Last update 30 Jun 2025

RP-L401

Overview

Basic Info

Drug Type
Gene therapy, Hematopoietic stem cell therapy
Synonyms
RP L401
Target-
Action-
Mechanism
Gene transference(Gene transference)
Therapeutic Areas
Congenital DisordersSkin and Musculoskeletal DiseasesOther Diseases
Active Indication-
Inactive Indication
Osteopetrosis
Originator Organization
Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc.
Active Organization-
Inactive Organization
Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc.
License Organization
University of LundRocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc.
Drug Highest PhaseDiscontinuedPhase 1
First Approval Date-
Regulation-

Related

1
Clinical Trials associated with RP-L401
NCT04525352
/ TerminatedPhase 1
A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis (IMO) to Evaluate the Safety and Preliminary Efficacy of Autologous CD34+ Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene
100 Clinical Results associated with RP-L401
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100 Translational Medicine associated with RP-L401
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100 Patents (Medical) associated with RP-L401
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100 Deals associated with RP-L401
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
OsteopetrosisPhase 1
United States
Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc.
19 Nov 2020
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

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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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