ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
April 30, 2020
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Phase 2/3 Randomized Controlled Trial of Remestemcel-L in 300 Patients With COVID-19 Acute Respiratory Distress Syndrome Begins Enrollment
April 30, 2020 – (Mesoblast) – More than 20 medical centers across the United States will participate in the trial, which is expected to complete enrollment within three to four months, with interim analyses planned.
FDA Official Steps Into Vaccine Vacuum After Shakeup
April 29, 2020 – (Politico) – Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research at the FDA, has been asked to accelerate vaccine development work and help coordinate between agencies, said a former government official familiar with the discussions.
New Capricor Data Reports 100 Percent Survival in Critical COVID-19 Patients Treated with CAP-1002
April 29, 2020 – (Capricor Therapeutics) – Of the six patients treated, four of them have been discharged. Following a review of the available data, the US FDA approved the company’s expanded access protocol to treat up to 20 additional COVID-19 patients.
Commission Issues Guidance to Mitigate Clinical Trial Disruption in the EU
April 28, 2020 – (European Commission) – The updated guidance reflects feedback from sector stakeholders, including ARM. Key revisions include updated guidance on the distribution of medicines to patients in clinical trials; remote source data verification; and communicating changes to clinical trial protocols to regulatory authorities.
These Kids With Rare Genetic Diseases Were Part Of Research Into New Treatments. The Coronavirus Has Stopped All Of It
April 28, 2020 – (Buzzfeed News) – The pandemic has shut down research across the world, from the basic research on cells in labs to large scale trials of new drugs involving thousands of patients. The closures have crippled efforts to develop cutting-edge genetic treatments for children with rare diseases in particular, leaving their families with little hope.
Their Three Children Have a Fatal Disease, and They Refused to Let the Coronavirus Take Away Their Only Chance at Saving Them
April 25, 2020 – (Washington Post) – Before their children, who have GM1 gangliosidosis, could receive an experimental gene therapy, the couple had to get them to the NIH in Maryland – and they had to do that in the narrow two-day window between when Trump announced he was implementing the ban on travel from Europe and when it went into effect.
83% Survival in COVID-19 Patients with Moderate or Severe ARDS Treated in New York with Mesoblast’s Cell Therapy Remestemcel-L
April 24, 2020 – (Mesoblast) – Mesoblast announced 83% survival in ventilator-dependent COVID-19 patients with moderate or severe ARDS treated with Mesoblast’s allogeneic mesenchymal stem cell product candidate remestemcel-L within the first five days. In contrast, there was only 12% survival among ventilator-dependent COVID-19 patients at a second major referral hospital network in New York City during the same period.
Pluristem Secures €50M Non-Dilutive Financing From the European Investment Bank to Support its COVID-19 Project and Phase 3 Studies
April 24, 2020 – (Pluristem) – The financing will support Pluristem’s research and development in the EU to further advance its regenerative cell therapy platform, and to assist moving the products in its pipeline to market, with a special focus on clinical development of PLX cells as a treatment for complications associated with COVID-19.
resTORbio and Adicet Bio Announce Merger Agreement to Advance Allogeneic Gamma Delta CAR-T Therapy Technology
April 29, 2020 – (Adicet Bio) – Under the terms of the agreement, Adicet would merge with a wholly-owned subsidiary of resTORbio in an all-stock transaction, and the equityholders of Adicet will become the majority owners of resTORbio’s outstanding common stock upon the close of the merger.
Bone Therapeutics Secures €11M Financing
April 29, 2020 – (Bone Therapeutics) – The financing will be used to advance the company's key assets, ALLOB and JTA-004, through late stage clinical development. Bone Therapeutics intends to pursue a capital raise when favorable market conditions are met.
Kiadis Pharma Announces €12M Private Placement With a US Healthcare Investor
April 28, 2020 – (Kiadis Pharma) – The proceeds will be used to fund the development of Kiadis’ K-NK cell therapy programs, including the development of K-NK002 for patients with blood cancer undergoing HSCT, K-NK003 for the treatment of acute myeloid leukemia, and the company’s K-NK platform for solid tumor indications.
Taysha Gene Therapies Launches with $30M Seed Financing and Strategic Partnership with the UT Southwestern Gene Therapy Program
April 28, 2020 – (Taysha Gene Therapies) – The company's pipeline includes 15 AAV gene therapy programs, with options to an additional four programs. Under the partnership, UT Southwestern will conduct discovery and preclinical research, lead IND-enabling studies, provide clinical GMP manufacturing, as well as execute natural history studies.
Caladrius Biosciences Closes $5M Registered Direct Offering
April 27, 2020 – (Caladrius Biosciences) – The company currently intends to use the net proceeds from the offering for working capital and general corporate purposes, including the advancement of its CD34+ technology-based clinical programs.
LifeSprout Closes $28.5M Series A Financing
April 27, 2020 – (LifeSprout) – LifeSprout, a privately-held regenerative medicine company founded with technology licensed from Johns Hopkins University, has closed a $28.5M Series A financing. The company is using proceeds to support clinical development of novel therapeutic products from its Regenerative Matrix platform.
Vertex Pharmaceuticals and Affinia Therapeutics Establish Multi-Year Collaboration to Discover and Develop Novel AAV Capsids for Genetic Therapies
April 27, 2020 – (Vertex Pharmaceuticals) – The collaboration will accelerate the development of genetic therapies for people affected by Duchenne muscular dystrophy, myotonic dystrophy type 1, and cystic fibrosis. Affinia Therapeutics will be eligible to receive over $1.6B, including $80M in upfront payments and research milestones that will be paid during the research term.
ExCellThera Announces EMA Orphan Designation for ECT-001 Cell Therapy as Treatment in Hematopoietic Stem Cell Transplantation
April 30, 2020 – (ExCellThera) – ExCellThera expects that enrollment in Phase 2 trials of its ECT-001 cell therapy will be completed in the following months and intends to continue dialogue with the FDA under the auspices of the RMAT designation on a pivotal registration-enabling study.
Neon Therapeutics Announces Acceptance of European CTA Application for NEO-PTC-01
April 30, 2020 – (Neon Therapeutics) – The Phase 1 dose-finding clinical trial in metastatic melanoma will be conducted in collaboration with the Netherlands Cancer Institute and is expected to begin in the third quarter of 2020.
Marker Therapeutics Receives FDA Orphan Drug Designation for Its Multi-Antigen Targeted T Cell Therapy for Acute Myeloid Leukemia
April 29, 2020 – (Marker Therapeutics) – Marker Therapeutics announced that the US FDA has granted Orphan Drug designation to MT-401, a multi-tumor-associated antigen-specific T cell product for the treatment of patients with acute myeloid leukemia following allogeneic stem cell transplant.
Off-the-Shelf CAR-T Therapy Helps Leukemia Patients in a Small, First-in-Human Trial
April 28, 2020 – (STAT News) – Of the five patients with T-cell acute lymphoblastic leukemia treated with Gracell Biotechnologies' CAR-T therapy, four had so few leukemic cells four months after the CAR-T infusion they were barely detectable.
Positive Opinion for Orphan Drug Designation for ADP-A2M4 in the European Union for the Treatment of Soft Tissue Sarcoma from EMA
April 28, 2020 – (Adaptimmune) – Adaptimmune’s SPEARHEAD-1 trial with ADP-A2M4 for people with synovial sarcoma and myxoid/round cell liposarcoma is actively enrolling at approximately 25 clinical sites in Canada, France, Spain, the United Kingdom, and the US.
SNUBH Team Finds Modified Adenovirus Halts Pancreatic Cancer Progression
April 28, 2020 – (Korea Biomedical Review) – The cancer did not progress further in any of the nine patients who had been treated for 12 weeks. The median progression-free survival was 11.4 months.
