Researchers have started a new gene therapy clinical trial to treat X-linked RP. The trial is being run by Nightstarx Ltd, a biopharmaceutical spinout company of Oxford the University of Oxford developing gene therapies for inherited retinal diseases, and researchers from the University of Oxford.
Self-administered weekly transcorneal stimulation in patients with retinitis pigmentosa was found safe and tolerable over one year of treatment, but the study failed to meet its primary endpoint of enlarged visual field area.
Researchers show that GARP2 accelerates retinal degeneration in mice, and have made an important step toward creating a standardized nomenclature between mice and humans for a measurement of retinal degeneration.
The US Food & Drug Administration has granted GenSight’s developing drug, GS030, Orphan Drug Disease Designation for the treatment of retinitis pigmentosa.
A novel gene-editing method has been found to partially restore sight in rats bred with RP.
A stem cell-based transplantation approach that restores vision in blind mice moves closer to being tested in patients with end-stage retinal degeneration, according to a study published January 10 in Stem Cell Reports.
The US Food & Drug administration has granted orphan drug designation (ODD) to MeiraGTx’s adeno-associated virus (AAV)-mediated gene therapy product candidate for treatment of X-linked retinitis pigmentosa (XLRP).
Ten people with an inherited form of blindness will be fitted with retinal implants in a procedure funded by NHS England.
Eight patients with retinitis pigmentosa have received a stem cell injection in a 28-person safety study.
The Guardian reports blind animals have had their vision partially restored using a DNA editing technique that scientists say could in future be applied to a range of genetic diseases.