The Bio Report

New gene editing technologies are expanding the ease and power with which scientists can manipulate biological systems with the promising of addressing not only human health issues, but problems the plant faces with regards to food, fuel, and the environment. But while much of the concerns raised about the potential consequences of this technology have focused on its use in humans, Elizabeth Alter, assistant professor of biology at City University of New York’s York College, argues its potential environmental implications will likely be far more significant. We spoke to Alter about her recent op-ed in The New York Times, the need for public discussion about the technology, what should be done today as we work through broader questions of policy.

The U.S. Securities and Exchange Commission last week finalized rules on crowdfunding that opens the door for the participation of non-accredited investors. The rules complete a long process for the commission set into motion by the passage of the JOBS Act. We spoke to Richard Swart, director of research for the Program for Innovation in Entrepreneurial and Social Finance at the University of California at Berkeley and chief strategy officer for the crowdfunding investment site NextGen Crowdfunding, about the new rules, how this will change the investment landscape, and what it all means for the biotech industry.

Growing challenges in the areas of health, food, energy, and the environment have increased efforts to harness biology to create sustainable solutions to global problems. With advances in the ability to engineer microbes to perform desired tasks, the rapidly evolving area of synthetic biology is expected to fundamentally reshape industrial processes and give rise to a new bioeconomy. Leading scientists, entrepreneurs, and investors will gather in San Francisco November 4 through 6 for SynBioBeta SF 2015 to explore the state of synthetic biology. We spoke to Richard Kitney, professor of biomedical systems engineering at Imperial College London and one of the leading scientists behind the United Kingdom’s efforts in synthetic biology, about the upcoming conference, where the state of the science is today, and why scaling up to industrial sized processes remains a big challenge.

A new class of immunotherapies is promising to radically alter the treatment of cancers and has generated excitement among investors for their groundbreaking potential. Now the Loncar Cancer Immunotherapy ETF provides a way for investors to bet on the sector through an exchange traded fund that consists of both Big Pharma and emerging growth biotechs leading the sector. We spoke to Brad Loncar, CEO of Loncar Investments and creator of the ETF, about the fund, why the focus on this narrow slice of the biotech world, and why he thinks immunotherapies will dramatically reshape cancer care in the years ahead.

Wall Street doesn’t like uncertainty and there are a number of policy issues now brewing that threaten to create some uncertainty for the biotechnology industry. As the BIO Investor Forum kicks off in San Francisco October 20 and 21, bringing together investors and CEOs for two days of panels and presentations, the conference will turn its attention to policy issues and the effects they have on valuations within this industry. We spoke to Sara Radcliffe, president and CEO of the California Life Sciences Association and a policy panelist and the BIO Investor Forum, about policy issues the industry faces, the growing controversy over pricing, and what policy matters investors should be watching.

The medtech industry has enjoyed robust M&A activity, strong financings, and a rise in R&D investments, but other numbers point to troubling developments that threaten the future health and growth of the industry, according to a new report from EY. We spoke to Ellen Licking, EY Life Sciences lead analyst, about the report, concerns about the venture capital industry’s move away from the sector, and questions about who will fund early-stage innovation that will be necessary for the future growth of the industry.

Vtesse, a rare disease drug development company, this week announced that it was initiating a late-stage pivotal trial for its lead experimental therapeutic to treat Niemann-Pick Type C1 disease. The start of the trial for the nine month old company represents a major milestone and suggest its history with the National Institutes of Health and the rare disease drug accelerator Cydan Development may point to new ways of cutting the time and cost of advancing a drug to market. We spoke to Ben Machielse, CEO of Vtesse, about the rapid pace at which the company has been able to move, the role NIH has played, and whether this points to new ways to accelerate the drug development process.

The U.S. Department of Health and Human Services has been working to move Medicare from fee-for-service to value-based payments. It’s seeking to get 90 percent of payments to being value-based by 2018. The problem, according to a recent Viewpoint in JAMA, is that when addressing life-ending chronic conditions faced by older patients, traditional professional standards that drive today’s metrics don’t effectively address patient desires. We spoke to Joanne Lynn, director of the Altarum Institute’s Center for Elder Care and Advanced Illness and lead author of the JAMA Viewpoint, about what value-based care means for patients near the end of their lives, the need to recognize the great variance in patient desires, and how healthcare systems will need to change to account for this.

