Choroideremia (CHM) research hub

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study (SOLSTICE)

This study is a long-term follow-up for men who have already received gene therapy for rare inherited eye conditions (choroideremia or X-linked retinitis pigmentosa). Researchers want to track their eye health and sight over several years to ensure the treatment remains safe and effective.