Complement Therapeutics Receives FDA Clearance to Initiate Clinical Trial of CTx001 for Geographic Atrophy
Complement Therapeutics GmbH has announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CTx001, the company’s lead gene therapy candidate. The clearance allows for the initiation of Opti-GAIN, a Phase I/II clinical trial evaluating CTx001 in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
About CTx001 and Its Therapeutic Target
CTx001 is an adeno-associated virus (AAV)-based gene therapy designed to deliver a truncated form of Complement Receptor 1 (mini-CR1). This approach is intended to provide long-term modulation of both the classical and alternative complement pathways. GA, an advanced form of dry AMD, leads to progressive and irreversible vision loss. Current treatment options remain limited, highlighting the need for durable therapies.
CEO Perspective
“FDA clearance of the IND for CTx001 is a major milestone for Complement Therapeutics and our mission to transform the treatment landscape for GA,” said Dr. Rafiq Hasan, CEO of Complement Therapeutics. “It is a testament to the dedication, talent, and vision of our team that we’ve progressed from a university spinout to a clinical-stage company in just four years. With Opti-GAIN, we are entering the clinic with a highly innovative gene therapy candidate that has the potential to deliver durable, one-time treatment benefits for patients affected by this devastating disease.”
About the Opti-GAIN Trial
The Opti-GAIN (Optimised Geographic Atrophy INterventional) study is an international, first-in-human, open-label Phase I/II clinical trial. It is designed to assess the safety, tolerability, and preliminary efficacy of CTx001. The trial is expected to enroll participants at leading retinal centres, with first patient dosing anticipated in the U.S. in Q1 2026.
The trial design has been guided by insights from i-GAIN, a natural history study involving over 230 participants. This earlier study provided foundational data on disease progression, imaging biomarkers, and patient stratification, informing the design and endpoints of the upcoming clinical trial.
