ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
April 9, 2020
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Orbsen Therapeutics Launches Clinical Trial to Address Leading Cause of Death Among COVID-19 Patients
April 8, 2020 – (Orbsen Therapeutics) – The Phase 2a clinical trial, funded by the Wellcome Trust, has already begun enrolling patients. Orbsen’s ORBCEL recently completed a successful Phase 1 trial in patients with moderate to severe ARDS.
Oxford Biomedica Joins Consortium to Rapidly Develop a COVID-19 Vaccine Candidate
April 8, 2020 – (Oxford Biomedica) – The Oxford vaccine candidate relies on adenoviral vector technology, ChAdOx1, which has been shown to generate a strong immune response from one dose and has demonstrated a good safety profile in pre-clinical and clinical trials conducted to date. No financial terms were disclosed.
COVID-19 Economic Fallout May Delay Cell and Gene Therapy Reimbursement Solutions
April 7, 2020 – (Pink Sheet - Subscription Only) – It will take longer than previously expected for payers to implement innovative approaches to managing the high cost of cell and gene therapies because of the economic burden imposed by the COVID-19 pandemic, Blue Cross Blue Shield of Massachusetts chief physician executive and senior VP for health and medical management Bruce Nash predicts.
FDA Clears IND Application for Mesoblast to Use Remestemcel-L in Patients With ARDS Caused by COVID-19
April 6, 2020 – (Mesoblast) – Remestemcel-L is being developed for various inflammatory conditions, and is believed to counteract the inflammatory processes implicated in these diseases.
GenSight Biologics Provides Update on Operations in the Context of COVID-19
April 6, 2020 – (GenSight Biologics) – In order to protect patients, GenSight will be delaying recruiting new patients into the third cohort until the COVID-19 situation has improved. The company remains on track to file their MAA for their gene therapy LUMEVOQ with the EMA in Q3 2020.
Lion TCR Joins the Efforts to Develop New Strategies for Protection and Treatment in COVID-19
April 6, 2020 – (Lion TCR) – Lion TCR will use its technology platform to isolate and characterize new T cell receptors specific for SARS-CoV-2 that can be used to engineer T cells capable of detecting and recognising SARS-CoV-2 infected cells.
Capricor Initiates Compassionate Use Program for Severe COVID-19 Patients Using CAP-1002, Its Novel Cell Therapy
April 3, 2020 – (Capricor) – Two patients were treated last week at a leading healthcare center in Los Angeles, California with additional patients planned in the coming weeks. Additionally, Capricor has submitted an expanded access IND application to investigate the use of CAP-1002 in certain COVID-19 patients.
Voyager Therapeutics Provides Update on the RESTORE-1 Parkinson’s Disease Trial
April 3, 2020 – (Voyager Therapeutics) – Voyager and its collaborator Neurocrine Biosciences have temporarily paused screening of new patients in the RESTORE-1 trial to allow clinical trial sites to assess the impact of the COVID-19 pandemic.
Orchard Therapeutics Outlines Business Impact of COVID-19
March 31, 2020 – (Orchard Therapeutics) – The company now believes it is likely to submit the BLA for OTL-200 to treat metachromatic leukodystrophy with the US FDA in the first half of 2021 due to COVID-19 related impacts.
AVROBIO Outlines Response to COVID-19 and Current Assessment of Business Impact
March 30, 2020 – (AVROBIO) – AVROBIO reports that, due to the temporarily hold on elective medical procedures in many geographies, they are currently not dosing new patients in clinical trials of their investigative gene therapies. Subsequent new patient dosing is anticipated in the second half of the year, as hospitals allow.
Sangamo Announces Closing of Biogen Collaboration Agreement
April 9, 2020 – (Sangamo) – Under the terms of the collaboration, Sangamo has received $225M from the purchase by Biogen of newly issued Sangamo stock and will receive an upfront license fee of $125M from Biogen no later than May 8, 2020. Additionally, Sangamo is eligible to earn up to $2.37B in other development, regulatory, and commercial milestone payments.
