Another day, another CRISPR announcement!

CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated announced yesterday that they will be entering into a 105 million USD strategic research collaboration focused on developing therapeutics for genetic disease.In more detail, what does that mean? A number of genetic mutations are responsible for inherited diseases, and two examples of this are cystic fibrosis and sickle cell anaemia.

Cystic fibrosis (CF) is caused by mutations within the CFTR gene which lead to a broken or non-folding protein, this in turn prevents the proper dilution of mucosal secretions and thus leads to numerous pathologies. Sickle cell anaemia/disease, by contrast, is due to mutations within the gene coding for haemoglobin leading to protein aggregation and disruption of red blood cell structures. It is predominantly found in descendants from malaria-prone areas, as the mutation – at least in heterozygotes – provides innate resistance against malarial infection.

The collaboration between Vertex and CRISPR will initially focus on developing therapeutics for cystic fibrosis and sickle cell anaemia, with other unspecified genes also on the target list. The agreement provides Vertex with an option to take an exclusive license on up to six of the treatments which come out of this collaboration, so there are at least four more interesting targets waiting in the wings. Interestingly, the press release comments that there is a specified number of other genetic targets – someone at Vertex is thus presumably frantically ranking their candidate genes to find the best short list. Hopefully they do it well, Vertex has agreed to pay up to 420 million USD plus royalties per licensed treatment.Vertex Pharmaceuticals is a publically listed company with a market cap of over 28 billion USD (listed as NASDAQ:VRTX, not to be confused with the currently rather controversial Valeant Pharmaceuticals, NASDAQ:VRX). Amongst their many products Vertex are well known for production of the highly effective (and expensive!) cystic fibrosis drug ivacaftor – otherwise known as Kalydeco, and thus have both the experience and necessary cash to further develop gene-editing CF therapeutics.CRISPR Therapeutics matches this with their extensive knowledge and IP portfolio covering the use of CRIPSR/Cas9 for human therapeutics. The costs of the work will be predominantly borne by Vertex, although in a two-stage model. All costs for the CF discovery process will fall on Vertex, while CRISPR will perform the majority of the discovery work, but the companies will equally split costs and sales for sickle cell/haemoglobinopathy research and development.

That of course is in the future, but what is happening now? Vertex will pay CRISPR 75 million USD in cash up front, alongside a separate 30 million USD investment into CRISPR Therapeutics. This is an equity investment, providing an observer seat on the board, and which also means that Vertex joins other pharmaceutical companies such as Celgene and GSK in holding portions of the company. Given that the last funding round raised 64 million USD for CRISPR, they seem to be going from strength to strength.

It is, of course, a hot area. CRISPR Therapeutics was founded by one of the first publishers of the CRISPR/Cas9 discovery, Dr Emmanuelle Charpentier. Coincidentally one of her co-authors, Dr Jennifer Doudna, was also recently in the news as her company Caribou Biosciences announced a CRISPR/Cas alliance with DuPont. One looming question, of course, is: do they even own the rights to this IP? The current patent fight between the University of California and MIT is causing consternation on a number of fronts, and may in fact be holding back wide-scale licensing of the technology. It will be interesting to see how this one pans out.