Drug intelligence / Profile preview

IG-002

Development stage
Preclinical
Lead developer
Intergalactic Therapeutics
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Subretinal
01

Overview

IG-002 is an investigational non-viral gene therapy being developed by Intergalactic Therapeutics to treat ABCA4-related retinopathies, including Stargardt disease, cone-rod dystrophy, and retinitis pigmentosa, which are caused by mutations in the large ABCA4 gene and currently lack approved treatments.[1][5][13][8] The therapy uses Intergalactic’s proprietary C3DNA platform—covalently closed circular DNA vectors encoding full-length human ABCA4—delivered locally to the retina via COMET, an electro-transfer-based, non-viral delivery system designed to overcome the cargo size and safety limitations of AAV gene therapy and potentially allow redosing.[5][13][8] In preclinical porcine and non-human primate studies, a single subretinal administration of IG-002 achieved durable ABCA4 expression in photoreceptors for at least 6–12 months with a favorable safety profile, supporting ongoing IND-enabling work and plans for first-in-human clinical trials.[5][13][8]

02

Targets

ABCA4

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