Broad Institute will retain CRISPR patents, ruling says

The Broad Institute in Cambridge, Massachusetts, will retain potentially lucrative rights to a powerful gene-editing technique that could lead to major advances in medicine and agriculture, the federal Patent and Trademark Office ruled Wednesday.

The decision, in a bitterly fought dispute closely watched by scientists and the biotechnology industry, was a blow to the University of California, often said to be the birthplace of the technique, which is known as CRISPR-Cas9.

An appeals board of the patent office ruled that the gene-editing inventions claimed by the two institutions were separate and do not overlap.

The result is that the Broad Institute, a research center affiliated with MIT and Harvard, gets to retain more than a dozen patents it has already been granted on the use of the Crispr technique to modify DNA in the cells of humans, animals and plants.

“It seems to be a decisive victory for the Broad Institute,” said Jacob S. Sherkow, an associate professor at New York Law School who has followed the case closely.

The Broad Institute, in a statement, said it agreed with the decision. Officials at the University of California said they were considering an appeal, although they noted the ruling left the door open for the university to obtain its own patents covering the use of Crispr for all types of cells.

“They have a patent on green tennis balls; we will have a patent on all tennis balls,” Jennifer Doudna, a professor at the University of California, Berkeley, said in a phone call with reporters. She has been widely credited as an inventor of the gene-editing technique.

Ultimately, companies wanting to apply Crispr for use in medicine, agriculture or other fields might need licenses from both the Broad Institute and the University of California, a lawyer for the university said. However, Sherkow said he was doubtful that the university could obtain a broad patent, given the wording of Wednesday’s decision.

Crispr has generated excitement among biologists because the technique makes it relatively simple to change particular letters in an organism’s DNA, much like using word processing software to search for particular words and then deleting or replacing them.

That could allow for the development of new disease treatments that would fix defective genes in the human body. Crispr could also make it more practical to alter the DNA of human embryos, making changes that could be passed to future generations.

An influential national advisory panel said Tuesday that such heritable changes should be allowed only in the narrowest of circumstances, to prevent infants from acquiring genes that would cause serious diseases, and only when there was no alternative.

If Crispr lives up to its promise, the patents on the technique could be worth hundreds of millions of dollars, or even more. However, related techniques, such as gene therapy and RNA interference, have also been described as having great promise and have resulted in few or no medical treatments so far.

Several companies are trying to develop medical treatments using Crispr, although they are in the early stage of development.

The stock of Editas Medicine, which holds licenses to the Broad patents, shot up nearly 30 percent Wednesday. By contrast, shares of Intellia Therapeutics and Crispr Therapeutics, which have licenses to the intellectual property from Doudna and her colleagues, each fell nearly 10 percent.

Those two companies, and others, may now have to negotiate licenses to the Broad patents, most likely by paying fees and royalties. It is rare in the medical field for one patent holder to block another party from bringing a medical treatment to market.

There have been some exceptions, such as Amgen’s current effort to block a rival cholesterol-lowering drug. But the Broad Institute and the University of California have given every indication that they want Crispr to be applied widely for public benefit, so the ruling Wednesday is unlikely to slow down research.

Crispr is an acronym for a natural process used by bacteria to fight viruses. Doudna, along with Emmanuelle Charpentier, now of the Max Planck Institute for Infection Biology in Germany, helped figure out how the process worked and demonstrated that the system could be used to cut DNA at any particular point in an organism’s genome.

Their initial demonstration of the editing technique used chemicals in a test tube. Still, the publication of their paper in the summer of 2012 set off a rush to apply the technique to plant, animal and human cells.

Several months later, Feng Zhang, a young scientist at the Broad Institute, was one of the first to accomplish the task. In a surprise to researchers in the field, the Broad Institute was granted a series of patents covering the use of the technique in cells that have nuclei, including human cells and plant cells.

The University of California, along with Charpentier and the University of Vienna, where she once worked, challenged the patent award, and the patent office began a procedure, called an interference, to determine the rightful inventor.

The University of California and its allies argued that once the technique had been demonstrated in the test tube, it was obvious that Crispr editing could be applied in human and plant cells with a little tweaking. The Broad Institute countered that significant inventive work was required by Zhang to get the technique to work in cells with nuclei.

On Wednesday, a panel of three judges of the Patent Trial and Appeal Board sided with the Broad Institute, saying that the paper by Doudna and Charpentier did not provide a “reasonable expectation of success” that the technique would work in plant, animal and human cells.

In a 51-page decision, the judges cited statements made in 2012 by experts in the field, including Doudna, saying that it was not yet known if the technique would work in human cells.

Still, Doudna and Charpentier have won various awards, a sign that many peers consider them to be pioneers in Crispr gene-editing. It remains to be determined if they can get their own patents and, if so, how broad they will be.

© 2017 New York Times News Service