ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
June 18, 2020
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EU Proposes Emergency Leeway on Gene Engineering Rules for Coronavirus Vaccines
June 17, 2020 – (Reuters) – A proposal from the European Commission would temporarily relax rules on trials of drugs involving genetically engineered organisms as an emergency measure to speed the development of a vaccine against the new coronavirus.
  • ARM applauded the EC's decision as a positive first step towards creating a much-needed dialogue on how to best facilitate the development of innovative therapies without negatively impacting patient safety or environmental health.
For Some Biotech Employees in the Age of COVID-19, the Workday Starts at the Bathroom Sink
June 16, 2020 – (Boston Globe) – While many executives have adapted to tapping on laptops at their dining room tables, working remotely has been hard, if not impossible, for bench scientists who need to run experiments. So a number of biotech companies have begun to let more of them back into the lab.
BridgeBio Pharma and University of Florida Establish Collaboration to Advance Therapies for Genetically Driven Diseases
June 18, 2020 – (BioBridge Pharma) – BridgeBio will provide sponsorship to select research programs around diseases with a genetic basis, including gene therapies and large and small molecules.
Catalyst Biosciences Announces Pricing of Public Offering of Common Stock
June 18, 2020 – (Catalyst Biosciences) – The gross proceeds to Catalyst from this offering are expected to be approximately $30M. 
CHA Biotech to Raise $62M to Bolster CDMO Business
June 18, 2020 – (Pulse Business News) – CHA Biotech aims to raise a total of 75B won ($62M) through issuances of bonds with option to equity swap to strengthen contract development and manufacturing servicing for global gene therapy developers.
Emergent BioSolutions to Invest $75M in Canton Site and Expand Viral Vector and Gene Therapy Capability
June 18, 2020 – (Emergent BioSolutions) – The investment will include a state-of-the-art, multi-suite operation up to 1000L scale. Advanced therapy CDMO drug substance manufacturing services are expected to be available beginning in 2023.
Lyell Immunopharma and PACT Pharma Announce Research and Clinical Development Partnership
June 18, 2020 – (Lyell Immunopharma) – Lyell and PACT announced a strategic partnership to jointly develop and test a next generation personalized anti-cancer T cell therapy against solid tumors. The program resulting from the partnership, which includes upfront and milestone payments to PACT, will be jointly owned and each party will share profits equally.
Sarepta Therapeutics and Selecta Biosciences Enter Into License and Option Agreement for Selecta’s ImmTOR Immune Tolerance Platform in Neuromuscular Diseases
June 18, 2020 – (Sarepta Therapeutics) – Sarepta will evaluate the utility of ImmTOR to minimize or prevent the formation of neutralizing antibodies to AAV in connection with the administration of Sarepta’s DMD and LGMD gene therapy candidates. Under the terms of the research license and option agreement, Sarepta will make an initial payment to Selecta, and Selecta is eligible to receive certain pre-clinical milestone fees.
IVERIC bio Announces Pricing of Upsized Public Offering of Common Stock and Pre-Funded Warrants
June 18, 2020 – (IVERIC bio) – The aggregate gross proceeds from the public offering and the concurrent private placement are expected to be approximately $143.9M.
BioCardia Announces Pricing of $10M Public Offering
June 17, 2020 – (BioCardia) – The company intends to use the net proceeds from this offering to complete enrollment in the ongoing CardiAMP Cell Therapy pivotal trial for the treatment of heart failure, fund clinical development, and pursue regulatory approval for product candidates.
CF Foundation Invests Up to $14M in Effort to Solve Key Challenges to Gene Delivery in Cystic Fibrosis
June 17, 2020 – (Cystic Fibrosis Foundation) – The Cystic Fibrosis Foundation has announced an investment of up to $14M in 4D Molecular Therapeutics to develop a customized vehicle to deliver a gene therapy into the lung cells of people with cystic fibrosis. The delivery is one of the key hurdles to developing an effective treatment.
Orca Bio Emerges With Nearly $300M to Transform Allogeneic Cell Therapy
June 17, 2020 – (Orca Bio) – The financing will support the continued advancement of Orca Bio’s cell therapy pipeline and its novel manufacturing platform, which sorts blood with single-cell precision and a high level of purity and speed to create optimal therapeutic mixtures of immune and stem cells.
4D Molecular Therapeutics Raises $75M in Series C Financing
June 16, 2020 – (4D Molecular Therapeutics) – The proceeds from this financing will be used to advance three of 4DMT’s AAV gene therapy product candidates through initial clinical proof-of-concept in patients, to advance 4DMT’s proprietary product pipeline and next-generation therapeutics platform, and to expand its internal GMP manufacturing capabilities.
