Complement Therapeutics, a University of Manchester spinout, has received UK Clinical Trial Authorisation (CTA) to begin a Phase I/II clinical trial of its lead gene therapy programme, CTx001, for Geographic Atrophy secondary to age-related macular degeneration (AMD).
The approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) enables the launch of the Opti-GAIN clinical trial in the UK and follows recent FDA IND clearance and Fast Track Designation in the United States, marking a major milestone in the company’s multinational clinical development programme.
CTx001 is an investigational AAV-based gene therapy designed to modulate multiple pathways of the complement system — a key driver of disease progression in Geographic Atrophy, a leading cause of irreversible vision loss for which treatment options remain limited.
The Opti-GAIN study is a first-in-human, open-label Phase I/II trial and will enrol patients at leading retinal centres in the UK and US, with first patient dosing expected in Q1 2026.
Complement Therapeutics was founded on pioneering research from the University of Manchester and is developing next-generation therapies for complement-mediated diseases.
