HTT Overview
Molecule Name
Huntingtin
Synonyms
HTT,Htt,Huntingtin
Drug Development Progress
Drugs in Clinical Trials10
Approved Drugs0
Highest Development StagePhase 3 Clinical
Target Synonym
Huntingtin，HD protein，HD，HTT，Huntington Disease Protein，IT15，Huntingtin (Huntington Disease)，LOMARS，Huntingtin Protein
Background
Huntingtin is the protein coded for by the HTT gene. Autosomal dominant mutation in the Huntingtin (Htt) protein is the cause of Huntington’s Disease (HD). In HD, the polyglutamine (polyQ) domain in the N-terminal sequenceof the protein is expanded beyond a threshold of 36 glutamines. mutant polyQ expansion strongly correlates in an inverse manner to disease age of onset.
HTT Product List
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Clinical Drug Information
| English Name | Research Code | Highest Development Stage | Company | Indication | Clinical Trial |
|---|---|---|---|---|---|
| Tominersen | RG-6042; RO-7234292; ISIS-443139 | Phase 3 Clinical | Ionis Pharmaceuticals Inc | Huntington Disease | Details |
| SKY-0515 | SKY-0515 | Phase 3 Clinical | Skyhawk Therapeutics Inc | Huntington Disease; Nervous System Diseases | Details |
| Votoplam | PTC-518; PTC518 | Phase 3 Clinical | Ptc Therapeutics Inc | Huntington Disease | Details |
| WVE-003 | WVE-003 | Phase 2 Clinical | Wave Life Sciences Ltd | Huntington Disease | Details |
| NA-841 | NA-841; NA841 | Phase 2 Clinical | Biomed Industries Inc | Huntington Disease | Details |
| SPK-10001 | SPK-10001; RG-6662; HTT miRNA GT | Phase 2 Clinical | Spark Therapeutics Inc | Huntington Disease | Details |
| ER-2001 | ER-2001; ER2001 | Phase 2 Clinical | Shenzhen Aima Biotechnology Co Ltd | Huntington Disease | Details |
| ARO-HTT | ARO-HTT; SRP-1005 | Phase 1 Clinical | Arrowhead Pharmaceuticals Inc | Huntington Disease | Details |
| ALN-HTT-02 | ALN-HTT-02; ALN-HTT02 | Phase 1 Clinical | Alnylam Pharmaceuticals Inc | Huntington Disease | Details |
| PTC-607 | PTC-607 | Phase 1 Clinical | Ptc Therapeutics Inc | Huntington Disease | Details |