Complement Therapeutics Announces FDA Fast Track Designation for CTx001 for the Treatment of Geographic Atrophy Secondary to AMD
- FDA Fast Track designation has been granted to CTx001 for Geographic Atrophy secondary to Age-related Macular Degeneration
- CTx001 is an investigational AAV-based gene therapy designed to modulate multiple pathways of the complement system
- CTx001 has previously received FDA IND clearance and will be evaluated in Opti-GAIN, a first-in-human Phase I/II clinical trial
Munich, Germany – 8th Jan 2026 – Complement Therapeutics GmbH (CTx), a clinical-stage biotechnology company developing next-generation therapeutics for complement-mediated diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTx001, the company’s lead gene therapy candidate for the treatment of Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). The Investigational New Drug (IND) application for CTx001 was previously cleared to proceed by the FDA, enabling advancement of the programme into clinical development.
Fast Track designation is intended to facilitate the development and expedite the review of drugs that treat serious conditions and have the potential to address unmet medical needs. It is designed to support more frequent interactions with the FDA and bring important new therapies to patients sooner.
CTx001 is a novel adeno-associated virus (AAV)-based gene therapy designed to deliver a truncated version of Complement Receptor 1 (mini-CR1), with the potential for long-term potent modulation of the classical and alternative pathways of the complement cascade. GA is an advanced form of dry AMD that leads to irreversible vision loss, with limited therapeutic options.
“FDA Fast Track Designation for CTx001 is an important regulatory milestone recognising the seriousness of Geographic Atrophy, the unmet medical need faced by patients as well as the innovative potential for CTx001 to meaningfully address this need” said Dr. Rafiq Hasan, Chief Executive Officer of Complement Therapeutics. “Together with the recent IND clearance, this designation supports our efforts to efficiently advance CTx001 into clinical development as we work to address a leading cause of irreversible vision loss.”
CTx001 will be evaluated in the Opti-GAIN (Optimised Geographic Atrophy Interventional) first-in-human Phase I/II clinical trial in patients with Geographic Atrophy secondary to AMD, with first patient dosing expected in the United States 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.
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For more information please visit: https://complementtx.com/
Company Contact
Dr Rafiq Hasan, CEO and Managing Director
Complement Therapeutics GmbH Email: info@complementtx.com