Retinitis pigmentosa emerges as next genetic medicine test bed
As therapies for one form of retinitis pigmentosa near the market, new technologies put subtype-agnostic therapies in reach
Nearly a decade after Luxturna was expected to open the door for gene therapies across inherited retinal diseases, retinitis pigmentosa is again becoming a proving ground for the field’s most advanced genetic medicine technologies.
The near-term test is X-linked retinitis pigmentosa, where Beacon Therapeutics Holdings Ltd. and MeiraGTx Holdings plc (NASDAQ:MGTX) each have an AAV gene therapy in late-stage development for disease caused by RPGR mutations. Their progress could show whether developers have solved enough of the delivery, manufacturing, endpoint, and regulatory challenges that slowed ophthalmic gene therapy after Luxturna...