This is a guest post from Susan K Finston, President of Finston Consulting. Do you have a response to Susan’s post? Respond in the comments section below.
A common first reaction to crowdfunding for life sciences may be to dismiss it out of hand. Given the undeniable chill in early stage funding, it is worth giving the idea a more serious look. Or, in the immortal words of Sherlock Holmes, “When you have eliminated the impossible, whatever remains, however improbable, must be the truth.”
The improbable truth is that Kickstarter has helped to raise millions through crowdsourcing to fund creativity in the arts and digital sciences. In 2012 alone, Kickstarter helped to raise over $100 million for new games projects. Most recently, The Veronica Mars Movie Project closed out its Kickstarter funding campaign successfully, far exceeding its goal with pledges of over $5.7 million raised from fans in 30 days. Every Kickstarter project, though, must fit into fixed categories. Under the heading of “What is not allowed,” Kickstarter states that “Projects cannot offer financial, medical, or health advice.” Like Seinfeld’s Soup Nazi, Kickstarter has ‘no soup’ for projects related to the innovative life sciences.
So crowdfunding portals for bio-pharma and medical devices are springing up like new grass after the rain in the U.S. and overseas, hoping to replicate Kickstarter’s success. As expressed by Sarah Lacy of Pandodaily:
“Hey, it worked for watches and robots on Kickstarter. And if we can donate $100 to finance a watch we may never see or an indie movie that does little for mankind, is it so crazy to take a flier on cancer research?”
The real challenge is how to choose among the alternatives – mainly recent entrants without a track record of raising the kinds of funds needed for translational research. indiegogo, although not a science-dedicated site, may have raised the most for R&D, helping the iCancer project to raise over $150,000 (though falling far short of a $1m goal, the non-profit has received funds pledged). The most well-wired and promoted of the dedicated crowdfunding sites may be MedStartr, which has adopted the “Startr” moniker that Kickstarter began with (before adding the “e”). Other U.S.-based dedicated crowdfunding sites that are science-dedicated include Microryza iAMscientist, PetriDish (primarily natural sciences) and SciFlies. Based on my initial review of the alternatives, Microryza stands out from the crowd due to the direct experience of the founders that led to the start of the company, and with an approach that is both engaging/accessible and provides gravitas to research projects on the site.
Time will tell whether bio-entrepreneurs can leverage crowdfunding for successful fundraising, and which of the current (or future) crop may become the Kickstarter of innovative life science. Stay tuned!
Susan K. Finston is President of Finston Consulting LLC, and, together with biotechnology pioneer Ananda Chakrabarty, is co-founder of Amrita Therapeutics Ltd., an emerging biopharmaceutical company based in India with cancer peptide drugs entering in vivoresearch. She is currently preparing to launch her first Crowd Funding campaign for Amrita Therapeutics first-ever therapeutic oncology medical device. For more information see AmritaTherapeutics.com or FinstonConsulting.com.
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ABSTRACT: The two leading therapeutic areas for biological products, in terms of current sales and pipeline focus, are oncology and AIID (arthritis, immune and inflammatory disorders). Datamonitor's biotechnology strategic market analysis team recently analysed the AIID market, since this sector is currently powering biotechnology market growth, owing to high demand for biologicals to treat rheumatoid arthritis and psoriasis (Belsey, M...
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This is a guest post by Morrison & Foerster‘s Marc A. Hearron, James J. Mullen, III and Matthew I. Kreeger. Do you have a response to this post? Sound off in the comments section below.
The Supreme Court of the United States recently heard oral argument in Association for Molecular Pathology v. Myriad Genetics, Inc. (No. 12-398) to decide the question, “Are human genes patentable?” The Court’s decision in Myriad could have broad implications for biotechnology companies. Morrison & Foerster was present at the argument.
Although one must be cautious about reading tea leaves from oral argument, a majority of Justices at the argument seemed skeptical that isolated human genes are patentable subject matter.
Myriad Genetics, Inc. is the patentee of several U.S. patents with claims directed to the human genes BRCA1 and BRCA2. The presence of mutations in these genes is highly correlated with the risk of developing breast or ovarian cancer. A coalition of groups and individuals brought a declaratory-judgment action over the patentability of the BRCA1 and BRCA2 claims.
