The Bio Report

The production of biologics is costly and takes time. Dyadic International has developed a proprietary manufacturing process that replaces the use of Chinese hamster ovary cells, long used to produce protein therapeutics, with a fungus that has a long history in industrial biotechnology applications. The company believes its process can produce drugs faster, in greater volumes, and at significantly lower costs than biotech processes in use today. We spoke to Mark Emalfarb, CEO of Dyadic, about its manufacturing process, the benefits it could bring, and why it may have big implications for drugmakers.

Alzheimer’s disease is the sixth leading cause of death and it continues to rise. Today it is costing the U.S. healthcare system $200 billion a year and there are no therapies that prevent, halt, or reverse the disease’s progression. United Neuroscience is taking a new approach to the disease with its endobody vaccines, a class of synthetic biologics it is developing to treat Alzheimer’s and other CNS conditions. We spoke to Ajay Verma, chief medical officer of United Neurosciences, about Alzheimer’s, the company’s platform technology, and the promise of enlisting the immune system in the fight against CNS diseases.

Gene therapy represents an expanding area of potential to correct and modulate the activities of genes driving diseases. One of the major challenges these groundbreaking technologies face, though, is delivering them to where they need to go within the body to be effective. Nanogenic Solutions believes its solved the problem with its LipTide technology that marries a payload carrying-lipid with synthetic peptides that target cell suface receptors and allow DNA or RNA into the cell. We spoke to Simon Newman, director of preclinical development for Nanogenic Solutions, about the challenges of gene therapy, the LipTide technology, and what it could mean for advancing a range of RNA and DNA therapies.

Brexit, the United Kingdom’s plan to withdraw from the European Union, carries a long list of unintended consequences, one of which is the relocation of the European Medicines Agency. As many as 20 cities are competing to become the new home of the drug regulator. We spoke to Rory Mullen, International Investment Executive for IDA Ireland, about Dublin’s efforts to woo the EMA, the process going forward, and why its viewed as an economic prize.

Nearly one in three drugs has a postmarket safety event, according to a study published earlier this month in JAMA. The study looked at 222 novel therapies approved between 2001 and 2010. We spoke to Nicholas Downing, clinical fellow in the Department of Medicine at Brigham and Women's Hospital and lead author of the study, about the findings, the limits of clinical trials, and whether the U.S. Food and Drug Administration needs to do more to improve surveillance of drugs once they are approved.

Climate change is not often viewed as a public health threat, but it can have unexpected consequences on the spread of infectious diseases. As changing temperatures make new areas more hospitable to mosquitos, its can raise the risk of mosquito-borne diseases, such as dengue, chikungunya, and zika in areas not usual thought as prone to outbreaks of tropical disease. We spoke to Erin Mordecai, assistant professor of Biology at Stanford University and lead author of a recent study in PLoS Neglected Tropical Diseases that looks at how climate change may affect the spread of mosquito-borne disease. We spoke to Mordecai about the study, what it means for potential infectious disease outbreaks, and how public health officials and drugmakers in the developed world may need to think differently about neglected tropical diseases.

Ten years ago, a group of San Francisco State University alumni working at Genentech got together with the Michael Goldman, the university’s chairman of the biology department, to establish the school’s annual Personalized Medicine Conference. This year, the conference will revisit topics from its past ten years as well as exploring the future of personalized medicine ask speakers to reflect on how personalized medicine has changed their lives. We spoke to SF State’s Goldman and Dan Maher, one of the alumni who drove the creation of the conference, about the state of personalized medicine, the pace of advances, and what’s surprised them most about where we are today.

The promise of precision medicine to provide more effective and safer cancer therapies that target the genetics driving each patient’s disease has been hindered by the lack of understanding of the specific genetic alterations underlying many cancers. Elana Fertig thinks math can solve that problem. Fertig, with a nod to the algorithm Netflix uses to help recommend movies users might like, is working to identify the genes that drive an individual’s cancer. Fertig, assistant professor of Oncology Biostatistics and Bioinformatics at Johns Hopkins Kimmel Cancer Center, discussed the proliferation of genetic data relating to cancer, how researchers may be able to capitalize on that, and how such an approach may also bring a new understanding of why patients suffer relapses and develop resistance to cancer therapies.

For anyone who has ever sought to find the price of healthcare services before obtaining them it will come as little surprise that information is not readily available. A recent study from Public Agenda, with support from the Robert Wood Johnson Foundation and the New York State Health Foundation, set out to find how Americans seek and use healthcare price information. We spoke to David Schleifer Director of Research for Public Agenda about what extent healthcare consumers seek price information, the implications of having a system with little price transparency, and what can be done to fix the problem.

As the Trump Administration calls for significant increases in military spending, it is also seeking steep cuts to the National Institutes of Health, the Centers for Disease Control and Prevention and programs used to address issues of global health. Michael Osterholm director of the Center for Infectious Disease Research and Policy at the University of Minnesota, in a recent op-ed in The New York Times argues that issues such as vaccine development, the need to combat antibiotic resistance, and respond to new infectious disease outbreaks are fundamental issues of national security. We spoke to Osterholm about global trends fueling the threat of infectious disease, the dangers of proposed budget cuts, and why the administration needs to invest in new ways to respond to the threats we face.

