Key takeaways:

• CTx001 gene therapy for geographic atrophy was well tolerated.
• Treated eyes exhibited slowed lesion progression and stabilized microperimetry.

LAS VEGAS — Customized microperimetry helped researchers track geographic atrophy in patients undergoing CTx001 gene therapy, according to a presentation at Clinical Trials at the Summit.

Arshad Khanani, MD, MA, FASRS, of Sierra Eye Associates in Reno, Nevada, presented initial findings from the first cohort of patients enrolled in the phase 1/2 Opti-GAIN study. The primary endpoints are safety and tolerability of CTx001 (Complement Therapeutics) as well as change in ellipsoid zone attenuation at 52 weeks vs. baseline, according to the presentation.

In this video, Arshad Khanani, MD, MA, FASRS, discusses CTx001, a gene therapy for the treatment of geographic atrophy.

Khanani said the study implemented one of the first prospective, ellipsoid zone-based perilesional microperimetry.

Even with short follow-ups of about 1 month after a low dose of CTx001, imaging for one patient demonstrated slowed lesion progression in the study eye and lesion growth in the fellow eye. Eyes in the first cohort also exhibited stabilization of microperimetry, Khanani said.

“This trial is powered to show a change in ellipsoid zone, and it involves a 6-month run-in followed by looking prospectively at the connection between structure and function using ellipsoid zone image-guided microperimetry,” Khanani told Healio. “To date, CTx001 has been well tolerated in the low dose of 5E10, and other doses are currently being evaluated.”