A majority of patients with age-related macular degeneration treated with investigational gene therapy GT005 experienced increases in complement factor I levels in an ongoing phase 1/2 clinical trial.
GT005 is an AAV2-based one-time investigational gene therapy for geographic atrophy secondary to AMD designed to restore balance to an overactive complement system, according to a press release from Gyroscope Therapeutics.
The interim results of the open-label FOCUS clinical trial showed GT005 resulted in sustained increases in vitreous complement factor I (CFI) levels and was well tolerated in
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