The Bio Report

Nucleoside analogs are a widely used class of chemotherapeutic agents, but the ability of cancer cells to evade them and develop resistance limits their benefits and increases their side effects. NuCana, which is developing a new class of cancer agents dubbed ProTides believes it can address this problem with nucleoside analogs by delivering them efficiently into cancer cell and preventing their degradation before they can act. We spoke to Hugh Griffith, founder and CEO of Nucana, about the pr oblems of resistance, how the company’s ProTide technology works, and the pipeline the company is advancing in the clinic.

Synlogic is using synthetic biology to engineer probiotic bacteria to deliver therapeutic benefit. By targeting the microbiome of the gut, the company believes it can produce systemic effects to treat metabolic and other diseases with its new class of therapies it dubs Synthetic Biotics. We spoke to Aoife Brennan, interim CEO and chief medical officer of Synlogic, about the company’s platform technology, how its seeking to exploit the microbiome, and how its engineered bacteria may offer a safer and more effective way to treat certain conditions.

One way to improve drug discovery and development is to work with more accurate models of human biology and human disease. Organ-on-a-chip technology that provides three-dimensional cell models in conventional microfluidic plates is helping to do that. Mimetas is one company that’s delivering this technology to the hands of researchers today. We spoke to Jos Joore, co-founder and CEO of Mimetas about the technology, how its changing drug development, and whether organ-on-a-chip technology may one day find its way into the clinic.

Insilico Medicine is working to harness artificial intelligence to address diseases of aging and in the process reinvent the way new drugs are discovered and developed. Its AI platform is integrated into the continuum of the discovery and development process and seeks to improve target identification, the selection of drug candidates, and predict clinical trial outcomes. In addition to working in collaboration with the large pharmaceutical companies, Insilico is pursuing internal drug discovery programs in range of diseases of aging. We spoke to Alex Zhavoronkov, CEO of Inisilco Medicine, about the company’s platform technology, the potential for AI to transform the discovery and development of drugs, and why Insilico focuses its efforts on diseases of aging.

Digital health technologies are providing new ways of monitoring patients and delivering care. In the realm of clinical trials, they provide a way to remove geographic barriers to patient participation, improve compliance, and reduce costs while also creating a way to capture real-world data. We spoke Bryan Silverman, CEO of ObvioHealth, about the company’s ClamiIt platform, the workings of siteless clinical trials, and the potential for digital technologies to address a variety of challenges trial sponsors face.

The convergence of artificial intelligence and the life sciences is promising to revolutionize the entire healthcare continuum including the way drugs are discovered, how clinical trials are conducted, the methods physicians use to diagnose patients, and how patients are monitored and treated. The life sciences investment firm Foresite Capital, armed with a new $668 million fund, is helping to bring about this change by backing innovative companies working at this nexus information technology and biotechnology. We spoke to Brett Zbar and Vik Bajaj, managing directors of Foresite, about their new fund, what constitutes a Foresite investment, and why they believe this is not just the latest fad, but a meaningful change in the way life sciences companies seek to address the challenges in all aspects of healthcare.

In this encore edition of The Bio Report, we revisit an interview that original ran in December 2017. Climate change and the increasing frequency and intensity of extreme weather events carry a toll on human health. Not only do floods, hurricanes, and other similar phenomenon cause death and injury, they also create long-term health effects. Jesse Bell, a research scientist at the North Carolina Institute for Climate Studies at North Carolina State University recently examined the issues in an article in the Journal of the Air & Waste Management Association. We spoke to Bell about the health consequences of these weather events, the challenges they create for public health systems to plan and prepare, and why new research is needed to better understand the relationship between these events and human health.

One of the reasons for the high cost of drug development is that most drugs fail in clinical development. Even though preclinical testing can provide a good insight into whether a drug hits an intended target, once in the body, drugs can interact with a large number of proteins and have unintended consequences. A-Alpha Bio, a spinout from the University of Washington's Institute for Protein Design and Center for Synthetic Biology, is developing a platform that can measure thousands of protein interactions simultaneously and how drugs affect them. We spoke to David Younger, co-founder and CEO of A-Alpha, about the company’s platform, the bottleneck in drug development it is addressing, and the business model it is pursuing.

