A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Treated Previously with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) in an Antecedent Study (SOLSTICE)
Sponsor: NightstaRx, Ltd, a Biogen Company
Principal Investigator: Kimberly Stepien, MD
Study Coordinator: Nickie Stangel
Long-term follow up evaluating the safety and efficacy of AAV2-REP1 used in antecedent choroideremia treatment studies.
The study will consist of up to 9 visits over a maximum 48-month study period. Subjects will rollover into the current study when they complete the antecedent study. No further gene therapy will be given in this protocol.
Have received a sub-retinal injection of AAV2-REP1 for CHM in an antecedent study