Biotech
Complement Therapeutics’ gene therapy gains FDA fast-track for vision loss treatment
CTx001 targets Geographic Atrophy (GA) secondary to Age-related Macular Degeneration
- By IPP Bureau
| January 12, 2026
Complement Therapeutics GmbH (CTx), a clinical-stage biotech pioneering treatments for complement-mediated diseases, announced that the US FDA has granted Fast Track Designation for its lead gene therapy candidate for vision loss treatment.
CTx001 targets Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). The therapy’s Investigational New Drug (IND) application had previously been cleared, clearing the path for clinical development.
In fact l, CTx001 is an adeno-associated virus (AAV)-based gene therapy engineered to deliver a truncated version of Complement Receptor 1 (mini-CR1), offering long-term modulation of key pathways in the complement cascade. GA, an advanced form of dry AMD, leads to irreversible vision loss and currently has limited treatment 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 enter the Opti-GAIN (Optimised Geographic Atrophy Interventional) Phase I/II trial, the first human study in GA patients. First patient dosing in the US is expected in Q1 of this year.
