The FDA has issued draft guidance for developers of personalized genome editing therapies for rare genetic diseases, and CMS said it will expand its Cell and Gene Therapy Access Model beyond sickle cell disease.
The FDA’s guidance, “Individualized Human Genome Editing Products: Considerations for BLA Submission and Postapproval Management,” covers how to structure a biologics license application for therapies that vary from patient to patient, according to an Oct. 27 news release. It centers on a concept called the Individualized Feature Space, a set of predefined limits on how a genome editing product may vary across patients while staying within a single application. When a new patient-specific variant falls within those limits and lab assays show adequate safety and activity, sponsors could add it to the application without new clinical trials, the agency said.
CMS said it will invite manufacturers of therapies for other conditions to apply to its Cell and Gene Therapy Access Model, a voluntary program in which CMS negotiates outcomes-based agreements with manufacturers for state Medicaid programs. The model’s first evaluation found CMS-led negotiations for sickle cell disease therapies eased states’ administrative burden and strengthened their leverage by pooling beneficiaries. In the coming months, CMS will release a request for applications designed to allow negotiations to start sooner after a therapy is approved, and it will accept applications for therapies already on the market.
The steps build on a series of FDA rare disease efforts. In February, the agency unveiled a pathway for ultra-rare disease therapies, and this month it launched a rare disease framework initiative. Philadelphia-based Children’s Hospital of Philadelphia treated the first patient in the world with a personalized CRISPR therapy in 2025, and the FDA this year expanded approval of Casgevy to children as young as 2. The CMS model launched with sickle cell gene therapies Casgevy and Lyfgenia in 2025, and 33 states, D.C. and Puerto Rico, covering about 84% of Medicaid beneficiaries with the condition, signed agreements to participate.
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