Stem cell therapies represent an area of great promise for treating intractable eye diseases, but there’s growing concern about clinics that promote costly stem cell treatments that have not been approved by the U.S. Food and Drug Administration and have not been proven to be safe or effective. This is already a multi-billion business and researchers have found some disturbing practices that have resulted into harm to patients. We spoke to Ajay Kuriyan, assistant professor at the Flaum Eye Institute and University of Rochester Medical Center, about his research into these clinics, how they sometimes disguise their work as legitimate clinical trials, and why this represents a threat not only patients, but this emerging therapeutic area.

Say “Oklahoma” and someone might think of wheat fields, natural gas, or the wind sweepin' down the plain. Cutting edge biomedical research, though, is probably not the first thing that comes to mind. Nevertheless, the Oklahoma Medical Research Foundation has for more than 70 years been conducting innovative scientific work that has forged new understandings of disease and made discoveries that have led to new drugs and diagnostics. We spoke to Manu Nair, vice president of Technology Ventures for the Oklahoma Medical Research Foundation, about the work it does, its efforts to commercialize its discoveries, and the challenges of getting on industry’s radar when you are outsides of a major biotech center.

Recursion Pharmaceuticals is reinventing the drug discovery process by turning biology into a data science problem. The company has set an audacious goal for itself of developing 100 drugs in 10 years. Though Recursion initially focused on repurposing existing drugs to treat rare diseases, it has expanded its work into new disease areas and is looking at new chemical entities as well. We spoke to Chris Gibson, founder and CEO of Recursion, about the approach the company is taking, the challenges of mixing biologists and data scientists together, and why he’s holding fast to his goal of attaining an unparalleled level of drug development efficiency through the use of artificial intelligence.

The blood-brain barrier provides essential protection against pathogens while allowing needed oxygen and nutrients to pass. However, one challenge it presents is getting therapeutics delivered to the brain and central nervous system. Bioasis Technologies has developed a way to attach fusion proteins to drugs to allow them to pass the blood-brain barrier. We spoke to Mark Day, CEO of Bioasis, about its platform technology, what is known about it from testing to date, and the potential therapeutic implications of being able to deliver drugs systemically that can reach the central nervous system and brain.

There’s been much made of the potential of the microbiome to address disease and promote wellness. While much of the therapeutic efforts in this area have focused on the microbiome of the gut, Azitra has developed a platform for selecting bacteria native to the skin and engineering it to produce therapeutic proteins. We spoke to Travis Whitfill, chief science officer of Azitra, about the company’s platform, why it may be preferable to apply bacteria to the skin that can produce therapeutic proteins where they are needed, and why some of the biggest opportunities for the technology may be in the health and beauty markets.

Incubators and accelerators come in many flavors. Mass Innovation Labs is seeking to distinguish itself with its approach to providing research scientists with infrastructure comparable to what a discovery and development team might have at a mature pharmaceutical company. We spoke to Amrit Chaudhuri, CEO of Mass Innovation Labs, about what it does to accelerate the movement of its client companies down the path to commercialization, what its track record has been, and how it differs from the competition.

Intrexon has been at the forefront of efforts to harness biotechnology to address significant issues in health, energy, food, and environmental sustainability. The company is applying synthetic biology to a wide range of applications from halting the spread of mosquito-borne disease to preventing apples from browning. We spoke to Tom Schrader, vice president of communications and strategy for Intrexon, about the company’s use of acquisitions and collaborations, the challenge the industry has faced in scaling production to commercial scale, and why synthetic biology will have implications for industries not traditionally thought of using biotechnology.

The field of behavioral economics is premised on the fact that people are irrational. By harnessing tools, such as incentives, healthcare providers and drugmakers are trying to change patient behavior as a way to improve outcomes and keep people heathy. Susan Garfield, principal in the life sciences practice of EY, discussed the opportunities for the application of behavioral economics in the healthcare realm, the role digital technologies can play in enabling this approach, and what companies are doing today to move this from academic to industry practice.

Dmitry Kaminskiy does not have a lot of kind words for aging. In fact, the general partner at Deep Knowledge Ventures and managing trustee of the Biogerontology Research Foundation has called it the “ultimate evil.” As part of his ongoing war against aging the venture capitalist is offering a $1 million prize to the first person to reach the age of 123. We spoke to Kaminskiy about the longevity industry today, how a shift in medicine from treatment to prevention will alter lifespans, and what he’s trying to accomplish with his longevity prize.

