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Why Is Editas Going Public?

Posted by Pete Shanks on January 14th, 2016

Editas logo

Editas, the gene-editing company founded by several of the scientists who developed CRISPR technology, announced on January 4th that it had filed preliminary paperwork for a public offering of stock. The filing with the Securities and Exchange Commission is extremely long, but lacks certain vital details, For instance, some clearly unanswered questions are:

  • How much cash does Editas hope to raise? There is a placeholder number of $100 million, but that is very likely to change dramatically.
  • When will this take place? "As soon as practicable after this Registration Statement is declared effective."
  • Will anyone be cashing in? "A significant portion of our total outstanding shares is restricted from immediate resale but may be sold into the market in the near future."

The Economist response seems acute:

As difficult sales pitches go, this one is hard to beat. This biotech company has burned through $75m in the past few years and has not yet started clinical work on a drug candidate. It says it will be many years, "if ever", before it has something ready to commercialise. If this were not enough, not only is there a thorny patent thicket to manage but the firm must fight and win a case seeking to overturn its own intellectual-property claims on the ground that it was not the first to invent them.

The prospectus does include some new information, including the gossipy history that the company was originally incorporated as Gengine. (Gene-engine? Could we have been spared the whole "editing" metaphor? Probably not.) There is certainly more detail about its product plans and, if you can read the tables correctly, current shareholders, the largest of which, per Xconomy's summary, are all venture capital funds:

16.6%   Flagship Ventures
15.6%   Third Rock Ventures
15.6%   Polaris Venture Partners
  9%      Bng0 (a Bill Gates-affiliated fund)
  5.7%   Viking Global
  5.7%   Fidelity
  5.7%   Deerfield
  4.8%   CEO Katrine Bosley

The prospectus confirms that Editas hopes to begin clinical trials on a therapy for Leber congenital amaurosis in 2017. That disease, which affects 2–3 per 100,000 newborns, is listed by NIH as being associated with at least 14 genes. Mutations in CEP290 (the Editas target, also known as LCA10) account for 15–22% of cases.

Being able to claim that the blind shall see is of course a great selling point, but even if the proposed treatment works, no price has been set for it. (Spark Therapeutics, which may be a competitor, has in the pipeline at least one gene therapy product for LCA blindness that seems likely to cost $500,000 per eye.)  Presumably this is more of a proof of concept for Editas than a big moneymaker.

Editas is not the only gene-editing firm considering raising money on the stock market. Intellia, one of the companies founded by Jennifer Doudna, co-author of the first published paper on the technology, has been rumored to be "IPO-ready." CRISPR Therapeutics, founded by Doudna's co-author Emmanuelle Charpentier, is at least considering one, according to CEO Rodger Novak, who noted wryly that

Coming late to this party is not very smart.

Meanwhile, the patent wars are coming to a head. In headline terms, that's a fight between Feng Zhang of Editas on one side, and Doudna (and Charpentier) on the other. Doudna was also a co-founder of Editas, along with Zhang, George Church and others, but withdrew when the patent dispute arose. The Patent Office has officially declared an "interference" and Doudna seems to be a slight favorite at present. (UC Berkeley is favored over the Broad Institute and MIT.) Both sides have stated that the technology will be freely available to researchers, but commercial licenses could be very, very lucrative. When this all ends is unclear.

The business of business is, of course, business, and far be it for those not expert in such matters to criticize decisions about going public. But Editas is said to have at least two years' cash on hand, and the current investors might even snap up the shares on offer.

So why now? Is this all about striking while the publicity is hot?

Previously on Biopolitical Times:

Coming Up at CGS in 2016

Posted by Jonathan Chernoguz on January 14th, 2016

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As 2016 gets underway, we’re noticing how deeply last year’s events are shaping the start of this new year. You can catch up with developments in inheritable genetic modification, genetic testing & biobanks, stem cells, synthetic biology and surrogacy in our Biopolitical News of 2015 blog post. We also compiled our favorite commentaries in Top Biopolitical Times blog posts of 2015.

Online conversations

In 2015 our Talking Biopolitics webinar series featured online interviews with

To watch any of these conversations, check out our YouTube page.

