BiogenBIIB said Wednesday its experimental treatment helped children with a devastating muscle-wasting disease even after they received an approved gene therapy from NovartisNVS.The company already ...

The Novartis Gene Therapies manufacturing facility on Tricenter Blvd. in Durham, N.C., just outside Research Triangle Park. Brian Gordon Swiss pharmaceutical giant Novartis has exited one of its ...

One could make a case that the sky-high one-time cost of gene therapy pays for itself by preventing the numerous emergency room visits and hospitalizations that it will eliminate. The estimated ...

Once Novartis acquired AveXis, it turned to setting a price for its much-anticipated gene therapy. Unlike other nations, the United States allows companies to charge whatever they want for new drugs.

Zolgensma® (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based prescription gene therapy used to treat paediatric patients with spinal muscular atrophy (SMA). Originally ...

He joins a team led by CEO Joe Nolan, formerly general manager of U.S. operations at AveXis, the company that developed Zolgensma, the first gene therapy for children with spinal muscular atrophy ...

A group of former executives of AveXis, the developer of the gene therapy Zolgensma, is behind a new startup launching publicly on Thursday with plans to push genetic medicine in new directions. The ...

This decision was based partly on the success of Zolgensma, originally developed by AveXis, as a gene therapy for spinal muscular atrophy (SMA) that is approved in the U.S., Japan, Europe, and Brazil ...

Novartis acquired AveXis for $8.7 billion in April 2018, picking up the phase 3 gene therapy for spinal muscular atrophy that would become Zolgensma and establishing the company as a frontrunner ...

AveXis, a Novartis company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending ...

AveXis received its first U.S. Food and Drug Administration approval in May 2019 for the treatment of spinal muscular atrophy (SMA). AveXis is also developing therapies for other genetic diseases, ...