One of the companies that hope to turn a revolutionary gene-editing technology into reality just teamed up with a major pharmaceutical company. Bayer, a $96 billion German drug company, will provide CRISPR Therapeutics a minimum of $300 million over the next five years to develop the CRISPR-cas9 gene-editing technology to treat conditions such as blood disorders, blindness, and congenital heart disease.
Bayer is also acquiring a majority stake in CRISPR for $35 million.
CRISPR-Cas9 is a tool that allows scientists to swap a particular, potentially faulty gene with another, potentially healthy one. So far, the technology hasn't been used in people (except in nonviable human embryos).
"This is potentially game changing, but it will take time," Dr. Rodger Novak, CRISPR Therapeutics' CEO and cofounder, told Business Insider. "That’s why we did it with Bayer. We understand it will take time, but it would take much longer if we didn't have a partnership like this."
CRISPR Therapeutics was founded in 2014 by Novak, Shaun Foy, and Emmanuelle Charpentier, who is one of the scientists credited with discovering the CRISPR technique. It's based mainly in Cambridge, Massachusetts, where its research and development is located. For the first year or so, Novak said the company stayed "under the radar screen on purpose."But lately, that hasn't been the case.
In October, CRISPR Therapeutics made a $105 million deal with Vertex Pharmaceuticals, a partnership Novak said is very different from the partnership with Bayer. With Vertex, Novak said, the main focus was licensing the technology and allowing Vertex to develop their own possible drug targets. With the Bayer collaboration, Novak said, the two companies will go into studying conditions and diseases like blindness and blood disorders, where Bayer has a lot of expertise.
The race to be first But CRISPR isn't the only company that wants to be the first to use CRISPR in humans. Editas, another CRISPR company based in Cambridge, Massachusetts and founded by CRISPR pioneers Jennifer Doudna and Feng Zhang, wants to start using CRISPR in people by 2017.
"We're not a technology company," Novak said. "We have the ability of translating to clinical medicine in the near future."
Novak said his company plans to fall along the same timeline.
He's hoping to see the company start applying the gene-editing technology to human cells by the end of 2017. Currently, most work in CRISPR has been done by putting a Cas9 protein and a guide RNA into a set of cells outside the body, where the two work together to cut and paste the faulty DNA with healthy DNA.
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Ideally, though, CRISPR will be able to reach the cells scientists want to target on their own. Studies have shown this is a possibility, but it's still very challenging because it's hard to target the right cells. But Novak is optimistic. He wants his company to start working on it as early as three or four years down the road.
CRISPR-cas9 technology emerged just three years ago. "That's nothing," Novak said. "Translating such technology that rapidly, if we manage to do this, that would be really breaking a world record."
As far as funding goes, the deal puts CRISPR's funding neck and neck with Editas.
In August, Editas raised $120 million from investors including Bill Gates. The company has also teamed up with Juno Therapeutics, a company focused on cell therapies for cancer, which will be worth up to $230 million.
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