The Bio Report

As a medical student, David Fajgenbaum nearly died from Castleman disease, a rare autoimmune condition. He would suffer recurring bouts that carried him to the brink of death but was able to push the disease into remission by discovering a drug that could be repurposed to treat the disease. Fajgenbaum co-founded the Castleman Disease Collaborative Network and developed a unique approach to research that is now being adopted by other rare disease organizations. He tells his story in his book “Chasing My Cure.” When the COVID-19 outbreak began, Fajgenbaum recognized that the deadliest aspect of the disease—a hyperactive immune response known as a cytokine storm—shared a common link with Castleman disease. He hoped that a researcher would apply his approach to finding a potential drug to repurpose to treat the virus and soon enlisted his own team to do so. We spoke to Fajgenbaum, assistant professor at the Perelman School of Medicine at the University of Pennsylvania and co-founder and executive director of the

As some jurisdictions move to lift shelter in place orders and seek to restore economic life to normal, there’s growing concerns about the health consequences of moving too fast and the failure to make decisions without adequate testing to guide the process. Fred Brown, president and chief operating officer of the big data health consulting firm Fred Brown Management Consulting, discussed beating the COVID-19 pandemic ahead of a piece he’s writing for the fall issue of the Journal of Commercial Biotechnology. Brown offers a few scenarios but believes it will take time for life to return to what it was like. We spoke to Brown about the path to vaccines and therapies, what it will take to move beyond the pandemic, and what we’re learning from this pandemic that will help prepare us for the next one.

Targeting the epigenome—the regulators that turn on and off the activity of genes—has long been viewed as a promising way to treat cancer. But despite the promise of this approach, early efforts brought few successes in part because of the broad effects of hitting these targets. Constellation Pharmaceuticals is taking a next-generation approach to epigenetics, targeting what it calls the writer, reader, and eraser classes of epigenetic regulators to modulate gene expression in a highly selective manner. It believes its approach can be used to both induce cancer cell killing, as well as enhance anti-tumor immunity. We spoke to Jigar Raythatha, president and CEO of Constellation Pharmaceuticals, about epigenetics, the company’s approach to developing highly selective therapies that target gene regulators, and its programs in myelofibrosis and prostate cancer.

Acepodia has developed platform technology that allows it to chemically modify or conjugate living cell surfaces. The technology can be applied to any immune cell and any antibody or binding protein, without the use of genetic engineering. The company is working to apply the technology to create a family of cost-effective, off-the-shelf immunotherapies. We spoke to Sonny Hsiao, CEO of Acepodia, about the company’s platform technology, how it works, and it why it has the potential to change the cost and efficacy of immunotherapies.

One of the challenges to securing the participation of patients in clinical trials can be simple geography. Sanguine Biosciences is seeking to tear down that barrier to participation by using mobile technology to bring clinical trials to patients. The company recently partnered with Vir Biotechnology to complete a COVID-19 clinical study aimed at better understanding the biology of the disease by sending healthcare personnel to collect blood samples from patients at their homes. We spoke to Brain Neman, co-founder and CEO of Sanguine, about it use of digital health technologies, how the company works, and its recent collaboration with Vir Biotechnology for a COVID-19 study.

One of the challenges cell and gene therapies pose is how to control how much and when a desired protein is delivered. Obsidian Therapeutics has developed a platform that allows a small molecule drug to control with precision the timing and level of protein expression from these therapies. We spoke to Paul Wotton, CEO of Obsidian Therapeutics, about the company’s platform technology, how it works, and how it may improve the safety and efficacy of cell and gene therapies.

Patrick Soon-Shiong likes to talk about the “triangle offense,” the activation of a combination of macrophages, natural killer cells, and memory T-cells to battle cancer. He believes the same approach he has been working to train on cancer can be enlisted in the fight against COVID-19, the virus behind today’s global pandemic. We spoke to Soon-Shiong, chairman and CEO of NantKwest, about his approach to immunotherapy, what results he’s seen to date, and why he believes what he’s learned about harnessing the immune system in the fight against cancer can be applied to treating COVID-19.

The COVID-19 pandemic is threatening to stress healthcare systems throughout the world and it is making the development of a vaccine an important part of a strategy to arrest the virus. Though clinical trials for a vaccine are under way, creating a vaccine alone will not be enough. If those efforts are successful, there will be challenges ahead with manufacturing, distributing, and providing equitable access throughout the world. We spoke to Aurélia Nguyen, managing director for vaccines and sustainability for the The Global Alliance for Vaccines and Immunizations about the COVID-19 outbreak, how it may be playing out in different parts of the world, and what was learned from GAVI involvement in previous efforts to develop an ebola vaccine.

Early detection is a critical means of improving outcomes for cancer patients. When cancer is detected at stage I, patients have a 90 percent chance of survival. By contrast, if cancer is diagnosed at stage IV, patients have just a 5 percent or survival. The use of costly and invasive diagnostic approaches have been a barrier to early detection, but new technology has the potential to change that. Laboratory for Advanced Medicine is developing a simple blood-based test to detect cancer at its earliest stage. We spoke to Ken Chahine, CEO of Laboratory for Advanced Medicine, about the company’s blood-based test, how it works, and how it is prioritizing indications.

