Articles by tag: rocket-pharmaceuticals

Marne-Cel FDA approval: gene therapy for LAD-I

FDA approved Marne-Cel for children with severe LAD-I: 100% survival in trials. Learn how Rocket Pharmaceuticals turned rejections into a breakthrough.

FDA approved first gene therapy for LAD-I in children

FDA approved Kresladi (marnetegragene autotemcel) — the first stem cell gene therapy for children with severe LAD-I. Learn about the mechanism, precedents, and business model. Detailed analysis on the website.