The FDA released draft guidance aimed at enabling faster access to personalized gene therapies for rare diseases. The proposal focuses on treatments targeting different mutations within the same disease-causing gene, seeking to reduce the need for full new clinical trials when mutations are extremely rare. Under the approach, a manufacturer could gain approval for one personalized therapy through a more traditional development pathway, then later seek clearances for additional mutations using laboratory data rather than repeating the complete clinical study. The FDA described parameters for later approvals through an “individualized feature space” defined in the initial application. The draft is part of broader regulatory work tied to the FDA’s Rare Disease Framework Initiative, including an upcoming discussion at the agency on standards for specific conditions rather than a one-size-fits-all model. The policy direction is especially relevant to companies developing base-editing and other mutation-specific strategies.