Inherited retinal disease gene therapy widens past RPE65
X-linked retinitis pigmentosa, choroideremia, and additional inherited retinal disease gene therapy programs are reading out.
Inherited retinal disease gene therapy was a single-program category (voretigene neparvovec for RPE65-mediated disease) until recently. X-linked retinitis pigmentosa (RPGR-targeted), choroideremia, and additional inherited retinal disease gene therapy programs are now in late-stage trials. The genetic-testing infrastructure question (genotype-confirmed diagnosis in inherited retinal disease) is the addressable-population enabler.
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