Last update 08 May 2025

Recombinant adeno-associated virus vector (Spark Therapeutics)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
recombinant adeno-associated virus vector that contains a bioengineered capsid (AAV-Spark100) and a codon-optimized expression cassette to drive expression of a secretable form of alpha-galactosidase A
Target
GALA
Action
stimulants
Mechanism
GLA stimulants(α-Galactosidase stimulants)
Therapeutic Areas
Nervous System DiseasesCardiovascular DiseasesCongenital Disorders+ [2]
Active Indication
Fabry Disease
Inactive Indication-
Originator Organization
Spark Therapeutics, Inc.Spark Therapeutics, Inc.
Active Organization
Spark Therapeutics, Inc.Spark Therapeutics, Inc.
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States)

Related

100 Clinical Results associated with Recombinant adeno-associated virus vector (Spark Therapeutics)
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100 Translational Medicine associated with Recombinant adeno-associated virus vector (Spark Therapeutics)
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100 Patents (Medical) associated with Recombinant adeno-associated virus vector (Spark Therapeutics)
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100 Deals associated with Recombinant adeno-associated virus vector (Spark Therapeutics)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Fabry DiseasePreclinical
United States
Spark Therapeutics, Inc.Spark Therapeutics, Inc.
11 Aug 2023
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
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Deal

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Core Patent

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Clinical Trial

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Approval

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Regulation

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