News Related to RNA-Based Drugs

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FDA Proposes Accelerated Approval Pathway for Ultra-Rare Disease Gene Therapies

Feb 24, 2026

The FDA released draft guidance establishing a new approval framework for individualized gene-editing and RNA-based therapies targeting ultra-rare diseases, allowing approvals based on small studies when traditional trials are not feasible.

FDA Proposes Framework to Speed Rare Disease Gene Therapy Approvals

Feb 24, 2026

The FDA proposed a new framework to accelerate approvals of personalized treatments for rare genetic diseases, allowing drugmakers to rely on small studies when traditional trials are not feasible while requiring post-approval evidence collection.