Rocket gene therapy’s flight to approval delayed by FDA rejection over manufacturing questions

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.