The Bio Report

The recent late-stage failure of another high-profile Alzheimer’s disease drug candidate is a reminder of the challenges of developing drugs for neurological diseases. These challenges are driven by the fact that there are hundreds of different brain cell types and complex circuits and pathways in the brain that make it difficult to identify the right protein to target to treat a given disease. Cerevance says its proprietary platform overcomes the limitations of must approaches used today to study human brain cells. We spoke to Brad Margus, CEO of Cerevance, about the company’s platform technology, how it addresses the challenges drug developer face, and the programs it has advanced to date.

Intensity Therapeutics argues that cancer is both a micro and macro disease. Therapies need to act at the site of tumors, but they also need to treat the systemic effects of the disease that results from micro-metastases. Intensity is developing cancer therapies that are delivered directly into tumors, but also stimulate the innate immune system to address the more systemic effects of cancer. We spoke to Lewis Bender, CEO of Intensity, about the company’s platform technology, how it enlists the innate immune system in the battle against cancer, and the thinking behind the company’s approach.

SQZ Biotech believes its platform technology can enable the engineering of virtually any function into any cell type allow for a new world of cell therapies to revolutionize the treatment of diseases. As its name implies, it does this by squeezing a cell to momentarily disrupt its membrane to allow it to insert a range of substances. We spoke to Armon Sharei, CEO of SQZ Biotech, about the company’s platform technology, its initial focus on oncology and auto-immune diseases, and the broad potential for this approach.

As concerns grow about the rise of antibiotic resistant bugs, the ability to combat them is being undermined by the economics of antibiotic drug development. Many large pharmaceutical companies stepped away from the space and despite the passage of the GAIN Act in 2012 and government investment in antibiotic research and development, drug companies argue more action is needed. We spoke to Michael Dunne, chief scientific officer at Iterum Therapeutics, about the state of the antibiotic arsenal today, the challenges drug developers face, and whether Congress will be willing to provide the industry incentives to address societal needs at a time when there is growing animosity towards the industry over issues such as pricing.

Neurotrope is developing its experimental therapy bryostatin as a potential treatment for Alzheimer’s disease and other neurodegenerative conditions. The company believes the drug has multiple mechanisms of action and can stimulate synaptic growth factors to repair damaged synapses, form new ones, and prevent neuronal death. The company, though, found itself in a controversy over the way it characterized results from a phase 2 study of the drug in 2017. It is now conducting a second phase 2 study of the drug in the hopes of demonstrating meaningful benefits for patients. We spoke to Daniel Alkon, chief scientific officer of Neurotrope, about bryostatin, the controversy over the way it reported its previous results, and the path forward for the drug.

As people age, their bodies lose the ability to regenerate as well as they were able to when they were younger. Alkahest believes this is tied to changes in the mix of proteins that are present in the blood as we age. It is developing therapies for diseases of aging that seek to target specific proteins that are upregulated in diseases of aging, as well as providing a wide set of proteins drawn from purified human plasma as a therapeutic approach to counter the depletion of regenerative proteins. We spoke to Elizabeth Jeffords, chief commercial and strategy officer for Alkahest, about the science underlying the company’s approach, the controversy around others who have offered so-called “young-blood” transfusions, and the pipeline the company is pursuing.

There was a time when scientists could spend their careers talking only to other scientists about their work, but that’s changed. Whether it’s the current funding climate, the need to understand complex scientific issues underlying public policy debates, or opportunities to fund translational work and commercialize important discoveries, scientists are increasingly being tasked with addressing lay audiences. In “Championing Science: Communicating your Ideas to Decision Makers” the husband and wife team of Roger and Amy Aines offer a detailed guide for scientists on how to communicate effectively with non-scientists. Roger Aines is chief scientist of the energy program at Lawrence Livermore National Laboratory and Amy Aines is CEO of Damianakes Communications. Each bring their professional experience to the book. We spoke to the duo about their book, why it's needed, and why they believe scientists not only need to learn how to communicate their ideas, but to compel action and change the world for the better.

Kleo Pharmaceuticals is a developing next-generation immuno-oncology drugs that are small-molecule compounds designed to act like biologics. Kleo’s compounds activate patients’ immune systems to target and destroy cancer cells but are faster and less costly to design and produce than biologics. We spoke to Doug Manion, CEO of Kleo, about the company’s platform technology, why he thinks it will produce safer and more effective immunotherapies, and the potential to use these compounds in conjunction with existing biologics to enhance their activity.

Josiah Zayner calls himself a biohacker. He’s got a Ph.D. in molecular biophysics from the University of Chicago and worked as a fellow in NASAs synthetic biology program. But it is Zayner’s evangelism for democratizing the tools of biotechnology, his flair for attention-grabbing self-experimentation, and efforts to share the knowledge and equipment necessary to perform procedures like gene editing that have given him some notoriety. As founder and CEO of The ODIN, Zayner is helping move biotechnology from the labs of universities and biopharmaceutical companies to high schools and garages. We spoke to Zayner about his efforts, how biotechnology is becoming accessible to laypeople, and how he sees this fueling innovation.