Children undergoing chemotherapy often face permanent hearing loss as a result of the toxicity of these treatments. The Children’s Cause for Cancer Advocacy is organizing a Patient Focused Drug Development meeting with the FDA in September in the hopes of getting the agency to put greater consideration into the harmful effects these drugs can have and what it means for patients to lose their hearing. We spoke to Nawal Ouzren, CEO of Sensorion, which will participate in the FDA meeting and is developing a treatment for sudden hearing loss that may be able to help these patients. We spoke to Ouzren about the problem of hearing loss for children receiving chemotherapy, why regulators and others may dismiss it as an unfortunate price to pay for a life-saving treatment, and how Sensorion’s experimental drug may help prevent these children from going deaf.

Alzheimer’s disease has been a costly and elusive area for drug developers. Despite many promising results in early-stage trials, drug companies have seen millions of dollars of R&D investment end in spectacular late-stage failures. While treatments for the disease are desperately needed, one of the barriers to success has been the availability of diagnostics that can detect the disease at early stages when therapeutic interventions may have their best chance for success. We spoke to Rachel Laing, managing partner of Bionest Partners about the problem, whether there are fundamental misunderstandings about the disease that have sent drug companies in pursuit of the wrong targets, and why diagnostics can play a critical role in reshaping the pursuit of an effective treatment for the neurodegenerative condition.

The lack of diversity in genomic data has been an issue of growing concern. It threatens to limit the benefits from the massive investment that has been made to date to transform biomedical research, drug development, and the clinical care of patients. We spoke to Jonas Korlach, chief scientific officer of Pacific Biosciences, about the problem, how it’s being addressed, and the role advancing technology can play in gleaning greater insights from the genomes that are analyzed.

Developers of cancer immunotherapies are in a race to find combinations that can distinguish their products for specific indications. Biomarkers, which have played an essential role in the development of targeted therapies, have proven a much more complex challenge in the realm of immuno-oncology. Nevertheless, biomarkers may be the key to winning the competitive battles in immuno-oncology. We spoke to Rachel Laing, managing partner of Bionest Partners, about the role for biomarkers in immuno-oncology, why immuno-oncology companies have much at stake in the hunt for biomarkers that can better select patients for their therapies, and the role biomarkers will play in the move toward real-time oncology.

As the value of the patient perspective gains greater recognition throughout the healthcare continuum, Wego Health is seeking to both tap and provide that perspective to guide a range of clients within the world of healthcare. The company has amassed a network of more than 100,000 people that includes patients, caregivers, thought leaders, and influencers. We spoke to Jack Barrette, founder and CEO of Wego, about the company, the changing role of patients, and how the patient voice is being harnessed to transform healthcare today.

It seems to be strange times when people feel the need to take to the streets to voice their support for science, but that’s what happened earlier this month as the second annual March for Science was held in Washington, D.C. and 250 other cities around the world. To mark the occasion, we spoke to Mary Woolley, CEO of Research!Amercia, about the public perception of science, the state of funding for science in the United States, and why scientist increasingly feel the need to step off the sidelines to advocate for what they do.

The global demand for meat is rising as is concerns about the health dangers, environmental toll, and animal welfare issues related to the way we produce meat today. The Good Food Institute is a nonprofit working with scientists, investors, and entrepreneurs to advance efforts to make clean meat and plant-based alternatives a commercial reality. We spoke to Liz Specht, senior scientist for The Good Food Institute, about the problems with meat production today, efforts to develop alternatives, and the role biotechnology can play in creating healthier, safer, and more humane sources of meat.

Familial hypercholesterolemia, or FH, is an inherited disorder that leads to premature cardiovascular disease. It can lead to heart attacks, strokes, and the narrowing of heart valves. In people with the condition, genetic mutations impair the ability of the liver to metabolize excess fats. While an estimated 1.3 million people in the United States have FH, only about 10 percent are diagnosed. We spoke to Katherine Wilemon, founder and CEO of the FH Foundation, about the condition, why so many people are undiagnosed, and why patients face significant barriers getting access to a new class of promising drugs to treat the condition.

