MedPath

WVE-003

Generic Name
WVE-003
Brand Names
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Drug Type
Biotech
Chemical Formula
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CAS Number
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Unique Ingredient Identifier
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Background

WVE-003 is an allele-selective antisense oligonucleotide that works to selectively reduce the toxic mutant huntingtin (mHTT) protein.

Associated Conditions
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Associated Therapies
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biospace.com
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After Decades of Failure, First Disease-Modifying Huntington's Treatment on the Horizon

New Huntington's disease therapies are nearing R&D finish lines, aiming to be the first disease-modifying treatments. Despite high-profile failures, companies like Wave, Prilenia, and uniQure are progressing towards regulatory approval. The field has gained momentum after decades of trial and error, with several mid-stage clinical trials underway. The ideal therapy would selectively lower mutant huntingtin and be widely distributed in the brain, though none currently exist.
journals.lww.com
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New Wave of Trials in Huntington's Disease Bring Hope to the...

Despite failed trials, hope is renewed in Huntington's disease treatment with a gene therapy showing 80% slowing of disease progression and another treatment seeking FDA accelerated approval.
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