The US Food and Drug Administration (FDA) has granted Fast Track designation to CTx001, an investigational gene therapy being developed by Complement Therapeutics GmbH for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The designation follows prior FDA clearance of the company’s Investigational New Drug application, allowing the program to proceed into clinical development.

Fast Track status is intended to facilitate the development and review of therapies for serious conditions with unmet medical needs and allows for more frequent interaction with the FDA.

CTx001 is an adeno-associated virus–based gene therapy designed to deliver a truncated form of complement receptor 1, known as mini-CR1. The approach aims to provide sustained modulation of both the classical and alternative complement pathways, which are implicated in the pathophysiology of GA. The therapy will be evaluated in Opti-GAIN, a first-in-human phase 1/2 clinical trial enrolling patients with GA secondary to AMD. Opti-GAIN will enroll patients at multiple centers in the United States and United Kingdom, with first dosing expected early this year. RP