ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
June 4, 2020
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Join ARM for Our Next Webinar: The Next Wave of Commercial Development
June 4, 2020 – (ARM) – The next wave of cell and gene therapies is poised to enter the market. How are developers shaping their commercial plans? Panelists will address how the landscape has changed since the first products were introduced, lessons from the first wave of products, how payors and regulators are evolving in their approach to new therapies, and how COVID-19 is affecting commercial strategies. Register today!
The Coronavirus Pandemic Claims Another Victim: Medical Research for Deadly Rare Diseases
June 4, 2020 – (Washington Post) – Since March, medical research on diseases other than COVID-19 has taken a huge hit, with countless experiments abandoned and clinical trials suspended or postponed. This singular focus will inevitably delay much-needed advances for other life-threatening ailments, including cancer, stroke, and heart disease, experts say.
Remestemcel-L Improves Respiratory and Functional Outcomes in Patients With Inflammatory Lung Disease
May 31, 2020 – (Mesoblast) – The Phase 2 trial in patients with COPD showed that remestemcel-L significantly improved respiratory and functional clinical outcomes in patients with elevated levels of the inflammatory biomarker C-reactive protein. Significantly elevated CRP levels are predictive of increased hospitalization and death in patients with COPD1, and are seen in various acute lung diseases, including ARDS caused by COVID-19.
VIVEbiotech Collaborates With Xyphos Biosciences for Clinical Development Support of Convertible CAR-T Cell Therapy
June 4, 2020 – (VIVEbiotech) – The collaboration will concentrate on the development and manufacturing of GMP-grade lentiviral vectors. VIVEbiotech will supply lentiviral vectors for Xyphos’s upcoming clinical trial of its lead convertible CAR-T program.
Avectas and UC Davis Collaborate on Cell Engineering Technology
June 3, 2020 – (Avectas) – The collaboration will involve the transfer of Avectas's technology to The Simon Laboratory where the expert group will engineer immune cells and study the delivery of cargoes such as DNA, mRNA, proteins, and gene editing tools to cells for autologous and allogeneic cell therapies.
CSL Behring and Seattle Children’s Research Institute to Advance Gene Therapy Treatments for Primary Immunodeficiency Diseases
June 2, 2020 – (CSL Behring) – Initially, the alliance will focus on the development of treatment options for patients with two rare, life-threatening primary immunodeficiency diseases, Wiskott-Aldrich Syndrome and X-linked Agammaglobulinemia.
Intellia Therapeutics Announces Pricing of Public Offering of Common Stock
June 2, 2020 – (Intellia Therapeutics) – The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, are expected to be $100M.
Iovance Biotherapeutics Announces Closing of $604M Common Stock Public Offering
June 2, 2020 – (Iovance Biotherapeutics) – Iovance intends to use the proceeds from this offering to support the potential commercial launch of lifileucel for advanced melanoma and LN-145 for advanced cervical cancer; to initiate a program directed at registration of Iovance’s tumor infiltrating lymphocyte therapies in non-small cell lung cancer; continued support of commercial manufacturing activities; and for other general corporate purposes.
Allogene Therapeutics Announces Pricing of Public Offering of Common Stock
June 1, 2020 – (Allogene) – Allogene Therapeutics announced the pricing of an underwritten public offering of its common stock, with gross proceeds expected to be approximately $550M.
Adaptimmune Therapeutics Prices Public Offering of American Depositary Shares
June 1, 2020 – (Adaptimmune) – The gross offering size is expected to be $225M. Net proceeds will be used to advance the development of Adaptimmune’s immunotherapies into and through clinical trials as well as for other general corporate purposes.
AveXis Community Statement on the Global Managed Access Program for AVXS-101
June 1, 2020 – (AveXis) – AveXis has updated its global managed access program, which provides the one-time transformative therapy to patients prior to regulatory approval via a free program, and now requires physicians to attest that their patient does not have access to, or is not medically eligible for, available treatment alternatives.
