The Bio Report

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.