Bayer and CRISPR Therapeutics launch gene drug discovery venture

Bayer is to set up a joint venture with CRISPR Therapeutics and acquire a minority stake in the firm, one of the pioneers in the potentially revolutionary gene-editing technology.

The partnership will be a joint venture (JV) which the German pharma company will manage through its new Bayer LifeScience Centre (BLSC) unit, combining the 'molecular scissors' gene-editing technology of CRISPR-Cas9 techniques with its own protein engineering and disease knowledge.

The JV will focus on discovering and developing new drugs for conditions including haemophilia, heart disease in infants and a form of blindness called Stargardt.

Bayer is just the latest in a string of big pharma companies to invest in firms with CRISPR-Cas9 expertise, of which CRISPR Therapeutics is one of four leading players.

CRISPR Therapeutics already has financial backing from Celgene and in October announced a discovery alliance with Vertex in cystic fibrosis. Novartis has a stake in rivals Intellia Therapeutics, and is also funding another firm in the field, Caribou Biosciences.

Bayer's venture will undoubtedly be playing catch-up, but it is likely to be a few years before the first CRISPR-Cas9 molecule for human therapy enters clinical trials.

Among the most hotly-tipped of the rivals is Editas, whose chief executive, Katrine Bosley, recently said it could begin clinical trials of its lead candidate by 2017. Its first disease target is a form of inherited blindness called leber congenital amaurosis (LCA).

The JV is the first investment by the newly-established BLSC, a standalone division which will foster 'novel strategic innovation' and report directly to Bayer´s management board.

Dr Marijn Dekkers, chief executive officer of Bayer said the firms were "philosophically and financially aligned" with the focus on "game-changing or possibly curative treatments" for serious human genetic diseases.

Bayer will provide a minimum of $300 million in R&D investments to the JV over the next five years, and will acquire a minority stake in CRISPR Therapeutics for $35 million in cash.

The JV will be led by Dr Axel Bouchon, head of the BLSC, on an interim basis as CEO, while Dr Rodger Novak, CEO and co-founder of CRISPR Therapeutics, will serve as the interim chairman of the newly-formed JV Board.

The company is headquartered in Basel, Switzerland and Cambridge, MA, with corporate offices in London.

Novak said the JV and Bayer partnership was 'game-changing' for his firm, allowing it to keep a 50% ownership in the area of blood disorders, blindness, and congenital heart diseases. All technology development and future IP developed by the JV will be exclusively available to the parent companies, and any new IP beyond the three core areas will be available exclusively to CRISPR Therapeutics for human use, and to Bayer for non-human use, such as agricultural applications.

Through the JV, Bayer may secure exclusive rights to use CRISPR Therapeutics' and the JV's proprietary CRISPR-Cas9 technology and intellectual property in the three targeted disease areas, including blood disorders, blindness and congenital heart diseases. CRISPR Therapeutics may gain exclusive access to Bayer's protein engineering know-how for use in CRISPR products as well as Bayer's extensive expertise and knowledge in the three targeted disease areas.

Axel Bouchon said CRISPR Therapeutics had built the most promising gene-editing technology on the market, adding that this was perfectly suited to fully leverage Bayer´s expertise in protein engineering and knowledge in the disease areas.

The name of the new JV will be announced shortly, and will be based in London, UK, with operations in Cambridge, Mass.