US RNA specialist Wave Life Sciences (Nasdaq: WVE) has presented positive results from its Phase Ib/IIa SELECT-HD trial of WVE-003, which is being developed as a potential disease modifying therapeutic for Huntington’s disease (HD).
WVE-003 is a first-in-class, allele-selective antisense oligonucleotide (ASO) designed to lower mutant huntingtin (mHTT) protein and preserve healthy, wild-type huntingtin (wtHTT) protein.
In the multidose portion of the SELECT-HD study, participants received either every-eight-week intrathecal doses of 30 mg WVE-003 or placebo, with 12 weeks of follow up.
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