The California legislature earlier this month passed a bill that would allow physicians to aid terminally ill patients who wanted to end their lives. California would become the fifth state to enact such legislation. We spoke to David Grube, national medical director of Compassion & Choices, a nonprofit working to expand end of life options, about the legislation, how attitudes among the public and physicians have changed, and what we’ve learned since Oregon passed the first such law 17 years ago.

The U.S. Food and Drug Administration recently released long-awaited draft guidance regarding the naming of biologics, biosimilars, and interchangeable biologics. At the same time the agency released a proposed rule to apply the naming scheme to six current biological products with, or expected to soon have, biosimilar competitors. We spoke to Gillian Woollett, senior vice president with the healthcare business strategy and public policy advisory firm Avalere Health, about the FDA’s actions, their implications, and some potential unintended consequences.

The National Comprehensive Cancer Network in October will release a new tool designed to help doctors understand the value of different cancer therapies by taking into account the costs of treatments. The effort from the influential group follows similar initiatives by Sloan Kettering Cancer Center and the American Society of Clinical Oncology. We spoke to Bob Carlson, CEO of the National Comprehensive Cancer Network, about the rising costs of cancer care, the new tool, and what impact it will likely have on drug pricing in the future.

President Jimmy Carter, at a recent press conference discussing his cancer diagnosis and treatment, expressed his wish to outlive the last Guinea worm. The Carter Center, since 1986, has led a global effort to eradicate Guinea worm disease with great success and its goal is within reach. We spoke to Ernesto Ruiz-Tiben, director of The Carter Center’s Guinea Worm Eradication Program, about its efforts, the history behind it, and what lessons can be drawn in combating other public health threats throughout the world.

Sprout Pharmaceuticals earlier this week won a controversial U.S. Food and Drug Administration approval of Addyi, the first drug approved in the United States to treat female sexual dysfunction. Following the news, Valeant Pharmaceuticals said it would acquire Sprout for $1 billion. We spoke filmmaker Liz Canner, director of the documentary Orgasm, Inc., about Addyi, the drug industry’s long standing pursuit of a female Viagra, and why its approval is troubling to many people.

Earlier this month in a preemptive challenge from Amarin Pharma against the U.S. Food and Drug Administration a federal judge ruled that the FDA cannot prohibit a drugmaker from promoting the off-label use of a drug if it does so through the dissemination of truthful and non-misleading information. The decision about the First Amendment Rights of a pharmaceutical company is seen as a significant ruling in a long-standing battle between the agency and the industry that has played out over the past 20 years. We spoke to John Kamp, executive director of the Coalition for Healthcare Communication, about the case, the issues behind it, and its implications for how the industry and agency will act going forward.

Drugmakers about 10 percent of the time fail to report serious adverse events to the U.S. Food and Drug Administration within the time required, according to a recent study in the Journal of the American Medical Association. We spoke to Pinar Karaca-Mandic, study co-author and associate professor of Health Policy and Management at the School of Public Health at the University of Minnesota, about the study, the concerns it raises, and whether regulators need to rethink the way adverse events are reported.

A growing problem with drug resistant infections acquired in hospitals is catching the eye of Consumer Reports, which has added the incidence of two common and deadly infections to their hospital ratings. The ratings come in the second part of a three-part investigation in the antibiotic crisis. We spoke to Doris Peter, director of Consumer Reports’ Health Ratings Center, about the study, what the highest rated hospitals are doing that the lower rated ones are not, and things patients can do to safeguard themselves when they face a hospital stay.

The controversy over the high price of new drugs and the question of the value they provide will come under increased scrutiny thanks to a grant to a Boston-based nonprofit that works to get at these questions. The Laura and John Arnold Foundation this week announced it is providing $5.2 million to the Institute for Clinical and Economic Review aimed at transforming the way new drugs are evaluated and priced. We spoke to Sarah Emond, COO of the institute, about the work it does, what this new grant will do to expand that work, and how to get a the question of the value of new drugs.

