Arshad Khanani, MD, on Restoring Aflibercept Expression in Wet AMD With Gene Therapy


The director of clinical research at Sierra Eye Associates discussed data from the phase 1/2 PRISM study.

“The exciting thing here is that with 4D-150, we are targeting the retina and not the front of the eye, as well as using very low doses, and our goal is to produce enough protein to control the disease while not having the complications that other programs have seen."

The gene therapy 4D-150 (4D Molecular Therapeutics) restored aflibercept expression to therapeutic levels in 5 patients with wet age-related macular degeneration (AMD) treated with a single intravitreal injection of 3E10 vg/eye of 4D-150. These data are from cohort 1 of the phase 1/2 PRISM trial (NCT05197270).

Principal investigator Arshad Khanani, MD, director, clinical research, and clinical associate professor, University of Nevada Reno School of Medicine, and colleagues found a 96.7% reduction in mean annualized anti-VEGF injection rate in treated participants, from around 11 in the year prior to enrollment. Most participants (80%) did not receive any supplemental aflibercept injections for around 40, 36, 32, or 16 weeks after treatment and therapeutic levels of aflibercept expression were found in the aqueous humor of the eye at 12 weeks after injection.

CGTLive spoke with Khanani to learn more about the PRISM study. He discussed the preliminary safety and efficacy data observed and noted that the dose-expansion phase of the trial should initiate in early 2023.

4D Molecular Therapeutics announces interim clinical data from on-going phase 1/2 clinical trial of intravitreal 4D-150 for wet age-related macular degeneration (wet AMD). News release. 4D Molecular Therapeutics. November 14, 2022.
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