Regeneron and Intellia Therapeutics Expand Collaboration to Develop CRISPR/Cas9-Based Treatments
June 1, 2020 – (Regeneron) – Regeneron Pharmaceuticals and Intellia Therapeutics announced an expansion of their existing collaboration to provide Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic targets and for the companies to jointly develop potential products for the treatment of hemophilia A and B. Intellia will receive an upfront payment of $70M, and Regeneron will make an additional equity investment in Intellia of $30M.
AgeX Therapeutics and Sernova to Collaborate to Engineer Universal Locally Immune Protected Cell Therapies for Type 1 Diabetes and Hemophilia A
May 29, 2020 – (Sernova) – The research collaboration will evaluate whether Sernova’s pluripotent stem cell-derived pancreatic islet beta cells engineered with AgeX’s UniverCyte technology can evade human immune detection. Under the terms of the agreement, Sernova has been granted a time-limited, non-exclusive research license by AgeX.
Stempeutics India Signs an Agreement With Novumcella Japan for Development of Stempeucel Product to Treat Critical Limb Ischemia Due to Buerger’s Disease
May 28, 2020 – (Stempeutics) – Stempeutics is evaluating accelerated development of Stempeucel in Japan with support from Novumcella.
FDA Approves Elixirgen Therapeutics IND Application for Therapy for Telomere Biology Disorders With Bone Marrow Failure
June 4, 2020 – (Elixirgen Therapeutics) – The FDA's approval allows Elixirgen Therapeutics to proceed with its planned Phase 1/2, open label, single center clinical trial to assess the safety and tolerability of EXG34217, an autologous cell therapy.
First Patient Treated in Phase 1 Clinical Trial of Gamma Delta T Cell Immunotherapy for Patients With Newly Diagnosed Glioblastoma
June 2, 2020 – (Incysus Therapeutics) – Incysus’s innovative DRI approach leverages conventional chemotherapy to modify the tumor microenvironment and activate immunity through the upregulation of the DNA damage response pathway while simultaneously delivering a large number of a type antitumor white blood cells directly to the tumor site.
NKMax America Receives IND Clearance for Phase 1/2a Trial of Cell Therapy in Combination With Trastuzumab or Cetuximab for the Treatment of Advanced/Metastatic Cancers
June 2, 2020 – (NKMax America) – The company plans to initiate a Phase 1/2a open-label, multi-center trial to evaluate the safety and anti-tumor activity of its expanded autologous natural killer cells in combination with trastuzumab or cetuximab in third quarter of this year.
Celyad Provides Update on Allogeneic CAR-T Franchise at the 2020 ASCO Virtual Scientific Program
June 1, 2020 – (Celyad) – Encouraging anti-tumor activity was observed in the trial with two patients who achieved a confirmed partial response. In addition, nine patients achieved stable disease, with seven patients demonstrating disease stabilization lasting more than or equal to three months of duration.
Lineage Cell Therapeutics Reports Regeneration of Retinal Tissue in Patient Treated With OpRegen RPE Cells for Dry AMD With Geographic Atrophy
June 1, 2020 – (Lineage Cell Therapeutics) – Specifically, the area of GA assessed at 9 months was approximately 25% smaller than the patient’s pre-treatment baseline and it grew approximately 50% slower than its historical rate during the subsequent six months.
BioMarin Provides Highlights of 4 Years of Clinical Data From Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
May 31, 2020 – (BioMarin) – The four-year update for the 6e13 vg/kg and three-year update for the 4e13 vg/kg cohorts demonstrated that all subjects in both cohorts remain off prophylactic Factor VIII treatment since receiving their single dose of valoctocogene roxaparvovec. Cumulative mean annualized bleed rates remain less than one in both cohorts and below pre-treatment baseline levels.