Researchers at the University of California, Berkeley and the Gladstone Institutes have grown beating cardiac tissue from stem cells in work that may lead to new ways to quickly screen for drugs likely to cause birth defects in the heart and identify drugs that may be dangerous during pregnancy. We spoke to Bruce Conklin, a senior investigator at the Gladstone Institute of Cardiovascular Disease, about the work, which was published in the journal Nature Communications, the promise it has for providing more accurate insights than animal models, and whether the approach could be expanded to other cell types to screen for drug toxicity to other organs.

For many patients with rare and difficult to diagnose conditions, it can take many years and many doctors to find a correct answer. CrowdMed is trying to offering an alternative to patients by allowing them to tap the wisdom of crowds and letting medical detectives who sign on to the site try to find the right answer. We spoke to Jared Heyman, founder and CEO of CrowdMed, about the problem with the traditional way doctors diagnose patients, the wisdom of crowds, and the case for making medicine a team sport.

Last year, the biotechnology industry set records across the board for financial metrics, a reflection of product success, new drug approvals, and free-flowing investment capital. Despite the record performance, concerns continue about growing pricing pressure and maturing pipelines that represent challenges with which the industry must contend. We spoke to Glen Giovannetti, EY’s Global Life Sciences Leader about his firms recently released Beyond Borders report, what the numbers tell us, and what the industry will need to do to keep the good times rolling.

A detailed view of funding of emerging therapeutic companies over the past ten years shows despite a rebound in venture financing, companies continue to struggle to find early-stage money. Nevertheless, the report shows the overall health of investment in the sector is thriving. We spoke to Dave Thomas, senior director of industry research and analysis for the Biotechnology Industry Organization and co-author of the report, about BIO’s findings, what therapeutic areas attracted the most financing, and what impact the capital markets have had on partnering and licensing activity.

To improve the long-term value of biopharmaceutical companies, management should learn to think more like activist investors, according to a new report from EY. The report argues that capital allocation and strategic decision-making could benefit from company leaders setting aside their assumptions and challenging themselves by thinking more like outsiders. We spoke to Jeff Greene, Global Life Sciences Transaction Advisory Services Leader for EY, about the report, what industry executives could learn from activists, and whether shareholder activists indeed have a track record worth emulating.

Bernard Muller was a successful businessman in the maritime and oil industry, but when he was diagnosed with the neurodegenerative disease ALS in 2010, he turned his entrepreneurial skills toward developing new therapies to treat the disease. Muller co-founded the world’s largest genetic research project for ALS, project MinE, and launched Treeway, a biotech company developing new therapies for ALS. As the Biotechnology Industry Organization kicks off its BIO 2015 International Convention in Philadelphia June 15, Muller is a finalist as one of the organization’s Everyday Superheroes in the pharma/biotech category. We spoke to Muller about his decision to launch Treeway, the active role he sees for patients, and why he thinks traditional approaches to drug development and clinical trial design have not served ALS patients well.

Sleep researchers and clinicians will be gathering in Seattle June 6 - 10 for SLEEP 2015, a joint meeting of the American Academy of Sleep Medicine and the Sleep Research Society. It is the largest gathering of sleep medicine physicians, sleep and circadian researchers, and health professionals in the sleep field. We spoke to Chris Winter, a fellow of the American Academy of Sleep Medicine and a board-certified sleep specialist with a background in neurology, about our understanding of sleep today, how genetics and digital health technologies may be altering our understanding of sleep, and what we should do to get a decent night’s rest.

Wall Street’s attention will turn to Chicago as the annual meeting of the American Society of Clinical Oncology runs from May 29 to June 2. Some 5,000 abstracts became available mid-month and investors have been sifting through them to gain insights into which drugmakers will rise and fall on news from the meeting. We spoke to Adam Feuerstein, senior columnist for TheStreet, about the ASCO meeting, what the early abstracts say, and who will likely be making headlines at the meeting this year.

