"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."
"Researchers at UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) have grown ‘mini eyes', which make it possible to study and better understand the development of blindness in a rare genetic disease called Usher syndrome for the first time....The 3D ‘mini eyes’, known as organoids, were grown from stem cells generated from skin samples donated by patients at Great Ormond Street Hospital for Children (GOSH)....These ‘mini eyes’ are an important step forward because previous research using animal cells couldn’t mimic the same sort of sight loss as that seen in Usher syndrome."
"WILMINGTON, Mass. & DALLAS, Tx. – October 18, 2022 – Charles River Laboratories International, Inc. and Nanoscope Therapeutics, Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced a comprehensive manufacturing collaboration utilizing Charles River’s extensive contract development and manufacturing (CDMO) services in both plasmid DNA and viral vectors."
Once our gene therapies are ready to leave the lab and enter preparation for human testing, clinical grade viral vectors will be needed.
Summary: Administering a chemical compound called synthetic retinoids to the retina helped restore brain networks associated with vision and prompted the growth of two times more neurons, effectively restoring vision in adult mouse models of the genetic visual disorder LCA.
While not Usher 1F, this is encouraging research for all types of inherited retinal diseases.
"Artificial retinas made in space appear to be better than retinas made on Earth — suggesting that a cure for a leading cause of blindness could be one of the first products manufactured on tomorrow’s commercial space stations."
"Kiora Pharmaceuticals (NASDAQ:KPRX) expects to release Phase 1b interim data at the beginning of 2023 from its flagship intravitreal injection to potentially restore vision in patients with retinitis pigmentosa, a rare genetic eye disease where cells in the retina breakdown over time, causing vision loss.Kiora Pharmaceuticals (NASDAQ:KPRX) expects to release Phase 1b interim data at the beginning of 2023 from its flagship intravitreal injection to potentially restore vision in patients with retinitis pigmentosa, a rare genetic eye disease where cells in the retina breakdown over time, causing vision loss."
"In many cases, the only thing a patient can do to manage their disease is go to an ophthalmologist and have their case followed and monitored, but without any effective treatment. A versatile gene-editing platform that could treat many of these diseases would completely transform the lives of these patients. The motivation behind this project is to develop a gene-editing approach that may have the potential to treat more of these diseases, and thereby improve the current treatment landscape and the outcome for the individual patient in the future."
"A new approach leveraging high-yield CRISPR could make it easier to re-engineer massive quantities of cells for therapeutic applications."