ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
April 23, 2020
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Caladrius Biosciences Plans to Assess Its CLBS119 Cell Therapy for Repair of COVID-19 Induced Lung Damage
April 23, 2020 – (Caladrius Biosciences) – Caladrius Biosciences announced that the US FDA has authorized its investigational new drug application for the study of CLBS119, a CD34+ cell therapy, for repair of COVID-19 induced lung damage.
The Coronavirus Pandemic Could Shrink Some Biotech Startup Valuations
April 23, 2020 – (STAT News) – The coronavirus pandemic means every step in that process has become far more uncertain in the past two months – and that added uncertainty means some biotech companies may be worth less on paper than they were before.
OPINION: People Are Dying From Coronavirus Because We’re Not Fast Enough at Clinical Research
April 22, 2020 – (STAT News) – This does not mean that it would be better to provide people with unproven therapies – or that COVID-19 studies should be accelerated so fast that we draw the wrong conclusions or put people at risk. But our inability to start and run clinical trials faster is a legacy of our decision not to develop the technologies and approaches that would make doing so easier.
Biotech Listings Help Prop Up Anemic US IPO Market
April 21, 2020 – (Bloomberg) – Biotechnology offerings are one of the few bright spots in an otherwise dreary market for initial public offerings and could hold steady for the rest of the year, even as the pandemic keeps listings in other sectors shut.
Duke-NUS Scientists Explore Using ‘Own’ Immune Cells to Target Infectious Diseases Including COVID-19
April 21, 2020 – (Duke-NUS Medical School) – Scientists from Duke-NUS Medical School are exploring the use of CAR-T and TCR therapies as a potential strategy for controlling infectious diseases, including COVID-19.
White House, Congress Reach Deal to Replenish Small Business Loan Program
April 21, 2020 – (The Hill) – The deal totals more than $480B and appropriates $320B for the Small Business Administration’s Paycheck Protection Program, $60B of which is set aside for small lenders and community financial institutions.
What's in Trump's Three-Phase Reopening Plan
April 16, 2020 – (POLITICO) – President Donald Trump's new guidelines for reopening parts of the country recommend states and localities confirm a two-week downward trend in coronavirus symptoms and documented cases before starting to ease lockdowns while assuring hospitals have adequate capacity and robust testing in place.
COVID-19 ARDS Patients Added to Healios One-Bridge Study
April 13, 2020 – (Healios) – The company intends to verify the results of the approximately five newly added COVID-19 ARDS cases separately from the 30 originally planned ONE-BRIDGE patients. The addition of this COVID-19 cohort will have no effect on the progress of the originally planned clinical trial.
Predictive Submits Emergency Use Authorization Application for Treatment of ARDS Secondary to COVID-19 With Umbilical Cord Mesenchymal Stem Cells
April 13, 2020 – (Predictive Biology) – The proposed IND clinical trial will utilize Predictive’s proprietary core technology of naturally occurring MSCs derived from umbilical cord tissue to assess the efficacy as an add-on therapy to standard treatment of patients with severe ARDS secondary to COVID-19.
SwanBio Therapeutics Announces Series A Expansion to Fund Its Lead Program and Grow Pipeline in Gene Therapy for Neurological Diseases
April 23, 2020 – (SwanBio Therapeutics) – SwanBio Therapeutics announced the successful completion of a $52M expanded Series A financing. The funding will help rapidly progress the understanding and development of AAV-based gene therapies, advance manufacturing and development capabilities, and allow the execution of a robust hiring plan to meet the demands of the pipeline.
AskBio Acquires BrainVectis to Expand Its Clinical Pipeline for Neurodegenerative Diseases
April 22, 2020 – (AskBio) – The company’s lead gene therapy candidate, BV-CYP01, has shown proof-of-concept in various animal models of Huntington’s disease and received Orphan Drug Designation from the European Commission in April 2019.
Evotec Regains Global Rights to Beta Cell Replacement Therapy
April 22, 2020 – (Evotec) – The beta cell program has already achieved preclinical data demonstrating that they are functionally equivalent to primary human islets in their ability to normalize blood glucose levels in in vivo models over several months.
Myosana Therapeutics Raises Up To $1M from CureDuchenne Ventures
April 22, 2020 – (Myosana Therapeutics) – Myosana will use this initial funding to advance development of its innovative non-viral gene therapy to deliver full length dystrophin for Duchenne muscular dystrophy.
Gilead, Kite and oNKo-innate Announce Research Collaboration to Discover Cancer Immunotherapies Focused on Natural Killer Cells
April 22, 2020 – (Gilead) – Under the terms of the agreement, oNKo-innate will receive an upfront payment and will be eligible to receive additional payments based on achievement of certain clinical, regulatory, and commercial milestones.
MilliporeSigma Boosts Commercial Viral Vector and Gene Therapy Manufacturing Capacity
April 21, 2020 – (MilliporeSigma) – MilliporeSigma announced a second Carlsbad, California-based facility for its viral and gene therapy service offering. The new $110M commercial facility is expected to open next year.
