A day after experts weighed in on the ethical concerns of CRISPR, the U.S. Patent Trial and Appeal Board (PTAB) has delivered its ruling on the high-profile patent interference case shadowing the field.
The three judges determined that there was “no interference-in-fact.”
This means that although the Broad patents were filed after the University of California’s, they are deemed to cover their own intellectual real estate. The two patents can theoretically coexist.
Wednesday’s decision is a win for The Broad Institute and MIT, the institutions representing the work of Feng Zhang. If the verdict had gone the other way, their patents could have been invalidated.
It also comes as a major relief for Editas Medicine, the now-public company resting on the Broad’s intellectual property. Editas shares spiked nearly 30 percent following the news.
For the companies that have licensed Jennifer Doudna and Emmanuelle Charpentier’s foundational CRISPR discovery, the outcome is less clear-cut.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
“To us, the ruling by the PTAB today is a narrow ruling,” said Samarth Kulkarni, chief business officer of CRISPR Therapeutics. “All they’ve said is that the eukaryotic application or practice is separately patentable. This does not mean they have priority. It also does not mean that they have taken anything away from us.”
Brent Babcock, a partner at the intellectual property law firm Knobbe Martens, shared a similar sentiment in a separate call.
“This is more of a middle-of-the-road decision where Broad keeps what it has, Berkeley keeps what it has, and if someone wants to practice both they have to go to both.”
Doudna and Charpentier are widely credited as the inventors of CRISPR-Cas9 through their work at the University of California at Berkeley. They filed patents describing the use of the gene-editing technique in cell-free systems.
Zhang’s team filed later but made an important leap, outlining how CRISPR can be applied to eukaryotic cells. It is that step that enables the development of human therapeutics, the highest-potential field.
Despite the slow start, the Broad’s patents were issued before the University of California’s. This lead to the interference, which sought to determine if there were intellectual grounds for both patents to exist.
In fact, there is.
Per the judges’ order, companies developing human therapies that rely on CRISPR-Cas9 will likely need to license technology from both sides of the table.
The markets reacted quickly and sent CRISPR Therapeutics’ shares down nearly 30 percent. They recovered significantly over the course of the afternoon.
There are many mitigating factors that dulled the blow for companies aligned with the University of California.
The first is that the ruling applies only to the United States. The rest of the world practices a first-to-file system, which recognizes Doudna and Charpentier as the undisputed inventors.
For both sides, an R&D exemption is in place to allow the companies to progress their science irrespective of the patent disputes. Any IP restrictions would only arise once a drug is approved — likely not until the early 2020s. There are plenty of arguments to be heard before then.
The R&D agreement also hints at a larger theme. While a winner-takes-all, cut-throat battle might make for a good story, the CRISPR contest has been pragmatic.
Doudna and Zhang cofounded Editas Medicine together in 2013 before the patent interference began.
In 2014, Zhang told Bio-IT World that he felt it was important to keep the technology open for the wider scientific field.
“Something like CRISPR is a foundation tool,” Zhang said. “I think making sure these foundation tools are open is important… What my lab has done is try to make the information, the technical know-how, as well as the physical reagents, as accessible to everybody as possible.”
A similar sentiment was echoed in the Broad Institute’s statement on the ruling.
“We believe CRISPR should continue to be available to the global scientific community to advance our understanding of the biology and treatment of human disease, and to help lay the groundwork for a new generation of therapies.”
Kulkarni did not sound concerned on the phone. His company may have to cross-license technology from The Broad, but it has also secured its own IP.
Science involves standing on the shoulders of other scholars. This means most inventions are incremental and the resulting patents are a blurry field.
With CRISPR, there is enough potential to go around. There may be a few licensing fees to pay, but it remains an open field. The science will succeed.
Photo: Chris Ryan, Getty Images