Recent mumps outbreaks are raising questions about strategies to combat the spread of the virus. One issue of concern is that the current vaccine is designed to protect against a different strain of the virus than the one that is sparking the outbreaks. Though the vaccine can help prevent some people from getting the disease or minimize its effects, the outbreaks have raised questions about whether a new vaccine is needed. We spoke to Stanley Plotkin, an expert on vaccines and a member of the editorial board of the journal Clinical and Vaccine Immunology, about what’s driving the outbreak, whether boosters can adequately address the problem, and why developing a new vaccine is not an easy solution to the problem.

The landmark legislation known as the Genetic Information Nondiscrimination Act of 2008, or GINA, anticipated growing concerns about genetic privacy, although it left much work undone. Now, though, as genetic information proliferates, proposed legislation would weaken protections by allowing employers to compel employees to share their genetic information in workplace wellness programs. Though voluntary, those who choose not to share this information could face thousands of dollars more for health insurance, according to reports. We spoke to Jeremy Gruber, past president of the Council for Responsible Genetics and someone credited with helping enact GINA, about the proposed legislation, what more needs to be done to protect genetic information, and how to best balance privacy concerns with the opportunities to leverage genetic data to better understand health and wellness.

By the time signs of Alzheimer’s disease manifests in the form of memory problems, behaviorial changes, or loss of executive function, years of irreversible damage to the brain has already occurred. In the latest annual report from the Alzheimer’s Association, the organization looks at the potential for biomarkers to diagnose the disease at its earliest appearance and allow for treatments to hold the disease in check. We spoke to Heather Snyder, senior director of medical and scientific operations for the Alzheimer’s Association about the report, where efforts to validate biomarkers of early-stage Alzhimer’s disease stand, and why this holds the potential to change the way Alzheimer’s disease is viewed and treated much in the way diagnostic tools have turned heart disease into a chronic condition.

The Personalized Medicine Coalition’s recently issued its 2017 report on the opportunities and challenges for the industry. Chris Wells, communications director for the organization, will be kicking off The 4th Annual Business of Personalized Medicine Summit in South San Francisco March 28, drawing from the new report to discuss trends and the pace of advances. We spoke to Wells about the state of personalized medicine, the obstacles to greater clinical adoption, and what the growing legislative battles in Washington will mean for the industry.

Orphan drugs, therapeutics that target small patient populations, have become an enticing area for Big Pharma as companies are attracted to the premium prices, lower development costs, and faster path to market. A new EvaluatePharma report on the Orphan Drug market forecasts a compounded annual sales growth rate of more than 11 percent through 2022 with orphan drug accounting more than 20 percent of total worldwide prescription sales by then. We spoke to Jon Gardner, U.S. editor for EP Vantage, about the growth of orphan drugs, what’s driving the sector, and whether the way the industry is taking advantage of the Orphan Drug Act will cause policymakers to push back.

The Broad Institute has emerged victorious in a battle with researchers at the University of California, Berkeley over patents covering breakthrough gene-editing technology that allows scientists to easily and inexpensively alter genetic material with precision. Last month, the Patent Trial and Appeal Board of the U.S. Patent and Trademark Office ruled that patents held by the Broad Institute relating to certain aspects of CRISPR-Cas9 gene editing did not interfere with those being sought by UC Berkeley researchers. We spoke to Kevin Noonan, Partner and Chair of the Biotechnology & Pharmaceuticals Practice Group of McDonnell Boehnen Hulbert & Berghoff and founding author of the Patent Docs blog, about the decision, what it means, and to what extent it resolves patent issues regarding the gene-editing technology.

Earlier this month the National Academy of Sciences and the National Academy of Medicine issued a report that considered the scientific, ethical, and governance issues surrounding human genome editing. The report comes as new gene editing technologies have reduced the cost and increased the ease of manipulating the human genome. We spoke to Alta Charo, co-chair of the study committee that wrote the report and professor of Law and Bioethics at University of Wisconsin-Madison, about the study, the potential for this science, and where the committee thought limits should be imposed.

The emergence of cancer immunotherapies, drugs that activate and enlist the immune system to fight cancer, has emerged as a promising approach to combat the disease in its many forms. Lawrence Fong, co-leader for the Cancer Immunotherapy Program at the University of California, San Francisco, stands at the intersection of several initiatives involving UCSF to better understand the immune system’s response to cancer and develop more effective drugs in the battle against it. We spoke to Fong about the potential of immunotherapies, the various efforts underway at UCSF, and what we known and don’t know about the immune system.

The start of Trump administration, a new cabinet, and a new Congress are raising concerns within the scientific community about how the changes in Washington will affect the health of science and innovation in the United States. At issue is not only funding for research, but a range of policy decisions relating to everything from public health to climate change that some fear will not be informed by science. We spoke to Mary Woolley, president of Research!America, about the new administration, why it is critical for scientists to become advocates, and the planned March for Science in Washington, D.C. this April.

For a number of diseases that require chronic administration of a substance naturally produced by the body, patients may have to rely on regular injections or infusions. Sernova is working to free patients from these types of regimes with it combination device and cell therapy that implants a pouch that contains living cells that produce the missing hormone, factor or other substance. We spoke to Phil Toleikis, CEO of Sernvoa, about its technology, the challenges involved in such an approach, and the company’s initial focus on diabetes and hemophilia.