A divided Federal Circuit panel held that claims covering isolated DNA sequences are patentable subject matter under 35 U.S.C. § 101. In March 2012, the Supreme Court sent the case back to the Federal Circuit for that court to reconsider its decision in light of the Supreme Court’s decision in Mayo Collaborative Services v. Prometheus Laboratories, Inc., 132 S. Ct. 1289 (2012). In Mayo, the Supreme Court reaffirmed the principle that laws of nature are not patentable.
On remand, the Federal Circuit wholly reaffirmed its prior ruling. The challengers petitioned the Supreme Court for review. The Supreme Court granted certiorari to decide one question: “Are human genes patentable.”
THE BRIEFS BEFORE THE SUPREME COURT
The plaintiffs assert that Myriad “did not invent any genes or variants or cause their significance” and that Myriad’s patents “cover the BRCA genes of every person in the United States, even genes that Myriad has never seen.”
Because human genes and genetic variants of those genes are “products of nature,” they are not eligible for patenting. The fact that the BRCA genes have been isolated from the human body makes no difference because under that rationale, “a kidney ‘isolated’ from the body would be patentable, gold ‘isolated’ from a stream would be patentable, and leaves ‘isolated’ from trees would be patentable.” The challengers’ position is supported, in whole or in part, by 24 amicus briefs, including briefs by the American Medical Association, AARP, and the American Intellectual Property Association.
Myriad contends that the claimed isolated DNA molecules “fall on the inventive side of the line” drawn by Section 101 and Supreme Court precedent. According to Myriad, “(o)nly by human intervention have the claimed molecules come about.” “Where others failed, Myriad identified the BRCA genes, and then, using information it had collected and discerned from studying the genes, characterized, defined, and isolated these particular molecules. The creation of new molecules never before available to the public is invention.”
Myriad’s position is supported by 26 amicus briefs, including briefs by the American Bar Association, the Biotechnology Industry Organization (BIO), the Pharmaceutical Research and Manufacturers of America (PhRMA), and a number of pharmaceutical companies.
The Solicitor General filed an amicus brief on behalf of the United States, nominally supporting neither party, but which, in practical terms, is seen as supporting the challengers. In a departure from the position of the Patent Office, the Solicitor General argues that “isolated but otherwise unmodified DNA is not patent-eligible.” According to the Solicitor General, the “public’s ability to study and use native DNA would be unduly compromised if changes caused by the extraction of naturally-occurring substances from their native environments were sufficient to trigger patent-eligibility.” The Solicitor General departs from the challengers when it comes to “complementary DNA” (cDNA) molecules, which the government describes as “synthetic molecules built by scientists to include, in a single contiguous DNA segment, only the exons of a naturally occurring gene, without the introns and regulatory regions that are normally interspersed with exon sequences in genomic DNA.”
The Justices’ questioning at oral argument suggests that a majority of Justices seem inclined to agree with the challengers’ argument that isolated human genes are not patent-eligible. As to cDNA, however, several Justices suggested that it is the product of human invention and would be eligible for patenting under Section 101, although it may or may not be patentable under other doctrines such as obviousness.
The Challengers’ Argument
Counsel for the challengers began his argument by asserting that Myriad invented “nothing.” The decisions as to what the genes contained were “made by nature,” not by Myriad. Myriad merely “unlocked the secrets” of the genes; it did not invent them.
A number of Justices asked the challengers to clarify exactly what they contend is and is not patentable. Justice Sotomayor asked why the test for the presence of the BRCA genes had not been patented. Justice Scalia asked why the method of isolating the genes was not patented. Justice Kennedy asked whether the challengers were asserting that the process of “tagging” the isolated DNA was not patentable. Counsel answered that the method was patented but had been freely licensed for years, and he clarified that the challengers were not asserting that the process of tagging the DNA could not be patented.
Justice Alito was one of the only Justices to ask questions at oral argument suggesting outright agreement with Myriad that isolated genes can be patented. Justice Alito asked how the isolated DNA was different from a plant in the Amazon that is discovered to have therapeutic properties but which requires a chemical to be extracted and concentrated. Counsel agreed that the process of concentrating the substance might make it patentable. Justice Alito suggested that that was no different from isolating the gene because both the isolated gene and the concentrated plant substance have a different “function” and are in a new “form.”
Several Justices, including Justices Scalia, Kennedy, and Kagan, asked whether there would be sufficient incentives for biotechnology companies to perform the type of work that Myriad performed if the genes are not patentable. Justice Sotomayor stated that the isolated gene itself “has no value,” but rather it is “the use you put the isolation to” that has value.
