IG-HD02

IG-HD02 is an experimental, preclinical gene editing therapy developed by Incisive Genetics for the treatment of Huntington's Disease. The therapy employs a non-viral delivery system consisting of CRISPR ribonucleoprotein (RNP) complexes encapsulated within lipid nanoparticles (LNPs). IG-HD02 is designed for allele-specific editing, specifically targeting a single nucleotide polymorphism (SNP 2) within the huntingtin (HTT) gene. This approach allows for the selective modification or silencing of the mutant HTT allele while preserving the expression of the wild-type allele, thereby addressing the toxic gain-of-function associated with the CAG repeat expansion that causes the neurodegenerative disorder.