We’re looking forward to kicking off this year’s Talking Biopolitics on Tuesday, January 26 with Paul Knoepfler, author of the just-released GMO Sapiens: The Life-Changing Science of Designer Babies. Paul will discuss the book and its implications with historian of science Nathaniel Comfort. You can RSVP here, and check out the Facebook event for updates.

Film series

Coming up next in our Being Human in a Biotech Age film series at UC Berkeley is No Más Bebés. The film documents the coercive sterilization of Mexican immigrant women in 1960-70s Los Angeles, and the landmark lawsuit they brought against those responsible. The screening will take place on Tuesday, February 16th at 4 pm in 470 Stephens Hall. We are very fortunate that we’ll be joined in person by filmmakers Renee Tajima-Pena and Virginia Espino for a Q&A following the screening. You can learn more about the screening at the Facebook event.

On Tuesday, April 12, we’ll be screening DNA Dreams. This documentary explores the inner workings of Shenzhen BGI (formerly Beijing Genomics Institute), which calls itself "The World’s Largest Genomics Organization,” and its animal cloning and cognitive genomics projects.

If you’re interested in other films with biopolitical themes, the earlier screenings in the Being Human series were FIXED: The Science/Fiction of Human Enhancement, Made in India, and Surviving Eugenics.

Job openings at CGS

The new year will also bring a new position to CGS. We have been selected as a host organization for the the American Council of Learned Societies Public Fellows Program, which allows us to seek a Project Director on Race, Genetics, and Society. The ACLS fellowship application process is open for recent PhDs in the humanities or humanistic social sciences. The fellowship competition will accept applications between January 14 and March 24; all applications must go through ACLS. The Project Director on Race, Genetics, and Society will plan, coordinate, and implement CGS’s programmatic work related to the impacts of genetic research, technologies, products, and services on social understandings of race and on racial justice, with the goal of tracking and contesting the re-emergence of race as biological rather than sociopolitical category.

Other positions will be posted soon. For more information, visit our Jobs and Internships page.

Image via Flickr/Dafne Cholet.

Previously on Biopolitical Times:

The Third Rail of the CRISPR Moonshot: Minding the Germline

Posted by Elliot Hosman, Biopolitical Times on January 13th, 2016

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As the 2015 news cycle ground down and rebooted for the new year, a wide swath of news publications—industry, research, scientific, and popular—declared CRISPR gene editing to be one of 2015’s biggest stories. In the new year, an ongoing CRISPR concern is how we can strengthen and brighten the line of policy and practice that cautions against creating genetically modified human babies.

Much of the news since the #GeneEditSummit in December has focused on a very different application of CRISPR: producing therapies for patients living with genetic conditions. Jaw-dropping investment news is issuing forth as multiple biotech firms team up with drug companies and venture capitalists to bring the CRISPR moonshot of gene-editing therapies into view.

While CRISPR coverage doesn’t always make it clear, many of the leading gene-editing companies have clearly stated that they’re aiming to treat genetic disease in one consenting patient at a time, not on a population level, and not in a fertility clinic for prospective parents seeking to tailor the genetic variants they pass on to their future children. Several key players in this lab-to-market push have spoken out forcefully:

Sangamo Biosciences (key figure(s): Edward Lanphier, CEO/president)

Early in 2015 as rumors were circulating that scientists were experimenting with the CRISPR/Cas9 technology on human embryos, some biotech figures stepped up proactively to make their concerns heard.  Edward Lanphier, CEO/president of Sangamo Biosciences (using older gene-editor Zinc Fingers to develop HIV/AIDS gene therapies), published an article in Nature with colleagues from the Alliance for Regenerative Medicine entitled “Don’t edit the human germline.” The article describes the use of CRISPR  gene editing in embryos to create edited humans as “dangerous and ethically unacceptable” and says “[w]e are concerned that a public outcry about such an ethical breach could hinder a promising area of therapeutic development, namely making genetic changes that cannot be inherited.” Recently, Sangamo announced that the FDA had approved its new hemophilia drug application for what could be the first in vivo clinical trial of a gene editing technology.

