The California Institute for Regenerative Medicine approved a $100 million RAPID initiative to fund platform‑based approaches for in‑vivo genetic therapies targeting rare diseases. The program will support projects that have engaged with the FDA and can scale IND‑enabling work through first‑in‑human studies, with a focus on metabolic, immune, retinal and hearing disorders. CIRM framed RAPID as a move away from single‑asset funding toward modular, repeatable platforms that can decrease development time and cost for many rare conditions. The agency cited recent examples of personalized genetic therapies and rising regulatory openness to platform strategies as rationale for the investment. Why it matters: Platform funding at scale can lower technical and regulatory barriers for rare‑disease developers, accelerate patient access to gene therapies and reshape translational priorities among academic startups and CDMOs.
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