Last update 01 Nov 2024

Marnetegragene autotemcel

Overview

Basic Info

Drug Type
Gene therapy, Hematopoietic stem cell therapy
Synonyms
LAD-1-gene-therapy-Rocket-Pharmaceuticals, Leukocyte-adhesion-deficiency-1-gene-therapy-Rocket-Pharmaceuticals
+ [2]
Target
Mechanism
ITGB2 gene stimulants(integrin subunit beta 2 gene stimulants), Gene transference
Inactive Indication-
Active Organization
Inactive Organization-
Drug Highest PhaseNDA/BLA
First Approval Date-
RegulationPRIME (EU), Rare Pediatric Disease (US), Orphan Drug (EU), Fast Track (US), Advanced Therapy Medicinal Products (EU), Regenerative Medicine Advanced Therapy (US), Priority Review (US)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Leukocyte Adhesion Deficiency Type 1NDA/BLA
US
02 Oct 2023
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 1/2
9
(tmxdmyajug) = sokfflhvxz czikbojwte (gwewtxieqf )
Positive
10 May 2024
Phase 1/2
9
adzcpugpph(cwfbrqijmc) = ckiqxxfbmp mtpbzlmmrb (hmzpawkmrx )
Positive
07 May 2024
Phase 1/2
9
(cazobcarqw) = The safety profile of RP-L201 has been highly favorable in all patients with no RP-L201-related serious adverse events to date glwzzgblhe (secepnkosl )
Positive
19 May 2022
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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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