GenSight Biologics, a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, announced UK Medicines and Healthcare Regulatory Agency (MHRA) acceptance of the Company’s Clinical Trial Application (CTA) to initiate the PIONEER Phase I/II study of GS030 in patients with Retinitis Pigmentosa (RP).
Category Archives: Gene therapy
Visual Activity Regenerates Neural Connections Between Eye and Brain
A study in mice shows for the first time that high-contrast visual stimulation can help damaged retinal neurons regrow retinal ganglion cell axons. In combination with chemically induced neural stimulation, axons grew further than in strategies tried previously. Treated mice partially regained visual function. The study also demonstrates that adult regenerated central nervous system (CNS)
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A gene therapy platform for macular degeneration patients
Physics researchers at The University of Texas at Arlington have developed a new platform that uses ultrafast near-infrared lasers to deliver gene therapy to damaged areas of the retina to enable vision restoration in patients with photo-degenerative diseases such as macular degeneration. Samarenda Mohanty, assistant professor of physics and head of UTA’s Biophysics and Physiology
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RPE65 is most frequently mutated gene in Leber Congenital Amaurosis in Denmark
Leber congenital amaurosis (LCA) represents the most severe form of inherited retinal dystrophies with an onset during the first year of life. Currently, 21 genes are known to be associated with LCA and recurrent mutations have been observed in AIPL1, CEP290, CRB1 and GUCY2D. In addition, sequence analysis of LRAT and RPE65 may be important
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Researchers Grow Retinal Nerve Cells in the Lab
Researchers have developed a method to efficiently turn human stem cells into retinal ganglion cells. Death and dysfunction of these cells cause vision loss in conditions like glaucoma and multiple sclerosis.
Ocular treatment likely to become first Gene Therapy approved in U.S.
Spark Therapeutics has announced positive results from the Phase 3 pivotal trial of its lead gene therapy product candidate, SPK-RPE65, for the treatment of RPE65-mediated inherited retinal dystrophies (IRDs). These results represent the first successful randomized, controlled Phase 3 trial ever completed in gene therapy for a genetic disease.
Gene therapy for neovascular age-related macular degeneration safe at 1 year
Results from a single-center, phase 1, randomized controlled clinical trial show that ocular gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration was found to be safe and well tolerated. The study enrolled patients with wet age-related macular degeneration at the Lions Eye Institute and the Sir Charles Gairdner Hospital in Nedlands, WA, Australia. Eligible
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AMD likely to be more in people with deficient vitamin D status
A multi-institutional team reports that vitamin D may play a significant role in eye health, specifically in the possible prevention of age-related macular degeneration, or AMD, among women who are more genetically prone to developing the sight-damaging disease. In a paper published in JAMA Ophthalmology online, the team found that women who are deficient in
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RetroSense Therapeutics Gets Approval for Clinical Trials in Retinitis Pigmentosa
RetroSense Therapeutics’ Investigational New Drug (IND) application for gene therapy based clinical trial for their product RST-001 has received clearance from the US Food and Drug Administration. RetroSense is developing RST-001 for the treatment of retinitis pigmentosa, a genetic condition that leads to the progressive degeneration of rod and cone photoreceptors (cells found in the retina that sense light),
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