Drug intelligence / Profile preview

GT011

Development stage
Unknown
Lead developer
Novartis
Modality
Gene Therapies
Administration
Subretinal
01

Overview

GT011 is a subretinal adeno-associated virus (AAV) gene therapy candidate being developed by Gyroscope Therapeutics (a Novartis company) for the treatment of geographic atrophy secondary to dry age-related macular degeneration, with the goal of providing durable, one-time treatment by modifying complement activity in the retina.[1][5][15] It is administered via subretinal delivery (using either a transvitreal or suprachoroidal cannulation approach) and is being evaluated in the FOCUS (phase 1/2), EXPLORE (phase 2), and HORIZON (phase 2) clinical trials, which collectively assess safety, dose escalation, and effects on geographic atrophy lesion growth.[1][5] Public clinical-trial summaries and reviews consistently describe GT011 as a subretinal gene therapy for geographic atrophy but do not yet disclose its precise transgene or molecular target, and it remains an investigational product without regulatory approval.[1][5][15]

02

Targets

FHL1 (Four and a half LIM domains protein 1)

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