Autosomal dominant inheritance
Autosomal dominant inheritance means that the faulty gene is on an autosome (any chromosome other than the sex chromosomes) and that only one faulty copy is needed to cause sight loss.
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Significantly worse vision found in female carriers of X-linked RP and Choroideremia
It was previously believed that female carriers of X-linked inherited retinal diseases (IRDs) like X-linked retinitis pigmentosa (RP and Choroideremia) remained unaffected by sight loss.
SB-007 update: dose-expansion portion of SpliceBio’s phase 1/2 trial underway with first participant dosed at Oxford
SpliceBio has begun the dose-expansion portion of their phase 1/2 clinical trial of SB-007, a dual adeno-associated viral vector gene therapy for Stargardt disease.
Research news round-up – Spring 2024
A round-up of the latest research into inherited retinal conditions - February 2024.
From Lab to Clinical Trial: First Healthy Volunteer Dosed with OCT-980
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).
RTx-015: Exploring Optogenetic Gene Therapy for RP and Choroideremia
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.
Brain stimulation may reduce hallucinations
UK researchers have discovered that passing a weak electrical current between electrodes on a person’s scalp may lead to a reduction in frequency of the visual hallucinations experienced by some people living with sight loss.
Antabuse moves into clinical trial for people with RP
Disulfiram (Antabuse), FDA-approved for deterring alcohol, is in a phase 1 trial at the University of Washington to improve vision in retinitis pigmentosa.
Cell-ebrating the dosing of patients with jCell in a phase 2 clinical trial for RP
The latest research news from Retina UK.
Slowing sight loss in stargardts: Ocugen’s gene therapy trial offers promising early results
Ocugen’s gene therapy candidate, OCU410ST, is an experimental treatment designed to slow vision loss in Stargardt disease, an inherited retinal disease (IRD) with no approved therapies currently.