(Bloomberg) — In a single year, Rodger Novak's gene-editing startup raised $89 million in venture funding, got $105 million to enter a partnership with big drugmaker Vertex Pharmaceuticals Inc. — and, this week, announced a deal with Bayer AG worth $335 million.
Crispr Therapeutics Ltd. won't start human trials of its therapies until 2017, but the company is at the forefront of one of the hottest technologies in biotech. Investors and drugmakers from Johnson & Johnson to Merck & Co. are flocking to a potent tool called Crispr, saying its precise DNA editing capabilities could yield treatments for conditions as diverse as blood diseases, cancers, auto-immune disorders and genetic eye disorders.
Gene-editing companies have drawn more than $1 billion in venture dollars since 2013, according to Boston Consulting Group, from traditional biotech venture capitalists like Deerfield Management Co. and Polaris Partners, and names better known in in the tech world, such as Bill Gates and Khosla Ventures. Demand to participate in Crispr Therapeutics' funding round was so strong that the company turned away some blue-chip investors, Novak said in an interview.
"Coming late to this party is not very smart," he said by telephone.
Like many promising new technologies that came before it, Crispr has yet to prove it will be effective at creating new medicine. And the idea of editing genes has made some ethicists queasy, though the companies getting funding aren't making alterations in human sperm, eggs or embryos, which would be controversial.
Tough disorders
Still, researchers and investors are excited by the prospects of the technique to treat diseases that have stymied the field of medicine for ages. Crispr's most obvious application is in repairing mutated genes that are linked to disease. Basel, Switzerland-based Crispr Therapeutics, along with Editas Medicine Inc., Intellia Therapeutics Inc., and Poseida Therapeutics Inc., all closely held biotech companies, are tackling disorders including sickle cell anemia, the blood disorder beta thalassemia, and inflammatory and autoimmune diseases.
While gene-editing technology has existed for more than 10 years, older methods were expensive and slow. Crispr, which scientists began to use in human cells about two years ago, has impressed researchers with its low cost and ease of use.
Crispr, often called Crispr-Cas9 by researchers, stands for clustered regularly interspaced short palindromic repeats. It acts like a pair of molecular scissors, allowing scientists to precisely cut out faulty sections of DNA that can lead to serious illnesses and replace them with healthy ones. In a two- part process, an RNA "guide" molecule first locates the targeted part of the genome, then a Cas9 protein attaches to the DNA to make the cut. In some cases, researchers just want to remove a bad section; in other cases, they can replace it with a new, good strand of DNA.
"The old technology was clunky and it literally might take us a year to get to a point that Crispr could do in a month — the difference is an order of magnitude," said Paul Knoepfler, an associate professor of cell biology at the University of California at Davis. "I've never really seen something spread that fast and become so ubiquitous in science."
'Knock-out' animals
Beyond fixing mutations directly, Crispr and other gene-editing technologies have far broader applications. Researchers use Crispr to make "knock-out" animals to study what happens when specific genes aren't working. Merck and AstraZeneca Plc use Crispr to produce cells in which certain genes are switched off, letting the drugmakers test new treatments or learn more about the repercussions of a gene variant.
