Complement Therapeutics Secures FDA IND Clearance for CTx001 in Phase I/II Opti-GAIN Trial for Geographic Atrophy
Thursday, October 09, 2025
Complement Therapeutics GmbH (CTx), a biotechnology company focused on complement-mediated diseases, has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for its lead gene therapy candidate, CTx001. This clearance allows the company to initiate the Opti-GAIN Phase I/II clinical trial in patients with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD), a condition affecting around 5 million people globally, including 1.5 million in the United States.
CTx001 is an adeno-associated virus (AAV)-based gene therapy that delivers a truncated form of Complement Receptor 1 (mini-CR1) to provide long-term modulation of both the classical and alternative complement pathways. GA represents an advanced stage of dry AMD that causes irreversible vision loss, with few effective treatment options currently available.
The Opti-GAIN (Optimised Geographic Atrophy INterventional) trial is an international, open-label, first-in-human Phase I/II study designed to assess the safety, tolerability, and preliminary efficacy of CTx001. Patients will be enrolled across leading retinal centres, with first dosing in the United States expected in the first quarter of 2026.
Trial design has been guided by findings from i-GAIN, a natural history study involving over 230 participants, providing key insights into disease progression, imaging biomarkers, and patient stratification in GA.
FDA IND clearance marks a significant step for Complement Therapeutics, advancing the company’s mission to develop innovative therapies that could provide durable, one-time treatment benefits for patients with this debilitating eye condition.
Source: prnewswire.com
