J&J gene therapy fails in rare eye disease

US healthcare giant Johnson & Johnson (NYSE: JNJ) has presented data from its Phase III LUMEOS study and disclosed that the trial did not meet its primary endpoint. 

The data in X-linked retinitis pigmentosa (XLRP), a rare inherited eye disease that causes progressive vision loss, were presented at the Retina Therapeutics Innovation Summit on Friday. The condition often manifests in childhood and mainly affects boys, with the LUMEOS study only enrolling a handful of women with the disease.

J&J acquired the drug in question— - botaretigene sparoparvovec (bota-vec) - in a deal with MeiraGTx (Nasdaq: MGTX) worth $130 million upfront and up to $415 million in total, in December 2023, following on from an original collaboration that began in 2019 when J&J's Janssen unit paid $100 million upfront as part of a deal to develop gene therapies for inherited retinal diseases.