FDA Approval Precedent (for gene therapy)

**What it means:** The fact that seven AAV-based gene therapies have received FDA approval provides a regulatory pathway that the MEF2C gene therapy team can follow. FDA has extensive experience reviewing AAV gene therapies for safety and efficacy, which means the review process for an MEF2C therapy is well-understood — it's just that the specific therapy still needs to be developed and tested. **Key precedent papers:** Luxturna (inherited retinal disease, 2017) was the first FDA-approved gene therapy for an inherited condition. Zolgensma (spinal muscular atrophy, 2019) showed that AAV gene therapy could work for a neurological condition — highly relevant to MHS since both target brain tissue.