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ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
February 20, 2020
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Immatics and GSK Partner to Develop Novel Adoptive Cell Therapies
February 20, 2020 – (Immatics) – Under the terms of the agreement, Immatics will receive an upfront payment of €45M ($50M) for two initial programs and is eligible to receive over $550M in development, regulatory and commercial milestone payments for each product.
Axovant Gene Therapies Announces Pricing of $65M Public Offering
February 19, 2020 – (Axovant Gene Therapies) – Axovant Gene Therapies announced the pricing of its underwritten public offering of 14,031,336 of its common shares at a price to the public of $3.75 per share.
bluebird bio, Cleared for Zynteglo Launch, Aims to Dose First Patients Soon
February 19, 2020 – (Fierce Pharma) – With a manufacturing green light in hand, bluebird bio is hoping to make good on its commercial hopes in the coming months. bluebird plans to dose its first commercial patients for Zynteglo in Germany in the first half of the year, the drugmaker said Tuesday.
Caribou Biosciences and ProMab Biotechnologies Announce Sale and Assignment Agreement for Humanized scFv Targeting BCMA
February 19, 2020 – (Caribou Biosciences) – Caribou Biosciences and ProMab Biotechnologies announced a sale and assignment agreement under which Caribou gains access to a ProMab humanized single-chain variable fragment (scFv) targeting BCMA. Caribou intends to utilize this scFv in the development of its CB-011 program, an allogeneic CAR-T therapy targeting BCMA-positive tumors including multiple myeloma.
Genprex Prices $17.5M Common Stock Offering
February 19, 2020 – (Genprex) – The company intends to use the net proceeds to advance its lead clinical programs in non-small cell lung cancer and for working capital and general corporate purposes.
Orgenesis Announces Collaboration With Johns Hopkins University for the Development and Processing of Cell- and Gene-Based Clinical Therapeutics
February 19, 2020 – (Orgenesis) – Orgenesis has entered into a collaboration agreement with the Johns Hopkins University to utilize Orgenesis’ POCare platform to develop and supply a variety of cell and gene therapies and technologies, including cell-based immunotherapy technologies. 
SanBio and Nippon Medical School Enter Into Joint Research Agreement to Investigate the Efficacy of SB623 in the Treatment of Traumatic Brain Injury at the Subacute Phase
February 19, 2020 – (SanBio) – The two parties will investigate whether the use of SB623-embedded dural matrix during cranioplasty can lead to restoration of brain functions.
Audentes Therapeutics Announces Plans to Build New State-of-the-Art Gene Therapy Manufacturing Facility in Sanford, North Carolina
February 18, 2020 – (Audentes Therapeutics) – Audentes Therapeutics announced that it plans to invest $109M to build a new gene therapy manufacturing facility in Sanford, North Carolina. The initial phase of the capital investment is planned to occur over approximately 18 months, and the plant is expected to be operational in 2021.
AVROBIO Announces Closing of $100M Public Offering
February 18, 2020 – (AVROBIO) – The company intends to use the net proceeds from the offering to fund current programs in Fabry disease, cystinosis, Gaucher disease, and Pompe disease; external and internal manufacturing and process development activities; and research and development activities that relate to its current and future clinical and preclinical activities.
Cellectis and Servier Expand Collaboration on UCART19 Products
February 18, 2020 – (Cellectis) – Under the term sheet, Cellectis will grant to Servier an expanded exclusive worldwide license to develop and commercialize all next generation gene-edited allogeneic CAR T-cell products targeting CD19. Financial terms will be improved to include an additional $27.6M (€25M) upfront payment, as well as up to $410M (€370M) in clinical and commercial milestones.
Univercells Secures Up to €50M Investment From KKR Platform to Speed Delivery of Manufacturing Technologies
February 18, 2020 – (Univercells) – With the new financing, Univercells will focus on scaling the NevoLine platform and the scale-X portfolio, and supporting continued expansion into the fast-growing gene therapy sector.
First Patients Begin Gene Therapy Treatment for Blindness as Part of NHS Long Term Plan
February 17, 2020 – (NHS) – Patients in the UK will benefit from Luxturna after NHS England reached a NICE-endorsed deal with manufacturer Novartis to fund the drug. The treatment for adults will initially be available from three national specialist centres in Manchester, London, and Oxford, with the option to roll-out the treatment to other hospitals.