Mogrify and Sangamo Announce Collaboration and Exclusive License Agreement for Mogrify’s iPSC- and ESC-Derived Regulatory T Cells
April 21, 2020 – (Sangamo) – Under the terms of the agreement, Sangamo will pay Mogrify an upfront payment. Mogrify is also eligible to receive potential additional payments related to development and regulatory milestones, and product sales.
New iPSC Master Cell Banks to Accelerate Development of Novel Cell Therapies
April 21, 2020 – (panCELLa) – panCELLa and CCRM have executed a collaboration agreement using novel technology to generate commercial-use and clinical-grade iPSC lines which will be made available to academia and industry to enable the development of new therapies.
Athersys Announces Closing of Public Offering of Common Stock
April 20, 2020 – (Athersys) – Gross proceeds to Athersys from the offering are approximately $58M. 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 initiation of a Phase 2/3 pivotal study for COVID-19 induced ARDS patients.
MicrofluidX Raises £1.4M in Seed Funding to Develop Novel Cell Therapy Bioprocessing Technology
April 20, 2020 – (MicrofluidX) – MicrofluidX announced it has raised £1.4M in seed funding from leading seed investors UK Innovation & Science Seed Fund, Longwall Ventures, and Moulton Goodies Limited with angel contributions from 88 Capital and Cambridge Angels.
Sofinnova Partners Announces Third Investment From Its Italian Fund
April 20, 2020 – (Sofinnova Partners) – Sofinnova Partners announced an investment in gene therapy company Genespire. The biotech was founded last month by Naldini, professor Alessio Cantore, The Telethon Foundation and San Raffaele Hospital and is a spin-off of SR-Tiget.
Novartis Kymriah Receives FDA Regenerative Medicine Advanced Therapy Designation in Follicular Lymphoma
April 22, 2020 – (Novartis) – The potential approval in r/r FL will be the third indication for Kymriah, which also has indications in r/r pediatric and young adult acute lymphoblastic leukemia, and r/r adult diffuse large B-cell lymphoma.
REGENXBIO Announces Additional Positive Long-Term and Interim Phase 1/2a Trial Update for RGX-314 for the Treatment of Wet AMD
April 22, 2020 – (REGENXBIO) – Positive long-term potential efficacy signals were sustained over two years in Cohort 3. The mean change in visual acuity across all six patients in Cohort 3 was markedly improved over two years, with a mean BCVA improvement of +14 letters.
FDA Grants Passage Bio Orphan Drug Designation for PBGM01 for Treatment of Infantile GM1 Gangliosidosis
April 21, 2020 – (Passage Bio) – The company plans to submit an IND for PBGM01 in the second quarter of 2020 and to initiate a Phase 1/2 trial in the second half of 2020 with clinical data expected in the first half of 2021.
WindMIL Therapeutics Opens Combination Therapy Portion of Its Phase 2a Clinical Trial of Marrow-Infiltrating Lymphocytes for Treatment of Patients with Non-Small Cell Lung Cancer
April 21, 2020 – (WindMIL) – WindMIL plans to treat approximately 20 patients with MILs - NSCLC plus nivolumab in this next part of the study. The study’s primary endpoint is objective response rate, with duration of response, progression free survival and overall survival serving as secondary endpoints.
Mustang Bio Receives Advanced Therapy Medicinal Product Classification from EMA for MB-107 Lentiviral Gene Therapy for X-Linked Severe Combined Immunodeficiency
April 20, 2020 – (Mustang Bio) – The ATMP program provides specific regulatory guidelines for preclinical development, manufacturing and product quality testing of ATMPs and offers incentives, including fee reductions for regulatory advice, recommendations and evaluation and certification of quality and non-clinical data.
Diabetes Reversed in Mice With Genetically Edited Stem Cells Derived From Patients
April 22, 2020 – (Washington University St. Louis) – Using iPSCs produced from the skin of a patient with a rare genetic form of insulin-dependent diabetes, researchers transformed the human stem cells into insulin-producing cells and used the gene-editing tool CRISPR-Cas9 to correct a genetic defect that had caused the syndrome. They then implanted the cells into lab mice and cured the unrelenting diabetes in those mice.
Study Shows Glaucoma Could Be Successfully Treated With Gene Therapy
April 21, 2020 – (University of Bristol) – Using CRISPR, researchers targeted a gene called Aquaporin 1 in the ciliary body. Inactivating the gene led to reduced eye pressure. Researchers then demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue.
CRISPR-Based ‘Discovery Engine’ for New Cell Therapies to Advance Cancer Treatments
April 17, 2020 – (University of California San Francisco) – Researchers have developed a breakthrough technique in which they use electroporation to enable CRISPR gene-targeting technology to quickly and efficiently reprogram T-cells with new functions. They applied this technique to power a high-throughput platform to evaluate the specificity and potency of many different potential cell therapies simultaneously.
Simulations Show How to Make Gene Therapy More Effective
April 17, 2020 – (University of Groningen) – Researchers used simulations to investigate the use of lipoplexes as a gene therapy delivery method. The simulation results could be used to improve the therapy's efficiency.
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
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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