Achilles Therapeutics Doses First Patient in Phase 1/2 Study in Recurrent or Metastatic Malignant Melanoma With First TIL Therapy to Specifically Target Clonal Neoantigens
May 29, 2020 – (Achilles Therapeutics) – The trial is expected to recruit approximately 20 patients and report initial data in the first half of 2021. Recruitment is ongoing across sites in the UK, with additional sites to open in the US and Europe.
Allogene Therapeutics and Servier Report Positive Results From Phase 1 Study of ALLO-501 in Relapsed/Refractory Non-Hodgkin Lymphoma at ASCO Annual Meeting
May 29, 2020 – (Allogene) – Responses were observed across all cell doses and tumor histologies, with an overall response rate of 63% and complete response rate of 37%.
Autolus Therapeutics Presents Additional Data on AUTO3 in DLBCL During the ASCO20 Virtual Scientific Program
May 29, 2020 – (Autolus) – At the recommended Phase 2 dose range, six out of eight patients achieved a complete response or partial response, and five out of eight patients achieved a complete response.
Iovance Presents Updated Clinical Data for Tumor Infiltrating Lymphocyte (TIL) Therapy Lifileucel in Advanced Melanoma at ASCO Scientific Program
May 29, 2020 – (Iovance Biotherapeutics) – Lifileucel shows a 36.4% overall response rate and a disease control rate of 80%. Median duration of response was not reached at 18.7 months of median study follow up.
Marker Therapeutics Reports Interim Results of Its MultiTAA-Specific T Cell Therapy in Patients With Pancreatic Adenocarcinoma at the 2020 ASCO Virtual Annual Meeting
May 29, 2020 – (Marker Therapeutics) – Out of the 13 evaluable patients, four patients experienced objective responses after administration of MultiTAA cells.
SPEAR T-Cells Targeting MAGE-A4 Demonstrate New Responses in Esophagogastric Junction, Lung, and Head and Neck Cancers
May 29, 2020 – (Adaptimmune) – In the SURPASS trial, three patients have responded out of the first four treated with ADP-A2M4CD8, including one confirmed and two unconfirmed partial responses. 
Yescarta Demonstrates High Rates of Response in Relapsed or Refractory Indolent Non-Hodgkin Lymphoma
May 29, 2020 – (Gilead Sciences) – After a single infusion of Yescarta, 93 percent of patients responded, with 80 percent of patients achieving a complete response as assessed by an independent review committee.
Ziopharm Oncology Presents Encouraging Clinical Data for Controlled IL-12 for the Treatment of Recurrent Glioblastoma at the 2020 American Society of Clinical Oncology
May 29, 2020 – (Ziopharm Oncology) – Subjects who received low-dose corticosteroids during veledimex dosing had a median overall survival of 17.8 months.
Gene Therapy With a New Base Editing Technique Restores Hearing in Mice
June 3, 2020 – (Harvard University) – With this technique, researchers repaired one single error in the Tmc1 gene known to cause a hereditary form of deafness. While a similar approach has been used previously for other forms of hearing loss, this is the first time base editing has been used for a genetic sensory disorder.
Engineered T Cells for Type 1 Diabetes Move Closer to Clinic
June 3, 2020 – (Seattle Children's Hospital) – Researchers have devised a way to genetically engineer a patient's T cells so they function like normal Treg. The hope is that when transferred back into the patient, these edited regulatory-like T cells enter the pancreas, where they can help to suppress the overactive immune response, sustaining and protecting the function of the islet cells.
Study Demonstrating Role of IdeS in Enabling of Gene Therapy in the Presence of Neutralizing Anti-AAV Antibodies Published in Nature Medicine
June 1, 2020 – (Spark Therapeutics) – New research published in Nature Medicine demonstrated that treatment with immunoglobulin G-degrading enzyme of Streptococcus pyogenes resulted in rapid and transient reduction of neutralizing anti-AAV antibodies and restored gene therapy efficacy in controlled laboratory tests of animal models.
Director, Communications
Alliance for Regenerative Medicine – Washington, DC
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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