A recent article in Nature Chemical Biology that shows it is possible to convert sugar into morphine with genetically engineered yeast has sparked public attention over the potential illicit use of the technology and the need for regulation. The work, though, also opens up significant possibilities for producing a wide range of drugs and the discovery of new ones to treat everything from cancer to infectious diseases. We spoke John Dueber, assistant professor of bioengineering at the University of California, Berkeley and one of the authors of the study, about the work, its implications, and what role biologists need to play in regulating themselves.

Universities are moving away from a passive approach to technology transfer to engage with industry in new partnerships, put a greater emphasis on translational research, and nurturing technologies so they can give rise to new products and companies. We spoke to Eric Tomlinson, chief innovation officer of Wake Forest Baptist Medical Center, about these changes, the approach Wake Forest is taking, and how it is forging new relationships with industry and the regional economy.

With the mapping of the human microbiome, a new understanding is emerging of the complex relationship between the microorganisms that live in the human gut, skin, and elsewhere on the body, and the role they play both in disease and in maintaining health. Along with growing concerns about drug resistant bacteria, this is giving rise to opportunities for narrow spectrum antibiotics. We spoke to David Martin, founder and CEO of AvidBiotics, about the problems of antibiotic resistance, the benefits narrow spectrum therapies offer, and how the sequencing of the microbiome is leading to new approaches to not only treat infectious diseases, but other diseases not traditional thought of as being driven by microorganisms.

AgeneBio is developing drugs to treat the pre-dementia stage of Alzheimer’s disease, and other neurological and psychiatric conditions. The company announced encouraging mid-stage results for its lead therapeutic, which works by quieting hyperactivity in the hippocampus portion of the brain, which plays a critical role in the formation of memory. We spoke to Jerry McLaughlin, CEO of AgeneBio about the company’s efforts, why this approach holds promise in delaying the onset of dementia in Alzheimer’s patients, and whether this approach may have implications for other neurological and psychiatric diseases.

In April, the IMS Institute released a new report that U.S. drug spending in 2014 rose 13.1 percent to $373.9 billion, the largest single year increase in spending since 2001. A number of factors drove the increase including the launch of innovative new therapies such as Gilead’s hepatitis C drug Sovaldi. We spoke to Murray Aitken, executive director of IMS Institute for Healthcare Informatics, about the new report, the confluence of factors that drove spending higher, and what the outlook is for 2015 and beyond.

AARP, the advocacy group serving people over the age of 50, is taking steps to ensure that digital health technologies best serve its membership. The organization has embarked on an initiative to test the design and functionality of digital health devices for older people and provide feedback to the marketplace. It’s enlisted partners United Healthcare and Pfizer as part of its effort. We spoke to Jeff Makowka, director of thought leadership for AARP, about its efforts, how it’s going about testing devices, and what it hopes to accomplish. We had some technical difficulties on this podcast that were not apparent during the interview. We’ve done our best to clean it up, but you will hear some static early in the recording. We apologize to our listeners and our guest, who we think you’ll find nonetheless interesting.

A newly discovered archive of documents reveal the sugar industry’s efforts to shape the national research agenda away from the effects of sugar on tooth decay and push for programs to focus on alternatives to reducing consumption. We spoke to Cristin Kearns, a University of California, San Francisco postdoctoral scholar who discovered the papers, about her research, how the agenda of the National Institute of Dental Health became aligned with the sugar industry’s, and how industry can subvert research agendas to protect their economic interests at the expense of public health.

Drug resistant bacteria cause 2 million illnesses and approximately 23,000 deaths each year in the United States, according to the U.S. Centers for Disease Control and Prevention. The problem of resistance also limits the ability to treat patients using certain medical procedures, such as chemotherapy, surgery, and organ transplants. Last week, the Obama Administration released its National Action Plan for Combating Antibiotic Resistant Bacteria, a multipronged approach to cut inappropriate use of antibiotics, improve surveillance, and develop new drugs. We spoke to Amanda Jezek, vice president of public policy and government relations at the Infectious Diseases Society of America, about the administration’s plan, whether it offers any new ideas, and what needs to be done to ensure its success.

