Gene Therapy Reaches Conditions With No Prior Treatment
Hereditary optic neuropathies were untreatable for as long as they have been described, and mitochondrial agents plus adeno-associated viral gene therapy have changed that within a decade. Fourteen active clinical programmes now target hereditary optic nerve disease, several addressing the specific mitochondrial mutations responsible for the most common inherited form. Durability remains the open question, since a single administration priced against a lifetime of blindness avoided must actually last a lifetime. This segment grows at 14.1% against a market at 9.4%, and it grows from a base of essentially nothing.
Market Impact: Therapy costs 320,000 dollars annually