AgeX Therapeutics and Pluristyx Announce Manufacturing, Marketing, and Distribution Agreement for Clinical-Grade Human Pluripotent Stem Cells for Therapeutic Applications
June 16, 2020 – (AgeX Therapeutics) – The agreement is a key step in AgeX’s licensing and collaboration strategy to facilitate industry and academic access to its cell lines, technology, and manufacturing platforms. 
Genprex Expands Manufacturing Program With Aldevron to Advance Oncoprex Clinical Development
June 16, 2020 – (Genprex) – The new agreement provides for production of TUSC2 plasmid DNA, the active agent in Oncoprex, at full commercial scale and should result in significantly lower costs per unit of product manufactured.
Xenetic Biosciences Announces Collaboration With Pharmsynthez and Multiple Academic Institutions in Russia and Belarus to Advance Development of XCART Platform
June 16, 2020 – (Xenetic Biosciences) – The initial stage of the collaboration will include an exploratory trial to evaluate and refine the XCART front-end process of target identification, screening, and lead characterization in a real-world clinical setting.
Magenta Therapeutics and Beam Therapeutics Announce Collaboration to Evaluate Targeted MGTA-117 as Conditioning Regimen for Base Editing Therapies
June 15, 2020 – (Magenta Therapeutics) – Beam will be responsible for clinical trial costs related to development of Beam’s base editors when combined with MGTA-117, while Magenta will continue to be responsible for all other development costs of MGTA-117.
Sernova Completes Acquisition of Cellular Local Immune Protection Technology
June 15, 2020 – (Sernova) – Pursuant to the asset acquisition, Sernova acquired all intellectual property associated with Converge’s conformal coating cell encapsulation technology.
Bit Bio Secures $41.5M of Funding From Top Life Sciences Investors
June 14, 2020 – (Bit Bio) – Its platform technology, opti-ox, enables precise reprogramming of stem cells and induces them to take on a new identity and is intended to produce human cells at a higher scale, speed, and consistency.
Kite Receives European Medicines Agency Approval for CAR-T Cell Therapy Manufacturing Facility in Europe
June 12, 2020 – (Gilead Sciences) – Kite, a Gilead Company, announced it has received approval to implement a variation to the Yescarta Marketing Authorization from the European Medicine Agency for end-to-end manufacturing.
Generation Bio Announces Pricing of $200M Initial Public Offering
June 11, 2020 – (Generation Bio) – Generation Bio announced the pricing of its initial public offering of 10,526,316 shares of its common stock.
Lineage Cell Therapeutics Reinitiates Patient Enrollment in Clinical Study of OpRegen for the Treatment of Dry AMD With Geographic Atrophy
June 18, 2020 – (Lineage Cell Therapeutics) – The company anticipates that it will complete enrollment of the last 3 patients in Cohort 4 of the OpRegen clinical study in Q3 2020.
Pfizer and Sangamo Announce Updated Results From 3e13 VG/KG Cohort in Phase 1/2 Trial of Giroctocogene Fitelparvovec Gene Therapy
June 18, 2020 – (Sangamo) – All five patients with severe hemophilia A who received the 3e13 vg/kg dose showed sustained factor VIII (FVIII) activity levels, and no patients experienced bleeding events or required FVIII infusions.
Sernova Highlights Positive Results Presented at the American Diabetes Association's Virtual 80th Scientific Sessions
June 18, 2020 – (Sernova) – The data presented clinically demonstrate that the vascularized Cell Pouch provides a consistently safe and biologically suitable, retrievable environment for the transplantation and survival of functional islets.
Atara Biotherapeutics Announces First Patient Enrolled in Randomized Placebo-Controlled Study of Allogeneic T-Cell Therapy ATA188 in Progressive Forms of Multiple Sclerosis
June 17, 2020 – (Atara Biotherapeutics) – Progression into the randomized, placebo-controlled study of ATA188 is supported by data from the Phase 1a study, which demonstrated that ATA188 was safe and well-tolerated across all four dose cohorts.
BioMarin Provides Additional Data from Recent 4 Year Update of Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
June 17, 2020 – (BioMarin) – During the four years following treatment with valoctocogene roxaparvovec, the cumulative mean ABR was 0.8, which represents a 95% reduction from baseline. There was a 96% reduction in mean Factor VIII usage to 5.4 infusions per year cumulatively over four years from the baseline of 135.6 infusions per year. 
