News

Astena Theraputics' investigational gene therapy, ATSN-101, has shown promising long-term results in an early clinical trial for LCA1, a sever inherited retinal disease caused by mutations in the GUCY2D gene. 

Beacon Therapeutics has announced positive results from its phase 2/3 VISTA trial of laru-zova, an experimental gene therapy for X-linked retinitis pigmentosa (XLRP). The findings mark a significant step forward for a condition that currently has no approved treatments.

Promising early clinical trial results suggest that ATSN-201, an investigational gene therapy for X-linked retinoschisis (XLRS), could offer a potential new treatment approach for this rate inherited retinal condition.

Ray Therapeutics, a clinical stage biopharmaceutical company, is currently investigating their lead optogenetic therapy candidate, RTx-015, in early stage clinical trials for those with RP and Choroideremia.

A consortium of researchers based at the Institute for Bioengineering of Catalonia (IBEC) have demonstrated that specially designed light-activated drugs can restore useful visual function in blind animals by making surviving retinal nerve cells respond to light.

Researchers have recently presented promising clinical data at the International Society for Stem Cell Research demonstrating that transplanted human neural progenitor cells survived for at least one year in participants with Retinitis Pigmentosa (RP).

Catherine L’Estrange, aged 11 from London has become the first individual in the UK to receive an investigative gene therapy treatment for retinal dystrophy associated with the BBS10 gene.

Octant, a clinical stage drug discovery company, has recently announced the dosing of the first healthy volunteer in their Phase 1a clinical trial of OCT-980 for Rhodopsin-associated autosomal dominant Retinitis Pigmentosa (RHO-adRP).

Biotechnology company Ocugen has announced the enrolment completion of their phase 3 clinical trial for OCU400.