FDA Clears Complement Therapeutics’ IND Application for CTx001 Gene Therapy in GA

Complement Therapeutics has announced that the FDA has cleared its investigational new drug (IND) application for CTx001, the company’s lead gene therapy candidate. The clearance paves the way for the initiation of Opti-GAIN, a phase 1/2 clinical trial in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

CTx001 is an adeno-associated virus (AAV)-based gene therapy engineered to deliver a truncated form of Complement Receptor 1 (mini-CR1). The therapy is designed to provide long-term modulation of both the classical and alternative complement pathways, addressing the overactivation of the complement system implicated in GA pathogenesis.

The Opti-GAIN (Optimised Geographic Atrophy INterventional) study is an international, first-in-human, open-label phase 1/2 trial aimed at assessing the safety, tolerability, and preliminary efficacy of CTx001. The trial will recruit participants across leading retinal research centers, with first patient dosing anticipated in the US in Q1 2026.

Trial design and patient stratification are informed by insights from the i-GAIN natural history study, which enrolled over 230 participants. The study provided critical data on disease progression, imaging biomarkers, and complement-driven pathways relevant to GA.

“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, Chief Executive Officer 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.”

For more information, visit complementtx.com