Gene therapy receives rare pediatric disease designation for inherited retinal dystrophy

The FDA granted rare pediatric disease designation for HORA-PDE6b, a gene therapy for patients with inherited retinal dystrophy caused by mutations of the PDE6B gene, according to a press release from eyeDNA Therapeutics.

The AAV5-based gene therapy is designed to delay or halt retinal degeneration in PDE6B-deficient patients through delivery of a non-mutated copy of the PDE6B gene into the subretinal space. The designation will allow eyeDNA Therapeutics to receive a priority review voucher if HORA-PDE6b is approved that can be used to advance an additional program or be sold to another company.

The gene therapy is supported by positive 24-month safety and efficacy data presented at the Association for Research in Vision and Ophthalmology 2024 meeting.

“This rare pediatric disease designation for HORA-PDE6b from the FDA recognizes the urgent need for effective treatments for this devastating disease where early treatment could slow or halt disease progression and have a profound impact on patients’ quality of life,” Rodolphe Clerval, CEO of Coave Therapeutics, parent company of eyeDNA Therapeutics, said in the release. “We look forward to providing further data updates from our ongoing phase 1/2 trial with HORA-PDE6b, and we are particularly excited to see initial results in younger patients where HORA-PDE6b could produce the greatest clinical benefit.”

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