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State of the Huntington’s disease pipeline

Setbacks for HTT-targeting programs have put alternative therapeutic approaches out in front

January 21, 2022 11:25 PM UTC

Though many view the mutant gene that causes Huntington’s disease as key to treating patients, setbacks in programs targeting HTT have enabled alternative therapeutic approaches to take the lead.

The clinical pipeline for Huntington’s disease contains at least 17 candidates, the most advanced of which — Ingrezza valbenazine from Neurocrine Biosciences Inc. (NASDAQ:NBIX) and pridopidine
from Prilenia Therapeutics B.V. — do not target HTT (see Figure 1)...