- cafead Jun 28, 2024 at 11:32: AM
via Rocket Pharmaceuticals’ flight path to approval for Kresladi has been delayed again after the FDA issued a complete response letter (CRL) requesting additional manufacturing information.
The biotech submitted an application in October 2023 for the gene therapy, which is also known as marnetegragene autotemcel or marne-cel, to treat severe leukocyte adhesion deficiency-I. The therapy contains patient-derived hematopoietic stem cells that have been genetically modified with an LV vector to deliver a functional copy of the ITGB2 gene.
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The biotech submitted an application in October 2023 for the gene therapy, which is also known as marnetegragene autotemcel or marne-cel, to treat severe leukocyte adhesion deficiency-I. The therapy contains patient-derived hematopoietic stem cells that have been genetically modified with an LV vector to deliver a functional copy of the ITGB2 gene.
article source