Last update 21 Nov 2024

AAV9-hASM

Overview

Basic Info

Drug Type
Gene editing
Synonyms-
Target
SMPD1
Mechanism
SMPD1 inhibitors(sphingomyelin phosphodiesterase 1 inhibitors)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease
Active Indication-
Inactive Indication
Niemann-Pick Diseases
Originator Organization
Virovek, Inc.Virovek, Inc.San Francisco Conservatory of MusicSan Francisco Conservatory of MusicUniversity of California, Santa BarbaraUniversity of California, Santa Barbara
+ [1]
Active Organization-
Inactive Organization
University of California, Santa BarbaraUniversity of California, Santa BarbaraSan Francisco Conservatory of MusicSan Francisco Conservatory of MusicSanofi (Hangzhou) Pharmaceuticals Co., Ltd.Sanofi (Hangzhou) Pharmaceuticals Co., Ltd.
+ [1]
Drug Highest PhasePendingPreclinical
First Approval Date-
Regulation-

Related

100 Clinical Results associated with AAV9-hASM
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100 Translational Medicine associated with AAV9-hASM
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100 Patents (Medical) associated with AAV9-hASM
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100 Deals associated with AAV9-hASM
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Niemann-Pick DiseasesPreclinical
US
University of California, Santa BarbaraUniversity of California, Santa Barbara
21 Aug 2019
Niemann-Pick DiseasesPreclinical
US
Virovek, Inc.Virovek, Inc.
21 Aug 2019
Niemann-Pick DiseasesPreclinical
CN
Sanofi (Hangzhou) Pharmaceuticals Co., Ltd.Sanofi (Hangzhou) Pharmaceuticals Co., Ltd.
21 Aug 2019
Niemann-Pick DiseasesDiscovery
US
San Francisco Conservatory of MusicSan Francisco Conservatory of Music
21 Aug 2019
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

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Approval

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Regulation

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