New FDA Plausible Mechanism Framework Spurs Development of Personalized In Vivo Prime Editing Platform for Urea Cycle Disorders
PR Newswire —
Guidance opens doors for rare disease treatments, but academic teams will likely need industry partners to meet rigor required for FDA approval PHILADELPHIA, March 31, 2026 /PRNewswire/ -- Researchers from Children's Hospital of Philadelphia (CHOP) and Penn Medicine, along with...