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Hap10 is a biotechnology company dedicated to developing precision medicines for Huntington’s Disease (HD) through allele-specific silencing. The company’s platform focuses on targeting Single Nucleotide Polymorphisms (SNPs) that are uniquely associated with the mutant Huntingtin (mHTT) gene, allowing for the selective degradation of the toxic protein while sparing the healthy, wild-type Huntingtin protein. This approach addresses a critical challenge in HD research: the need to maintain the neuroprotective functions of normal Huntingtin. Founded by Dr. Michael Hayden, a pioneer in HD research, Hap10 is advancing a pipeline of antisense oligonucleotides (ASOs) designed to cover the majority of the HD patient population. The company aims to provide a safer and more effective therapeutic option compared to non-selective Huntingtin-lowering strategies.
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