Last update 05 Jul 2025

Adeno-associated virus serotype 9 vector-delivered SMN1 gene therapy (Genzyme/University of California San Francisco/Emory University/Georgetown University)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
adeno-associated virus serotype 9 vector-delivered SMN1 gene therapy (Genzyme/University of California San Francisco/Emory University/Georgetown University)
Target
SMN1
Action
stimulants
Mechanism
SMN1 gene stimulants(survival of motor neuron 1, telomeric gene stimulants)
Therapeutic Areas
Nervous System DiseasesOther Diseases
Active Indication-
Inactive Indication
Spinal Muscular Atrophy
Originator Organization
Genzyme Corp.Genzyme Corp.
Active Organization-
Inactive Organization
Genzyme Corp.Genzyme Corp.The University of California, San FranciscoThe University of California, San FranciscoEmory UniversityEmory University
+ [1]
License Organization-
Drug Highest PhasePendingDiscovery
First Approval Date-
Regulation-

Related

100 Clinical Results associated with Adeno-associated virus serotype 9 vector-delivered SMN1 gene therapy (Genzyme/University of California San Francisco/Emory University/Georgetown University)
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100 Translational Medicine associated with Adeno-associated virus serotype 9 vector-delivered SMN1 gene therapy (Genzyme/University of California San Francisco/Emory University/Georgetown University)
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100 Patents (Medical) associated with Adeno-associated virus serotype 9 vector-delivered SMN1 gene therapy (Genzyme/University of California San Francisco/Emory University/Georgetown University)
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100 Deals associated with Adeno-associated virus serotype 9 vector-delivered SMN1 gene therapy (Genzyme/University of California San Francisco/Emory University/Georgetown University)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Spinal Muscular AtrophyDiscovery
United States
Georgetown UniversityGeorgetown University
-
Spinal Muscular AtrophyDiscovery
United States
Genzyme Corp.Genzyme Corp.
-
Spinal Muscular AtrophyDiscovery
United States
Emory UniversityEmory University
-
Spinal Muscular AtrophyDiscovery
United States
The University of California, San FranciscoThe University of California, San Francisco
-
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Clinical Result

Indication
Phase
Evaluation
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Phase
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Deal

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Clinical Trial

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Approval

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Regulation

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