These include valoctocogene roxaparvovec for Hemophilia A (NCT03370913, NCT03392974), etranacogene dezaparvovec for Hemophilia B (NCT03569891), onasemnogene abeparvovec‐xioi for spinal muscular atrophy (SMA) (NCT03505099, NCT03837184), lenadogene nolparvovec for Leber Hereditary Optic Neuropathy (NCT03406104), and AAV5‐RPGR for X‐linked Retinitis Pigmentosa (NCT04671433). The gene discussed is RPGR; the disease is proximal spinal muscular atrophy.