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IG-SCD 1

Development stage
Preclinical
Lead developer
Incisive Genetics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

IG-SCD 1 is a preclinical gene-editing therapy developed by Incisive Capital for the treatment of Sickle Cell Disease and Thalassemia. The drug utilizes a non-viral delivery system consisting of lipid nanoparticles (LNPs) to transport CRISPR ribonucleoprotein (RNP) complexes. The therapeutic mechanism involves the targeted activation of fetal hemoglobin (HbF) expression, which serves to compensate for the defective or absent adult hemoglobin characteristic of these hematological disorders. By employing a non-viral, LNP-based approach for the delivery of CRISPR components, IG-SCD 1 aims to provide a safer and more efficient alternative to traditional viral vector-mediated gene editing strategies.

02

Targets

Hb (Hemoglobin)BCL11A (B-cell CLL/lymphoma 11A)

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