Orchard Therapeutics secured an exclusive license for an ex vivo autologous hematopoietic stem cell gene therapy program for the treatment of MPS-I developed by SR-Tiget.
The U.S. Food and Drug Administration approved Zolgensma, a gene therapy that Novartis called a life-changing medicine, for children younger than 2 years of age who have been diagnosed with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene.
Akcea Therapeutics Inc. and Ionis Pharmaceuticals Inc. announced that Waylivra received conditional marketing authorization from the European Commission as the only therapy for FCS.
Novartis AG, which previously announced positive interim trial results for the company’s experimental gene therapy for spinal muscular atrophy, said an investigation is underway into whether a second trial death could be related to the treatment.
Regeneron Pharmaceuticals is investing $800 million in Alnylam Pharmaceuticals to develop treatments for eye and central nervous system diseases using gene-silencing RNA interference technology.
Biogen Inc. should slash the price of the spinal muscular atrophy (SMA) drug Spinraza, and the $4 million to $5 million Novartis has said the company’s experimental gene therapy Zolgensma for the disease is worth is excessive, an independent U.S. organization that reviews the value of medical treatments said.
The U.S. Food and Drug Administration approved ADMA Biologics’ Asceniv to treat primary humoral immunodeficiency disease (PIDD or PI) in adults and adolescents.
An advisory committee of the EMA recommended conditionally approving a drug from Akcea Therapeutics and Ionis Pharmaceuticals that aims to treat a rare genetic disease.
Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.