The next generation of Operation Warp Speed is coming soon, and this time it’s going to take aim at rare diseases, Peter Marks, the director of the FDA’s Center for Biologics Evaluation and Research, told attendees of the Biopharma Congress in Washington, DC on Monday.
“We’re getting ready to pilot that program,” Marks said, making clear the goal is to help areas with high unmet needs.
"The gene-independent treatment approach activates endogenous retinal stem and progenitor cells, which differentiate into photoreceptors to possibly preserve or restore visual function, the release said."
While the title is a bit misleading, this article describes the development of a new and better way to deliver corrected genes to the eye to treat Usher syndrome/retinitis pigmentosa.
Lab grown retinal photoreceptor cells have the potential to replace cells lost in the eyes of those with RP/Usher to restore vision already lost.
This is further evidence of the benefits of potent antioxidants for RP. Oxidative stress plays a role in retinal degeneration, and so these compounds offer protection. NAC/NACA, both in clinical trial are another example, as is the compound our University of Oregon researcher is testing on our Usher 1F zebrafish with promising results.
"Today we’re excited to take the covers off of our first flagship product development program: the Science Eye, a visual prosthesis targeted at retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD), two forms of serious blindness presently without good options for patients. The Science Eye is a combination device that uses an optogenetic gene therapy targeted at the cells of the optic nerve (the retinal ganglion cells) in conjunction with an implanted flexible thin-film, ultradense microLED display panel inserted directly over the retina."
"Scientists at Stanford University have developed a retinal prosthesis with five times the resolution of the most advanced prosthetics currently used in clinical studies. By redesigning the electric circuitry of an existing prosthesis, researchers were able to match the natural vision of rats."
"In 2001, the first large animal was successfully treated with a gene therapy that restored its vision. Lancelot, the Briard dog that was treated, suffered from a human childhood blindness called Leber’s congenital amaurosis type 2. Sixteen years later, the gene therapy was approved by the U.S. Food and Drug Administration. The success of this gene therapy in dogs led to a fast expansion of the ocular gene therapy field. By now every class of inherited retinal dystrophy has been treated in at least one animal model and many clinical trials have been initiated in humans. In this study, we review the status of viral gene therapies for the retina, with a focus on ongoing human clinical trials. It is likely that in the next decade we will see several new viral gene therapies approved."
"A pair of phase 3 trials of Neurotech Pharmaceuticals’ encapsulated cell therapy implant have met their primary endpoints, positioning the biotech to talk to the FDA about bringing the treatment for a cause of central vision loss to market."
"Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) ("Kiora" or the "Company") announced today the dosing of the first patient in a first-in-human open-label clinical trial for KIO-301, intended to restore vision loss in patients with Retinitis Pigmentosa (RP). RP is a rare, inherited genetic eye disease resulting in degeneration of the retinal photoreceptors (rods and cones) and often significant loss of functional vision."
