Content matching 'gene therapy'
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.
Researchers received the go-ahead from the FDA to test a gene-editing technique in humans and produce a "functional cure" for HIV.
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.
NightstaRx Ltd, a one-year old gene therapy company, has received £5 million in new venture finance to expedite the development of a treatment for choroideremia, an inherited cause of progressive blindness. This brings total funding for the company to £17 million.
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.