4D Molecular Therapeutics (4DMT) announced that the first patient has been dosed in the phase 1 clinical trial of 4D-110, a Roche-licensed product candidate, delivered by a single intravitreal injection for Choroideremia. Choroideremia is a blinding and currently untreatable X-linked inherited retinal disease.
“Dosing the first patient in the phase 1 clinical trial of 4D-110 marks the first of three Therapeutic Vector Evolution pipeline candidates expected to enter the clinic in 2020,” David Kirn, MD, co-founder, chairman and chief executive officer of 4DMT, said in a company news release. “4DMT harnesses the power of directed evolution to develop precision-guided AAV gene medicines. In the case of 4D-110, this proprietary and optimized AAV vector is designed to provide targeted delivery to the retina of a functional copy of the CHM gene by intravitreal injection, a routine clinical route of administration. Ultimately, our aim is to alter the course of this debilitating disease and to treat patients with all stages of the disease. I would like to thank the Choroideremia patient community, their families and caregivers, the Choroideremia Research Foundation, and the clinical trial physicians and staff, without whom 4DMT would not have reached this stage.”
“With no currently approved therapies available for patients impacted by Choroideremia, gene therapy represents a promising therapeutic approach,” said David Birch, PhD, Scientific Director, Retina Foundation of the Southwest and a principal investigator for the study. “Due to its optimized vector, 4D-110 is a novel gene therapy approach that shows promise in safely treating a broad region of the retina and in a broad range of patients. The potential to slow-down or halt the debilitating visual field constriction seen in this disease is an exciting opportunity for patients.”
The phase 1 open-label, dose-exploration and dose-expansion study is expected to enroll up to 15 patients with Choroideremia. The study is designed to assess the preliminary safety, tolerability and biological activity of a single intravitreal injection of 4D-110. In addition, the clinical trial will evaluate the effect of 4D-110 on the visual function and retinal degeneration.
“On behalf of the Choroideremia Research Foundation and Choroideremia patients internationally, I am extremely excited by the initiation of 4DMT’s clinical trial,” said Christopher Moen, MD, Chief Medical Officer of the Choroideremia Research Foundation. “This pivotal milestone brings us one step closer to a transformative treatment which has the potential to end blindness from Choroideremia.”