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Huntington's Disease

Autosomal dominant disorder caused by CAG repeat expansion in the HTT gene. 100% penetrant. Gene-silencing approaches developed here are directly transferable to ALS SOD1 programs.

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312
Papers (5yr)
4
Active trials
2
Tracked biomarkers
1
Related diseases
Research signals

HTT gene-silencing trials use delivery mechanisms nearly identical to ALS SOD1-ASO programs, a high-value cross-program collaboration opportunity identified by NeuropathIQ. View full research signals ->

Tracked biomarkers
NfL (CSF)Strong evidence

Tracks neurodegeneration and correlates with disease stage.

Mutant HTT protein (CSF)Moderate evidence

Direct target engagement marker for HTT-lowering therapies.

Active clinical trials
NCT IDDrugPhaseStatusSponsor
NCT05686551HTT antisense oligonucleotidePhase IIRecruitingIonis Pharmaceuticals

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Related diseases — shared mechanisms

Phase 1 primary cohort + monitored diseases

ALS
Shared: RNA delivery vectors (AAV9/ASO)
Research and educational use only. Information does not constitute medical advice, diagnosis, or treatment recommendations. Verify with primary literature. AI disclaimer →