LSIPR 50 2018: It’s not like selling cars

Managing and licensing the IP portfolio of a biomedical research centre as prestigious as the Broad Institute of MIT and Harvard may come with any number of challenges, but for Issi Rozen there is one key question he tackles in his day-to-day work as chief business officer: how can the Broad’s proprietary technology have the maximum impact possible, in a responsible way?

“Our mission is to propel the understanding of the human genome and how we understand human health and disease,” he says, explaining that when the Broad issues a licence it has to be sure that its use will support that mission.

Rozen previously worked in the biotech industry, including in startups such as venture-backed Resolvyx Pharmaceuticals, where he led the business development and partnering efforts.

“Most of the time the mission was to maximise profit, but it turns out it’s much more complex to maximise impact,” Rozen says. Maximising profit is “easy, if the numbers are right”, but there are lots of different frameworks to consider and balance when trying to maximise impact.

For example, considering whether a right should be exclusive, who could be the best party to develop something, and how the relevant data will be made available as soon as possible are just some of the elements the Broad has to assess when working out how its research tools can have the biggest impact.

CRISPR responsibility

The Broad is renowned for its proprietary CRISPR technology, which allows researchers to alter DNA sequences and modify gene function. The potential of the tool is huge: it can accelerate life sciences research, improve biotechnology, and treat human diseases.

But when licensing the technology, the Broad is conscious of the fact that it could have potentially negative uses.

The institute doesn’t grant CRISPR licences for germline DNA editing, for example, and licences relating to agricultural applications of the technology do not permit purposes relating to tobacco or sterile seeds.

“We place restrictions on certain licences to make sure our technology is used in a socially responsible way,” he explains.

An equally important consideration relating to the Broad’s social responsibility centres on whether licences should be exclusive. Rozen explains that the institute has many policies around this, including the inclusive innovation model.

Under this model, CRISPR/Cas9 technology was licensed to primary licensee Editas Medicine. Editas had the right to exclusively use the technology on targets of its choosing for the development of genomic medicines.

When that period of exclusivity expired, other parties applied to license the technology for use against genes of interest which are not being pursued by Editas.

The model incentivised Editas to devote investment into developing CRISPR-based genome-editing technology to treat a multitude of diseases by increasing the efficiency and accuracy of gene insertion or correction, while also supporting the wider development of other medicines which will reach a wider pool of patients.

Editas currently uses CRISPR technology to treat rare diseases such as Leber congenital amaurosis type 10, an inherited eye disease which causes significant vision loss, and has also been experimenting with the tool to develop gene editing strategies for haematological disorders such as sickle cell disease.

Balancing the incentive to innovate with wide-scale access to the Broad’s research tools is the “safe and responsible” way to maximise the impact of CRISPR technology by creating “a much more robust availability for development”, according to Rozen.

Called into question

In January this year, the Broad’s patent portfolio suffered a setback in Europe when one of its patents covering the CRISPR/Cas9 technology was revoked by the European Patent Office (EPO), as it found the Broad could not claim two key priority dates.

Rozen says that while “it’s always disappointing when a patent is called into question”, the EPO revoked only one patent, in Europe, and the Broad is appealing what it has termed merely a “technical” decision.

The decision doesn’t affect the Broad’s US patent with the same claims, and the remainder of the Cas9 series of patents in Europe is also not impacted by the decision. It doesn’t affect the Broad’s other series of CRISPR technology, CPS1, so although the decision is disappointing, Rozen says that overall “most of the portfolio is untouched”.

In February 2017, the US Patent Trial and Appeal Board (PTAB) ruled that its CRISPR patent, which had been challenged by the University of California, Berkeley, was valid. The board said the Broad’s patent, which covered CRISPR editing in eukaryotic cells, did not interfere with patent claims filed by UC, Berkeley and the University of Vienna.

Both parties were able to retain their CRISPR patents, allowing the Broad to “continue to optimise and improve the technology” in the US. UC, Berkeley has appealed the finding.

Partner up

A key part of Rozen’s day-to-day work centres on developing partnerships between the Broad and those in industry. By collaborating with pharmaceutical companies, the Broad is able to combine its technologies with the strength of industry to develop novel targets for drug discovery, ultimately advancing the early stage entry of drugs on to the market.

Rozen says genomics data analysis is becoming more important, and the Broad has numerous collaborations with data technology companies.

“The world is exploding with data,” he says, and the Broad is “developing the right systems to be able to store, analyse, and use” that data to develop products that help patients.

In October last year the Broad announced it had teamed up with DuPont Pioneer. Under the agreement, the parties will jointly offer non-exclusive licences to foundational CRISPR technology for use in commercial agricultural research.

The partnership is expected to prompt progress in the industry which will ultimately result in benefits for the global food supply, such as higher and more stable yields for farmers and a more sustainable agricultural system overall.

Rozen says the way Broad partners with other organisations, and elects to share its innovations non-exclusively, is contributing to the rise of responsible licensing. Going forward, he would like to see responsible licensing applied more broadly by those who own the rights to impactful inventions such as CRISPR.

“Life sciences is a unique place,” he says. “On the one hand, biotech companies are for-profit entities with shareholder responsibilities, but on the other they’re developing products which have a major impact on health.”

Balancing the duty to shareholders with the public responsibility to allow access to lifesaving treatments is something most companies take seriously, Rozen says. Overall he thinks the industry is doing a “pretty good job” of aligning the incentives with the public responsibility.

Whatever the outcome of the disputes at the PTAB and the EPO, there is no doubt that the institute will continue to develop strategic partnerships and leverage its IP portfolio to ensure that CRISPR technology has the most positive impact possible, in the most responsible way.

“We’re not selling cars, we’re selling products that will save lives,” concludes Rozen.