The FDA unveiled draft guidance aimed at expanding access to personalized gene therapies for rare diseases by allowing a “plug and play” approval approach. Under the proposal, sponsors could obtain approval for one mutation-correcting therapy using a traditional development program, then later seek clearances for additional mutations using laboratory data instead of repeating full clinical trials. Regulators said they plan to define the scope for supplemental clearances through an “individualized feature space” included in the original application. The strategy is designed to address a recurring bottleneck in rare diseases where individual mutations are too uncommon to support standalone clinical trials. The FDA also tied the proposal to broader efforts under its Rare Disease Framework Initiative, including standards that focus on individual conditions rather than a one-size-fits-all model. Beam Therapeutics and Prime Medicine were flagged as potentially relevant beneficiaries due to their base-editing and gene therapy platforms. The agency is holding related discussions on rare disease initiatives on its White Oak campus as it evaluates how to operationalize the framework.