Gene Therapies

BioMarin Collaborates with Allen Institute to Develop Gene Therapies for Rare Brain Diseases

Shots: BioMarin will receive an exclusive license to each program for research, development, and commercialization of the gene therapies The collaboration will utilize Allen institute technologies to develop new gene therapies for rare genetic diseases of CNS by targeting gene expression to specific cells Allen Institute has developed modified engineering AAVs to carry genes on …

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AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases

AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, and Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today announced a partnership to support the development and manufacturing of AavantiBio’s gene therapies, …

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Vertex Signs a License Agreement with Obsidian Therapeutics to Discover Regulated Gene Editing Therapies

Shots: Obsidian to receive $75M in equity, up front, research milestones and is eligible to receive ~$1.3B as research, development, regulatory, and commercial milestones across up to five potential programs. Additionally, Obsidian will receive royalties on sales of products that emerge from the collaboration Obsidian will deploy its cytoDRiVE platform to discover controllable genetic therapies …

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Almirall and Tyris Collaborate to Develop Next Generation Gene Therapies

Shots: Tyris to receive up front, research milestone along with research funding and will be responsible for generating clinical lead candidates and will further progress them to clinical development Almirall to get an exclusive option to acquire gene therapies and progress them through clinical development to commercialization The collaboration will integrate Tyris’s non-viral based gene …

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Getting gene therapies to market faster

The FDA approval process is slow. Even for COVID-19 vaccines, the process can be time consuming. However, regulators around the world do have some expedited programs. However, a number of countries have programs that expedite review, particularly for the diseases with unmet need, orphan drugs, or when new treatments represent a significant advance over the …

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