May 10, 2024

ARVO 2024 Highlight: Positive Two-Year Results from PDE6B Gene Therapy Clinical Trial

Eye On the Cure Research News

Mutations in PDE6B are a leading cause of retinitis pigmentosa.

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EyeDNA Therapeutics, a subsidiary of Paris-based Coave Therapeutics, reported encouraging 24-month results for 17 adult patients in its Phase 1/2 PDE6B gene therapy clinical trial underway at Clinique Ophthalmologique, CHU de Nantes, in France. The results were reported by Dr. Jean-Baptiste Ducloyer at the 2024 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) held May 5-9 in Seattle. The company plans to talk with regulators to establish a path for advancing the emerging gene therapy.

According to Dr. Ducloyer, mutations in PDE6B cause autosomal recessive retinitis pigmentosa in approximately 4,500 people in the US, EU, and Japan. PDE6B expresses an enzyme that is part of the phototransduction cascade, the biochemical process in the retina that makes vision possible.

Vision improvements in the trial were observed for patients who received the high and low dose of the gene therapy, which is known as HORA-PDE6B. Five-year follow-up data showed that vision in patients receiving the low dose treatment had stable best-corrected visual acuity (BCVA). In comparison, BCVA consistently declined in untreated eyes. A full-field stimulation test using blue light indicated that rod function also improved in patients' treated eyes. Rods provide peripheral vision and vision in dark and dim settings.

EyeDNA is also evaluating its HORA-PDE6B gene therapy in as many as six younger patients, 13 to 17 years old, who have earlier stage disease. Three younger patients are enrolled thus far.

All patients in the Phase 1/2 clinical have had one eye treated. Dr. Ducloyer said that most patients want their second eye treated.

There were five eye-related serious adverse events in patients that were potentially due to the investigational treatment, and two of these were resolved. 

HORA-PDE6B is administered by an injection underneath the retina. The gene therapy uses a human-engineered adeno-associated virus to deliver healthy copies of the PDE6B gene to the photoreceptors in the retina to augment the mutated gene copies.

EyeDNA is developing and commercializing HORA-PDE6B with Théa Open Innovation.