Casgevy: Innovative Medicinal Products Require Innovative Approaches to Regulatory Assessment

• मुख्य लेखकों: Essam Kerwash (लेखक), John D. Johnston (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: MDPI AG, 2024-07-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: Connect to this object online.

Casgevy (exa-cel) is an autologous cellular therapy modified ex vivo by a CRISPR-Cas9-mediated gene-editing technology. For Casgevy to be granted the indication in transfusion-dependent β-thalassemia, one single-arm trial was submitted which was not amenable to conventional statistical analysis of &...