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eyeDNA Therapeutics

Ownership: Private
Development stage: Phase 2

Overview

Positive 24-month follow-up results from Phase I/II study (NCT03328130) of HORA-PDE6b gene therapy in patients with retinitis pigmentosa caused by bi-allelic mutations in PDE6b gene. The treatment was administered to 17 evaluable patients aged 18 and above with advanced forms of PDE6b RP, using two ascending doses in four consecutive cohorts. Results affirmed the positive interim analysis at the 12-month follow-up and are paving the way for a registrational trial.[6][8]

Therapeutic areas: Ophthalmology
Modalities: Gene therapy (AAV5-based gene replacement therapy)
Industry: Biotech

Drug pipeline

1 asset

HORA-PDE6b

Phase 2

Indications: Retinitis pigmentosa, PDE6b-related inherited retinal dystrophy

Partnerships

Théa Open Innovation (TOI) for development and commercialization of HORA-PDE6b

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