Adverse events from drugs cost the U.S. healthcare system an estimated $25 billion in 2013, but data from adverse events reporting is generally not factored into payer and provider decisions about what drugs are most cost-effective. The healthcare analytics firm AdverseEvents is trying to change that by turning adverse events data gathered by the U.S. Food and Drug Administration into actionable information. We spoke to Brian Overstreet, CEO of AdverseEvents, about its RxCost offering, why payers and providers rely on mostly pre-approval clinical data in their drug decision making, and why it’s important to consider the broader costs associate with a drug.

Tomasz Sablinski believes the drug development process is broken and has sought to reinvent it. His company, Transparency Life Sciences, relies on crowdsourcing to design its clinical trials, makes all of its data public, and employs digital technologies to remotely monitor participants and dramatically reduce the costs of studies. Now, several years into his efforts, we checked in with Sablinski, CEO of Transparency, about the progress he’s made, what barriers his encountered, and whether his success is having any impact on the way other companies are conducting drug development today.

The biopharmaceutical industry has long been considered immune to the threat of patent trolls, patent holders who seek to monetize the value of a patent through enforcement rather than productive use. But a paper by UC Hastings Robin Feldman and Harvard Fellow Nicholson Price argues that the biopharmaceutical industry should be worried and that steps should be taken now to counter the threat before it blossoms into an industrywide problem. We spoke to Feldman, professor of Law and Director of the Institute for Innovation Law at the UC Hastings College of Law about the study, why the biopharmaceutical industry should be concerned, and what steps can be taken to deter abusive behavior while protecting innovation.

A first of its kind study of venture investment in therapeutics by disease area and innovation finds that venture investment following the Great Recession has not yet recovered to the levels seen in the years leading up to the financial crisis. In addition, the study shows great disparities in the level of funding of disease categories and finds that chronic diseases with large patient populations have seen some of the biggest drops in investment. We spoke to Dave Thomas, one of the authors of the study from the Biotechnology Industry Organization, about its findings, where dollars are going and where they are not going, and what effect recent policies may have had in attracting investment to certain areas.

At the end of January, after nearly a year of hearings, roundtables, and white papers, the House Energy and Commerce Committee’s Subcommittee on Health released a draft of the much anticipated 21st Century Cures Act. The draft, nearly 400 pages long, addresses a broad range of issues in the drug and device development and review process. We spoke to Nick Manetto, principal with the national advisory and advocacy firm FaegreBD, about the legislation, where the points of controversy lay, and whether despite its bipartisan birth political brawling is ahead.

There’s no shortage of data created in the world of healthcare, but harnessing it to improve care and reduce costs remains a challenge. Apervita, backed with $18 million recent venture investment from GE Ventures, Baird Capital, and others is a marketplace for people to buy and sell their healthcare analytics. We spoke to Paul Magelli, CEO of Apervita, about the pressures on healthcare providers today, the challenges to integrating analytics into practice, and how Apervita hopes to change that.

President Obama unveiled the details of his $215 million precision medicine initiative, the centerpiece of which is a 1 million person study that seeks to correlate genetic data, with health records, lifestyle information, and more to better understanding wellness and disease and fuel the development of new therapies. We spoke to Amy Miller, executive vice president of The Personalized Medicine Coalition, about the initiative, if the funding for it will match its ambition, and whether concerns about privacy will stand as a barrier to its success.

CAR-T cell immunotherapies seek to harness the body’s immune system to fight tumor cells. The promise of this new class of therapies has ignited investor’s imaginations, but a new report from EP Vantage argues that the enthusiasm that has driven valuations of CAR-T companies should be tempered by an eye towards the risks. We spoke to Jacob Plieth, report author and senior reporter for EP Vantage, about the promise of these therapies, what we know about their safety and efficacy, and why the muddy intellectual property landscape is a concern.

