4D-175: A Promising Treatment for Geographic Atrophy (GA)

4D-175, a vector-based intravitreal genetic medicine, has gained attention as a potential treatment for geographic atrophy (GA). Recently, 4DMT Molecular Therapeutics announced that the US Food and Drug Administration (FDA) approved the Investigational New Drug Application (IND) for 4D-175 for the treatment of GA. This approval means that the company can move forward with the GAZE clinical trial, an upcoming Phase 1 open-label trial in patients with GA secondary to age-related macular edema (AMD).

What Is 4D-175?

4D-175 is a vector-based intravitreal genetic medicine that combines a vector known as R100 and a codon-optimized transgene encoding a highly functional shortened form of human complement factor H (sCFH). According to the company, sCFH is an optimized version of CFH that can fit into adeno-associated virus (AAV) vectors with robust expression and full functionality in human cells in vitro and multiple preclinical animal models and species in vivo. Furthermore, the 4D-175 is designed to provide durable transgene expression in the retina without significant inflammation following a single, low-dose intravitreal injection.

The GAZE Clinical Trial

The GAZE clinical trial is an upcoming Phase 1 open-label trial that will evaluate 4D-175 in patients with GA secondary to age-related macular edema (AMD). According to the company, the trial will consist of a sequential cohort dose exploration stage, with patients receiving a single intravitreal injection of 4D-175 at one of three dose levels. The aim of this trial is to explore safety and transgene expression levels to select doses for Phase 2.

Potential Benefits of 4D-175

Based on the information provided by 4DMT, 4D-175 has the potential for durable clinical benefit with a single intravitreal injection, which could greatly reduce the current treatment burden for patients. This reduction may lead to better long-term vision outcomes. Additionally, the optimized version of CFH may lead to better outcomes in patients with geographic atrophy.

Conclusion

Overall, 4D-175 presents a promising option for treating geographic atrophy, and the approval of the IND by the FDA is a significant step forward. Although the GAZE clinical trial will not begin until the latter half of 2024, the potential for a single, low-dose intravitreal injection to provide durable clinical benefit is exciting. As always, additional research and evaluation will be necessary, but the potential benefits make 4D-175 worth monitoring.

Keywords:

4D-175
GAZE clinical trial
geographic atrophy
age-related macular edema
vector-based intravitreal genetic medicine

Originally Post From https://www.ophthalmologytimes.com/view/4dmt-receives-ind-clearance-for-4d-175-for-treatment-of-geographic-atrophy

“4DMT’s 4D-175 receives IND clearance for treatment of geographic atrophy”