eyeDNA Therapeutics has been granted a Rare Pediatric Disease Designation by the FDA for HORA-PDE6b, its novel gene therapy for patients with inherited retinal dystrophy caused by mutations of the PDE6b gene, the company said in a press release. 

PDE6b Retinitis Pigmentosa (PDE6b RP) is a rare inherited retinal dystrophy that is estimated to affect up to 3,000 people in the United States and 4,000 people in Europe, accounting for 1- 3% of all RP cases. The disease is caused by mutations of the PDE6b gene resulting in dysfunctional PDE6b protein, disrupting the conversion of light into electrical signals and leading to the progressive loss of photoreceptors and vision. Symptoms often start in childhood and lead to blindness by midlife. There are currently no approved treatments for PDE6b RP, the company said in the press release. 

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, the company said. 

eyeDNA presented positive 24-month follow-up results¹ from its Phase 1/2 study (NCT03328130) evaluating the safety and efficacy of HORA-PDE6b at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting.

eyeDNA said it is exploring the optimal paths to making HORA-PDE6b available to PDE6b RP patients in the United States and in Europe via an accelerated approval process.

Reference
1. JB Ducloyer, et al. 12-month Safety and Efficacy Evaluation of HORA-PDE6b, a Gene Therapy Targeting Patients with Retinitis Pigmentosa Due to Biallelic PDE6B Gene Mutation (2024) Abstract 2134, Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting: Seattle, WA, USA