The Patent Fight Over Gene Editing Technology

March 13, 2017

In biotechnology, the ability to program genes is big business.  A classic example is synthetic insulin for diabetics, which is made in bacteria using a circular piece of DNA called a plasmid.  The engineered plasmid contains the DNA sequence for human insulin and tells bacterial cells how to use that DNA to produce the hormone.

CRISPR, short for Clustered Regularly Interspaced Short Palindromic Repeats, is an assortment of enzymes isolated from bacteria that allow scientists to “cut and paste” DNA sequences in a very accurate and precise manner. In contrast to the insulin plasmid example, CRISPR can be used to edit the chromosomal DNA of an organism allowing a scientist to rely on the cell’s natural capabilities to express its genes. 

While CRISPR technology holds great promise, there is a dispute between two groups as to who owns the rights to exploit it.  The University of California group, led by Dr. Jennifer Doudna and Dr. Emmanuelle Charpentier, filed a patent application in May 2012, claiming the application of CRISPR in prokaryotic cells, that is, simple cells like bacteria. The MIT group, led by Dr. Feng Zhang, filed a patent application in December 2012, claiming the use of CRISPR in certain eukaryotic cell types, such as mice or human cells.  Both patent applications were limited to a variant called “CRISPR-cas9,” which refers to an enzyme used to cut DNA.

Through a quirk in patent procedure, the MIT patent was granted first, despite being filed later. MIT’s patent issued on April 15, 2014, while the California group’s earlier-filed application remains pending.

Since the America Invents Act took effect in 2013, the patent office presumes that the first inventor to file a patent application is entitled to a patent. Here, both applications were filed prior to 2013 and were thus subject to the old rule that the first inventor, not necessarily the first one to file, is entitled to the patent. The patent office’s interference proceeding is designed to resolve such priority disputes. 

In the CRISPR case, the University of California group argued that MIT’s patent claimed the same material as the UC patent application. The patent office determined that the parties claimed distinct technologies, and terminated the proceeding.  The UC group is entitled to appeal, but have not announced a decision to do so.

The patent office’s ruling allows both patents to co-exist. With billions of dollars tied up in CRISPR research, patent licensing gets messy, particularly where the patent claims are similar. Each group already has licensing arrangements in place with other entities. MIT created Editas Medicine to exploit the CRISPR technology.  The UC group has created Caribou Biosciences to do the same.  For companies that already have licenses from one group, the patent office’s decision raises the possibility that additional licenses may need to be negotiated from the other group to avoid infringement. 

Basic research may be impacted if universities must obtain costly licenses. In the past, the University of California has opted to provide free patent licenses to non-profit research entities, while charging a fee to for-profit enterprises.  MIT and the University of California may wish to create a cooperative licensing arrangement to make sure that research can continue unencumbered.  In other industries, licensing groups of patents directed at the same technology is a common way to promote efficient commercialization and to avoid wasteful infringement litigation.

That said, scientific progress will likely mitigate any restrictive effects as well. CRISPR-cas9 is already not the only game in town. Research on CRISPR-cpf1, which uses a different DNA-cutting enzyme, has yielded promising results, and is not covered by either of the CRISPR-cas9 patents.