Last update 11 Oct 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
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication-
Inactive Indication
Lysosomal Storage DiseasesNeuronal Ceroid-Lipofuscinoses
Originator Organization
Feinberg School of MedicineFeinberg School of Medicine
Active Organization-
Inactive Organization
Feinberg School of MedicineFeinberg School of MedicineThe University of Texas Southwestern Medical CenterThe University of Texas Southwestern Medical Center
License Organization-
Drug Highest PhasePendingPhase 1
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

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Neuronal Ceroid-LipofuscinosesPhase 1
United States
The University of Texas Southwestern Medical CenterThe University of Texas Southwestern Medical Center
04 May 2021
Neuronal Ceroid-LipofuscinosesPhase 1
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
Phase
Evaluation
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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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