CRISPR: A licence to heal

Few areas of emerging research have generated as much excitement in the life sciences community as CRISPR/Cas9, a gene-editing system which relies on the cas9 enzyme to alter DNA. But who invented it and who owns it?

Life sciences experts are convinced by the revolutionary technology’s ability to put gene-editing at the heart of healthcare, making the associated IP an extremely valuable asset. Claims of ownership are far from resolved and have been the subject of protracted legal battles in the US and Europe.

An impending interference proceeding at the US Patent and Trademark Office (USPTO) will be critical in determining who lands the US rights to a key branch of the technology. There are few more qualified to shed some light on the matter than Eric Rhodes, chief executive officer of ERS Genomics.

Based in Dublin, ERS licenses patent rights to CRISPR/Cas9 and is a key player in the CRISPR IP sphere.

“In its simplest terms, what CRISPR allowed us to do is modify DNA sequences within cells and organisms,” Rhodes says. While CRISPR is not the first genome-editing tool of this kind, its efficiency and ease of use set it apart, he explains.

The legal superstructure that has been constructed around CRISPR and its associated IP can appear at a glance to be as complex as the science itself. For a start, who invented the technology and who owns the basic foundational IP is the subject of protracted legal battles.

On one side, there is the University of California (UC), the University of Vienna, and Emmanuelle Charpentier; on the other, the Broad Institute, a collaboration between the Massachusetts Institute of Technology and Harvard University.

Each side owns its own patents, and each side has assigned those patents to a curated network of licensing companies that manages the rights to CRISPR for different applications. One of those companies is ERS Genomics, which was created by Charpentier to provide “broad access” to CRISPR/Cas9.

"There's always been the questions of whether, in addition to the UC portfolio, you also need a licence to the Broad Institute's patents." - Eric Rhodes, ERS Genomics

ERS doesn’t own the rights to the technology—the company has been assigned Charpentier’s patent rights for all applications of CRISPR/Cas9 other than as a human therapeutic.

While human therapeutics are arguably the “most important” application of CRISPR, Rhodes acknowledges, the applications covered by ERS’s licenses have groundbreaking potential.

CRISPR’s ease of use and efficiency, Rhodes says, have allowed gene-editing to broaden out into a range of applications that were previously too expensive or technically challenging.

These include areas such as livestock, plant agriculture, and veterinary medicine, and there could be direct benefits to human medicine as well. Rhodes cites one example of CRISPR being used to develop rodents with immune systems that mirror that of humans.

Licensing focus

Amid the ongoing battle between scientists and research institutions, all seeking to prove who first invented and developed the technology, ERS stands out as a key pillar in the CRISPR landscape, but one which does not engage in scientific research.

“We don’t have labs, we don’t do R&D—we do licensing,” Rhodes says. As the global battle for patent ownership shows, however, IP is arguably the most valuable asset associated with CRISPR.

“We take the patents and make the rights available,” he says. In Rhodes’ words, the company sees itself as simply “enabling” others to commercialise Charpentier and her co-inventors’ work and bring it to market.

ERS lives and breathes IP. In spite of this, however, it doesn’t have an in-house patent team of its own. Rather, it shares external counsel with some of the other companies in the UC network and works closely with in-house counsel at Caribou, Intellia, and CRISPR Therapeutics.

“We’re essentially five or six people; we have fewer internal resources compared to some of the human therapeutic focused companies,” Rhodes insists.

Close cooperation between this group is essential the context of the ongoing patent battle with the Broad Institute. The latest battleground between the factions is an interference proceeding instituted by the USPTO in June.

Under this interference the USPTO will rule on which side first invented CRISPR/Cas9 technology for use in eukaryotic cells (cells which have a nucleus enclosed inside a membrane).