Last update 24 May 2025

SGT-001

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
SGT 001
Action
stimulants
Mechanism
micro-dystrophin stimulants(micro-dystrophin stimulants)
Inactive Indication-
Originator Organization
Active Organization
Drug Highest PhasePhase 1/2
First Approval Date-
RegulationFast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Muscular Dystrophy, DuchennePhase 2
United States
06 Dec 2017
Muscular DystrophiesDiscovery
United States
-
Muscular DystrophiesDiscovery
United States
-
Muscular DystrophiesDiscovery
United States
-
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
SGT-001 low-dose (5E13 vg/kg)
wevvtbcgqe(nxasrdpfli) = frmyrugven yoghgbjdsi (zwwrhmivdj )
-
03 Mar 2024
SGT-001 high-dose (2E14 vg/kg)
wevvtbcgqe(nxasrdpfli) = lctmtuxbbs yoghgbjdsi (zwwrhmivdj )
Phase 1/2
3
mnxnqvheoq(hdurotenke) = fzfyhtctec yvphvydwyx (ibvsqnnvrv )
Positive
14 Mar 2022
Phase 1/2
-
SGT-001 at 5E13 vg/kg
myxxqgzolc(rvkhlkpxll) = xmgeopbasz qopxvungjw (dviqyxrbyx )
Positive
27 Apr 2021
SGT-001 at 2E14 vg/kg
myxxqgzolc(rvkhlkpxll) = kdmgjojpmh qopxvungjw (dviqyxrbyx )
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Clinical Trial

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Regulation

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