Complement Therapeutics Receives UK CTA Approval to Advance CTx001 into Opti-GAIN Phase I/II Clinical Trial in Geographic Atrophy Secondary to AMD
- CTx001 is an investigational AAV-based gene therapy designed to modulate multiple pathways within the complement system
- UK Clinical Trial Authorisation (CTA) approval enables initiation of the Phase I/II Opti-GAIN clinical trial in the United Kingdom
- The approval follows recent FDA IND clearance and Fast Track Designation for CTx001, supporting a multinational first-in-human clinical development programme
Munich, Germany – 29th Jan 2026 – Complement Therapeutics GmbH (CTx), a clinical-stage biotechnology company developing next-generation therapeutics for complement-mediated diseases, today announced that it has received Clinical Trial Authorisation (CTA) approval in the United Kingdom for CTx001, the company’s investigational gene therapy programme for Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD).
The CTA application for CTx001 has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA). This approval builds on Complement Therapeutics’ recent U.S. Food and Drug Administration (FDA) Investigational New Drug (IND) clearance, for which the programme also holds Fast Track Designation in the United States. Together, these regulatory milestones enable acceleration of the company’s clinical development programme across sites in both the US and UK.
“UK CTA approval enables us to initiate an innovative first-in-human clinical development programme for CTx001 in Geographic Atrophy,” said Dr. Rafiq Hasan, Chief Executive Officer of Complement Therapeutics. “The Opti-GAIN study has been carefully designed to leverage patients’ natural history data and incorporates novel imaging and functional endpoints to better characterise disease progression and treatment response. Together with our recent FDA IND clearance and Fast Track Designation, this positions us to evaluate CTx001 through a rigorous, multinational clinical programme across the US and UK.”
CTx001 is an investigational adeno-associated virus (AAV)-based gene therapy designed to deliver a truncated version of Complement Receptor 1 (mini-CR1), enabling long-term modulation of the classical and alternative pathways of the complement cascade. Given its small size, mini-CR1 has the potential to penetrate different compartments of the eye and thereby address the inflammation associated with GA in a more robust manner. Dysregulation of the complement system is recognised as a key driver of disease progression in GA, an advanced form of dry AMD that leads to progressive and irreversible vision loss and for which treatment options remain limited.
The Opti-GAIN (Optimised Geographic Atrophy INterventional) trial is an international, first-in-human, open-label interventional Phase I/II study that leverages data from an extensive natural history programme. The clinical design of Opti-GAIN enables a detailed assessment of disease progression and treatment response using innovative endpoints, including evaluation of ellipsoid zone (EZ) attenuation and focal microperimetry. The study is therefore well placed to evaluate the safety, tolerability and preliminary efficacy of CTx001 in patients with GA secondary to AMD. The trial will enrol patients across leading retinal centres with first patient dosing expected in Q1 2026.
About Complement Therapeutics GmbH:
Complement Therapeutics GmbH (CTx) is a German headquartered clinical-stage biotechnology company focused on the research and development of novel therapeutics for complement-mediated diseases. The Company is a spinout from the University of Manchester and is based on the pioneering research of its founders into novel targets within the complement cascade.
Our lead investigational product (CTx001) is being evaluated as a potential gene therapy for GA, a leading cause of blindness. Additional programs will evaluate potential therapeutic opportunities in other complement-mediated conditions.
The Company has subsidiaries in the UK (Complement Therapeutics Ltd) and in the USA (Complement Therapeutics Inc) as well as research laboratories in Stevenage, UK.
About Geographic Atrophy
Geographic Atrophy (GA) is a leading cause of blindness in the elderly and represents the advanced stage of dry age-related macular degeneration. It is characterised by the progressive degeneration of photoreceptors, retinal pigment epithelium, and choriocapillaris, resulting in irreversible vision loss. GA affects over 5 million people globally and remains a significant unmet clinical need.
–Ends–
For more information please visit: https://complementtx.com/
Company Contact
Dr Rafiq Hasan, CEO and Managing Director
Complement Therapeutics GmbH
Email: info@complementtx.com