Beacon Therapeutics
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
This is a Phase 2, open-label, multicenter study to evaluate the safety of 2 doses of AGTC-501 administered as a single subretinal injection in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein. The trial includes a screening period of up to 60 days and a 5 year study period. Each participant will receive a single subretinal injection of one of two dose levels of AGTC-501 in their previously untreated eye. There will be 3 groups. Group 1 will receive the high dose and include up to 12 participants, Group 2 will rec…
Inclusion Criteria: * Be ≥12 years of age * Have one eye previously treated with an AAV vector-based gene therapy designed to provide full-length functioning RPGR protein. * Have a BCVA no better than 78 letters and no worse than 34 letters * Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability and fixation, per the Investigator's discretion. * Have detectable baseline mean macular sensitivity measured by MAIA microperimetry, as determined by the Investigator and confirmed by the Central Reading Center (CRC). * Have dete…
Adeno-associated virus vector expressing a human RPGR gene
Adeno-associated virus vector expressing a human RPGR gene
Adeno-associated virus vector expressing a human RPGR gene