Major GAIN for Complement Therapeutics with FDA IND clearance
CTx, which is developing nextgen therapeutics for complement-mediated diseases, reveals that the FDA has cleared its Investigational New Drug application for CTx001, the company’s lead gene therapy candidate.
The IND clearance enables initiation of the Opti-GAIN Phase I/II clinical trial in patients with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD) - a condition that has a global prevalence of five million, affecting 1.5 million people in the US alone.
CTx001 is an 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. GA is an advanced form of dry AMD that leads to irreversible vision loss, with limited therapeutic options currently available.
“FDA clearance of the IND for CTx001 is a major milestone for Complement Therapeutics and our mission to transform the treatment landscape for GA,” said Dr. Rafiq Hasan, Chief Executive Officer of Complement Therapeutics.
“It is a testament to the dedication, talent, and vision of our team that we’ve progressed from a university spinout to a clinical-stage company in just four years. With Opti-GAIN, we are entering the clinic with a highly innovative gene therapy candidate that has the potential to deliver durable, one-time treatment benefits for patients affected by this devastating disease.”
The Opti-GAIN (Optimised Geographic Atrophy INterventional) trial is an international first-in-human, open-label, Phase I/II study designed to evaluate the safety, tolerability, and preliminary efficacy of CTx001. The trial will enrol 75 patients across leading retinal centres with first patient dosing expected in the US in Q1 2026.
The trial design is informed by data from i-GAIN, a natural history study involving over 230 participants, which has provided valuable insights into disease progression, imaging biomarkers, and patient stratification in GA.
Complement Therapeutics is a University of Manchester Innovation Factory spinout, headquartered in Germany. The company focuses on researching and developing novel therapeutics for complement-mediated diseases, building on the pioneering work of its founders into new targets within the complement cascade.
Complement Therapeutics’ UK research activities include research labs at Stevenage Bioscience Catalyst, a leading cluster for cell and gene therapy and early-stage therapeutics. As part of SBC’s collaborative ecosystem, the company benefits from proximity to GSK, access to specialist lab infrastructure and tailored support designed to accelerate the translation of scientific innovation into patient impact.
The company has subsidiaries in the UK (Complement Therapeutics Ltd) and in the US (Complement Therapeutics Inc) in addition to its Stevenage facilities.