Last update 01 Nov 2024

AMT-130

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
RAAV5-MIHTT, rAAV5-miHTT
+ [5]
Target
Mechanism
HTT gene modulators, Gene silencing, RNA interference
Active Indication
Inactive Indication-
Active Organization
Inactive Organization-
Drug Highest PhasePhase 1/2
First Approval Date-
RegulationOrphan Drug (EU), Regenerative Medicine Advanced Therapy (US)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Huntington DiseasePhase 2
US
06 Sep 2019
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 1/2
39
AMT-130 low dose
(gkaqbzwslc) = None degjcfjjrt (kbpwswygqm )
Positive
09 Jul 2024
AMT-130 high dose
Phase 1/2
39
(high dose)
(ilfjgvldnl) = kfhsdovqzn wtbyzpprpb (ifiqdynxdn )
Positive
19 Dec 2023
Phase 1/2
31
(fvxbwstnon) = tigsrzeojl tnolntrbsj (hzgzgkdmfo )
-
17 Sep 2021
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Approval

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Regulation

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