Last update 20 Mar 2025

scAAV2/MFSD8 gene replacement therapy (Northwestern University Feinberg School of Medicine/UT Southwestern)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms-
Target
MFSD8
Action-
Mechanism
MFSD8 gene transference(Major facilitator superfamily domain-containing protein 8 gene transference)
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic Disease
Active Indication
Lysosomal Storage Diseases
Inactive Indication
Neuronal Ceroid-Lipofuscinoses
Originator Organization
Feinberg School of MedicineFeinberg School of Medicine
Active Organization
The University of Texas Southwestern Medical CenterThe University of Texas Southwestern Medical CenterFeinberg School of MedicineFeinberg School of Medicine
Inactive Organization-
Drug Highest PhasePreclinical
First Approval Date-
Regulation-
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Related

100 Clinical Results associated with scAAV2/MFSD8 gene replacement therapy (Northwestern University Feinberg School of Medicine/UT Southwestern)
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100 Translational Medicine associated with scAAV2/MFSD8 gene replacement therapy (Northwestern University Feinberg School of Medicine/UT Southwestern)
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100 Patents (Medical) associated with scAAV2/MFSD8 gene replacement therapy (Northwestern University Feinberg School of Medicine/UT Southwestern)
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100 Deals associated with scAAV2/MFSD8 gene replacement therapy (Northwestern University Feinberg School of Medicine/UT Southwestern)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Neuronal Ceroid-LipofuscinosesPreclinical
United States
The University of Texas Southwestern Medical CenterThe University of Texas Southwestern Medical Center
04 May 2021
Neuronal Ceroid-LipofuscinosesPreclinical
United States
Feinberg School of MedicineFeinberg School of Medicine
04 May 2021
Lysosomal Storage DiseasesPreclinical
United States
Feinberg School of MedicineFeinberg School of Medicine
-
Lysosomal Storage DiseasesPreclinical
United States
The University of Texas Southwestern Medical CenterThe University of Texas Southwestern Medical Center
-
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Clinical Result

Indication
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Approval

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Regulation

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