SKY-0515 is Skyhawk's investigational small molecule RNA splicing modifier developed through the company's novel RNA-splicing ...

LETI-101 has demonstrated therapeutically relevant reductions of mutant huntingtin (HTT) protein, exceeding 80%, while preserving essential wild-type HTT in a clinically relevant Huntington’s disease ...

LETI-101 has demonstrated therapeutically relevant reductions of mutant huntingtin (HTT) protein, exceeding 80%, while preserving essential wild-type HTT in a clinically relevant Huntington’s disease ...

LETI-101 combines Life Edit's proprietary CRISPR nuclease with AAV5 delivery to selectively target the T allele of an exonic SNP in the HTT gene to treat HD. In preclinical studies, LETI-101 has ...

SKY-0515 is designed to reduce both HTT protein and PMS1 protein, an additional key driver of somatic CAG repeat expansion and HD pathology. "We are excited to present on our Huntingtin-lowering ...

SKY-0515 is Skyhawk’s investigational small molecule RNA splicing modifier developed through the company’s novel RNA-splicing platform. SKY-0515 is designed to reduce both HTT protein and PMS1 protein ...

SKY-0515 is Skyhawk’s investigational small molecule RNA splicing modifier developed through the company’s novel RNA-splicing platform. SKY-0515 is designed to reduce both HTT protein and PMS1 protein ...