While most drug developers have sought to target aberrant genes or the proteins driving diseases, Syros Pharmaceuticals has developed a proprietary platform that targets the regulatory region of DNA to not only turn genes on or off but modulate their activity as well. The company is initially focusing on cancer and monogenic diseases. We spoke to Nancy Simonian, CEO of Syros, about the company’s platform technology, the opportunities in targeting regulators of genes, and why this could represent a broad new approach to treating diseases.

A sharp spike in generic drug prices that triggered an investigation in Connecticut continues to deepen. The antitrust case that alleges price-fixing and widespread collusion between generic drug companies to divvy up markets and avoid competition has now grown to include attorneys general in 49 states seeking action against 18 companies and two executives for activity involving an expanding list of widely-used drugs. We spoke to Joseph Nielsen, assistant attorney general in the Antitrust Department of the Connecticut Office of the Attorney General, about the case, how it has evolved, and its implications for generic drugmakers, consumers, and the healthcare system.

Rural counties in the United States have seen a steady loss of hospital-based obstetric services. A new study published in JAMA that looked at nearly 5 million births in almost 1,100 rural U.S. counties found a connection between the loss of these services and eroding outcomes for newborns and their mothers. We spoke to Carrie Henning-Smith, deputy director of the University of Minnesota’s Rural Health Research Center and co-author of the JAMA study, about the findings, their implications, and what can be done to address eroding healthcare for women giving birth in rural America.

The convergence of technology and life sciences, changing consumer behavior, and increasing pricing pressures on drug and device makers are forcing these companies to rethink business models and how they derive value from their products, according to a new EY Progressions 2018 report. The report argues that life sciences companies will no longer be able to rely exclusively on product-centric innovations, which face diminishing returns as health systems wrestle with cost constraints. We spoke to Pamela Spence, EY global life sciences industry leader, about the report, what a growing trend in collaborations between technology and healthcare companies may say about where the industry is heading, and why the future of life sciences companies may be as health technology companies.

Matthew Levy worked in the biopharmaceutical industry in human resources when he was confronted with a life-changing event. A doctor told him he had advanced cancer and just 90 days to live. An aggressive treatment regimen saved him, but it also led Levy to rethink his life. Now a motivational speaker and executive coach, Levy is trying to help others who may have settled into safe and comfortable jobs but find them unfulfilling. We spoke to Levy about his experiences, how it changed him, and what others could do to make turning points in their lives without having to face death.

Despite the often remarkable advances in cancer therapies, radiation is still widely used to treat a variety of cancers. Nanobiotix believes it can make radiation therapy more effective by injecting its nanomedicines into tumors to amplify the effects of radiation without damaging healthy tissue. We spoke to Laurent Levy, CEO of Nanobiotix, about the company’s technology, how it works, and why it may be broadly applicable to a wide range of cancers.

As medical advances have helped extend lifespans, older people still experience a decline in quality of life in their later years as a result of diseases associated with aging. This can cause a loss of independence, isolation, and suffering. The Biogerontology Research Foundation, a United Kingdom-based nonprofit, is funding work to understand the molecular and cellular processes of aging and develop biotechnological interventions to halt or reverse damage from it. We spoke to Franco Cortese, deputy director of The Biogerontology Research Foundation, about a new report it’s issued that characterizes the longevity and geroscience landscape today, areas of research that hold promise, and why aging might best be thought of as a disease.

Caelum Biosciences, a Fortress Biotech company, is a clinical-stage biotech developing treatments for rare and life-threatening diseases. It’s lead therapeutic in development is a novel antibody to treat patients with amyloid light chain amyloidosis, a rare disorder of the bone marrow that causes misfolded proteins to accumulate in tissue and organs throughout the body and cause damage. We spoke to Michael Spector, president and CEO of Caelum, about the company’s business model, its lead therapeutic, and how the competitive landscape for a rare disease therapy may shape development strategies.

Incubators create success stories, but usually aren’t thought of as success stories themselves. The newly renamed MBC Biolabs, which started with humble beginnings in a utility closet as the first technology incubator in the University of California system, has been a big success by meeting the needs of entrepreneurial scientists. We spoke to Doug Crawford, managing director of Mission Bay Capital and General Manager MBC Biolabs, about its recent rebranding, how it operates, and its plans for expansion.