ReNeuron Announces Regulatory Approval From the FDA and MHRA for an Expanded Phase 2a Study in RP Patients
June 17, 2020 – (ReNeuron) – The company expects to present further data from the expanded Phase 2a clinical trial during the next twelve months and expects to have sufficient data from the study to enable it to seek approval in the second half of 2021 to commence a single pivotal clinical study with its hRPC cell therapy candidate in RP.
Bionic Sight Doses First Patient in a Phase 1/2 Clinical Trial of a New Investigational Treatment for Blindness Caused by Retinitis Pigmentosa
June 16, 2020 – (Bionic Sight) – The first patient was dosed in early March, 2020, but application of the therapy to subsequent patients was postponed due to the COVID-19 pandemic. Bionic Sight expects to resume enrollment in the second half of 2020.
Bellicum Receives FDA IND Clearance to Initiate a Phase 1/2 Clinical Trial for BPX-603, a Dual-Switch GoCAR-T
June 15, 2020 – (Bellicum Pharmaceuticals) – The company intends to initiative the Phase 1/2 trial with BPX-603 targeting solid tumors that express HER2 later this year.
Genocea Files IND Application for GEN-011, a T Cell Therapy Designed to Improve on Current Limitations of TIL Therapy
June 15, 2020 – (Genocea) – Unlike TILs, GEN-011 does not require extra surgery or collection of viable tumor samples and uses peripheral blood in a rapidly scalable manufacturing process. This may enable access to a broader population of patients with advanced cancers, and may result in a cost of therapy that is favorable compared to TIL-based approaches.
Autolus Therapeutics Presents AUTO1 and AUTO3 Data at the 2020 EHA25 Virtual Congress
June 12, 2020 – (Autolus Therapeutics) – Of the 19 patients treated with AUTO1 for acute lymphoblastic leukemia, 16 (84%) achieved MRD-negative complete response. 
CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001 in Severe Hemoglobinopathies
June 12, 2020 – (CRISPR Therapeutics) – New data presented today show that at 15 months after CTX001 infusion, Patient 1 was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL, and F-cells of 100%. At 5 months after CTX001 infusion, Patient 2 was transfusion independent and had total hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL, and F-cells of 99.4%.
New Data Show Near Elimination of Vaso-Occlusive Crises and Acute Chest Syndrome in Phase 1/2 Study of bluebird bio’s LentiGlobin Gene Therapy for Sickle Cell Disease
June 12, 2020 – (bluebird bio) – There was a 99.5% mean reduction in annualized rate of vaso-occlusive crises and acute chest syndrome among the 14 patients who had at least six months of follow-up and a history of these complications.
A New Target for CAR T Cells: Senescence-Related Diseases
June 17, 2020 – (Memorial Sloan Kettering) – Researchers successfully eliminated senescent cells from two different mouse models of liver fibrosis using CAR-T cell engineered to recognize uPAR, a molecule present on the surface of senescent cells.
Light-Activated CRISPR Triggers Precision Gene Editing and Super-Fast DNA Repair
June 17, 2020 – (Johns Hopkins Medicine) – The team found that within 30 seconds of shining the light on the cells, the CRISPR complex had cut more than 50 percent of its targets.
Treating Ovarian Cancer With iCAR Cells
June 15, 2020 – (Center for iPSC Research & Application) – The iCAR cells, which utilize iPSCs, were differentiated into natural kill cells and administered into mice in which ovarian cancer cells were injected days earlier, extending the lifespan of the animals by 50%.
Putting ‘Super’ in Natural Killer Cells
June 11, 2020 – (UC San Diego) – Researchers deleted a gene called CISH in stem cell-derived NK cells, removing an internal checkpoint that is normally activated or expressed when NK cells are stimulated by cytokines. This may improve NK cell activation and function.
IU Researchers Model Human Stem Cells to Identify Degeneration in Glaucoma
June 4, 2020 – (Indiana University) – The researchers used gene editing technology to introduce a genetic mutation commonly associated with glaucoma into existing lines of the stem cells for disease modeling, as well as to correct the gene defect in patient-derived cells. 
'Medicaid Best Price' Changes Aimed at Value-Based Gene Therapy Contracts
June 17, 2020 – (Reuters) – The changes proposed include calculating the best price based not just on one discount, but as a comprehensive blend of prices. They would also allow for price calculations outside of the current three-year window.
Director, Communications
Alliance for Regenerative Medicine – Washington, DC
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
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
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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