January 09, 2026
1 min read
Key takeaways:
- CTx001 is an investigational adeno-associated virus-based therapy for geographic atrophy.
- Patient dosing in the Opti-GAIN phase 1/2 clinical trial is expected to begin in the first quarter.
The FDA granted fast track designation for CTx001, an investigational gene therapy candidate for geographic atrophy secondary to age-related macular degeneration.
According to a press release from Complement Therapeutics, CTx001, an adeno-associated virus-based therapy, is designed to treat GA by delivering a truncated version of complement receptor 1, with the aim of yielding “long-term potent modulation of the classical and alternative pathways of the complement cascade.”
CTx001 is an investigational adeno-associated virus-based therapy for GA.
The FDA grants fast track designation to expedite the development and review of therapies that treat serious conditions and address unmet medical needs. The FDA previously cleared the investigational new drug application for CTx001, which enabled the advancement of the therapy into clinical development.
Patient dosing in the Opti-GAIN phase 1/2 clinical trial investigating CTx001 is expected to begin in the first quarter, according to the release.
“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,” Rafiq Hasan, MBBS, CEO of Complement Therapeutics, said in the release. “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.”
Perspective
The FDA fast track designation for CTx001 represents a meaningful and encouraging development in the evolving treatment landscape for geographic atrophy secondary to age-related macular degeneration.
GA remains a major cause of irreversible vision loss, and while recent therapies have demonstrated the ability to slow progression, the unmet need for durable, disease-modifying approaches remains substantial. Complement Therapeutics’ approach is meaningful in that it solves a major challenge, which is the frequency of dosing patients with current GA therapeutics.
The combination of prior investigational new drug clearance and fast track designation from the FDA underscores the seriousness of GA and the promise of this investigational therapy. Results from the upcoming Opti-GAIN phase 1/2 trial will be closely watched by the retina community as gene-based strategies move closer to clinical reality.
Rishi P. Singh, MD
Disclosures: Singh reports consulting for Apellis and Astellas.
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