To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA) Clinical Trial
Official title:
An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy (LX101) in Patients With Leber's Congenital Amaurosis (LCA)
To evaluate the scaling clinical trail of AAV2-RPE65 gene therapy agent (LX101) in patients with congenital amaurosis (LCA).
This study evaluated the overall safety and initial efficacy of RPE65 mutant congenital amaurosis (RPE65-LCA) in subjects treated with a single, subretinal injection of LX101 (containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65) in the second eye 1 year after treatment. ;