AAV-RPE65

Unassigned

New Medicines

Leber congential amaurosis caused by mutations in the RPE65 gene

Information

Advanced therapy medicinal product (ATMP)
MeiraGTx
MeiraGTx

Development and Regulatory status

Phase II Clinical Trials
None
Phase II Clinical Trials
Yes
Yes

Category

An adeno-associated virus (AAV) gene therapy
LCA is the severest of all retinal dystrophies. Affected individuals usually present in the first year of life with profound sight impairment, roving nystagmus, variable retinal pathology and occasionally other systemic pathology. It is known to be caused by at least six genes. Estimated prevalence is 1 per 50,000-100,000 [1].
Leber congential amaurosis caused by mutations in the RPE65 gene
Subretinal injection
Parenteral