Investment in biomedical research in the United States is declining at a time when other countries have been increasing their spending. While this is raising concerns about the threats this poses to the nation’s economic competitiveness, a new study in the Journal of the American Medical Association suggests new strategies will be necessary to fund research and development if the clinical value of past investments and opportunities to improve care are to be fully realized. We spoke to study co-author Ray Dorsey, Professor of Neurology and Co-Director of the Center for Human Experimental Therapeutics at the University of Rochester Medical Center, about the findings, what strategies can be employed to reverse the trends, and why new investment alone is not the answer.

It was an unprecedented year for M&A activity in the life sciences, but even though Big Pharma returned to dealmaking after largely spending 2013 on the sidelines, it’s been unable to close its growth gap through acquisitions. Specialty Pharmaceutical and Big Biotech have been building muscle and key acquisitions that could address growth for Big Pharma continue to be snapped up by competitors. We spoke to Jeff Greene, EY’s Global Life Sciences Transaction Advisory Services Leader, about his firm’s new M&A report, at what point the growing price of assets becomes too rich, and what the outlook is for 2015.

Ron Cape, co-founder, chairman and CEO of Cetus, the first biotechnology company, died earlier this month at 82. Cetus, which developed PCR technology, as well as Betaseron and Interleukin-2,has faded from the minds of industry watchers, but its impact has been lasting. The company not only provided scientific, but financial and cultural innovations that helped shaped the industry. Chiron acquired the company in 1991. We spoke to Mark Jones, director of research for the Life Sciences Foundation about Cape, the innovations he made, and his lasting contributions.

The rapidly growing area of regenerative medicine is promising to replace chronic therapies with cures. As the biotechnology industry gathers in San Francisco for a big week of meetings, the Alliance for Regenerative Medicine is readying to deliver its annual State of the Industry Briefing. We spoke to Edward Lanphier, president and CEO of Sangamo Biosciences and the newly elected chairman of the alliance, about the transformation of medicine that’s underway, the particular excitement among investors and industry over gene therapy, and why he’s confident payers are ready to embrace the new technology.

As the biotech industry ramps up for the JPMorgan Healthcare conference, we continue our conversation with TheStreet.com’s Adam Feuerstein. In part two of our conversation, we look ahead to what’s in store for the biotech industry in 2015. We get Feuerstein’s thoughts on who and what to watch in the new year, what Big Pharma company may be the target of a takeover, and whether fights over drug pricing will put an end to biotech’s bull market.

The year 2014 was one for the record books for the biotech industry. In part one of a two-part podcast, we take a look back at the year that was with Adam Feuerstein, senior columnist for TheStreet.com. Feuerstein discusses the growing controversy over drug pricing, the newsmakers of 2014, and lessons from the Dendreon bankruptcy.

Two-thirds of Americans say they are willing to anonymously share their health information with researchers, but only about 1 in 5 is willing to provide their purchase history or social media activity, according to a recent Truven Health Analytics-NPR Health Poll. The findings suggests that while there is growing comfort with electronic health records, privacy concerns may hinder the ability of Big Data to deliver on its full promise to improve the health of individuals. We spoke to Michael Taylor, chief medical officer of Truven Health Analytics, about the survey, the level of theft and misuse of health data, and who people trust the most with their information.

The cost of drug development, a metric that underlies discussions ranging from investment in startups to drug pricing, is a matter of great interest and controversy within the pharmaceutical industry. The Tufts Center for the Study of Drug Development recently completed a new estimate of the cost of developing a new prescription drug and raised it to $2.6 billion, up from $802 million in 2003. We spoke to Joe DiMasi, director of economic analysis at the Tufts center for the Study of Drug Development and principal investigator for the study, about the findings, why there’s been dramatic growth in cost despite efforts to reinvent drug development, and whether there’s reason to think the trend will reverse itself anytime soon.

News last month of the Cystic Fibrosis Foundation’s $3.3 billion sale of royalty rights to Kalydeco and other Vertex Pharmaceuticals drugs it helped fund served as a bold statement to the growing prominence and power of venture philanthropy. We spoke to Margaret Anderson, executive director of FasterCures, about the transaction, the role new funding and collaboration models are playing in accelerating drug development, and what traditional investors and disease groups are learning from each other.