Intellia Therapeutics, Caribou Biosciences (Jennifer Doudna) and CRISPR Therapeutics (Emmanuelle Charpentier)

Intellia Therapeutics and CRISPR Therapeutics, two companies founded by CRISPR co-discoverers, released a statement [pdf] on the first day of the National Academies’ summit on human gene editing that said in part:

[G]ermline gene editing is outside of the scope of our companies’ research and development. We are dedicated to discovering and developing gene editing-based treatments for serious diseases using only non-germline somatic cells. This is the greatest area of patient need, where the benefits and risks are best understood, and where the ethical support is unambiguous. … [W]e are committed to … [r]efraining from directly modifying germline cells, including sperm, egg or embryonic tissue, or developing any clinical applications of germline gene editing.

Jennifer Doudna and Emmanuelle Charpentier have held this view for some time.

A few weeks after the Sangamo et al. Nature article, Doudna joined a cautious-yet-optimistic statement with other scientists that asked for a pause in CRISPR germline research in order to engage in broad public debate. In Doudna’s personal and professional capacity since “A prudent path forward for genomic engineering and germline genetic modification,” [pdf] she has expressed more extensive reservations. There’s the Hitler dream she recalled to Michael Specter in The New Yorker, and the article she published in Nature on the first day of the #GeneEditSummit that argued against editing the human germline because of “the unknown social consequences” and our limited knowledge of the “technology” and “the human genome.”

Emmanuelle Charpentier has gone further, telling BBC in September “Personally I don't think that it is acceptable to manipulate the human germline for the purposes of changing some genetic traits that will be transmitted over generations,” and telling New Scientist in December: “I hope that using the technology with the idea of changing human characteristics will not be pursued. …  Philosophically and sociologically speaking, I have lots of issues with this.”

In New Scientist, Charpentier also noted, “[T]here is money involved, whether I like it or not.” Indeed. A few weeks later news broke that Charpentier’s company CRISPR Therapeutics had penned a five-year $350 million joint venture with German drug firm Bayer to develop “new delivery technologies” to overcome a big gene therapy obstacle: getting CRISPR into the cells of targeted tissues. Doudna’s firms, meanwhile, are also teaming up with major investors. Caribou has formed “strategic alliance[s]” with two giants, chemical manufacturer DuPont to develop a variety of fields including industrial, agricultural, animal and antimicrobial applications of CRISPR, and drug developer Novartis which will be working with Caribou’s offshoot company Intellia to pursue human therapeutics, while also collaborating with Caribou in research.

Editas Medicine (Feng Zhang, CEO Katrine Bosley)

The first CRISPR company to file to go public still hasn’t made it clear where it stands on the germline controversy. Asked by Nature in May [pdf], Editas co-founder and CRISPR co-discoverer Feng Zhang noted, “[G]iven that many diseases might be treatable through somatic cell genome editing, it is unclear whether germ line editing is an appropriate solution.” In the same interview, Editas CEO Katrine Bosley stated,

The current question about CRISPR and germline engineering is far more complex [than mitochondrial replacement or 3 person IVF], and we don’t have a sense of the breadth of the implications, and we don’t understand the risks well. The technology’s progress now demands us to confront these questions, but that can’t be done quickly.

In recent coverage, Zhang is paraphrased as saying that “the importance of germline editing varies between groups of people, such as potential parents and policy-makers,” while noting that as a researcher, “we are not ready to use [CRISPR] for medical treatment, because there are issues with specificity and efficiency.” Yet neither Zhang nor Editas has voiced principled objections to allowing scientists, private companies, or others to engineer the genes we pass on to future generations. With money rolling in, they may not be worried about the fears of investors, but as a company racing to be the first to begin human clinical trials of a CRISPR gene therapy, they should probably be concerned about how the public will view their ambivalence on the germline question.

  * * *

Minding the Germline

Gene therapy companies know that there are numerous obstacles to overcome if they are to translate shiny and powerful new nano-engineering tools like CRISPR into accessible medical treatments down the line. Many remember Jesse Gelsinger, a teenager who died after a gene therapy trial gone wrong, and are aware that this tragedy cautions against the breakneck speed that the market dynamics of drug development engender. Researchers working in stem cell therapeutics—many of whom, like the scientists and biotech figures cited above, have called for a moratorium on germline applications of CRISPR—are also familiar with this tale.

Concerns about the safety and effectiveness of this new kind of gene therapy have been voiced by many, though hyperbole about Eradicating! All! Genetic! Disease! can still be found. Less widely acknowledged are questions about whether any treatments that are successfully developed will be affordable. In California, billions of dollars of taxpayer money have been invested into the California Institute for Regenerative Medicine (CIRM) in hopes of developing hugely hyped but so far nonexistent therapies; after ten years, two late-stage clinical trials are ongoing and may produce medically relevant results, but at sky-high prices.

