Novartis Receives Approval From Japanese Ministry of Health, Labour and Welfare for Zolgensma, the Only Gene Therapy for Patients With Spinal Muscular Atrophy
March 19, 2020 – (Novartis) – Approximately 15-20 SMA patients in Japan are expected to be eligible for treatment each year. Reimbursement with MHLW is expected by the end of 1H20 and, pending agreement, Zolgensma will be available at that time.
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Univercells Launches CDMO Exothera With GMP Capabilities in 15,000m² Site in Jumet, Belgium
March 19, 2020 – (Univercells) – Univercells has launched a contract development and manufacturing organization, Exothera, to support cell and gene therapy developers with process development and production of viral vectors.
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AskBio Enters Research Collaboration and Licensing Agreement With University of North Carolina for Angelman Syndrome
March 18, 2020 – (AskBio) – A UNC School of Medicine team has generated preclinical evidence that gene therapy may help individuals with Angelman syndrome by improving seizure and motor outcomes. The financial terms of the agreement were not disclosed.
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CERo Therapeutics Announces Research Collaboration With Lyell Immunopharma and Completion of a $40M Series A Financing
March 18, 2020 – (CERo Therapeutics) – Under the terms of the agreement, CERo and Lyell will collaborate to pursue proof-of-concept studies for a new class of cell-based therapeutics directed against solid tumors.
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Hebrew University to Collaborate with Cytovia Therapeutics in Strategic Partnership to Develop Cancer Immunotherapies Targeting Natural Killer Cells
March 18, 2020 – (Cytovia Therapeutics) – Under the collaboration, Cytovia will sponsor a research program for the development of multi-specific antibodies targeting both NK cells and the tumor antigen.
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HORAMA Signs Exclusive License Agreement With Leiden University Medical Center Targeting CRB1 Gene Mutations to Treat Inherited Retinal Dystrophies
March 18, 2020 – (HORAMA) – Under the agreement, HORAMA will receive an exclusive worldwide license to certain patent rights and know-how for the therapeutic candidate. In return for these rights, LUMC will receive an undisclosed upfront payment, milestone payments, and royalties on net sales of products.
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Orchard Therapeutics Appoints Company Founder and Gene Therapy Pioneer Bobby Gaspar, M.D., Ph.D., as New CEO
March 18, 2020 – (Orchard Therapeutics) – Orchard Therapeutics announced that company founder and gene therapy pioneer Dr. Bobby Gaspar has been named CEO, effective immediately. Dr. Gaspar, previously president of research, chief scientific officer, and a member of the Orchard board of directors, succeeds Mark Rothera, who has served as the company’s chief executive officer since 2017.
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Oxford Biomedica Signs License and Clinical Supply Agreement With Juno Therapeutics, a Bristol-Myers Squibb Company, for LentiVector Platform for CAR-T and TCR-T Therapeutics
March 18, 2020 – (Oxford Biomedica) – The agreement grants Juno Therapeutics a non-exclusive license to Oxford Biomedica’s LentiVector platform for its application in CAR-T and TCR-T programs in oncology and other indications. Oxford Biomedica will receive an upfront payment of $10M and potentially up to $86M upon achievement of certain development and regulatory milestones.
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AGC Announces the Launch of a Voluntary Tender Offer Over All Ordinary Shares of MolMed
March 17, 2020 – (MolMed) – MolMed announced that AGC Inc. declared its intention to launch – directly or through a company of the same Offeror Group or through a newly fully owned incorporated company – a voluntary tender offer over 100% of the company's ordinary shares. The company aims to obtain all of the issued shares of Molmed for approximately €240M.
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Immatics Announces Business Combination with Arya Sciences Acquisition Corp, Creating a Publicly Listed Leader in TCR-Based Immunotherapies
March 17, 2020 – (Immatics) – Immatics will receive gross proceeds of up to $252M at the closing of the transaction. The company is developing novel T cell receptors to enable a robust and specific T-cell response against cancer cells.
