SNITTS conference, Innovation by Collaboration September 22 nd, 2017, Stockholm Theme: Innovative processes for knowledge utilization

Transcription

1 SNITTS conference, Innovation by Collaboration September 22 nd, 2017, Stockholm Theme: Innovative processes for knowledge utilization Knut J. Egelie, PhD candidate CIP NTNU Head of IPR Management, NTNU Technology Transfer

2 CRISPR and patents Licensing - a tool for access to university knowledge

3 Overview 1. What is CRISPR and why is it important? 2. What is the status of CRISPR intellectual property rights? 3. How is licensing of the CRISPR system being conducted? 4. Should a different approach be taken for the licensing of CRISPR? 5. Do past practices provide useful perspectives for licensing CRISPR? 6. What possibilities exist for future dissemination of CRISPR?

4 Access to disruptive technologies are research critical

5 Access to biomedical research is a current challenge Egelie et al, Nature Biotechnology Oct. 2016

6 The rise of biotechnology, accompanied by a rise in patent filings especially by universities. University patenting has created concerns about their effect on progress of science yet, established grounds on which to develop products and services of enormous benefit to society

7 What is CRISPR and why is it important? (Clustered, Regularly Interspaced, Short Palindromic Repeats) Acronym for component of a bacteria system that enables single-celled organisms to defend themselves against invading bacteriophages A genome editing tool: The Holy Grail of biotechnology. Can be programmed to target ANY possible matching DNA sequence in the genome of ANY species, and initiate a wide range of changes in the genome. CRISPR will affect almost every aspect of life, and provide inspiration for future technological breakthroughs Jennifer Doudna, professor, UC Berkeley, and one of the inventors of CRISPR gene editing

8 (Clustered, Regularly Interspaced, Short Palindromic Repeats) Cheap easy safe fast Global investment community anticipates a multi-billion dollar industry to be developed Patents are at the core Image credit: Genome Research Limited.

9 The conflict over foundational CRISPR patents between Berkeley and MIT/Broad Institute A patent dispute between three inventors and their institutions is making concerns in the biotech community Understanding several facets of innovation barriers, patent law and history may provide some lessons about the probable - and best - outcome for the dispute.» J. S. Sherkow, Nature 2015 = uncertainty during the formative period of technology adoption regarding who, if anyone, holds rights to the core CRISPR invention and thus control over the technology platform. How is this solved? We propose through various licensing schemes.

10 What is the status of CRISPR intellectual property rights?

11 High intensive patent landscape We found in total 1,694 CRISPR/Cas9 patent families, by end 2016

12 Top CRISPR patent assignees (patent owners) FENG ZHANG, Broad Institute and MIT JENNIFER DOUDNA, UC Berkeley, and EMMANUEL CHARPENTIER, University of Vienna

13 How is licensing of the CRISPR being offered? Both Broad Institute and UC Berkeley, offer free use for academic research purposes through a nonprofit clearinghouse.

14 How is licensing of the CRISPR being offered? Patents has been licensed to several companies. Greatest commercial potential in the field of human therapeutics. Broad Institute and UC Berkeley has only granted exclusive licenses in this field of use. Exclusive license agreement between Broad Institute and Editas Medicine for human therapeutics, and that exclusive license has granted only one sublicense. UC Berkeley and the University of Vienna granted an exclusive license in all fields of use to Caribou Biosciences, and that licensee granted an exclusive sublicense to Intellia Therapeutics in the field of human therapeutics.

15 How is licensing of the CRISPR being offered? University of Vienna co-owns patents licensed by UC Berkeley to Caribou Biosciences. Granted exclusive license to ERS Genomics in all fields of use except human therapeutics and exclusive license to CRISPR Therapeutics for human therapeutics. The licensing landscape is complex for many uses for CRISPR within human therapeutics. In each instance, the exclusive licensing to a spin off company from the university and one or more of its researchers has concentrated control of the use of CRISPR in a for-profit entity. Both short- and long-term goals likely to be in conflict with the broad dissemination of the CRISPR-Cas9.

