The Bio Report

Delivering biologics orally rather than through injection has been an intriguing goal but has proven difficult. Most efforts have focused on finding ways to turn these large protein molecules into formulations where they would not breakdown in the along the digestive tract before they can be absorbed and provide a therapeutic benefit. Rani Therapeutics has taken an unusual tact. Rather than reconceiving the biologic, Rani has reconceived the pill itself. The company has developed what it calls a “robotic” pill that carries the therapeutic to the gut where it injects the drug into the wall of the intestines. We spoke to Mir Imran, chairman and CEO Rani Therapeutics, about how the Rani Pill delivers biologics orally, the technology underlying it, and how the company thinks about the opportunities it will pursue.

Luis Alvarez, a West Point graduate who earned a Ph.D. in bioengineering from MIT, served 20 years in the military including time as an intelligence officer in Iraq. He saw injured soldiers who doctors were able to save, only to later have their limbs amputated because of the inability for injuries to heal properly. The experience led him to develop a means of turning recombinant proteins into a form that allows them to be used as coatings that act like paint and can be applied to implants to promote growth and other benefits. We spoke to Alvarez, founder of Theradaptive, about his journey from the battlefield to the lab, how his company’s platform technology works, and the range of applications to which it may be applied.

Last month, Canadian regulators provided clearance for Appili Therapeutics to begin a phase 2 study of an approved antiviral therapy as a potential preventative treatment against COVID-19 outbreaks. The study will enroll 760 participants who are in long-term care facilities in Ontario. Though others are looking at the drug as a possible treatment for COVID-19, this is the first study to consider its potential to prevent outbreaks. We spoke to Armand Balboni, CEO of Appili, about drug, how it works, and its potential to prevent outbreaks of COVID-19 in high-risk populations.

The urgency to find treatments for the COVID-19 virus has allowed researchers to set aside institutional bureaucracy and companies to apply their technologies in new ways. One example of this is Scipher Medicine’s collaboration with Northeastern University’s Barabasi Labs, Harvard Medical School, and Network Science Institute. The company is using its artificial intelligence platform to help identify existing therapies that might be repurposed as treatments for COVID-19. We spoke to Alif Saleh, CEO of Scipher, about the collaboration, the approach Scipher is using to identify drug candidates, and how this might expand on the company’s business strategy.

As a researcher, Joe Garcia applied functional genomics to understanding genes that contribute to inflammatory disorders such as acute respiratory distress syndrome, or ARDS. As founder and CEO of the biotech company Aqualung Therapeutics, he’s working to advance therapies to hit these novel targets to treat uncheck inflammation with the company’s lead experimental therapeutic candidate targeting ARDS. We spoke to Garcia about the company’s ARDS therapy, how it works, and why it’s a timely focus given the COVID-19 pandemic.

Bacteriophages have long been used to treat infections. These naturally occurring virus are capable of killing bacteria, but each strain of phage is highly specific. Because of their unique mechanism of action, they provide a potential to address the growing threat posed by multidrug-resistant bacteria, but to treat someone, the right phage must be matched to each patient’s infection. Adaptive Phage Therapeutics believes it’s found a way to create phage therapies suited to treat patients with drug-resistant infections by building a bank of targeted and genomically-screened bacteriophage and testing individual patient’s bacterial colony against that to determine the appropriate phage to treat them. We spoke to Greg Merril, co-founder and CEO of Adaptive Phage Therapeutics, about the origins of the company, how its technology works, and the regulatory hurdles for producing customized therapies to treat individual patients.

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.