4DMT receives IND clearance for 4D-175 for GA treatment
The company expects enrollment of the GAZE phase 1, open-label clinical trial, to begin in the second half of 2024.
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4DMT Molecular Therapeutics announced the US Food and Drug Administration (FDA) has approved the company’s Investigational New Drug Application (IND) for 4D-175 for the treatment of geographic atrophy (GA).
4D-175 is a vector-based intravitreal genetic medicine that combines the intravitreal vector, R100, and a codon-optimized transgene encoding a “highly functional shortened form of human complement factor H (sCFH).”1
The company noted in its release, sCFH is an engineered and optimized version of CFH that can fit into adeno associated virus (AAV) vectors with robust expression and full functionality confirmed in human cells in vitro, as well as in multiple preclinical animal models and species in vivo.1
Furthermore, according to the company, the 4D-175 is designed to provide durable transgene expression in the retina without significant inflammation following a single, low-dose intravitreal injection.
GAZE, an upcoming Phase 1 clinical trial, will access 4D-175 in patients with GA secondary to age-related macular edema (AMD). According to the company, the trial will be open-label and consist of a sequential cohort Dose Exploration stage. Patients will receive a single intravitreal injection of 4D-175 at 1 of 3 dose levels. The clearance of the IND from the FDA allows 4DMT to continue with the GAZE clinical trials, and the company expects enrollment to begin in the second half of 2024.1
“4D-175 has the potential for durable clinical benefit with a single intravitreal injection, greatly reducing the current treatment burden for patients, which may lead to better long-term vision outcomes. In Phase 1, we aim to explore safety and transgene expression levels to select doses for Phase 2,” said David Kirn, MD, co-founder and CEO of 4DMT.
References:
4DMT Announces FDA Clearance of IND Application for 4D-175 Genetic Medicine for the Treatment of Geographic Atrophy. Press Release; June 24, 2024. Accessed June 27, 2024. https://www.globenewswire.com/news-release/2024/06/24/2902932/0/en/4DMT-Announces-FDA-Clearance-of-IND-Application-for-4D-175-Genetic-Medicine-for-the-Treatment-of-Geographic-Atrophy.html
Preclinical Characterization of 4D-175, a Novel AAV-based Investigational Intravitreal Gene Therapy for Geographic Atrophy. Accessed June 27, 2024. https://4dmoleculartherapeutics.com/wp-content/uploads/Presentation_ARVO-2024_Calton_4D-175-Preclinical-Characterization_Final_20Apr2024.pdf
