ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
May 21, 2020
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Join ARM on May 27 for Our Next Webinar: Cell and Gene Therapy Market Access in a COVID-19 World
May 21, 2020 – (ARM) – On Wednesday, May 27 at 12pm ET, ARM will host experts from national and regional commercial payors in a webinar on market access to cell and gene therapies. Our panel of speakers will address questions such as: How might patient access to cell and gene therapies be impacted by COVID-19? How do we navigate the balance between transformative effect and cost in relation to other therapies? Register today!
Kleo Pharmaceuticals and Celularity Enter into Research Collaboration to Rapidly Develop Allogeneic NK Cell Combination Therapies for COVID-19 and Multiple Myeloma
May 21, 2020 – (Kleo Pharmaceuticals) – Kleo Pharmaceuticals and Celularity announced a preclinical research collaboration to rapidly advance synergistic combinations of each company’s technology platform as potential treatments for COVID-19 and multiple myeloma.
NantKwest Announces FDA Authorization of IND Application for Mesenchymal Stem Cell Product for the Treatment of Severe COVID-19 Patients
May 18, 2020 – (NantKwest) – NantKwest announced it has received authorization from the FDA for an IND application to treat patients with acute respiratory distress syndrome caused by COVID-19 with BM-Allo.MSC, an allogeneic mesenchymal stem cell product. NantKwest has entered into an agreement with Be The Match BioTherapies to provide donor material.
CODA Biotherapeutics Announces Research Collaboration With the Facial Pain Research Foundation
May 20, 2020 – (CODA Biotherapeutics) – As part of the agreement, the FPRF has provided an undisclosed grant to a leading academic-based research institution to support preclinical research of CODA’s chemogenetically controlled inhibitory ion channels in neuropathic pain.
Metcela Raised $4M in Series B Funding to Launch a Phase 1 Clinical Trial for Heart Failure Therapy
May 20, 2020 – (Metcela) – With this financing, Metcela plans to accelerate the clinical development of the fibroblast-based cell therapy product, MTC001, for the treatment of chronic heart failure and initiate a Phase 1 clinical trial.
Cowen Healthcare Investments Successfully Raises $493M for Third Fund
May 19, 2020 – (Cowen Healthcare Innovations) – CHI focuses its investment activity on established companies with outstanding management teams and disruptive approaches to disease that address significant unfulfilled medical needs.
Xenetic Biosciences Announces Research and Development Collaboration with Scripps Research to Advance XCART Platform
May 19, 2020 – (Xenetic Biosciences) – Under the terms of the Scripps Research agreement, Xenetic will be granted an exclusive license to certain intellectual property arising from the research program in the field of B-cell malignancies, as well as an option to acquire rights and licenses to new intellectual property outside of that field.
bluebird bio Announces Pricing of Public Offering of Common Stock
May 18, 2020 – (bluebird bio) – bluebird bio anticipates the total gross proceeds from the offering (before deducting underwriters’ discounts and commissions and estimated offering expenses) will be approximately $500M, excluding any exercise of the underwriters’ option to purchase additional shares.
Gamida Cell Announces Pricing of $60M Public Offering of Ordinary Shares
May 18, 2020 – (Gamida Cell) – Gamida Cell intends to use the net proceeds from this offering to fund the preparation of a BLA for omidubicel in high-risk hematologic malignancies, the continued clinical development of its product candidates, the expansion of its commercial manufacturing capabilities, and general corporate purposes.
Krystal Biotech Announces Pricing of $125M Public Offering of Common Stock
May 18, 2020 – (Krystal Biotech) – The company currently intends to use the net proceeds from this offering to advance B-VEC through a Phase 3 trial; advance the development of KB105, KB104, KB301, and KB407; complete development of a GMP certified manufacturing facility; and for general corporate purposes.
SQZ Biotech Closes $65M Series D Financing
May 18, 2020 – (SQZ Biotech) – The financing will support the advancement of SQZ’s current pipeline of multi-functional cell therapies, designed to direct specific immune responses, and its expansion into infectious diseases.
Oxford Biomedica Receives MHRA Approval for the First Two Manufacturing Suites in Oxbox
May 15, 2020 – (Oxford Biomedica) – Construction was completed at the end of 2019. This approval by the MHRA of the first two suites enables commercial production of batches to commence for partner programs within the coming weeks.
Passage Bio Receives Rare Pediatric Disease Designation for PBGM01 for Patients with GM1 Gangliosidosis
May 21, 2020 – (Passage Bio) – PBGM01 is an AAV gene therapy currently being developed for the treatment of infantile GM1 gangliosidosis and has previously been granted Orphan Drug designation. The company plans to initiate a Phase 1/2 trial in the fourth quarter of 2020 with initial safety and biomarker data expected late in the first half of 2021.
Fate Therapeutics Announces FDA Clearance of IND Application for FT538, First CRISPR-Edited, iPSC-Derived Cell Therapy
May 20, 2020 – (Fate Therapeutics) – The three functional components of FT538 are designed to boost the innate immune response in cancer patients, where endogenous NK cells are typically diminished in both number and function due to prior treatment regimens and tumor suppressive mechanisms.
