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Complement Therapeutics Receives FDA Clearance for CTx001 Gene Therapy IND to Treat Geographic Atrophy

08 Oct 2025
Admin
News Article

Complement Therapeutics Receives FDA Clearance for CTx001 Gene Therapy IND to Treat Geographic Atrophy

Complement Therapeutics GmbH (CTx), a biotechnology company developing next-generation therapies for complement-mediated diseases, 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. This clearance enables the initiation of the Opti-GAIN Phase I/II trial in patients with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD).

GA is an advanced form of dry AMD, affecting approximately 5 million people globally, including 1.5 million in the U.S., and leads to irreversible vision loss. Current treatment options for GA are extremely limited.

About CTx001

CTx001 is an AAV-based gene therapy designed to deliver a truncated version of Complement Receptor 1 (mini-CR1).
The therapy modulates both classical and alternative pathways of the complement system, aiming to address multiple mechanisms driving GA progression.
Intended as a one-time treatment, CTx001 seeks to provide durable benefits to patients with this degenerative eye disease.

About the Opti-GAIN Trial

Opti-GAIN (Optimised Geographic Atrophy INterventional) is an international, first-in-human, open-label Phase I/II study.
The trial will evaluate safety, tolerability, and preliminary efficacy of CTx001.
Patients will be enrolled across leading retinal centers, with first dosing expected in the U.S. in Q1 2026.
The study design builds on insights from i-GAIN, a natural history study of over 230 participants, which provided key data on disease progression, imaging biomarkers, and patient stratification.

Leadership Comment

Dr. Rafiq Hasan, CEO of Complement Therapeutics:

FDA clearance of the IND for CTx001 is a major milestone for our mission to transform GA treatment. In just four years, we’ve progressed from a university spinout to a clinical-stage company. With Opti-GAIN, we are advancing a highly innovative gene therapy with the potential for durable, one-time benefits for patients suffering from this devastating condition.