ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
February 6, 2020
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Sonoma Biotherapeutics Launches With $40M in Series A Funding to Advance Regulatory T Cell Therapy in Autoimmune and Degenerative Diseases
February 6, 2020 – (Sonoma Biotherapeutics) – Sonoma Biotherapeutics has entered into a strategic partnership with Lyell that provides both parties with access to technologies and know-how to enhance the durability, stability, and specificity of cell therapies in their respective indications of focus.
AGTC Announces Proposed Public Offering of Common Stock
February 5, 2020 – (AGTC) – Applied Genetic Technologies Corporation announced that it has commenced an underwritten public offering of 6,000,000 shares of its common stock.
Beam Therapeutics Announces Pricing of $180M Initial Public Offering
February 5, 2020 – (Beam Therapeutics) – Beam Therapeutics announced the pricing of its initial public offering of 10,588,236 shares of common stock at a public offering price of $17 per share.
Ophthalmic Gene Therapy Company Ikarovec Launches With £2.5M Seed Funding
February 5, 2020 – (Cambridge Network) – The primary use of funds is to take the company’s lead programme, a multicistronic gene therapy for diabetic macular edema (DME), to IND-enabling studies. The proceeds will also be used to develop the company’s three earlier stage novel gene therapy products for major ophthalmic indications, build the intellectual property portfolio, and establish its own laboratories.
Vineti Completes $35M in Series C Financing to Industrialize Personalized and High-Value Therapeutics
February 5, 2020 – (Vineti) – Vineti will use the funds to support further enhancement of the company’s automation and analytics offering, new software product development, and global commercial expansion.
WindMIL Therapeutics Announces Cancer Immunotherapy Pre-Clinical Research Collaboration with University of Pennsylvania
February 5, 2020 – (WindMIL Therapeutics) – The collaboration will leverage WindMIL’s and Penn’s respective expertise to conduct pre-clinical comparisons of the characteristics and functionality of CAR-T and CAR-MIL products.
Ziopharm Oncology Announces Pricing of Public Offering of Common Stock
February 5, 2020 – (Ziopharm Oncology) – The gross proceeds to Ziopharm are expected to be approximately $90.4M. Ziopharm intends to use proceeds from the offering for progressing its clinical programs towards commercialization, working capital needs, and general corporate purposes.
Avacta and Daewoong Agree to Collaboration and License Agreement with AffyXell Therapeutics
February 3, 2020 – (Avacta) – Avacta and AffyXell will now work together to develop Affimer proteins against a range of targets which, when produced by mesenchymal stem cells (MSCs), are intended to inhibit inflammatory and autoimmune pathways and improve the overall efficacy of MSCs, creating a next generation of stem cell therapies.
Catalent To Acquire Leading Cell Therapy Company MaSTherCell Global for $315M, Creating an Industry-Leading Cell & Gene Therapy Platform
February 3, 2020 – (Catalent) – Catalent and MaSTherCell Global announced that Catalent has agreed to acquire MaSTherCell Global, a company backed by Great Point Partners, SFPI-FPIM, and Orgenesis, for an aggregate of $315M in cash.
Gene Therapy Developer Passage Bio Files for $125M IPO
February 3, 2020 – (Genetic Engineering & Biotechnology News) – Passage Bio, a developer of gene therapies for rare monogenic CNS diseases, has disclosed plans to raise $125M through an initial public offering, according to a registration statement filed with the US SEC.
AnGes Initiates Phase 2b Study in the US of HGF Gene Therapy Product Collategene in Patients with Lower Limb Ischemic Ulcers
February 4, 2020 – (AnGes) – Collategene received approval in Japan in 2019 for the treatment of critical limb ischemia.
AskBio Announces First Patient Dosed in Phase 1 Trial Using AAV Gene Therapy for Congestive Heart Failure
February 4, 2020 – (AskBio) – NAN-101 is a gene therapy that aims to activate protein phosphatase inhibitor 1 to inhibit the activity of protein phosphatase 1, a substance that plays an important role in the development of heart failure.
Calibr’s ‘Switchable’ CAR-T Platform for Cancer Moves Forward With FDA Clearance of IND
February 3, 2020 – (Scripps) – The therapy leverages a patient’s own immune cells to treat cancer, putting them under the control of a novel molecular “switch” that seeks to eliminate the potentially life-threatening side effects of cytokine release syndrome.
Mesoblast Submits Completed Biologics License Application for Ryonciltm to US FDA
February 3, 2020 – (Mesoblast) – The company has requested Priority Review of the BLA by the FDA under the product candidate’s existing Fast Track designation for SR-aGVHD. If approved, RYONCIL is expected to be launched in the US in 2020.
Building a Safer CAR-T Therapy
February 3, 2020 – (Ludwig Institute for Cancer Research) – Researchers have devised a new type of CAR-T cell that can be switched on and off on demand. The technology could be used to lessen the risk of cytokine release syndrome.
Researchers Identify Possible New Combination Treatment for Advanced Melanoma
January 31, 2020 – (UCLA) – Researchers found that using NKTR-214 in combination with an infusion of anti-tumor immune cells may produce a stronger immune response. When tested in mice with melanoma tumors that were unlikely to stimulate an immune response, the approach increased the number of anti-tumor immune cells, and those immune cells lived longer and functioned better.
Scientists Boost Gene-Editing Tools to New Heights in Human Stem Cells
January 30, 2020 – (Arizona State University) – In a proof of concept study, researchers found that, unlike CRISPR, TREE could not only make single DNA edits to the APOE4 gene, but also make highly accurate corrections to both copies of the APOE4 gene that humans possess.
Broader Types of Data to Be Used in Development of NICE Guidance
February 4, 2020 – (NICE) – Data sources could include electronic health record data; ‘real-world data’ looking at health and social care practice outside of trials, such as registries and clinical audits; and any other relevant data that has been made available for others to use, such as data held by local authorities about public health and social care.
OPINION: We Need Better Regulation of Stem Cell Therapies, Especially Rogue Clinics
February 6, 2020 – (STAT News) – So far, only a few stem cell treatments have been proven to work, such as bone marrow transplants, and they have been authorized in only a few cases for blood disorders, including certain cancers. Yet hundreds of clinics in the US defiantly offer stem cell products and therapies that have never been approved by the FDA or been proven effective by any responsible laboratory or test.
OPINION: Unlawful Stem Cell Products Continue to Harm People as FDA Deadline Looms
February 2, 2020 – (The Hill) – Members of Congress can provide still greater public support for FDA’s efforts as November’s critical enforcement deadline approaches. Timely congressional hearings can shine a spotlight on the promise of regenerative medicine, the pitfalls of rushing treatments straight to consumers without rigorous testing, and the dangers posed by treatments that are not yet proved to be safe, effective, or beneficial.
OPINION: Managing Uncertainty In Drug Value: Outcomes-Based Contracting Supports Value-Based Pricing
January 30, 2020 – (Health Affairs) – Uncertainty regarding the magnitude and duration of benefit for products will likely become more common as the growing pipeline of genetic and cell therapies delivers an increasing number of therapies for rare disorders. These treatments may provide transformative benefits and should be supported by appropriate payment incentives when they do.
Director / Vice President, Finance and Operations
Alliance for Regenerative Medicine – Washington, DC
Vice President, Global Policy and Advocacy
Alliance for Regenerative Medicine – Washington, DC
Cell Therapy Product Manager (200752)
MilliporeSigma – Bedford, 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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