HORA-PDE6B / Coave Therap - LARVOL DELTA

EyeDNA Therapeutics Receives Rare Pediatric Disease Designation from FDA for its Investigational Gene Therapy HORA-PDE6b for Patients with Retinal Dystrophy due to PDE6b Gene Mutations (GlobeNewswire) - "eyeDNA Therapeutics...announces that it has been granted a Rare Pediatric Disease Designation (RPDD) by the US Food & Drug Administration (FDA) for HORA-PDE6b, its novel gene therapy for patients with inherited retinal dystrophy (IRD) caused by mutations of the PDE6b gene."

FDA event • Inherited Retinal Dystrophy • Ophthalmology

eyeDNA Therapeutics to Present 2-year Follow-up Phase I/II Data on its Investigational Gene Therapy HORA-PDE6b for Retinitis Pigmentosa at the ARVO 2024 Annual Meeting (GlobeNewswire) - "eyeDNA Therapeutics....announces that updated 12-month and new 24-month follow-up results from its Phase I/II study evaluating the safety and efficacy of HORA-PDE6b, its investigational gene therapy for retinitis pigmentosa (RP) caused by bi-allelic mutations in the PDE6B gene (PDE6b RP) will be presented during oral presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting in Seattle, WA, USA (May 5-9, 2024)."

P1/2 data • Ophthalmology • Retinitis Pigmentosa

12-Month Safety and Efficacy Evaluation of HORA-PDE6B, a Gene Therapy Targeting Patients with Retinitis Pigmentosa Due to Biallelic PDE6B Gene Mutation. (ARVO 2024) - "It employs an Adeno-Associated Virus serotype 5 (AAV2/5) vector carrying the human PDE6B isoform 1 cDNA, controlled by the photoreceptor-specific human rhodopsin kinase promoter that targets Rods photoreceptors. Over a short 12-month period, HORA-PDE6B has shown a favorable safety profile and potentially efficacy, encouraging the continued development of HORA-PDE6B."

Clinical • Gene therapy • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • Uveitis

Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene (clinicaltrials.gov) - P1/2 | N=23 | Recruiting | Sponsor: Coave Therapeutics | Active, not recruiting ➔ Recruiting | N=17 ➔ 23 | Trial completion date: Dec 2026 ➔ Dec 2029 | Trial primary completion date: Jan 2023 ➔ Dec 2029

Enrollment change • Enrollment open • Trial completion date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene (clinicaltrials.gov) - P1/2 | N=17 | Active, not recruiting | Sponsor: Coave Therapeutics | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2026 ➔ Dec 2026 | Trial primary completion date: Sep 2022 ➔ Jan 2023

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

Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene (clinicaltrials.gov) - P1/2; N=15; Recruiting; Sponsor: Horama S.A.; Trial completion date: Sep 2024 ➔ Sep 2026

Clinical • Trial completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene (clinicaltrials.gov) - P1/2; N=15; Recruiting; Sponsor: Horama S.A.; Trial completion date: Jun 2022 ➔ Sep 2024; Trial primary completion date: Jun 2020 ➔ Sep 2022

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