Poseida Therapeutics Announces Dosing of First Patient in Phase 1 Clinical Trial of P-PSMA-101 Autologous CAR-T for Metastatic Castration-Resistant Prostate Cancer
May 20, 2020 – (Poseida Therapeutics) – As part of its research, Poseida’s Phase 1 open label, multi-center, dose-escalating trial will include cohorts receiving single and multiple doses of P-PSMA-101, with the goal of determining the best dose with the fewest side effects.
AveXis Receives EC Approval and Activates “Day One” Access Program for Zolgensma, the Only Gene Therapy for Spinal Muscular Atrophy
May 19, 2020 – (AveXis) – AveXis, a Novartis company, announced the European Commission granted conditional approval for Zolgensma for the treatment of patients with certain forms of spinal muscular atrophy. Designed to work within existing local pricing and reimbursement frameworks, the “Day One” access program offers ministries of health and reimbursement bodies a variety of flexible options that can be implemented immediately to support swift access to and broad reimbursement of Zolgensma.
Bone Therapeutics Treats First Patients in Pivotal JTA-004 Phase 3 Knee Osteoarthritis Study
May 18, 2020 – (Bone Therapeutics) – Reporting of the topline results of the study on the 3-month primary endpoint and 6-month follow-up period is planned in the second half of 2021.
CARsgen Therapeutics Receives IND Clearance From the US FDA for CT041 CLDN18.2-CAR-T Cells
May 18, 2020 – (CARsgen) – CARsgen Therapeutics announced that its first-in-class drug candidate, CT041, an autologous CAR-T therapy for the treatment of patients with claudin18.2-positive adenocarcinoma of the stomach, gastroesophageal junction, or pancreas, has received IND clearance from the US FDA. CT041 is the first claudin18.2-targeted CAR-T therapy IND cleared for clinical trials in the world.
Kuur Therapeutics Announces Presentation of Phase 1 CAR-NKT Immunotherapy Data at ASGCT Meeting
May 15, 2020 – (Kurr Therapeutics) – Data from the study demonstrated that patient-derived CAR-NKT cells targeting GD2, an antigen expressed by almost all neuroblastomas, expanded post infusion and were able to localize to the site of neuroblastoma tumors. All five treated patients showed evidence of CAR-NKT expansion, peaking at three weeks post infusion, with expansion continued through week 4 in one patient.
Orchard Therapeutics Presents New Interim Data From OTL-203 Proof-of-Concept Study for MPS-I
May 15, 2020 – (Orchard Therapeutics) – Treatment with OTL-203 and the selected conditioning regime were well-tolerated across all eight patients and demonstrated rapid hematologic reconstitution, with neutrophil and platelet engraftment within 21 days following treatment, and supranormal IDUA enzyme expression in peripheral blood.
Pfizer’s New Phase 1b Results of Gene Therapy in Ambulatory Boys With Duchenne Muscular Dystrophy Support Advancement Into Pivotal Phase 3 Study
May 15, 2020 – (Pfizer) – The treatment provided durable and statistically significant improvements across multiple efficacy-related endpoints measured at 12 months post-infusion, including sustained levels of mini-dystrophin expression and improvements on the North Star Ambulatory Assessment rating scale. Three serious adverse events were recorded, all of which fully resolved within 2 weeks.
Caladrius Biosciences Reports Positive Full Results for CLBS16 From the ESCaPE-CMD Trial at SCAI 2020 Scientific Sessions
May 14, 2020 – (Caladrius) – Trial investigators observed that patients experienced a highly statistically significant (p=0.0045) increase in coronary flow reserve at six months after a single intracoronary administration of CLBS16. A single administration of CLBS16 resulted in statistically significant improvements in chest pain frequency, Canadian Cardiovascular Society angina classification, and Seattle Angina Questionnaire scores.
Incysus Treats First Patient in Phase 1 Study of Gamma Delta T Cell Immunotherapy in Leukemia and Lymphoma Patients Undergoing Allogeneic Stem Cell Transplantation
May 12, 2020 – (Incycus) – Incysus Therapeutics has developed this novel gamma delta T cell approach to allow haploidentical transplantation while providing immune protection to prevent leukemic relapse and infections during the period in which the patient recovers.
Animal Study Shows Human Brain Cells Repair Damage in Multiple Sclerosis
May 19, 2020 – (University of Rochester Medical Center) – Researchers found that when human glia progenitor cells are transplanted into adult mouse models of progressive multiple sclerosis, the cells migrated to affected areas in the brain, created new oligodendrocytes, and replaced the lost myelin. The study also showed that this process of remyelination restored motor function in the mice.
Organ-On-Chip Technology Makes Embryonic Blood
May 15, 2020 – (Center for iPS Cell Research & Application) – The research team mixed hemogenic endothelial prepared from human iPS cells with purchased endothelial cells and mesenchymal stroma cells in a chip. The porous membrane allowed the researchers to mimic the organization of the aorta-gonad-mesonephros.
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
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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