A few weeks ago I attended the Regenerative Medicine Industry Day at Edinburgh’s BioQuarter, much of which focused on the challenges SMEs involved in stem cell therapy face. These ranged from manufacturing stem cells and GMP, compliance with clinical trial regulations, and of course funding. One of the main issues investors seem to have is how do you value a company in the stem cell sector?
One of the problems with stem cells is that the related IP is complex. In October 2011 The Court of Justice of the EU handed down a decision on questions referred in the case of Brüstle v Greenpeace effectively prohibiting the patenting of inventions derived from human embryos. This decision confirmed that any human ovum after fertilisation is to be regarded as an embryo and that inventions requiring the destruction of human embryos or their use as base material are excluded from patentability. This ruling applied to totipotent stem cells, those cells taken from the early embryo with the potential to develop into any tissue. The IPO however can still issue patents for pluripotent stem cells, those cells that have lost the programming capability to form every tissue but still have the capacity to form any cell in a specific lineage. These cells can be derived from adult humans and so avoids the ethical issues involved with using human embryos.
The UK produces world class research at both the academic and industrial level in stem cell research. The ban could lead to British inventions being exploited in countries where totipotent cells can be patented.
Even for companies specialising in pluripotent stem cells with patent capability, the funding challenges still remain. Venture capital investment is lacking in the area because the research has not yet been fully validated and it could be years before a marketable product is available. Stem cell technology is regarded as too risky to be an attractive investment position. Large pharmaceutical companies have also been cautious in the past of stem cell research. To raise revenue some UK companies have licensed stem cell lines and reagents as research tools to fund longer term development of cell therapies. There is also great potential for in vitro drug screening and toxicity testing using stem cell lines. As I mentioned in a previous blog on personalised medicine, one application in this area would be to derive stem cells from individual populations for more accurate drug administration regimes.
For the UK’s strength in pioneering stem cell research to be fulfilled, the potential for commercial opportunity must be demonstrated. When the first stem cell based therapies become available the full potential of the sector be realised. But for the moment there is a large gap between the funding provided by research councils and charities and the elusive potential commercial funding that venture capitalists or large pharmaceuticals can provide.