CHM and choroideremia: There are also several ongoing phase 3/4 clinical trials using rAAV as the vector, including a study of long-term follow-up safety and efficacy study of spinal muscular atrophy Type 1, Type 2, or Type 3 (ClinicalTrials.gov: NCT04042025), a study to evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with Choroideremia (ClinicalTrials.gov: NCT03496012), and a study to demonstrate the efficacy of AAV5-hFIXco-Padua (AMT- 061) and to further describe its safety profile (ClinicalTrials.gov: NCT03569891).