiratory Distress Syndrome
April 13, 2020 – (Athersys) – The primary efficacy endpoint will be number of ventilator-free days through day 28 as compared to placebo, a well-established endpoint for ARDS trials that evaluates an intervention’s combined impact on survival and liberation from invasive mechanical ventilation.
Pluristem Expands Its Compassionate Use Program: Treated First COVID-19 Patient in US Under FDA Single Patient Expanded Access Program
April 13, 2020 – (Pluristem) – The patient was treated with PLX cell therapy at Holy Name Medical Center in New Jersey, an acute care facility that is currently an active site for Pluristem’s Phase 3 critical limb ischemia study.
ERC Launches Development Program for Novel Cell-Based Therapeutic and Prophylactic SARS-CoV-2 Vaccine
April 9, 2020 – (ERC) – The technology is based on ERC’s Gliovac/ERC1671 glioblastoma vaccine platform currently in an FDA approved clinical trial in the US. Researchers believe the vaccine could produce a therapeutic immune reaction that could resolve symptoms and cure infected patients.
MaxCyte Provides Business Update Related to COVID-19 Pandemic
April 9, 2020 – (MaxCyte) – CARMA Cell Therapies remains fully committed to the MCY-M11 clinical development program; however, timelines may be impacted due to the current deprioritization of non-COVID-19 clinical trials and restrictions on patient recruitment at the two clinical trial sites.
New Cell Therapy Trial Launches for Patients With Severe COVID-19
April 9, 2020 – (The Cure Alliance) – An international team of scientists has been granted immediate FDA authorization for a 24-patient clinical trial to test the safety and exploratory efficacy of umbilical cord-derived mesenchymal stem cells to block the life-threatening lung inflammation that accompanies severe cases of COVID-19.
Flexion Temporary Suspends Phase 1 Trial of Its FX201 Gene Therapy for the Treatment of Osteoarthritis of the Knee
April 1, 2020 – (Flexion Therapeutics) – Flexion is temporarily suspending its active clinical trials including the Phase 1 trial evaluating the safety and tolerability of FX201, its investigational intra-articular gene therapy product candidate, in patients with osteoarthritis of the knee. The decision was based on the recent guidance from the FDA on clinical trials during COVID-19.
Cobra Biologics and the Karolinska Institutet Collaborate to Develop COVID-19 Vaccine
March 30, 2020 – (Cobra Biologics) – Cobra Biologics and the Karolinska Institutet announced they have been awarded €3M in emergency funding by Horizon 2020 for the research, development, and Phase 1 clinical trial testing of a DNA vaccine against COVID-19.
Avectas Completes $20M Series C Funding
April 16, 2020 – (Avectas) – Avectas has completed a new Series C equity funding of approximately $20M, bringing the total equity invested in the business to date to $40M. The funds raised in this latest equity round will be used to accelerate the clinical translation and commercial scale-up of Avectas' proprietary cell engineering technology platform.
Athersys Announces Pricing of Public Offering of Common Stock
April 15, 2020 – (Athersys) – Gross proceeds to Athersys from the offering are expected to be approximately $50M. Athersys intends to use the net proceeds from the offering for working capital and general corporate purposes, including funding towards its ARDS clinical program, which includes a Phase 2/3 pivotal study for COVID-19 induced ARDS patients.
Chinese Investors Continue to Show Appetite for Biopharma and Med Tech, With Qiming’s $1.1B Fund
April 15, 2020 – (BioWorld) – Qiming Venture Partners said that it has established a new $1.1 billion fund to target early stage health care and technology investments, the latest good news for biopharma and med-tech startups.
InVivo Therapeutics Announces $3M Registered Direct Offering
April 15, 2020 – (InVivo Therapeutics) – The company intends to use the net proceeds from this offering for working capital, business development activities, and general corporate purposes.
TScan Announces Collaboration With Novartis on the Discovery and Development of Novel T Cell Receptor Therapies for the Treatment of Solid Tumors
April 15, 2020 – (TScan) – The collaboration includes an upfront technology access fee and research funding totaling $30M, and potential milestone payments contingent on clinical, regulatory and sales success that could aggregate in the hundreds of millions of dollars. TScan will identify and characterize TCRs, and Novartis will have the option to license and develop TCRs for up to three novel targets.