Adverum Biotechnologies Doses First Patient in Cohort 4 of OPTIC Phase 1 Clinical Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD
April 27, 2020 – (Adverum Biotechnologies) – The primary endpoint of the trial is the safety and tolerability of ADVM-022 after a single IVT administration. Adverum will report preliminary data from all four cohorts later this year.
CARsgen Therapeutics Receives Positive EMA Opinion on Orphan Drug Designation for CT053 for the Treatment of Multiple Myeloma
April 27, 2020 – (CARsgen Therapeutics) – CARsgen Therapeutics announced the EMA has granted Orphan Designation to the company's investigational CT053 CAR T-cell therapy, an anti-BCMA autologous CAR-T therapy for the treatment of multiple myeloma. CT053 was previously granted Orphan Drug Designation by the US FDA.
Iovance Details Early Responses to TIL Cell Therapy in Lung Cancer
April 27, 2020 – (Fierce Biotech) – Two of the patients had complete responses to the therapy that are ongoing one year later. Three other patients developed new lesions after undergoing the TIL therapy but went into remission after they received tumor-directed treatments.
Orchard Therapeutics Announces First Patient Dosed With OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome
April 27, 2020 – (Orchard Therapeutics) – The study is designed to evaluate safety, tolerability and clinical efficacy and is intended to enroll up to five patients between three months and 24 months of age who will be followed for three years.
'Backpacks' Boost Immune Cells' Ability to Kill Cancer
April 29, 2020 – (Wyss Institute) – These cytokine-secreting "backpacks" for macrophages keep the cells in their tumor-killing state for up to five days after they arrive at a tumor site, and have been found to slow tumor growth and reduce metastasis in mice with an aggressive form of breast cancer.
Beta Cells From Stem Cells: Potential for Cell Replacement Therapy
April 27, 2020 – (Helmholtz Zentrum München) – The researchers developed an approach to enrich the stem cell culture with highly specialized pancreas progenitors which might lead to a more targeted differentiation into beta cells.
Promising New Treatment for Recurrent Pediatric Brain Cancer
April 27, 2020 – (Baylor College of Medicine) – Researchers have developed a novel approach that delivers appropriately-targeted CAR-T therapy directly into the cerebrospinal fluid that surrounds the tumor. The approach was effective in treating medulloblastoma and ependymoma in mouse models of the diseases.
A Step Toward a More Efficient Way to Make Gene Therapies to Attack Cancer, Genetic Disorders
April 27, 2020 – (UCLA) – The technique uses high-frequency acoustic waves coupled with millions of cells that flow through an “acoustofluidic device” in a cell culture liquid. The procedure opens up pores along the cells’ membranes that allow DNA and other biological cargo to enter the cells, and it enables the researchers to insert the cargo without the risk of damaging the cells by contacting them directly.
EMA Warns Against Using Unproven Cell-Based Therapies
April 28, 2020 – (EMA) – The CAT’s advice is in response to individuals, companies, and hospitals promoting unproven cell-based therapies as cures for a broad range of conditions including cancer, cardiovascular diseases, autism, cerebral palsy, muscular dystrophy, and vision loss. These treatments can pose serious risks to patients for little or no benefit.
DeGette, Upton Unveil Next Steps for 21st Century Cures 2.0
April 27, 2020 – (Congresswoman Diana DeGette) – US Reps. Diana DeGette (D-CO) and Fred Upton (R-MI) released a concept paper for their bipartisan “Cures 2.0.” The 12-page concept paper unveils high-level areas of focus for Cures 2.0, which will look to safely and efficiently modernize the delivery of health care in the wake of the coronavirus pandemic.
What Is the Economic Value of Receiving CAR-T Therapy in an Outpatient Setting?
April 24, 2020 – (American Journal of Managed Care) – Researchers found that outpatient administration of CAR-T therapy in nonacademic specialty oncology networks was associated with a $32,987 (40.4%) reduction in total costs.
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
Business Manager (US)
Ovizio Imaging Systems – US East Coast
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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