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IG-AT01
- Development stage
- Preclinical
- Lead developer
- Incisive Capital
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
01
Overview
IG-AT01 is an investigational gene-editing therapy being developed by Incisive Capital for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The therapeutic approach utilizes CRISPR-Cas9 ribonucleoproteins (RNPs) delivered via a non-viral lipid nanoparticle (LNP) platform. By delivering the CRISPR machinery as an RNP complex rather than as DNA or mRNA, the therapy aims to achieve transient editing activity, which may reduce the risk of off-target effects and genomic integration associated with viral vectors. The program is designed to address the underlying genetic cause of AATD, typically involving mutations in the SERPINA1 gene, and is currently in the preclinical stage of development.
02
Targets
SERPINA1 (Alpha-1-antitrypsin)
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