FDA actions hit multiple gene therapy programs as uniQure, REGENXBIO, and Sarepta updates sharpen regulatory focus
SHERIDAN, WYOMING - March 04, 2026 - A cluster of U.S. FDA-driven developments is reshaping near-term operating plans across gene therapy developers, with uniQure disclosing a new Phase 3 requirement, REGENXBIO facing a complete response letter for its Hunter syndrome program, and Sarepta Therapeutics managing a leadership change alongside conservative 2026 sales expectations for Elevidys. The same news cycle also includes broader questions from regulatory experts about how decisions are being made inside the agency, including scrutiny around CBER leadership. For clinical, regulatory, and finance teams, the immediate takeaway is execution risk: trial design expectations, control strategy, and evidentiary standards are moving targets that can directly change timelines and cost.