An investigational allele-selective antisense oligonucleotide that targets a single-nucleotide polymorphism (SNP3) present on the mutant HTT allele in roughly half of HD patients, allowing it to selectively lower mutant huntingtin while sparing normal huntingtin protein — a potential advantage over non-selective approaches like tominersen, since wild-type huntingtin has important normal cellular functions.
Dosing Notes
Investigational; intrathecal injection, under evaluation in the SELECT-HD trial.
Treatment Type
pharmaceutical
Clinical Evidence
What the Evidence Shows
Each finding below is linked to its primary source. Confidence levels reflect the quality and quantity of available evidence — not CelluTarget's endorsement of any treatment.
High: Supported by multiple robust studies or regulatory approval
Moderate: Supported by limited controlled studies or consistent case series
Low: Based on case reports, expert opinion, or early-phase data only
Low ConfidenceBased on case reports, expert opinion, or early-phase data only
WVE-003 is designed to selectively lower mutant huntingtin while sparing the normal allele in patients carrying the targeted SNP3 variant (roughly half of the HD population), a potential safety advantage over non-selective ASOs. The SELECT-HD trial is in early-phase evaluation of safety, pharmacokinetics, and allele-selective knockdown.
Verified Jul 2026
Institutional Research
Huntington's disease: a therapeutic field on a bumpy ride