As to cDNA, the Justices were far less receptive to the challengers’ argument. Justice Sotomayor stated that cDNA is “not a product of nature; it’s a product of human invention.” Justice Breyer stated that there is “no such thing in nature” as cDNA and that cDNA has properties that are not true of the isolated DNA. Justice Kennedy suggested that cDNA has features that regular DNA does not. Counsel repeatedly tried to assert that cDNA is found in nature, but he appeared to make little headway.
The Solicitor General’s Argument
Solicitor General Donald Verrilli himself argued as amicus on behalf of the United States, reflecting the importance of this case. The Solicitor General’s middle-ground position—that isolated genes are not patentable but that cDNA is—appeared to be carrying the day with several of the Justices.
The Chief Justice queried whether patentability under Section 101 was the proper way to consider these issues, suggesting that the doctrine of obviousness was the better course. He stated that taking a small part of something bigger is obvious, and thus it would be obvious to take an isolated gene from an entire chromosome.
“I don’t understand how a small part of something bigger isn’t obvious,” remarked the Chief Justice.
Nevertheless, the Solicitor General urged the Court to focus on patentability under Section 101 as it did in Mayo.
Justice Alito pointed out that the government has changed its position and that there are conflicting opinions within the Executive Branch. The Solicitor General acknowledged as much.
Justice Kagan brought up Justice Alito’s hypothetical about the Amazonian plant. The Solicitor General contended that the use of the substance in the plant would be patentable but that the substance itself would not.
Counsel for Myriad withstood a barrage of questioning about the patentability of merely isolated human genes, which several Justices stated are “found in nature.”
Justice Sotomayor, for example, likened the case to a new recipe for improved chocolate-chip cookies, stating that the cookie might be patentable because the inventor has done something new with the ingredients but that the basic ingredients themselves—salt, flour, eggs, and butter—could not be patented.
Myriad’s counsel argued that there was human invention in the decision where to begin the gene and where to end the gene—i.e., where to snip the gene from the rest of the chromosome. He likened isolating the gene to a baseball bat that has been isolated from the rest of a tree, stating that a baseball bat is found in nature but the decision where to start and end it is decided by humans. Justices Scalia and Breyer resisted the analogy, stating that this DNA is found in the human body. The Chief Justice also stated that the baseball-bat analogy is “quite different” because that is not just snipping. “Here,” he stated, “what’s involved is snipping. You’ve got the thing there and you snip—snip off the top and you snip off the bottom and there you’ve got it.”
Justice Kennedy remarked that isolated DNA is not useful until “tags” are added to it. He thus suggested that DNA that is isolated but not tagged is not different from how it exists in the body.
Justice Breyer stated that “the patent law is filled with uneasy compromises.” Returning to the hypothetical about the plant in the Amazon, Justice Breyer stated that the historically recognized compromise is that processes to extract the substance from the plant are patentable, that newly discovered uses of the substance are patentable, but that the substance itself is not. This “hornbook patent law,” he suggested, keeps substances themselves free of patent restrictions but encourages innovation to develop new uses for those substances.
Myriad’s counsel urged the Court to defer to the views of the Patent Office, which “sits at the intersection of law and science.” He pointed out that the Patent Office did not join the Solicitor General’s brief and has adopted the position that isolated genes are patentable. Justice Ginsburg responded that the federal government has disavowed the Patent Office’s position, and that “the strength of the presumption would be diluted” as a result.
Justice Kagan referred to the Patent Office as “very patent happy.”Justice Kagan asked whether the “first person who found a chromosome and isolated it” from the body could have patented chromosomes. She also asked whether “the first person who found a liver” could patent the liver.
Myriad’s counsel answered that these would be patentable under Section 101 but might not be under other provisions, such as 35 U.S.C. § 103. Justice Breyer responded that “that’s the problem” because it would mean “(a)nything from inside the body that you snip out and isolate” could satisfy Section 101. Justice Sotomayor added that “if you cut off a piece of the liver or a piece of the kidney,” that does not make it patentable, suggesting that the same should be true of a piece of a chromosome.
In the most explicit signs that the Court might accept the Solicitor General’s view, Justice Kennedy asked Myriad’s counsel whether, if the Court were to agree with the government, it would “give the industry sufficient protection for innovation and research.” And in the challengers’ rebuttal, Justice Sotomayor asked whether there would be “some value to us striking down isolated DNA and upholding the cDNA.”
Myriad had argued in its brief that the Federal Circuit erred in concluding that any of the challengers has standing. At oral argument, none of the Justices asked any questions about Myriad’s standing argument, suggesting that standing likely will not be a basis for the Court’s decision in this case.
