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May 05, 2026
The United Arab Emirates has unveiled two gene therapy projects targeting autism, epilepsy, and rare brain disorders in children. The initiatives focus on underlying genetic causes and earlier diagnosis.
Mar 11, 2026
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
Feb 26, 2026
The FDA has proposed a new "plausible mechanism" pathway to approve customized treatments for rare diseases after testing in only a handful of patients, aiming to remove barriers for gene editing and other bespoke therapies that don't fit traditional approval systems.
Feb 24, 2026
The FDA has proposed new guidelines to create a pathway for approving bespoke therapies tested in small patient groups, specifically targeting rare genetic conditions and gene editing treatments that don't fit traditional approval systems.