Across the continuum of drug discovery, development, and delivery there’s a growing effort to incorporate the patient perspective into the process. Last year FasterCures, the nonprofit focused on accelerating and improving medical research, held a multi-stakeholder workshop to discuss the challenges and opportunities of patient reported outcomes and how to make them a more powerful tool for incorporating patients’ perspectives into R&D and care decision-making. A new report from the organization captures the results of that workshop. We spoke to Cynthia Grossman, director of science of patient input at FasterCures, about the report, why patient reported outcome measures and patient-centric outcomes are not one and the same, and what can be done to better reflect what matters to patients.

CellMax Life believes it can transform cancer diagnostics with its affordable non-invasive tests for early cancer detection. The company says with its platform it has been able to overcome the challenge of detecting rare circulating tumor cells in pre-cancer and early-stage cancer patients when the disease can be successfully treated. We spoke to Atul Sharan, co-founder and CEO of CellMax, about the challenges of liquid biopsies, how the company’s technology works, and why he thinks it has the potential to change the way cancer is diagnosed today.

The development of a biomarker to identify people with prostate cancer increased the diagnosis of the disease, but did little to suggest the appropriate course of treatment for individual patients. Kenneth Pienta, professor of urology and co-director of the Johns Hopkins University InHealth Signature Initiative, is trying to change that by using surveillance and extensive data gathering to continuously stratify patients and refine treatment approaches. Pienta, who will be speaking at the Precision Medicine World Conference in Mountain View, California January 22 to 24, discussed InHealth, how its transforming the treatment of prostate cancer patients, and why it may be a model for applying precision medicine approaches to a broad range of diseases.

A surge in merger and acquisition activity in the life sciences is expected this year, according to a new report from EY. The company annual M&A Firepower Report says increased competition, new sources of capital, and the change in U.S. corporate tax laws will drive greater dealmaking in the sector. We spoke to Jeff Greene, EY global life sciences transaction advisory services leader, about the report, the drivers of the activity, and why nontraditional players in the sector may be in a better position to make a splash in 2018.

We conclude our biotech review-preview series with our final installment this week as we take a look at the year ahead as the industry readies for the JPMorgan Healthcare Conference. We spoke to Jon Gardner, U.S. News Editor for EP Vantage, about the EP Vantage 2018 Preview(http://bit.ly/2CT2RDP), the outlook for big-value drug launches, and whether dealmaking or clinical success will drive stock prices in 2018. As a note, this podcast was recorded prior to Spark Therapeutics announcement of its pricing for its gene therapy Luxturna.

In the second installment of our three-part review-preview series of podcasts, we continue an annual tradition by sitting down with Adam Feuerstein, senior biotech writer for STAT, to discuss the year that was in biotech and what to look for in 2018. We talked to Feuerstein about the themes that emerged in 2017, his annual best and worst biotech CEOs, and what he’ll be watching at JPMorgan and beyond.

It’s that time of year when we begin to look back and think ahead. Starting with this week’s interview, we begin a three-part review-preview series to discuss the year in biotech and what to look for in 2018. In 2017, there were exciting developments in the area of immune-oncology with the approval of the first Car-T therapies and Gilead’s acquisition of Kite Pharma. As the year approached the finish line, investors got to view data from a range of studies at the American Society of Hematology meeting in Atlanta, setting the stage for 2018 when data from studies looking at combinations of immunotherapies will be closely watched. We spoke to Brad Loncar, CEO of Loncar investments, about the state of immunotherapies, what caught his attention at the ASH meeting, and what he’ll be watching in 2018.

Last month Otsuka Pharmaceutical and Proteus Digital Health won U.S. Food and Drug Administration approval for what’s being hailed as the first digital pill. Abilify Mycite, a drug-device combination that marries Otsuka’s Abilify, used to treat schizophrenia, with Proteus’ ingestible sensor, wearable sensor, and smartphone app intended to monitor and improve compliance. We spoke to George Savage, chief medical officer of Proteus, about the technology, other potential uses, and how it may help address the quality and cost of healthcare.

