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4D Molecular Therapeutics to Kick off Phase I Gene Therapy Trial for Geographic Atrophy

NEW YORK – The US Food and Drug Administration has cleared an investigational new drug application (IND) from 4D Molecular Therapeutics (4DMT) for its investigational gene therapy for a serious eye condition that can result in vision loss, the company said Monday.

Emeryville, California-based 4DMT is developing the drug candidate, 4D-175, as a treatment to slow progression of geographic atrophy, an advanced and severe form of age-related macular degeneration (AMD). 4D-175 contains an engineered and codon-optimized version of the CFH gene, mutations in which have been associated with AMD. About three-quarters of AMD patients carry a high-risk variant of CFH.

4D-175 is delivered intravitreally, or to the inside of the eye, and is designed to provide durable transgene expression in the retina. The transgene, referred to as sCFH, encodes for a shortened form of complement factor H, a protein that regulates part of the immune response.

Armed with the FDA's permission, 4DMT plans to launch an open-label Phase I clinical trial, dubbed GAZE, to assess the safety and tolerability of 4D-175 in patients with geographic atrophy secondary to AMD. Patients will receive a single injection of the drug at one of three dose levels. 4DMT said it expects to begin enrollment in the second half of the year.

The company will also evaluate transgene expression and biological activity in the Phase I clinical trial and use study results to inform dose levels for a potential Phase II clinical trial.