T-Cure Bioscience Signs Exclusive Worldwide License With NCI for the Development of T Cell Receptor Therapy for Treatment of Solid Tumors
April 8, 2020 – (T-Cure Bioscience) – T-Cure Bioscience announced that the company has entered into an exclusive, worldwide license with the National Cancer Institute for IP related to a TCR product candidate for the treatment of Kita-Kyushu lung cancer antigen 1 expressing malignancies. T-Cure anticipates the KK-LC-1 TCR therapy will enter a multi-site Phase 1 clinical study in Q3 2020.
Beam Therapeutics Licenses SIRION Biotech’s LentiBOOST Technology for Its CAR-T Pipeline
April 7, 2020 – (SIRION Biotech) – SIRION will be entitled to undisclosed upfront and milestone payments and is eligible to receive royalties on future product net sales plus license fees tied to commercial success.
GenScript ProBio and Eutilex Enter Into Exclusive Strategic Collaboration on Plasmid and Virus Process Development and Manufacturing for CAR-T Programs
April 7, 2020 – (GenScript ProBio) – Eutilex will use GenScript's high-quality one-stop plasmid and virus platform for plasmid and virus process development and manufacturing in its CAR-T programs.
Prime Therapeutics Launches Solution to Ease Cost Burden of Gene Therapy
April 7, 2020 – (Fierce Healthcare) – Blues plans and self-funded employers that join the new PreserveRx program will pay a per-member per-month fee and in exchange will be shielded from the high one-time cost of new therapies such as Zolgensma.
Quell Therapeutics Enters a Collaborative Research Agreement With the Hannover Medical School
April 7, 2020 – (Quell Therapeutics) – Under the terms of the agreement, Quell Therapeutics will collaborate in joint research with MHH to accelerate the discovery and validation of multiple CAR-Treg cell therapies.
Deerfield Closes $840M Healthcare Venture Fund
April 6, 2020 – (Deerfield) – Deerfield Management Company announced the closing of the Deerfield Healthcare Innovations Fund ll, which will invest in advancements in science that may lead to important therapeutic interventions and back new technologies and infrastructure to improve the way healthcare is delivered to patients.
Evotec and Takeda Enter Into Multi-Year Gene Therapy Research Alliance
April 6, 2020 – (Evotec) – Under the alliance, Evotec will support multiple Takeda programs targeting conditions aligned with Takeda’s four core therapeutic areas: oncology, rare diseases, neuroscience, and gastroenterology.
venBio Closes $394M Life Sciences Venture Capital Fund
April 3, 2020 – (venBio) – venBio Fund III will continue to invest primarily in therapeutics companies that are developing biopharmaceuticals for unmet medical needs. The venBio team typically leads or co-leads investments and takes an active role with each of their portfolio companies.
Arch Venture Partners Announces $1.46B Raised in Two New Funds to Invest in Transformative Biotechnology Companies
April 2, 2020 – (ARCH Ventures) – ARCH Venture Partners announced the closing of two new funds, ARCH Venture Fund X and ARCH Venture Fund X Overage, with a combined $1.46B to invest in transformative, early stage biotechnology companies.
Flagship Pioneering Completes Raising a New $1.1B Capital Pool
April 2, 2020 – (Flagship Pioneering) – This new capital pool will operate alongside separate Flagship funds totaling $1B raised in 2019 to support the financial needs of Flagship-originated companies after they spin out from Flagship Labs and enter their growth stage.
Fate Therapeutics Announces Worldwide Collaboration With Janssen for Novel iPSC-Derived Cell-Based Cancer Immunotherapies
April 2, 2020 – (Fate Therapeutics) – Fate Therapeutics will receive $100M upfront from Johnson & Johnson Innovation. Fate is also eligible to receive payments of up to $1.8B upon the achievement of development and regulatory milestones and up to $1.2B upon the achievement of commercial milestones, plus royalties on worldwide commercial sales.
