FDA grants Fast Track Designation to Complement Therapeutics’ CTx001 for geographic atrophy

The US Food and Drug Administration (FDA) has granted Fast Track Designation to Complement Therapeutics’ 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 from Complement Therapeutics for CTx001 was previously approved by the FDA in October 2025, which allowed the company to initiate the Opti-GAIN (Optimized Geographic Atrophy INterventional) phase 1/2 clinical trial.

Opti-GAIN is an international first-in-human, open-label, phase 1/2 study designed to evaluate the safety, tolerability, and preliminary efficacy of CTx001. First dosing is expected to begin in the US during Q1 of 2026.

Fast Track designation from the FDA is intended to facilitate the development and expedite 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.¹

Rafiq Hasan, MD, CEO of Complement Therapeutics, commented on the decision by the FDA in a press release from the company, saying, "FDA Fast Track Designation for CTx001 is an important regulatory milestone recognizing 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. 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."

The company defines CTx001 as an adeno-associated virus (AAV)-based gene therapy designed to deliver a truncated version of Complement Receptor 1 (mini-CR1). According to the company, this enables long-term modulation of the “classical and alternative pathways of the complement cascade.” The company describes the complement cascade as “part of the immune system that enhances (complements) the ability of antibodies and phagocytic cells to clear microbes and damaged cells from an organism, promote inflammation, and attack the pathogen’s cell membrane.”^1,2

Furthermore, the company notes that dysregulation of the complement system is implicated in the development of several diseases, including AMD, kidney disease, and various hematological conditions. The company also noted that research conducted by the founders of the company identified the pivotal role of factor H-related proteins in the development of AMD, providing the foundation for the therapeutic strategy being pursued by the company with CTx001.^1,2

References:

1. Complement Therapeutics Announces FDA Fast Track Designation for CTx001 for the Treatment of Geographic Atrophy Secondary to AMD. Published January 8, 2026. Accessed January 12, 2026. https://www.prnewswire.com/news-releases/complement-therapeutics-announces-fda-fast-track-designation-for-ctx001-for-the-treatment-of-geographic-atrophy-secondary-to-amd-302655523.html

2. Harp MD. FDA clears IND for Complement Therapeutics’ CTx001 gene therapy in geographic atrophy. Published October 8, 2025. Accessed January 12, 2026. https://www.ophthalmologytimes.com/view/fda-clears-ind-for-complement-therapeutics-ctx001-gene-therapy-in-geographic-atrophy