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.
The cohort of companies developing products for treating cancer with genetically engineered T-cells has increased with the announcement that Merck Serono and Intrexon Corp will collaborate to generate new drug candidates using the technology.
Research by Douglas Melton of Harvard University and colleagues showing that insulin-producing pancreatic beta cells can be created in a laboratory has reached the stage of commercialisation with the launch of a new company aimed at bringing a cell therapy for diabetes into human trials.
The annual Bio-Europe Spring meeting typically brings together business development directors from small and medium-sized European companies to discuss licensing or partnering deals with their counterparts at large pharma, or with venture capitalists.
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.
Canada-based EnGene Inc, which has developed a gene-based technology to treat autoimmune diseases, has raised $11.5 million in a Series B investment round. The funds will be used to advance the lead product which is intended to treat ulcerative colitis.
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.
A new technique for culturing human liver and pancreatic stem cells could pave the way for improved therapies for treating both liver disease and pancreatic cancer. This is one of the conclusions from two studies published in the journal Cell on 15 January.
Cardio3 BioSciences SA has acquired a portfolio of immuno-oncology assets in the US thereby expanding its therapeutic focus to cancer in addition to cardiovascular medicine. The Belgium-based company specialises in regenerative medicine.
The European Medicines Agency’s main scientific committee is recommending approval of a stem cell therapy to treat limbal stem cell deficiency. It is the first positive opinion for a stem cell therapy and the fifth for an advanced therapy medicinal product (ATMP).