IG-HD03

IG-HD03 is a preclinical gene editing therapy being developed by Incisive Genetics (also known as Incisive Capital) for the treatment of Huntington's Disease. The therapy utilizes CRISPR ribonucleoprotein (RNP) complexes delivered via lipid nanoparticles (LNPs) to perform allele-specific, non-viral gene editing. IG-HD03 is specifically designed to target a single nucleotide polymorphism (SNP 3) in the huntingtin (HTT) gene, allowing for the selective silencing or disruption of the mutant HTT allele while preserving the expression of the normal (wild-type) allele. This approach aims to provide a disease-modifying treatment for Huntington's Disease, an autosomal dominant neurodegenerative disorder caused by a CAG repeat expansion in the HTT gene. By reducing the levels of toxic mutant huntingtin protein, the therapy seeks to halt or slow disease progression.

Other names

IG-HD03-Incisive Capital-HTT (SNP 3)-CRISPR RNP-non-viral gene editing-LNP delivery-gene editing-Huntington's Disease