Retinitis pigmentosa

Endogena Therapeutics has completed the dose-escalation stage of its Phase I/IIa study of EA-2353 in retinitis pigmentosa (RP) with no clinically relevant or dose-limiting adverse events. Given the positive safety and tolerability profile, the study will now enrol patients into the expansion cohort, using the highest dose evaluated in the study to explore the potential […]

The US Food and Drug Administration (FDA) has designated the investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) as a Fast Track development programme.   Fast Track is a process designed to enable patients to benefit earlier from important new drugs for serious conditions.   EA-2353 takes a novel, small-molecule approach and selectively […]

Results from Phase I/II MGT009 study into investigational gene therapy botaretigene sparoparvovec suggest sustained vision improvement in patients with X-linked retinitis pigmentosa (XLRP).  The study found treatment with botaretigene sparoparvovec had an acceptable safety profile and demonstrated encouraging improvements in retinal sensitivity, visual function and functional vision.  The late-breaking results were revealed at the American […]