rAAV2-CB-hRPE65 / Beacon Therapeutics, UMass Chan Medical School - LARVOL DELTA

An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA (clinicaltrials.gov) - P=N/A | N=3 | Not yet recruiting | Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Gene therapy • New trial • Gene Therapies • Inherited Retinal Dystrophy • Retinal Disorders

RPE65 Gene Therapy （LX101）for Leber's Congenital Amaurosis (clinicaltrials.gov) - P=N/A; N=9; Not yet recruiting; Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Clinical • New trial • Gene Therapies • Inherited Retinal Dystrophy • Retinal Disorders

Optimal Inhibition of Choroidal Neovascularization by scAAV2 with VMD2 Promoter-driven Active Rap1a in the RPE. (PubMed, Sci Rep) - "scAAV2-VMD2, but not scAAV2-RPE65, specifically and efficiently transduced the RPE to increase active Rap1a protein in the RPE...Increased active Rap1a in the RPE in vivo or in vitro inhibited inflammatory and angiogenic signaling determined by decreased activation of NF-κB and expression of VEGF without causing increased cell death or autophagy measured by increased LCA3/B. Our study provides a potential future strategy to deliver active Rap1a to the RPE in order to protect against both atrophic and neovascular AMD."

Journal • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Gene Therapies • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Results at 5 Years after Gene Therapy for RPE65-deficient Retinal Dystrophy. (PubMed, Hum Gene Ther) - "There were no clinically significant adverse events. Visual acuity and static perimetry testing results suggest that treating patients at a younger age is associated with better visual function outcomes during 5 years after treatment."

Journal • Gene Therapies • Ophthalmology • Pediatrics

AGTC announces data evaluating novel AAV-based gene therapy as a potential treatment for Leber congenital amaurosis (LCA) and severe early-childhood-onset retinal degeneration (SECORD) (GlobeNewswire) - P=NA, N=12; "In the treated eye, best-corrected visual acuity (BCVA) increased in five subjects, static perimetry hill of vision measurements for the central 30° of the visual field increased in six subjects, total visual field hill of vision measurements increased in five subjects, and kinetic visual field area improved in three subjects. One study participant had a decrease in BCVA and two subjects had a decrease in kinetic visual field area. Common adverse events associated with the injection included subconjunctival hemorrhage in eight subjects and ocular hyperemia (redness) in five subjects. The investigators concluded that the treatment with rAAV2-CB-hRPE65 was not associated with serious adverse events and improvement in one or more measures of visual function was observed in 9 of 12 (75 percent) subjects."

Clinical data • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology

"Approved or near approval gene therapy list, with my edit. This list ignores approved antisense, oligos, and vaccines." - @antonioregalado

Anticipated regulatory • Pipeline update • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology

VMD2 Promoter-mediated Gene Therapy Optimizes Active Rap1a Expression in the Retinal Pigment Epithelium of Wild Type Mice (ARVO 2019) - "However, an autophagy regulator, LC3A/B protein in RPE/choroid was not increased in AAV2-VMD2-CARap1a injected eyes compared to either AAV2-VMD2-GFP or scAAV2-RPE65-CARap1a. Conclusions The VMD2 promoter has greater specificity for vector mediated expression in the RPE than the RPE65 promoter and increased expression of CARap1a without increasing autophagy."