Jun 1, 2023

Coave Reports Encouraging Phase 1/2 Clinical Trial Results for PDE6B Gene Therapy

Research News

The company is expanding the trial to enroll younger patients with less advanced disease

Coave Therapeutics, a French biotechnology company, announced that its gene therapy for people with retinitis pigmentosa (RP) caused by mutations in the gene PDE6B improved visual function at 12 months after dosing for a subgroup of six patients with less-advanced disease in a 17-patient, Phase 1/2 clinical trial.

The company said the results support preparation for a registrational trial for the PDE6B gene therapy. With success in a registrational or pivotal trial, the company would likely seek marketing approval from regulators such as the European Medicines Agency and/or the US Food & Drug Administration.

Coave has received approval from regulators to dose six additional patients, ages 13 to 25 years of age with less-advanced disease, in the current Phase 1/2 clinical trial.

Improvements for the trial subgroup were reported in: best-corrected visual acuity (BCVA); visual fields; microperimetry which measures sensitivity in the central retina; full-field sensitivity (FST), which measures general retinal sensitivity; and the ability to navigate a mobility course.

Two dose levels of the gene therapy were evaluated in the Phase 1/2 clinical trial. Only the patient’s worse-seeing eye was treated. Those patients with improved visual function received the higher dose.

Known as CTx-PDE6B, the gene therapy is administered by an injection underneath the retina. The gene therapy uses a human-engineered adeno-associated virus (AAV) to deliver copies of healthy PDE6B genes to the photoreceptors in the retina to augment the mutated gene copies.