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Eureka Therapeutics Completes $45M Series E Financing and Enters Into Strategic Collaboration With Lyell Immunopharma
March 17, 2020 – (Eureka Therapeutics) – The company will use proceeds from the Series E round to advance its proprietary TCR-mimic and antibody-TCR ARTEMIS programs, including Eureka’s anti-AFP ARTEMIS Phase I/II clinical trial in the United States for the treatment of hepatocellular carcinoma, the predominant type of liver cancer.
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Sigilon Therapeutics Announces $80.3M Series B Financing to Advance Shielded Living Therapeutics to the Clinic
March 17, 2020 – (Sigilon Therapeutics) – The funding will support the first-in-human clinical trial of Sigilon’s novel encapsulated cell therapy for hemophilia A, expected to begin in the first half of 2020, as well as continued advancement and expansion of Sigilon’s programs in rare blood disorders, lysosomal diseases and endocrine and immune disorders.
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Coronavirus Turmoil Throws Biotechnology IPOs Into Question
March 17, 2020 – (Wall Street Journal) – Unlike technology companies, biotech startups typically go public with little or no revenue because investors are betting on the promise of drugs in their pipelines. But as markets downshift, public investors will place increased value on biotechs with strong balance sheets.
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NantKwest Doses First Patient in Phase 2 Single-Arm Trial of Metastatic Merkel Cell Carcinoma Therapy With Off-the-Shelf CD16 Natural Killer Cells
March 19, 2020 – (NantKwest) – The trial will evaluate the company’s first-in-class, off-the-shelf CD16-targeted natural killer cells in combination with ImmunityBio’s IL-15 superagonist N-803 and Avelumab.
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Abeona Therapeutics Announces First Patient Treated in Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa
March 17, 2020 – (Abeona Therapeutics) – The VIITAL Phase 3 study is a multi-center, randomized clinical trial assessing EB-101 in up to 15 RDEB patients, with approximately 30 large, chronic wound sites treated in total. The primary outcome measure is wound healing, comparing treated with untreated wound sites on the same patient.
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AGTC Announces Completion of Enrollment in All Adult Dose Groups of Its Ongoing Phase 1/2 Clinical Trials in Patients with Achromatopsia
March 16, 2020 – (AGTC) – The company plans to report interim data from all adult dose groups in the second half of 2020 and to use the data to inform decision-making regarding readiness to move the product candidates to pivotal trials
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Could CRISPR Be Humanity's Next Virus Killer?
March 18, 2020 – (WIRED) – Stanford University researchers reported using the gene editing technology CRISPR to destroy coronavirus genes in infected cells. That result suggests that we may be entering an era of developing new CRISPR-based weapons against deadly viruses, from flus to coronaviruses.
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Gene Therapy May Hold Key to Treating Life-Threatening Cardiac Disease
March 18, 2020 – (UC San Diego) – In mice models of Danon disease, gene therapy expressed positive results in heart, liver, and muscle function. The heart’s overall function of ejecting blood and relaxing improved, as did the body’s ability to degrade proteins and metabolism
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FDA Unveils Guidance for Trials Impacted by COVID-19
March 18, 2020 – (Regulatory Focus) – While recognizing that protocol modifications may be required, and that there may be unavoidable protocol deviations due to COVID-19 illness and/or COVID-19 control measures, FDA urges sponsors and clinical investigators to engage with Institutional Review Boards/Independent Ethics Committees "as early as possible."
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Big Pharma Shied Away From Gene Therapy for Years. Academia Picked up the Slack
March 17, 2020 – (BioPharma Dive) – The time spent building the knowledge and expertise at universities or closely affiliated startups has been one of the reasons why big pharmas have rushed into the space. By advancing the technology, the universities reduced the risk of failure, making pharmas more willing to buy in.
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Vice President, Global Policy and Advocacy
Alliance for Regenerative Medicine – Washington, DC
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Manager, Clinical Quality
Abeona Therapeutics – Cleveland, OH
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Head of Clinical Pharmacology
Magenta Therapeutics – Cambridge, MA
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