PDE6A gene therapy / STZ eyetrial - LARVOL DELTA

Ocular Safety and Toxicology of Subretinal Gene Therapy With rAAV.hPDE6A in Nonhuman Primates. (PubMed, Transl Vis Sci Technol) - P1/2 | "This study demonstrates that treatment candidate rAAV.PDE6A was well tolerated in NHPs. Occurrence of retinal atrophy as a dose-limiting finding highlights the importance of further study into the mechanisms of atrophy induction after retinal gene therapy."

Journal • Gene Therapies • Genetic Disorders • Inflammation • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • ACACA • PDE6A

PDE6A Gene Therapy for Retinitis Pigmentosa (clinicaltrials.gov) - P1/2 | N=9 | Active, not recruiting | Sponsor: STZ eyetrial | Trial primary completion date: Jul 2023 ➔ Jul 2027

Gene therapy • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • PDE6A

PDE6A Gene Therapy for Retinitis Pigmentosa (clinicaltrials.gov) - P1/2 | N=9 | Active, not recruiting | Sponsor: STZ eyetrial | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2025 ➔ Jul 2027 | Trial primary completion date: Sep 2021 ➔ Jul 2023

Enrollment closed • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

PDE6A Gene Therapy for Retinitis Pigmentosa (clinicaltrials.gov) - P1/2; N=9; Recruiting; Sponsor: STZ eyetrial

New P1/2 trial • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa