Clinical-stage gene therapy company, eyeDNA Therapeutics has been granted a Rare Paediatric Disease Designation (RPDD) by the United States Food and Drug Administration (FDA) for HORA-PDE6b, its novel gene therapy for patients with inherited retinal dystrophy caused by mutations of the PDE6b gene.
PDE6b retinitis pigmentosa (PDE6b RP) is a rare inherited retinal dystrophy that accounting for between 1–3% of all cases of retinitis pigmentosa. The disease causes progressive loss of photoreceptors and vision. Symptoms typically begin in childhood and often progress to blindness by midlife. At present, no approved treatments are available for PDE6b RP.¹
HORA-PDE6b is an AAV5-based gene therapy designed to deliver a non-mutated copy of the functional human PDE6b gene into the subretinal space, where it rapidly induces transgene expression and synthesis of functional PDE6b proteins in photoreceptor cells. By effectively providing these cells with a functional protein, HORA-PDE6b may significantly delay or halt retinal degeneration in PDE6b-deficient patients.
eyeDNA presented positive 24-month follow-up results from its Phase I/II study evaluating the safety and efficacy of HORA-PDE6b last year.
According to the company, it is exploring the optimal paths to making HORA-PDE6b available to PDE6b RP patients in the US and in Europe via an accelerated approval process.
By effectively providing these cells with a functional protein, HORA-PDE6b may significantly delay or halt retinal degeneration
Reference
- Coave Therapeutics, 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 (media release, 17 Dec 2024), available at: globenewswire.com/news-release/2024/12/17/2998050/0/en/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-Mutat.html [accessed 20 Dec 2024].
