Investigational gene therapy well tolerated in patients with AMD
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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 the majority of patients. Decreases in downstream complement proteins associated with overactivation of the complement system were also observed.
“Our investigational gene therapy, GT005, is designed to restore balance to an overactive complement system and reduce inflammation by increasing production of the CFI protein. We are excited by these early results from the FOCUS trial that showed GT005 has been well tolerated to date, increased CFI levels in a durable manner and caused downregulation of an overactive complement system,” Nadia Waheed, MD, MPH, chief medical officer of Gyroscope Therapeutics, said in the release.
The clinical trial is evaluating the safety and dose response of three doses of GT005 given as a single subretinal injection. Cohorts 1, 2, 3, 5 and 6 are undergoing a dose escalation schedule, while cohorts 4 and 7 are undergoing a dose expansion schedule.
The interim results of cohorts 1 to 4 showed three doses of GT005 were well tolerated, with no signs of GT005-induced inflammation. No serious adverse events related to GT005 were recorded.
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