One-third of adults suffer from high blood pressure and 9.4 million people a year will die from complications relating to the condition. In fact, cardiovascular disease remains the leading cause of death worldwide. Quantum Genomics is developing a new class of drugs that target an enzyme in the brain for the treatment of high blood pressure and the prevention of related cardio-vascular risks. We spoke to J.P. Milon, CEO of Quantum Genomics, about its platform technology, how it works, and why it may have promise as an approach to treating both hypertension and heart failure.

Atrial fibrillation, a condition characterized by an irregular and rapid heartbeat, affects up to 6.1 million Americans and can cause strokes. It is not well addressed by current medical approaches. Chris Larson, an adjunct associate professor at Sanford Burnham Prebys Medical Discovery Institute is part of a team working to find new drugs to treat the condition by identifying every gene in the human genome that can affect the rhythm of the heart. We spoke to Larson about the condition, SBP’s drug discovery efforts, and why he left industry to work on drug discovery at a non-profit institute.

Current therapies for autoimmune diseases represent a $100 billion market, but available drugs often provide limited benefits to patients or cause problematic side effects. Landos Biopharma is targeting the area of autoimmune diseases with an initial focus on inflammatory bowel disease by pursuing therapies that target a novel pathway. We spoke to Landos founder and CEO Josep Bassaganya-Riera, about IBD, why new therapies are needed, and the approach Landos is taking.

The ability to print human tissue is changing the way drug research is conducted today and opens the possibility of one-day printing replacement organs with the technology. CellInk saw an opportunity in making bioinks and started to produce a line of lower-priced printers as well in the hopes of expanding the marketing by lowering the cost of the technology. We spoke to Erik Gatenholm, CEO and co-founder of CellInk, about the technology, how its being used today, and how he expects applications for it to evolve.

Bing Wang trained as an electrical engineer, but his experience as a cancer survivor led him into the world of healthcare investment banking and then to his current role as CEO of the cancer therapeutics company Refuge Biotechnologies. Refuge is leveraging CRISPR technology, but rather than editing genes, the company is using it as a way to activate or inhibit specific genes by harnessing it for its targeting mechanism. We spoke to Wang about his own journey from patient to CEO, the company’s technology, and why he believes this will lead to safer and more effective cancer therapies.

As the year draws to a close, we continue what’s become an annual tradition and look back on the year in biotech and ahead to the J.P. Morgan Healthcare Conference and beyond with Adam Feuerstein, senior biotech writer for STAT News. Adam tries to make sense of a puzzling year for biotech that seemed full of contradictions, offers some thoughts on the highs and lows of the past 12 months, and discusses what he’ll be watching for during the J.P. Morgan Healthcare Conference.

The ability to manipulate and understand biology at the molecular level coupled with powerful computing capabilities is opening up new possibilities to harness technology to improve human health. The OS Fund is focused on investing in entrepreneurs developing platform technologies that have the ability to address global problems. We spoke to Bryan Johnson, co-founder of the OS Fund, about his investment vision, what it takes to attract his eye, and how he sees these platform technologies reshaping the world healthcare and beyond.

Paul Keckley is a long-time healthcare policy analyst whose both worked within hospital systems and advised major healthcare organizations, but when he recently collapsed on a golf course due to severe dehydration, he was given an up-close and personal view of the murky world of hospital billing. We spoke to Keckley about his experience, the challenges of deciphering hospital bills, and why he thinks it will be market forces, rather than price transparency, that rein in healthcare costs.

Matt State is known for his work trying to understand the biology underlying psychiatric illnesses, but as the chairman of the University of California, San Francisco’s Department of Psychiatry, part of his job has been to help address the city’s homelessness crisis. Homelessness is a complex issue, but the role mental health plays in the problem is often overlooked. With about 10,000 people who are homeless in San Francisco, more than a third of these people have a psychiatric or substance abuse problem. We spoke to State about the connection between homelessness and mental health issues, UCSF’s efforts in this area, and how it fits with the mission of a leading public institution.

Gecko Biomedical’s first product Setalum is a sealant that prevents bleeding after the use of sutures to repair blood vessels, but the polymers underlying the product represent a platform technology that can be used to develop a broad array of tissue reconstruction offerings addressing bone, nerve, ophthalmic, and urological applications. We spoke to Christophe Bancel, CEO of Gecko, about the company’s platform technology, its versatility, and how the company is looking to partnering to expand its reach.

In 1988, researchers showed that they could extend the lifespan of worms by manipulating the genetics of these model organisms. The implications that the normal aging process could be altered ignited scientific research into the emerging area of geroscience. As the Buck Institute for Research on Aging readies to mark the anniversary of this landmark research with a day-long celebration, we spoke to Gordon Lithgow, chief academic officer of the Buck. Lithgow discussed how the field has advanced from that key discovery, our understanding of aging today, and what progress has been made to identify compounds that could extend healthy years of life.