Research Update

First Treatment Dose Administered to Patient in Phase 1 Trial for Choroideremia

The first human patient has received an investigative gene therapy in a Phase 1 clinical trial conducted by 4D Molecular Therapeutics to treat Choroideremia¹.

Choroideremia is a rare and inherited retinal disease (IRD) which is caused by degeneration of the light-sensitive photoreceptor cells within the retina, and can cause night blindness in its early stages when the rod photoreceptor cells die. Over time, the visual field narrows and progresses to tunnel vision and blindness commonly occurs in late adulthood. Given that no treatment or cure currently exists for Choroideremia, this research shows a lot of promise in finally developing a safe and effective therapy.

4D-110 is a novel gene therapy which has been designed to provide a functional CHM gene to replace the defective or mutated copy causing Choroideremia, and is delivered into the retina via intravitreal injection. 15 patients living with Choroideremia are expected to be enrolled in this phase 1 study which will assess the safety, tolerability and biological activity of the one-time injection, as well as the changes to visual function and retinal degeneration.

Principal investigator of the study, David Birch, PhD commented on the importance of this research, stating; “With no currently approved therapies available for patients impacted by Choroideremia, gene therapy represents a promising therapeutic approach,” and that 4D-110 “shows promise in safely treating a broad region of the retina and in a broad range of patients.”

References:

1. 4D Molecular Therapeutics Announces First Patient Dosed in Phase 1 Clinical Trial of 4D-110 by Intravitreal Injection for the Treatment of Choroideremia. Available at https://www.4dmoleculartherapeutics.com/4d-molecular-therapeutics-announces-first-patient-dosed-in-phase-1-clinical-trial-of-4d-110-by-intravitreal-injection-for-the-treatment-of-choroideremia/. Accessed July 2020.