Complement Therapeutics Receives FDA Clearance for Gene Therapy Trial in Geographic Atrophy

Oct 08 , 2025
share:

MUNICHOct. 8, 2025 /PRNewswire/ — Complement Therapeutics GmbH (CTx), a biotechnology company focused on complement-mediated diseases, announced today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CTx001, its lead gene therapy candidate.

The IND clearance permits the initiation of the Opti-GAIN Phase I/II clinical trial in patients with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). GA is an advanced form of dry AMD that causes irreversible vision loss and affects an estimated 1.5 million people in the U.S., contributing to a global prevalence of 5 million cases.

AAV Therapy Targets Complement Cascade

CTx001 is an adeno-associated virus (AAV)-based gene therapy designed to deliver a truncated version of Complement Receptor 1 (mini-CR1). The therapy’s goal is to provide long-term modulation of both the classical and alternative pathways of the complement cascade, which is implicated in the progression of GA. Current therapeutic options for the condition are limited.

Dr. Rafiq Hasan, CEO of Complement Therapeutics, highlighted the importance of the clearance: “FDA clearance of the IND for CTx001 is a major milestone for Complement Therapeutics and our mission to transform the treatment landscape for GA.” He added that the highly innovative gene therapy has the potential to deliver “durable, one-time treatment benefits” for patients.

Opti-GAIN Trial Details

The Opti-GAIN (Optimised Geographic Atrophy Interventional) study is an international, first-in-human, open-label Phase I/II trial. Its primary objective is to evaluate the safety, tolerability, and preliminary efficacy of CTx001.

The trial will enroll patients across leading retinal centers, with the first patient dosing expected in the U.S. in Q1 2026. The design of the trial was informed by data from i-GAIN, a natural history study that involved over 230 participants and provided insights into disease progression and patient stratification in GA.

Source:

https://www.prnewswire.com/news-releases/complement-therapeutics-receives-fda-ind-clearance-to-advance-ctx001-into-opti-gain-a-phase-iii-clinical-trial-in-geographic-atrophy-secondary-to-amd-302577045.html

About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Download

Login

Don't have an account? Please register
Account*
Password*
Code*
Refresh
Forgot password?
Logging in indicates that you have read and accepted the Registration Agreement and User Agreement
Log in with other accounts

New User Registration

Already have an account?
First Name*
Middle Name
Last Name*
Organization*
Organization Type*
Country/State*
Email Address*
Set Password*
Confirm password*
Refferal Code*

Reset Password

Return to
Email*
Code*
New password*
Confirm password*

Google Account Binding

Organization*
Organization Type*
Country/State*