SanBio Announces Business Alliance With Ocumension in the Research, Development, and Commercialization of Innovative Stem Cell Therapies for Ophthalmic Diseases
March 31, 2020 – (SanBio) – Pursuant to the terms of the agreement, Ocumension will fund the initial investment of $6M for the preclinical and manufacturing development. Ocumension obtains exclusive rights to develop and commercialize SB623 and MSC2 in Greater China for ophthalmic indications, and SanBio will be eligible to receive up to $71M in milestone payments.
HEALIOS K.K. Increases Investment in Athersys by Fully Exercising Warrant to Acquire Additional Shares
March 30, 2020 – (Athersys) – Athersys announced that its partner in Japan, HEALIOS K.K., exercised in full its warrant to purchase shares of Athersys common stock, representing an additional investment of approximately $7M in Athersys.
Kiadis Pharma Files First IND Application With the US FDA for Natural Killer Cell Therapy Produced With PM21
April 9, 2020 – (Kiadis Pharma) – Kiadis Pharma is developing K-NK002 as an adjunctive therapy to the current haploidentical HSCT standard of care with the goal of improving relapse rates.
Precision BioSciences Announces Dosing of First Patient in Phase 1/2a Clinical Trial of PBCAR20A for Relapsed/Refractory NHL, CLL, and SLL
April 7, 2020 – (Precision BioSciences) – In preclinical disease models, PBCAR20A demonstrated potent in vivo clearance of CD20+ tumor cells and overall tumor volume reduction, with no evidence of graft-versus-host disease.
Histogen Announces Investigational New Drug Application Amendment for HST 001
April 6, 2020 – (Histogen) – Histogen announced that it has submitted an IND application with the FDA for the initiation of a Phase 1b/2a clinical trial of its lead program, HST 001 for the treatment of androgenic alopecia in men.
Gracell Announces China MNPA Acceptance of IND Application for GC007g Cell Therapy for CD19 Positive Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia
April 3, 2020 – (Gracell Biotechnologies) – The company plans to initiate a Phase 1 clinical study in patients with CD19 positive relapsed or refractory B-cell acute lymphoblastic leukemia in Q2 2020.
IVERIC bio Announces Fast Track Designation From US FDA for Zimura for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
April 3, 2020 – (IVERIC bio) – Last October, IVERIC bio announced that Zimura met its pre-specified primary efficacy endpoint and reached statistical significance in a clinical trial in geographic atrophy secondary to dry AMD.
Mapping Kidney Development With Stem Cells
April 9, 2020 – (CiRA) – The cells self-organized into kidney organoids when mixed together in a manner consistent with that seen naturally in an embryonic kidney. Transplantation of the resulting kidney organoids into mice resulted in good engraftment, with blood vessels from the mice feeding the organoids.
Researchers Engineer Better Way to Target Leukemia Cells
April 6, 2020 – (Baylor College of Medicine) – The new CAR T cells, named TriCAR T cells, targeted CD19/20/22 and were significantly more effective than T cells that target CD19 alone. When the leukemia cells stopped producing CD19 and were no longer visible to CD19 CAR T cells, TriCAR T cells were still effective.
Bespoke Gene Therapies to Get Boost Under FDA, NIH Collaboration
April 2, 2020 – (Bloomberg Law) – Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said his agency is working toward developing a public-private partnership for individualized gene therapies with the NIH. The partnership aims to find a way to make gene therapies for rare disorders that are effective but generally don’t treat enough patients to be profitably made and sold.
Siddhartha Mukherjee and Ken Burns Present PBS Premiere of 'The Gene: An Intimate History'
April 3, 2020 – (Columbia University) – The Gene airs at a critical moment for the science community, as geneticists around the world grapple with the ethical questions these technologies raise.
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
Manager, Clinical Quality
Abeona Therapeutics – Cleveland, OH
Senior Research Scientist
Kytopen – Cambridge, MA
Head of Clinical Pharmacology
Magenta Therapeutics – Cambridge, MA
Medical Director / Senior Medical Director
Magenta Therapeutics – Cambridge, MA
Senior Research Associate, Immunology / Stem Cell Biology 
Magenta Therapeutics – Cambridge, MA
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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