The US Food and Drug Administration (FDA) has granted Fast Track Designation for a treatment for  geographic atrophy (GA) secondary to age-related macular degeneration (AMD). 

CTx001, developed by biotechnology company Complement Therapeutics, is a novel adeno-associated virus (AAV)-based gene therapy designed to deliver a truncated version of Complement Receptor 1 (mini-CR1). The fast-tracked therapy has 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. 

The FDA approval follows the previous decision to clear the Investigational New Drug (IND) application for CTx001. 

“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 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. 

Bruno Quinney, Content Team, DDW