4D Molecular Therapeutics has obtained FDA clearance for its Investigational New Drug Application for 4D-175, an intravitreal genetic medicine utilizing the R100 vector, intended for treating geographic atrophy (GA) in patients. GA is a severe type of age-related macular degeneration (AMD) that causes irreversible vision loss and affects millions globally.

The Phase 1 GAZE clinical trial aims to evaluate 4D-175 in GA patients secondary to AMD. This trial includes an open-label Dose Exploration stage where participants will receive a single intravitreal injection of 4D-175 at various doses. The objectives of the trial are to assess safety, define optimal dose levels for Phase 2, and evaluate transgene expression and biological activity. With FDA clearance secured, 4DMT plans to begin enrolling patients at clinical sites in the latter half of 2024.

4DMT, emphasized that 4D-175 has the potential to provide sustained clinical benefits with a single injection, thus reducing the treatment burden associated with current therapies. The therapy aims to restore normal complement regulation in the retina, potentially slowing disease progression and improving long-term vision outcomes.

The development of 4D-175 utilizes a proprietary vector, R100, and a codon-optimized transgene encoding a functional form of human complement factor H (sCFH). This genetic construct is designed to achieve sustained transgene expression in the retina while minimizing inflammation, addressing a significant unmet need in GA treatment.

 

Source: globenewswire.com

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