One should not read too much into the Justices’ questions at oral argument because the Court does sometimes rule differently from how observers expect it to rule based on questioning. Many Justices, however, did seem to be searching for a middle ground in which isolated human genes could not be patented but syntheticDNA is patent-eligible under Section 101. A decision is expected by the end of June.
About the authors:
Mr. Kreeger is San Francisco-based chair of Morrison & Foerster’s Patent Interferences practice, representing clients in technology, life sciences, and medical diagnostics. Mr. Mullen, managing partner of the firm’s San Diego office, represents IP clients across a range of cleantech and biotech industries, including medical therapeutics and diagnostics and nucleic and amino acid sequencing. The other co-author is MoFo associate Marc Hearron, a member of the firm’s Appellate and Supreme Court group in Washington.
This is a guest post from Susan K Finston, President of Finston Consulting. Do you have a response to Susan’s post? Respond in the comments section below.
Even if you have never heard of Thomas Kuhn, your thinking about science has most likely been effected by his theory of what causes paradigm shifts.
Over 50 years ago, Thomas Kuhn published The Structure of Scientific Revolutions (1961), one of the most cited academic books of all time. Kuhn’s theories about the fashion of research challenged the conventional wisdom of science as a logical, step-wise progression.
Kuhn demonstrated that rather than a step-by-step process, scientific research proceeds in fits and starts, with new, potentially important discoveries bumping up against established theories and prevailing fashions. He documented the empirical difficulties of establishing new science paradigms, where scientists may have a vested interest in preservation of the status quo.
Simply put, scientists with funding are loathe to discard research programs, regardless of whether they are breaking new ground – that is the human condition. And grant-making organizations and corporate funders similarly prefer the familiar over the unknown. This has the effect of pushing away new, possibly better ideas, even in the face of inconsistent data.
So until an overwhelming amount of these data points accumulate that cannot be reconciled with the existing paradigm, the science establishment resists change. Then once the tipping point is reached, we come to Kuhn’s paradigm shift where continuing the status quo becomes unacceptable and change is unavoidable.
Now we can add Cancer R&D models to the long list of Kuhn’s illustrations where a science revolution is long overdue and blocked by over-commitment to the status quo.
Given the vast financial resources needed to bring new cancer drugs to market, the substantial time required to move from in vitro to in vivo research and through the regulatory process, and the professional stake of countless scientists and institutions to the status quo, the area of cancer research may be the least open to science revolution.
This is a research environment that rewards predictable R&D over truly ground-breaking research models, and where over $100 billion dollars is spent on research yielding few new effective therapies or cures:
“Jim Watson, the Nobel-winning discoverer of DNA’s double-helix structure, caused a minor sensation recently by arguing that curing most metastatic cancers — cancers that spread in the body — remains more daunting than ever, while researchers pursue scientific dead ends. Lamenting a “conservative” research establishment that he suggested is reluctant to take scientific risks, he urged scientists to follow new, unexplored, yet more promising directions.”
Given recent high profile failures of late-stage cancer programs, even cancer research fast may be reaching the point where it is no longer possible to ignore the need for truly novel approaches.
Are you ready for the coming revolution in cancer R&D?
About the author:
President of Finston Consulting LLC since 2005, Susan works with innovative biotechnology and other clients ranging from start-up to Fortune-100, providing support for legal, transactional, policy and “doing business” issues. Susan has extensive background and special expertise relating to intellectual property and knowledge-economy issues in advanced developing countries including India and South Asia, Latin America and the Middle East North Africa (MENA) region. She also works with governments, s and NGOs on capacity building and related educational programs through BayhDole25. Together with biotechnology pioneer Ananda Chakrabarty, she also is co-founder of Amrita Therapeutics Ltd., an emerging biopharmaceutical company based in India with cancer peptide drugs entering in vivo research. Previous experience includes 11 years in the U.S Foreign Service with overseas tours in London, Tel Aviv, and Manila and at the Department of State in Washington DC. For more information on latest presentations and publications please visit finstonconsulting.com.
This guest post is from the BiotechBlog Intern, Fintan Burke. Fintan is a student at the School of Biotechnology at Dublin City University. Do you have a response to Fintan’s post? Respond in the comments section below.
