Retinitis pigmentosa

Topline results from a Phase I/II clinical trial have shown that Kiora’s light-restoring small molecule KIO-301 has the potential to meaningfully improve vision in patients with retinitis pigmentosa (RP). The results of the ABACUS study for Kiora’s intravitreal (IVT) molecular photoswitch were presented at the American Academy of Ophthalmology annual conference by Russell Van Gelder, […]

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 […]