Content matching 'gene therapy'
The buzz at the Phacilitate Cell & Gene Therapy Forum in Washington DC on 26 to 28 January was that gene therapy will become a mainstream activity in the global pharmaceutical industry over the next five years, driven in part by the new gene-modified cell therapies and genome editing.
UniQure NV has secured a gene therapy agreement with Bristol-Myers Squibb Company in cardiovascular disease, its first major commercial deal since winning regulatory approval in 2012 for Glybera, a gene therapy for lipoprotein lipase deficiency.
Pfizer Inc is prepared to commit up to $280 million to an experimental gene therapy for haemophilia which is under development by Spark Therapeutics in the US, while at the same time reinforcing its gene therapy research capacity in the UK.
The US Food and Drug Administration has granted ‘breakthrough therapy’ status to a gene therapy that is in Phase 3 development for patients with an inherited eye disorder that can lead to blindness. The developer is Spark Therapeutics of Philadelphia, Pennsylvania.