While CRISPR is ubiquitous in some circles, it still hasn’t hit the public fan like stem cells did back in the 2004 presidential election. It is heartening to see biotech companies come out in very public ways against research and development aimed at engineering the human germline, but questions remain. Will this long-anticipated reboot of gene therapy deliver safe and effective treatments? Will the hundreds of millions of invested dollars—private money to be sure, but money chasing a scientific advance made in large part at public universities—lead to treatments that are accessible and affordable?

Previously on Biopolitical Times:

Image via Flickr/Richard Masoner

False Inevitabilities and Irrational Exuberance

Posted by Gina Maranto, Biopolitical Times guest contributor on January 8th, 2016

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Scans of media coverage carried out by CGS and others after the National Academies of Medicine and Sciences co-sponsored International Summit on Human Gene Editing in early December revealed that, for editors at least, it was a confusing event.  Some stories ran under headlines signaling that gene editing research had been given a green light [Science]; others said scientists were seeking a moratorium [The New York Times].

Since then, several disquieting themes have emerged online in mainstream media and science blogs. These include the phenomenal medical gains to be had from gene editing for somatic therapeutic interventions, with the attendant piquing of interest among venture capitalists in search of the next big profit-taking opportunity in biomedicine. 

There is also ongoing discussion of the desirability of “fixing” the human genome through reproductive genetic interventions.  Disturbingly, some commentators are touting the “inevitability” of human germline.  And a few powerful voices in science and bioethics seem to be at pains to prove that CRISPR-Cas9 modifications that aim to “improve” resulting offspring—eugenics by any other name—would be categorically different from any previous efforts of that sort because they would be driven by public demand rather than state mandate.

Take, for example, the December 22 Quartz piece whose headline trumpets that 2015 was “the year it became OK to genetically engineer babies.”  The article itself, by Akshat Rathi, makes less forceful claims about the “okayness” of designer babies, but does argue

[W]hen historians of science look back decades from now, they may well mark 2015 as the year genetically engineering humans became acceptable. That’s because, while the world was paying attention to the gene-editing summit, a more momentous decision had been made just a month earlier in the UK.

That decision was the British Parliament’s approval of regulations allowing so-called “three-person IVF” which produces heritable alterations in embryos, though via a technique that’s very different from gene editing.  Rathi goes on to predict:

Based on past progress, it is likely that genetic enhancements to humans will become a reality step by step. Just like mitochondrial replacement therapy, they will first appear for a very narrow purpose, such as curing single-gene disorders, and then, likely over many decades, we might reach the stage of creating those fabled designer babies.

Rathi is not alone in proclaiming the coming of the new age of genetic tinkering. For example, Michael Specter in The New Yorker, writes of CRISPR-Cas9, “Inevitably, the technology will also permit scientists to correct genetic flaws in human embryos.”

But is reproductive human germline editing inevitable?  Rathi offers an ostensibly well-founded prediction on past evidence, but errs in globalizing from the case of Britain.  Specter forecasts from a more gee-whiz angle.

Such decontextualized and ahistorical rhetoric does no one a service.  At this date in the world’s history, it is fatuous to contend that all technologies must be used because they are developed.  Technology indeed has an internal momentum, as individuals and industries seek to refine existing techniques and products.  But even path dependence—the tendency for newer innovations to build upon older ones—is not a given.  The history of invention is littered with cases in which old forms are abandoned completely (think CDs and VHS) or technologies are simply not deployed.

And Britain cannot be taken as a norm of any sort with regard to genetic policy.  Since the 18th century, the British have been fascinated by animal and agricultural breeding.  In the 19th century, Galtonian eugenics sprang from a particular cultural ground, combining longstanding classist, racist, imperialist, and liberal capitalist notions with biology to yield scientistic social policies—policies that were different not in kind but in degree from previous approaches to controlling the lower classes.

Despite the claim by many that after WWII British recoiled from eugenics, we know that is not true.  IVF developers Patrick Steptoe and Robert Edwards both voiced eugenic aims for their IVF research, and in the past ten years or so, a vocal and influential group of neo-eugenicist philosophers and biologists there have pushed a eugenics agenda and acted as boosters for germline interventions. 