Axovant Announces Partnership With Invitae to Increase Access to Genetic Testing and Accelerate Diagnoses of GM1 and GM2 Gangliosidosis
April 15, 2020 – (Axovant) – The Detect program includes a specific LSD testing panel of 53 genes designed to provide patients and families accurate information quickly to preserve valuable treatment time.
Daiichi Sankyo, Mitsubishi UFJ Capital and Nagoya Institute of Technology Announce Open Innovation Research on Gene Therapy to Restore Vision
April 13, 2020 – (Daiichi Sankyo) – To carry out the research, a new company called OiDE OptoEye has been established and wholly funded by the OiDE Fund, operated by Mitsubishi UFJ Capital. If the pre-agreed goals of the three-year joint research are achieved, Daiichi Sankyo will purchase all of the stock of OptoEye in order to continue research for the project on its own.
Autolus Therapeutics Announces FDA Acceptance of IND Application for AUTO1 for Adult Acute Lymphoblastic Leukemia
April 16, 2020 – (Autolus Therapeutics) – The active IND allows initiation of the US sites in the company’s first pivotal study, AUTO1-AL1. The AUTO1-AL1 study clinical trial application was approved by the MHRA in January 2020 and the first site opened in the UK in March.
FDA Authorizes Initiation of a Phase 2 Trial Evaluating MultiStem Cell Therapy in Trauma Patients
April 15, 2020 – (Athersys) – Athersys announced that the FDA has authorized an IND application to initiate a Phase 2 clinical trial evaluating the company's MultiStem cell therapy for early treatment of traumatic injuries and the subsequent complications that result following severe trauma, including Systemic Inflammatory Response Syndrome (SIRS), which can contribute to organ failure or other severe complications.
GenSight Biologics Announces Positive DSMB Review of PIONEER Phase 1/2 Trial of GS030 Combining Gene Therapy and Optogenetics for the Treatment of Retinitis Pigmentosa
April 14, 2020 – (GenSight Biologics) – GenSight expects to complete enrollment in the second half of 2020, with early findings from the first two cohorts potentially available by the end of 2020, dependent on the course of the COVID-19 pandemic.
Kolon TissueGene Announces Plans To Resume US Phase 3 Clinical Trial For TG-C
April 13, 2020 – (Kolon TissueGene) – Kolon TissueGene announced that the clinical hold issued by the FDA in April 2019 has been lifted effective April 10, 2020, and that the company can continue with its knee osteoarthritis phase 3 clinical trial. Kolon TissueGene has begun activities to resume the Phase 3 trial and reinitiate enrollment later this year.
PolarityTE Announces Data From Head-to-Head Burn Study Demonstrating Graft Take and Wound Closure with SkinTE Treatment
April 3, 2020 – (PolarityTE) – AHSC-treated wounds had graft take and achieved closure by their last follow-up with a single application. There were no AHSC treatment related adverse events.
Fate Therapeutics Announces First Patient Treated in First-in-Human Clinical Trial of FT596
April 2, 2020 – (Fate Therapeutics) – Fate Therapeutics announced that the first patient has been treated in the company’s Phase 1 clinical trial evaluating FT596 in patients with B-cell malignancies and chronic lymphocytic leukemia. FT596 is an off-the-shelf CAR-NK cell cancer immunotherapy.
Researchers Restore Sight in Mice by Turning Skin Cells Into Light-Sensing Eye Cells
April 15, 2020 – (NIH) – iPS cell reprogramming protocols can take six months before cells or tissues are ready for transplantation. By contrast, the direct reprogramming described in this study coaxed skin cells into functional photoreceptors ready for transplantation in only 10 days.
Scientists Repair Stroke-Damaged Rat Brain
April 8, 2020 – (Lund University) – Researchers from Lund University have now succeeded in restoring mobility and sensation in rats that have suffered a stroke by transplanting neurons developed from reprogrammed human skin cells into the animals' brains.
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
Manager, Clinical Quality
Abeona Therapeutics – Cleveland, OH
Senior Research Scientist
Kytopen – Cambridge, MA
Senior Application Scientist – Cell Therapy
Treefrog – Pessac, France
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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