This month sees the introduction of the UK’s Open Access policy as recommended by the government-commissioned Finch Report into Open Access (OA) and initiated by Research Councils UK (RCUK). Under this new policy, any published scientific paper in the UK must be made OA immediately if it qualifies for Gold OA (an upfront payment to the publisher to allow access by anyone), or be made Green OA (have the article stored in some repository after being published in the journal) after 6-24 months embargo period. In order to pay for those Article Processing Costs (APCs) charged by journals, the RCUK is giving research institutions the cost of APCs as a subsidy as part of research grants to ensure that OA is achieved.
The original policy announced by Research Councils UK (RCUK) was drafted in March 2012, fuelled by the Minister for Science’s positive reaction to the Finch report into Open Access recommendations. Since this initial policy was announced, however, significant changes have been made to the policy which has attracted a variety of responses:
- After liaising with research organisations, learned societies and scientific publishers, July saw the final draft released. Although fundamentally unchanged, the ability for publishers to impose non commercial clauses on Green OA was met with some criticism from researchers who viewed this as impeding the ability of a paper to reach Open Access.
- By January 2013, RCUK announced that the embargo periods were not to be enforced, following up on an announcement in November that only 45% of APCs would be funded as Gold OA in the first year.
- In February the government’s own Lords Science and Technology Committee’s released a highly critical report of the RCUK’s actions. Among the critiques were a lack of clarity in the policy about which papers qualify, how long embargos could last until OA is allowed and why the RCUK did not carry out a full cost-benefit analysis for Gold OA funding.
- In response the RCUK adopted a “decision tree” developed by the UK’s Publisher’s Association in March in order to clarify which papers qualify for what type of access.
Instead of clarifying the matter, however, the revised policy guidelines drew renewed criticism from the research community. Paul Jump’s report at the Times Higher Education notes that spokespeople from the RCUK and the Publishers Association were encouraging different routes for Green or Gold options. Another article at The Scholarly Kitchen questions what procedures are in place to enforce the various avenues a researcher can go down for APC funding or publishing. Questions were also raised over the lack of enforcement for the proper management of the APC grants, given the chequered history of reporting NIH-sponsored trials.
When contacted, the RCUK was able to address the reasoning behind the block grant funding mechanics. “We see the early years of implementation as a journey so there will be a transition” said Alexandra Saxon, Head of Communications at RCUK. “As we recognise the differing nature of each research organisation, how they manage the funds is best left to them – however, we will be working with them to share best practice across the sector. How research organisations are managing the funds will also be an area of focus for the review in 2014 and subsequent reviews.” In terms of establishing any regulatory body for the block grants, Ms Saxon did not indicate any future plans for doing so, saying “we want to ensure that the monitoring of the policy does not become a burden to research organisations.” However, she also acknowledged that the open access arena is a “very fast moving landscape” and stressed the importance of monitoring various aspects of the policy within its 2014 review.
The UK’s approach to mandating Open Access is similar to the germinating sentiment internationally. An international coalition of academic and research libraries broadly welcomed the recommendations made in the Finch report, while Australia and Ireland have already mandated Green Open Access for some time. The US has also recently initiated plans to launch its own Open Access policy for federally-funded papers. (Ms Saxon notes that any international research collaboration that acknowledges RCUK funding “should be publish[ed] under the RCUK policy.”)
None of this is to say that publishers are not readying themselves for an open access transition. Dr Neil Henderson of Palgrave Macmillan notes that currently a hybrid publishing option exists for 41 of their titles, with other open publishing options being announced. “We launched OA for 20 titles in June 2011 and added a further 20 in January 2013. Those titles that we do not yet offer OA for are largely society journals where the discussions with the society is still ongoing.” While some may hold the notion that OA may someday reduce the need for costly scientific journals, Dr Henderson notes that current standards come at a price. “As soon as you add levels of service to the package (eg online submission and peer review systems) and do something with the content (eg copy edit and typeset it, adding DOIs, ensuring the content is fed to abstracters and indexers etc) there is a cost involved. Unless someone is going to do all of this work for free someone needs to pay for it.”
While the APC subsidy indicates change from the current library subscription model, the staggered development of the UK’s own OA policy – considered a front runner in Europe for Golden Access mandates – suggests a long path to a change in the current publishing model internationally. While the UK developments bring a cautious optimism among OA enthusiasts, Dr Henderson suggests “it will take quite some time though before any significant switch occurs.”
About the author:
Fintan Burke is a student at the School of Biotechnology at Dublin City University. His main fields of interest include biomedical therapies and recombinant organisms. Fintan may be contacted at email@example.com .