CMC Hurdles Are Now the Biggest Barrier to CGT Approval

Published on Drug Discovery News

Recent FDA rejections of three advanced therapies, Capricor’s CAP‑1002, Ultragenyx’s DTX401, and Rocket Pharma’s Kresladi, reveal a growing trend: cell and gene therapies are being stalled not by safety concerns, but by gaps in chemistry, manufacturing, and controls (CMC). From early INDs to late-stage BLA filings, manufacturing readiness now determines success. Experts note rising regulatory expectations for comparability data, process control, and scalability, pushing sponsors to rethink early-stage decisions and invest in CDMO partnerships, standardized platforms, and cross-functional alignment.

Read the full article on Drug Discovery News:

👉 Why gene and cell therapies are stalling at the FDA

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