16 Should a different approach be taken for the licensing of CRISPR? Enabling technology that provides methods and tools for gene editing broadly. Should such technology be licensed exclusively whatever company? Likely increased interest in licensing the UC Berkeley patents by any commercial entity that wants to utilize CRISPR. Industry wanting to use the UC Berkeley CRISPR in human therapeutics will have to approach Intellia Therapeutics or CRISPR Therapeutics for a sublicense. May be necessary for a commercial licensee also to obtain a sublicense from the Broad Institute s exclusive licensee, Editas Therapeutics. The various universities have concentrated control of the patents in three spin offs - their respective licensees in the field of human therapeutics.

17 Do past licensing practices provide useful perspectives? 1. Cohen and Boyer: recombinant DNA in bacteria, at Stanford and UC San Francisco, Axel: Genetic engineering and co-transformation of eukaryotic DNA in higher organisms, at Columbia University, RNAi: RNA interference to control gene expression, at four universities, In each of these cases, broad, non-exclusive licensing by the universities made the technologies available for both academic research use and commercial use (albeit, on differentiated terms).

18 The Stanford model Catalytic for the biotech industry. Recombinant DNA is one of the most important technologies licensed by Stanford. Under Stanford s licensing program patents were non-exclusively licensed to both large and small industries. Licenses were granted to 468 companies. Estimated that total sales volume of products based on this platform technology exceeded $35 billion. Stanford and UCSF has accrued $255 million in royalty income.

19 With the courtesy of Prof. Greg Graff Then and now Non-exclusive licenses Surrogate companies Cohen-Boyer recombinant DNA CRISPR gene editing Founded by Feng Zhang Founded by Jennifer Doudna Many competing non-exclusive licenses Few, field-specific exclusive licenses Few, field-specific exclusive licenses Founded by Herbert Boyer

20 The big question: What happens when researchers at other universities (like ) make potentially valuable discoveries of their own, using the CRISPR research tool they obtained for nonprofit- or research-use only? What are their options for commercializing follow-on inventions?

21 Possibilities for future dissemination of CRISPR Academic technology transfer stresses the creation of start-up companies where the inventions are exclusively licensed to the start-up company. Non-exclusivity Works for inventions that are developed by a single entity that can focus all of its efforts on one or a few projects. Non-exclusivity Such companies now play a major role in the development of human therapeutics that are then acquired and commercialized by major pharmaceutical companies. Non-exclusivity Does this make sense for an enabling technology like CRISPR? Non-exclusivity CRISPR should be widely disseminated to not only the non-profit but also the for-profit sector. Non-exclusivity Only then to expect a forecast explosion in gene editing, and only then is the promise of this ground-breaking technology likely to be realized. Non-exclusivity Hsu et al., Cell, 2014

22 Today None of the players involved with CRISPR are discussing plans for a broad, non-exclusive, commercial licensing program that would make the technology platform widely and efficiently available on fair and reasonable terms for the many competing commercial applications that are likely to arise.

23 Future concerns What would the world have looked like if Genentech had been granted exclusive use of the Cohen-Boyer technology from Stanford, and Genentech itself was responsible for extending any commercial sublicenses to other competing biotech companies? Would we have the same biotech industry that we do today?

24 Future concerns In the past, it was the university licensing offices that stood somewhat above the competitive fray and forged workable licensing programs that struck a balance, however, imperfect, between commercial control and academic access to the breakthrough technology

25 Future concerns In the case of CRISPR, it appears that the university licensing offices have already abdicated playing such a role, by handing over exclusive rights to venture-capitalbacked commercial entities.

26 Future concerns How likely is it that those commercial entities now in control will decide that broad, non-exclusive sublicensing is a viable business model? This is where universities and governments should explore access and sharing models in knowledge utilization.

27 Thank you Knut J. Egelie, PhD candidate CIP NTNU Head of IPR Management, NTNU Technology Transfer For more, see our article in October 2016 issue of Nature Biotechnology