Last update 21 Nov 2024

Volrubigene Ralaparvovec

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
+ [1]
Target
UGT1A1
Mechanism
UGT1A1 modulators(UDP-glucuronosyltransferase 1-1 modulators), Gene transference
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic DiseaseOther Diseases
Active Indication
Crigler-Najjar Syndrome
Inactive Indication-
Originator Organization
Astellas Gene Therapies, Inc.Astellas Gene Therapies, Inc.
Active Organization
Astellas Gene Therapies, Inc.Astellas Gene Therapies, Inc.
Inactive Organization
University of PennsylvaniaUniversity of Pennsylvania
Drug Highest PhasePhase 1/2
First Approval Date-
Regulation-
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Related

1
Clinical Trials associated with Volrubigene Ralaparvovec
NCT03223194 / TerminatedPhase 1
VALENS: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT342, an AAV8-Delivered Gene Transfer Therapy in Crigler-Najjar Syndrome Subjects Aged 1 Year and Older
100 Clinical Results associated with Volrubigene Ralaparvovec
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100 Translational Medicine associated with Volrubigene Ralaparvovec
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100 Patents (Medical) associated with Volrubigene Ralaparvovec
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100 Deals associated with Volrubigene Ralaparvovec
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Crigler-Najjar SyndromePhase 2-
Astellas Gene Therapies, Inc.Astellas Gene Therapies, Inc.
-
Crigler-Najjar SyndromePhase 2-
University of PennsylvaniaUniversity of Pennsylvania
-
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Clinical Result

Indication
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Clinical Trial

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Approval

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Regulation

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