Adverum Biotechnologies Announces Closing of $150M Public Offering of Common Stock
February 14, 2020 – (Adverum Biotechnologies) – Earlier this month, Adverum announced new interim data from their Phase 1 gene therapy trial in wet-AMD. All six patients in Cohort 1 are rescue-injection-free, with three patients at 52 weeks; 4 of 6 patients in Cohort 2 (lower dose cohort) are rescue-injection-free at 24 weeks.
Magenta Therapeutics Announces Updated Phase 2 Data on MGTA-456 Cell Therapy, Demonstrating Continued Durability in Inherited Metabolic Disorders
February 20, 2020 – (Magenta Therapeutics) – In patients with cALD, Loes scores remained stable over the follow-up period, consistent with a halt in disease progression. Patients with MPS I showed normalized levels of blood a-L-iduronidase by Day +42 and had decreased levels of Hurler-specific urine glycosaminoglycans.
Clinically Meaningful Outcomes Using Remestemcel-L in Patients With Chronic Graft Versus Host Disease
February 20, 2020 – (Mesoblast) – Mesoblast announced that the expanded access protocol using remestemcel-L for steroid-refractory chronic graft versus host disease has resulted in clinically meaningful outcomes in all three treated patients within 28 days. The investigator-initiated collaboration will be expanded to evaluate remestemcel-L in a pivotal trial for chronic GVHD.
AGTC Announces Completion of Enrollment in the Two Highest Dose Groups of Its Ongoing Phase 1/2 Clinical Trial in Patients with X-Linked Retinitis Pigmentosa
February 19, 2020 – (AGTC) – AGTC plans to report interim data from these two new dose groups and to report top-line 12-month data for the first four dose groups in the second half of 2020. The company intends to initiate a pivotal trial by the end of the year.
Mustang Bio Announces First Subject Treated in Phase 1/2 Trial with the Optimized CD20-targeted CAR T Cell Therapy MB-106
February 18, 2020 – (Mustang Bio) – Mustang Bio announced that the first subject treated with the optimized MB-106 manufacturing process, developed in collaboration between Mustang and Fred Hutchinson Cancer Research Center, has achieved a complete response at the lowest starting dose in an ongoing Phase 1/2 clinical trial.
Longeveron Announces Completion of Enrollment in Phase 2b Clinical Trial of Longeveron Allogeneic Mesenchymal Stem Cells in Aging Frailty
February 11, 2020 – (Longeveron) – It is anticipated that the final trial results will become available in the first half of 2021. The primary objective of the study is to assess the effect of LMSC treatment on exercise tolerance and endurance via the six-minute walk test.
Controlling CAR T-Cells With Light Selectively Destroys Skin Tumors in Mice
February 19, 2020 – (University of California San Diego) – By programming CAR T cells to switch on when exposed to blue light, researchers were able to control the cells to destroy skin tumors in mice without harming healthy tissue. In tests in mice, administering the engineered CAR T cells and stimulating the skin tumor sites with blue LED light reduced tumor size by eight to ninefold.
Gene Therapy Can Protect Against ALA and SMA-Related Cell Death
February 17, 2020 – (Karolinska Institutet) – In experiments with human motor neurons formed from iPS cells taken from ALS and SMA patients, the researchers were able to show that the introduction of SYT13 protects the cells from degeneration by reducing endoplasmatic reticulum stress and blocking programmed cell death.
Beta-Arrestin-2 Increases Neurotoxic Tau Driving Frontotemporal Dementia
February 18, 2020 – (University of South Florida) – Researchers found that genetically reducing β-arrestin-2 lessens tauopathy, synaptic dysfunction, and the loss of nerve cells and their connections in the brain.
New Institute Launched to Ensure the US Healthcare System Is Ready for Gene Therapies
February 19, 2020 – (Institute for Gene Therapies) – As part of IGT’s effort, experts from across the healthcare system will work together to ensure health policies reflect the latest medical advances, remove barriers that hinder patient access to gene therapies, and advocate for sustainable, long-term solutions.
Carl June on CRISPR, CAR-T and How the Vietnam War Dropped Him Into Medicine
February 14, 2020 – (Endpoints News) – In August of 2011, Carl June and his team published a landmark paper showing their CAR-T treatment had cleared a patient of cancer. A year later, Jennifer Doudna published the first major CRISPR paper. Last week, June, whose CAR-T work was eventually developed by Novartis into Kymriah, published in Science the first US paper showing how the two could be brought together. 
Director / Vice President, Finance and Operations
Alliance for Regenerative Medicine – Washington, DC
Vice President, Global Policy and Advocacy
Alliance for Regenerative Medicine – Washington, DC
Director, Regulatory Affairs
Cardinal Health – Remote
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 www.alliancerm.org.

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