One such advocate, Julian Savulescu of Oxford University, has even argued that to “save” humanity, we should pursue the elimination of “genes” for “aggression” and other “negative” traits.  Savulescu goes one step further by maintaining that would-be parents are morally obligated to make these kinds of interventions on embryos.

But when we look beyond Britain, the landscape appears quite different.  Modification of human embryos by whatever technique has been seen as problematic enough to have been prohibited in over 40 countries  and such interventions are anathema to many people, including scientists, in countries that do not yet have specific policies in place.  Rather than focus on inevitability, better to ask how, when, and by whom germline editing might be used.  Given the breadth of opposition, where would the drive come from for the wholesale policy changes that would need to happen to make germline editing a widespread reality?

So-called “patient demand” could be a factor.  As historian of science Daniel Kevles pointed out at the gene editing summit, eugenics was never limited to state interventions, but embraced widely by individuals.  In Politico recently, Kevles wrote,

What could happen now is likely to be far more bottom-up than the top-down, state-directed racial programs of the past—individuals and families choosing to edit their genes, whether to prevent illness or improve capacity or looks, and finding themselves encouraged to do so by what was absent in the era of eugenics: the biotechnology industry.

During and after the summit, reporters advanced the patient demand argument, seizing especially on the tearful plea during a comment session by Sarah Gray, from the American Association of Tissue Banks, who had lost a baby to anencephaly (a condition whose unclear genetic basis would make it ill-suited for gene editing), “If you have the skills and the knowledge to fix these diseases, then frickin’ do it.”

But is all patient demand really what it seems?  As Boston University sociologist Ruha Benjamin noted—referencing scientists’ campaign in 2004 for $3 billion in public funding for stem cell research in California—it is possible to leverage a “pro-cures” bias and to mobilize patients and their families to fight on behalf of scientists’ agenda.  At the gene editing summit, Benjamin suggested that patients could again be co-opted by scientists eager to move forward with both therapeutic and germline applications of CRISPR-Cas9.

The campaign in Britain over three-party embryos also prominently featured narratives of women afflicted with mitochondrial disease and their traumas and travails.  Such tales pull at the heartstrings and deflect attention from broader ethical considerations and, in some cases, facts.  As David King, who runs the watchdog group Human Genetics Watch, remarked when the UK’s fertility agency, the Human Fertilisation and Embryology Authority (HFEA), approved mtDNA work,

The decision is very disappointing, but comes as no surprise, since the HFEA can never say no to scientists.  These experiments are scientifically useless and morally very problematic. The research lobby has distorted the scientific facts in order to defuse criticism.

Although it has been described as exceedingly thorough—with several reviews by an expert panel, solicitation of views on ethical issues, surveys and calls for comment from the public, and debates in the House of Parliament—the HFEA process has also been deemed problematic by civil society groups in Britain and elsewhere.  And as CGS consultant Pete Shanks and CGS staffer Jessica Cussins found, the HFEA’s claim of “broad public support” for approving the techniques is misleading at best.

The gene editing summit, while billed as a “global discussion,” was also found wanting.  CGS’s Marcy Darnovsky and others in attendance (see, for example, presentations by Catherine Bliss and Ruha Benjamin) enumerated the many groups left out.  If the NAS is genuinely committed to “ongoing discussion,” as it has said it is, it should develop a robust framework for how and when those discussions will occur and implement measures for true inclusivity.  As David Corn of the Innovative Genomics Initiative at University of California has written, “We need to keep talking about gene editing. And by ‘we’, that means everyone, even across national boundaries. And everyone in all walks of life needs to be involved in the conversation.”


Gina Maranto is a fellow at the Center for Genetics and Society. She is Professor and Director of Ecosystem Science and Policy and Coordinator of the Environmental Science and Policy program at the University of Miami's Leonard and Jayne Abess Center. Her articles, opinion pieces, and reviews have appeared in Discover, The Atlantic Monthly, Scientific American, The New York Times, and other publications. She is the author of Quest for Perfection: The Drive to Breed Better Human Beings.

Previously on Biopolitical Times:

Composite image via Flickr/John Ward, Flickr/Rob Pearce, Flickr/Gennie Stafford



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