As the debate over drug pricing intensifies, biotech investor Peter Kolchinsky is weighing in with a proposed approach to balance access to medicines with the incentive for companies to invest in the development of innovative new therapies. In his new book The Great American Drug Deal, Kolchinsky makes the case for an approach to drug pricing that would ensure that the timely movement of innovative drugs to generic versions while also suggesting mechanisms for cutting the price of therapeutics after patents and exclusivity periods expire when competition fails to arise. We spoke to Kolchinsky about The Great American Drug Deal, his notion of a biotech social contract, and why it’s critical that the industry think differently than it has in the past about approaches to reform drug pricing.

There is growing scientific evidence suggesting that Alzheimer’s disease may be an autoimmune condition. Whether it is or not may be an unsettled issue, but targeting neuroinflammation associated with the disease is viewed by some as a potential therapeutic strategy. INmune Bio is developing an experimental, second-generation, selective TNF inhibitor that targets neuroinflammation. It believes this approach can slow or stop the progression of cognitive and psychiatric symptoms associated with the disease. We spoke to R.J. Tesi, CEO of INmune Bio, about whether Alzheimer’s disease is an autoimmune condition, the role of neuroinflammation in the progress of the disease, and how his company’s experimental therapy differs from existing TNF inhibitors today.

The Gloucester Marine Genomics Institute is seeking to harness biotechnology to discover new therapeutics by studying the DNA of marine life. At the same time, the institute hopes to breathe new life into a 400-year old fishing village that houses it and create new opportunities there. We spoke to Andrea Bodnar, science director at the Gloucester Marine Genomics Institute, about marine biotechnology, the ocean as a source for novel therapeutics, and the institute’s efforts to transform the economy along Cape Ann.

The latest reports on the coronavirus outbreak put the number of infections at nearly 75,000 and deaths at more than 2,100. Against the backdrop of the outbreak, we spoke to Evan Loh, chairman of the Antimicrobials Working Group and CEO of Paratek Pharmaceuticals, about the global changes that are fueling the threat of infectious disease outbreaks, the state of the antimicrobial arsenal, and what needs to be done to spur the development of new agents to combat the rise deadly bugs.

Orchestra BioMed may play at the intersection of drugs and devices, but’s its business strategy is clearly drawn from the biopharmaceutical industry. The company develops its pipeline and then leverages strategic alliances with global partners who can best commercialize its products and maximize their potential. We spoke to David Hochman, CEO of Orchestra BioMed, about the company’s therapeutic devices, the large market opportunities it is targeting, and how it seeks to rewrite the way medtech companies think about partnering.

Psychedelics have long been viewed as having potential to treat a range of mental health disorders including depression, addiction, PTSD, and ADHD. Government policies, though, have long impeded studies of their benefits. Mind Medicine is developing a pipeline of therapies based on psychedelics in the hopes of developing needed medicines for psychiatric conditions. Its lead experimental therapy is an ibogaine-derived molecule for the treatment of opioid addiction. We spoke to Steve Hurst, founder and CEO of MindMed, about the potential for psychedelic-based medicines, what’s known about them to date, and the challenges of working with these substances.

The difficulty in diagnosing Alzheimer’s disease and identifying it at its earliest stages when interventions offer the best opportunity for success, is one of the critical challenges in addressing the neurodegenerative condition. CorTechs Labs has developed quantitative analysis software that allows physicians to analyze brain images to diagnose and monitor patients with the diseases. We spoke to Chris Airriess, CEO of CorTechs, about the difficulty in diagnosing Alzheimer’s disease today, how its technology works, and its efforts to marry its software to genetic data as a way to identify and monitor people who may be at risk of developing the disease.

As people age, the ability of their eyes to focus on objects near to them weakens. The condition, known as presbyopia, affects more than 1.8 billion people worldwide. Though reading glasses provide a solution to the problem, Orasis Pharmaceuticals is developing a corrective eyedrop it says will provide an alternative. We spoke to Elad Kedar, CEO of Orasis, about aging eyes, its experimental eye drop intended to allow people to free themselves from a dependency on reading glasses, and how it works.

Ben Zeskind likens Immuneering’s platform technology to noise-cancelling headphones. Infact, the company’s use of the term “Disease Cancelling Technology” speaks directly to that. The approach, he says, allows the company to build a pipeline of drug candidate that address aspects of disease that have eluded tradition drug development approaches. We spoke Zeskind, CEO of Immuneering, about the company’s evolution from its roots in bioinformatics, its movement into drug development, and how its proprietary platform technology works.

One reason immunotherapies fail is because of the ability to tumors to alter the microenvironment in which they exist and hide themselves from detection by the immune system. In some indications, as few as 20 percent of patients benefit from checkpoint inhibitors. Oncolytics Biotech is developing pelareorep, an immune-oncolytic virus that activates the innate and adaptive immune systems, triggering inflammation in the tumor, and overexpressing checkpoints to increase the number of patients that can benefit from the use of checkpoint inhibitors. We spoke to Matt Coffey, president and CEO of Oncolytics Biotech, about Pelareorep, how it works to treat cancer, and why it can make checkpoint inhibitors more effective.

The brain converts short-term memories into long-term memories through the formation and stabilization of new connections between neurons. Tetra Therapeutics is working to treat cognitive impairment and memory loss from Alzheimer’s disease by developing an experimental therapy intended to stabilize these connections. We spoke to Mark Gurney, CEO of Tetra Therapeutics, about memory loss in Alzheimer’s disease, the company’s experimental therapy to treat the condition, and a recent study he was involved in that suggest TNF inhibitors may provide protection against the condition in patients with autoimmune conditions.