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IONIS-HTTRx (formerly ISIS-HTTRx)

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Company Ionis Pharmaceuticals Inc.
DescriptionAntisense molecule targeting huntingtin (HTT)
Molecular Target Huntingtin (HTT)
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentPhase I/II
Standard IndicationHuntington's disease (HD)
Indication DetailsTreat Huntington's disease (HD)
Regulatory Designation U.S. - Orphan Drug (Treat Huntington's disease (HD));
EU - Orphan Drug (Treat Huntington's disease (HD))
PartnerRoche

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