REPISTAT is a new molecule capable of slowing the progression of Retinitis Pigmentosa.
BlueRock came out of the work at David Gamm's lab at the University of Wisconsin using stem cells to restore vision in those with inherited retinal diseases. It is exciting that they are now applying to begin a clinical trial.
Another gene agnostic gene therapy:
"SKG1108 uses a recombinant adeno-associated virus to deliver DNA directly to the retina. This DNA encodes light-sensitive proteins that can help restore vision by creating new photo-sensing cells, which replace the lost rod and cone cells in patients with advanced retinitis pigmentosa."
A clinical trial for all types of retinitis pigmentosa using stem cells - BlueRock Therapeutics grew out of the work of Dr. David Gamm at the University of Wisconsin.
Innovative Treatment Approach: The research utilized intravitreal injection of RPCs in a well-established animal model of photoreceptor degeneration. The RPCs release neurotrophic factors which are required to maintain the health and function of retinal cells. This is a novel use of these cells and delivering them to the vitreous cavity greatly simplifies the treatment procedure versus more invasive alternatives.
Therapeutic Benefits: The RPC treatment demonstrated substantial preservation of both retinal structure and visual function.
Clinical Progress: Supported by this preclinical work in rats, jCell has progressed through multiple clinical trials in patients with retinitis pigmentosa (RP). jCyte held a successful post-phase 2 Type B meeting with the FDA in January 2024 marking a significant milestone towards a pivotal trial in RP.
Topline results from the Phase 2b/3 RESTORE trial showed high- and low-dose MCO-010 optogenetic therapy significantly improved best-corrected visual acuity (BCVA) in patients with retinitis pigmentosa. These data were presented at the American Society of Retina Specialists (ASRS) 42nd Annual Meeting.
OCU400 has previously been evaluated in a phase 1/2 trial, data from which suggests positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), and Low-Luminance Visual Acuity (LLVA) among treated eyes. In this study, most patients (89%; n = 16/18) demonstrated preservation or improvement in the treated eye either on BCVA or LLVA or MLMT scores from baseline. Most (78%; n = 18) also had preservation or improvement in treated eyes in MLMT scores from baseline.
Kiora Pharmaceuticals, Inc. recently shared additional data from their ABACUS-1 clinical trial for KIO-301, a small molecule photoswitch aimed at improving vision in patients with late-stage retinitis pigmentosa. The new findings highlight a significant increase in brain activity in the visual cortex as measured by functional MRI (fMRI), confirming the drug's potential in enhancing visual processing.
"Ocugen Inc announced the first patient has been dosed in its Phase 3 liMeliGhT clinical trial for OCU400—a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP)....89% (16/18) of RP patients demonstrated preservation or improvement in the treated eye."
A gene agnostic treatment for RP enters a phase 3 clinical trial.
