Spinout, Complement Therapeutics, Advances Gene Therapy into US Clinical Trial

Complement Therapeutics GmbH (CTx), a University of Manchester Innovation Factory spinout today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CTx001, the company’s lead gene therapy candidate.

Complement Therapeutics is a biotechnology company developing next-generation therapeutics for complement-mediated diseases.

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 5 million, affecting 1.5 million people in the US alone.

Wes Streeting MP, and Peter Kyle, Shadow Science, Innovation and Technology Minister, at Complement Therapeutics cutting-edge labs as The Labour Party launched its Life Sciences Industry Strategy last year

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.”

Complement Therapeutics Team

Complement Therapeutics is one of a growing portfolio of spinout companies created through the University of Manchester Innovation Factory, which specialises in translating world-class academic research into commercial ventures. The company’s journey from laboratory discovery to clinical-stage biotech exemplifies the Innovation Factory’s mission to support founders, attract investment, and deliver lasting health, economic and societal impact.

Read more: Complement Therapeutics Secures £72m in Series A Finance