WebDec 23, 2016 · Gene Therapy for Achromatopsia (CNGB3) (CNGB3) The safety and scientific validity of this study is the responsibility of the study sponsor and … WebMay 14, 2024 · Gene therapy offers first steps in treatment of achromatopsia. Mark Pennesi, MD, PhD, reports during ARVO 2024 that preliminary results have shown that AGTC-401 and AGTC-402 seem safe and well tolerated in patients with ACHM. The preliminary results of 2 ongoing phase 1/2 clinical trials evaluating the effects of …
Gene therapy offers first steps in treatment of achromatopsia
WebOct 17, 2016 · This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy. WebAchromatopsia is a genetic disorder in which a child is born with nonfunctioning cones. The cones are special photoreceptor cells in the retina that absorb different color lights. There are three types of cones … headset mpow air se
Gene therapy for achromatopsia Semantic Scholar
WebDec 2, 2024 · Achromatopsia is currently without a cure, but phase I/IIa clinical trials of gene augmentation therapy for CNGA3 and CNGB3 achromatopsia are underway (NCT02935517, NCT02599922, NCT03758404, and ... WebAug 24, 2024 · Achromatopsia is a severe monogenic heritable retinal disease that disrupts cone function from birth, leaving patients with complete colour blindness, low acuity, photosensitivity and nystagmus. While successful gene-replacement therapy in non-primate models of achromatopsia has raised widespread hopes for clinical treatment, it was yet … WebJan 5, 2024 · Using a novel multimodal approach, we demonstrate for the first time that gene therapy can successfully activate dormant cone-mediated pathways in children with achromatopsia (CNGA3- and … headset multilaser p2 preto - ph002