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IG-HD01 is an allele-specific gene editing therapeutic being developed by Incisive Genetics for the treatment of Huntington's Disease. The drug utilizes a CRISPR-Cas9 ribonucleoprotein (RNP) complex delivered via lipid nanoparticles (LNPs), a non-viral approach designed to overcome the limitations of viral vectors. IG-HD01 is specifically engineered to target a single nucleotide polymorphism (SNP 1) associated with the mutant huntingtin (HTT) allele. By selectively inactivating the mutant allele while sparing the wild-type HTT gene, the therapy aims to reduce the production of the neurotoxic huntingtin protein that drives disease progression. As of early 2024, IG-HD01 is in the IND-enabling stage of development.
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