Last update 16 May 2025

RP-L101

Overview

Basic Info

Drug Type
Gene therapy, Hematopoietic stem cell therapy
Synonyms
Fanconis anaemia gene therapy Rocket Pharmaceuticals, LP L01, RP L101
+ [1]
Target-
Action-
Mechanism
Gene transference(Gene transference)
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic DiseaseHemic and Lymphatic Diseases+ [1]
Active Indication-
Inactive Indication
Fanconi Anemia
Originator Organization
Fred Hutchinson Cancer Research CenterFred Hutchinson Cancer Research Center
Active Organization-
Inactive Organization
Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc.Fred Hutchinson Cancer Research CenterFred Hutchinson Cancer Research CenterNational Heart, Lung & Blood InstituteNational Heart, Lung & Blood Institute
+ [1]
License Organization-
Drug Highest PhaseDiscontinuedPhase 1
First Approval Date-
Regulation-
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Related

100 Clinical Results associated with RP-L101
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100 Translational Medicine associated with RP-L101
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100 Patents (Medical) associated with RP-L101
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100 Deals associated with RP-L101
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Fanconi AnemiaPhase 1
United States
National Heart, Lung & Blood InstituteNational Heart, Lung & Blood Institute
-
Fanconi AnemiaPhase 1
United States
Fred Hutchinson Cancer Research CenterFred Hutchinson Cancer Research Center
-
Fanconi AnemiaPhase 1
United States
National Cancer InstituteNational Cancer Institute
-
Fanconi AnemiaPhase 1
United States
Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc.
-
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

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Translational Medicine

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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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