HORA-PDE6b Gene Therapy Shows Promise for Retinitis Pigmentosa at 24 Months


eyeDNA Therapeutics, a subsidiary of Coave Therapeutics, has announced promising 24-month follow-up data from its ongoing Phase I/II clinical trial of HORA-PDE6b, an investigational gene therapy for retinitis pigmentosa caused by bi-allelic mutations in the PDE6b gene. The results, presented at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting, demonstrate the potential of HORA-PDE6b to provide long-term benefits for patients with this rare genetic eye disorder.

The Phase I/II study is evaluating the safety and efficacy of HORA-PDE6b, which utilizes Coave Therapeutics’ proprietary gene therapy platform to deliver a functional copy of the PDE6b gene to the retina. Retinitis pigmentosa is a progressive, inherited eye disease that leads to severe vision loss and blindness due to mutations in the PDE6b gene. By addressing the underlying genetic cause of the disease, HORA-PDE6b aims to slow or halt the progression of vision loss in affected patients.