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IG-SCD 2
- Development stage
- Preclinical
- Lead developer
- Incisive Capital
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
IG-SCD 2 is a preclinical gene-editing therapy candidate developed by Incisive Capital (Incisive Genetics) for the treatment of Sickle Cell Disease and Thalassemia. The therapy utilizes a CRISPR ribonucleoprotein (RNP) complex delivered via a non-viral lipid nanoparticle (LNP) system. The mechanism of action involves the activation of fetal hemoglobin (HbF) expression to compensate for the defective or deficient adult hemoglobin characteristic of these hemoglobinopathies. By employing a non-viral delivery platform, IG-SCD 2 aims to provide a durable, potentially curative treatment while avoiding the safety and manufacturing complexities associated with viral vectors.
02
Targets
HbF (Fetal hemoglobin)
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