Novartis grows its gene therapy ambitions

Nature Reviews Drug Discovery 17, 307 (2018). doi:10.1038/nrd.2018.68 Author: Asher Mullard Novartis is buying gene therapy company AveXis for US$8.7 billion, expanding its gene therapy capabilities.AveXis's lead candidate is AVXS-101, a recombinant AAV9-based gene therapy for the treatment of spinal muscular atrophy (SMA). SMA is a neuromuscular disease that is caused by genetic defects in
Source: Nature Reviews Drug Discovery - Category: Drugs & Pharmacology Authors: Tags: News and Analysis Source Type: research