Chemistry:Marnetegragene autotemcel
| Clinical data | |
|---|---|
| Trade names | Kresladi |
| Other names | RP-L201, Ladicell |
| AHFS/Drugs.com | kresladi |
| License data | |
| Routes of administration | Intravenous infusion |
| ATC code |
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| Legal status | |
| Legal status | |
| Identifiers | |
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Marnetegragene autotemcel, sold under the brand name Kresladi, is a gene therapy used for the treatment of severe leukocyte adhesion deficiency type I.[2]
Marnetegragene autotemcel consists of the recipient's hematopoietic (blood) stem cells, which are genetically modified to introduce functional copies of the ITGB2 gene. Following conditioning, a single dose of marnetegragene autotemcel is infused intravenously to address the underlying cause of severe leukocyte adhesion deficiency-1 by restoring CD18 and CD11a cell surface expression in white blood cells, including neutrophils.[3]
Marnetegragene autotemcel was approved for medical use in the United States in March 2026.[3]
Medical uses
Marnetegragene autotemcel is indicated for the treatment of children with severe leukocyte adhesion deficiency I due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.[1][3]
Society and culture
Legal status
Marnetegragene autotemcel was approved for medical use in the United States in March 2026.[2]
The US Food and Drug Administration (FDA) granted the application for marnetegragene autotemcel orphan drug, rare pediatric disease, regenerative medicine advanced therapy, and fast track designations.[3] The FDA granted accelerated approval of Kresladi to Rocket Pharmaceuticals.[3]
Names
Marnetegragene autotemcel is the international nonproprietary name.[4]
Marnetegragene autotemcel is sold under the brand name Kresladi.[2][3]
References
- ↑ 1.0 1.1 "Kresladi- marnetegragene autotemcel injection, suspension". 30 March 2026. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=97db9c3c-20c2-4dad-890c-bc821805421f.
- ↑ 2.0 2.1 2.2 "Kresladi". 26 March 2026. https://www.fda.gov/vaccines-blood-biologics/kresladi.
This article incorporates text from this source, which is in the public domain.
- ↑ 3.0 3.1 3.2 3.3 3.4 3.5 "FDA Approves First Gene Therapy for Severe leukocyte adhesion deficiency type I". U.S. Food and Drug Administration (FDA) (Press release). 26 March 2026. Retrieved 6 April 2026. This article incorporates text from this source, which is in the public domain.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87". WHO Drug Information 36 (1). 2022.
External links
- Clinical trial number NCT03812263 for "A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I" at ClinicalTrials.gov
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