Complement Therapeutics GmbH (CTx) has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for 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 for CTx001 was previously cleared to proceed by the FDA, enabling advancement of the programme into clinical development.
CTx001 is a novel adeno-associated virus (AAV)-based gene therapy designed to deliver a truncated version of complement receptor 1 (mini-CR1), with 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.
“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 Rafiq Hasan, CEO 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 (optimised geographic atrophy interventional) first-in-human phase I/II clinical trial in patients with geographic atrophy secondary to AMD, with first patient dosing expected in the US in Q1 2026.
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