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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
LX101 (Containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65 encoding human RPE65 gene).Specification: 0.2mL/bottle.Route of administration: subretinal injection, injection volume 0.3 mL/ eye
Local and systemic safety assessment
Eye and non-eye adverse events
Time frame: 52(+4)weeks
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