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HTT

—Huntingtin

Chromosome: 4

Location: 4:3041363-3243957

OMIM: 613004

Ensembl: ENSG00000197386

Official·OMIM:613004 Official·ENSG00000197386

Encodes huntingtin protein. CAG repeat expansion causes Huntington disease.

Treatment Roadmap — HTT

Preclinical

Phase 1

4 trial

Phase 2

3 trial

Phase 3

2 trial

Approved

Clinical trials have approximately 10% success rate. This roadmap shows the current status, not guaranteed outcomes.

Associated Diseases

Huntington Disease

Autosomal DominantPrevalence: ~30Kneurological

Official·OMIM:143100 Official·ORPHA399

Summary

Clinical Trial

Approved Therapy

Registered Variant