Regenerative Medicine

Juno Therapeutics Inc, which has technology for genetically engineering T cells, has teamed up with Fate Therapeutics Inc to find out whether there is a role for small molecules in improving the profile of engineered T cells as therapies. The four-year collaboration takes T cell immunotherapy one step further by potentially introducing new modulators into the mix of technologies that are now being investigated globally for a wide range of cancers. The collaboration was announced on 6 May.

Small and medium-sized enterprises (SMEs) play a special role in drug development particularly in the field of advanced therapy medicinal products (ATMPs). With small numbers of staff, many of whom are scientists, they are in a unique position to drive medical innovation.

One of Japan’s biggest industrial concerns, Fujifilm Holdings Corp, has expanded further into regenerative medicine with the acquisition of Cellular Dynamics International Inc, a developer of induced pluripotent stem cells (iPS). CDI was founded by the stem-cell pioneer James Thomson.

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.