Climate change and the increasing frequency and intensity of extreme weather events carry a toll on human health. Not only do floods, hurricanes, and other similar phenomenon cause death and injury, they also create long-term health effects. Jesse Bell, a research scientist at the North Carolina Institute for Climate Studies at North Carolina State University recently examined the issues in an article in the Journal of the Air & Waste Management Association. We spoke to Bell about the health consequences of these weather events, the challenges they create for public health systems to plan and prepare, and why new research is needed to better understand the relationship between these events and human health.

Tom Frieden, after eights years of running the U.S. Centers for Disease Control and Prevention, is turning his sights to global health. Frieden has set a goal of saving 100 million lives from cardiovascular disease in low- and middle-income countries by applying proven strategies used in developed countries. His program Resolve to Save Lives, an initiative of Vital Strategies, will also work to prevent infectious disease epidemics and pandemics by strengthening public health systems in these nations. We spoke to Frieden about the initiative, whether strategies that worked in the developed world can be translated to low- and middle-income countries, and what it will take to be successful.

A public-private consortium is seeking to cut the time it takes to discover and advance new cancer therapies to the clinic to one year from the six years it takes on average today. Accelerating Therapeutics for Opportunities in Medicine, or ATOM, brings together scientists from government, academia, and industry with the ambitious goal of harnessing supercomputers to transform cancer drug discovery into a rapid process that can determine molecules that will be safe and effective before advancing them to human clinical trials. We spoke to Michelle Arkin, a member of ATOM and associate professor of pharmaceutical chemistry at the University of California, San Francisco’s school of Pharmacy, about the consortium, the approach its taking, and why it may alter the way therapies for a much broader range of diseases than cancer are developed.

Merger and acquisitions may be a path to creating value for life science companies, but talks can breakdown because of flaws in management thinking that skew their sense of the value of their company. Oded Ben-Joseph, managing director of Outcome Capital, applied behavioral economics to the M&A front to discuss how cognitive biases can derail M&A transactions in an article in the September issue of In Vivo. We spoke to Ben-Joseph about cognitive biases, why the life sciences sector is particularly prone to the problem, and what executives can do to minimize their effects.

The growing problem of bacterial resistance to antibiotics represents a significant public health threat. That’s been made worse by the dearth of new therapies that have come to market. AmpliPhi Biosciences is developing bacteriophages, viruses that infect and kill bacteria, to provide a way to target drug-resistant bugs. We spoke to Paul Grint, CEO of AmpliPhi about the health need, AmpliPhi’s approach, and why harnessing these natural killers of bacteria may provide a promising source of new therapeutics.

Bioelectronic therapies are being developed to treat a number of conditions that currently can only be addressed using pharmaceutical interventions. Thync, a bioelectronics company, believes its technology that targets the cervical and thoracic spinal nerves to modulate the autonomic nervous system, can be used to treat variety of ailments including mental health, inflammatory disorders, and skin conditions. It points to a growing body of scientific literature that demonstrates the important role the nervous system plays in regulating the body’s immune response. We spoke to Thync CEO Isy Goldwasser about the technology, how it works, and why the company’s first clinical trial of it will be as a potential treatment for psoriasis.

Despite the growing volume of electronic health records, they have so far left the recruitment of clinical trial patients, clinical trial designs, and site selection largely unchanged. The result is that drug companies are often designing clinical trials with gaps in information about the patient population they are serving, the medical issues these patients face, and where they can find them. This adds to the high cost and long timelines required to move an experimental therapy through clinical development. TriNetX is trying to address this problem through its health research platform that allows drug developers to analyze large amounts of patient data from healthcare organizations within its network. We spoke to Gadi Lachman, CEO of TriNetX, about its platform, how it works, and why he believes this could lead to more efficient drug development.

Synthetic biology is promising to harness living organisms to replace industrial processes that rely on toxic chemicals, consume large amounts of energy and water, and leave environmental degradation in their wake. One such example of a company seeking to transform an industry in this way is Colorifix, which is developing a revolutionary dyeing process to help the textile industry dramatically reduce its environmental impact in a cost-effective way using a synthetic biology based approach. We spoke to Orr Yarkoni, founder and CEO of Colorifix, about the company, how it is using synthetic biology to change the process of dyeing materials, and why he expects it to change the environmental toll of the textile industry.