The Bio Report

A gatekeeper of cellular metabolism known as mTORC1 underlies a wide range of age-related diseases. Navitor Pharmaceuticals is developing therapies that can modulate the mTORC1 complex and allow for a new approach to treating a wide range of diseases including autosomal dominant polycystic kidney disease and major depressive disorders. We spoke to Tom Hughes, president and CEO of Navitor, about mTORC1, the company’s platform technology, and why it has implications for a wide range of seemingly unrelated conditions.

Whether it is cells engineered to provide therapeutic benefits or biomanufacturing processes to replace energy-intensive and toxic chemical byproducts of industrial manufacturing, getting the right cell for the job is essential. Berkeley Lights has developed platform technologies that allow researchers to rapidly screen large numbers of cells and analyze them to identify the best cells for their purposes. We spoke to Eric Hobbs, CEO of Berkeley Lights, about the company’s platform technology, how it works, and how it can help accelerate the emergence of the new bioeconomy.

Glen de Vries in his new book "The Patient Equation" says that despite the digital revolution in the way we can capture and analyze data, not much has changed for decades in the how clinical trials are conducted. We spoke to de Vries, co-founder and co-CEO of the clinical trials data platform Medidata, about how clinical trials need to evolve, how technology can be used to improve patient access, and how it can capture new types of data to better answer questions about the safety and efficacy of therapies.

While immunotherapies carry great promise for improving outcomes for people with cancer, the ability of cancers to evade the immune system and develop resistance limits their benefits as monotherapies. BioEclipse Therapeutics is developing therapies that marry activated immune cells with oncolytic viruses. Together, they provide a synergistic effect that attacks cancers while providing protection against relapse and recurrence. We spoke to Pamela Contag, co-founder and CEO of BioEclipse, about the ability of cancers to return after treatment with immunotherapies, BioEclipse’s efforts to develop a multi-mechanistic immunotherapy to overcome that challenge, and why its approach may have broad application across a range of cancers.

While the potential for precision medicine has excited drug developers and clinicians with the promise of delivering more meaningful therapies to patients, the advent of these medicines has largely come in the area of cancer. As the understanding of the biology of other diseases is better understood, efforts to develop precision medicines are advancing into new areas. We spoke to Rachel Laing, managing director of the life sciences consulting firm Bionest, about the state of precision medicine, what can be learned from the experience in cancer, and what it will take to make precision medicine approaches the way we treat diseases broadly.

Zea Biosciences is not a typical contract manufacturing organization. The company uses plants to grow recombinant proteins for biologic therapies. To produce a consistent and predictable product, Zea uses a data-intensive approach and grows plants in clean rooms. The end result is a high-scalable platform that is cost-efficient. We spoke to Jim Wilson, CEO of Zea Biosciences, about the company’s approach to producing biologics in plants, why it is a data-intensive process, and the advantages it provides over traditional biomanufacturing.

United Biomedical has long been in the business of producing animal vaccines, but in recent years it has turned its attention to developing vaccines for chronic human diseases including neurological diseases, diabetes, and other targets. More recently it has used its vaccine platform to develop a multitope peptide COVID-19 vaccine and spun it out into a division dubbed COVAXX. We spoke to COVAXX co-founder and CEO Mei Mei Hu and COVAXX co-founder and Chairman Peter Diamandis, about the company’s efforts to develop a COVID-19 vaccine, how its peptide-based vaccine works, and the case for this approach. Since recording this interview, COVAXX began dosing participants in its phase 1 trial September 28, 2020.

CAR-T therapies are an area of great promise for improving outcomes for cancer patients, but the process of preparing cells taken from a patient and genetically modifying them is time consuming and costly. Exuma Biotech believes it can address the cost and time involved in the preparation of these products with its rapid, point-of-care platform technology. We spoke to Greg Frost, chairman and CEO Exuma Biotech, about CAR-T therapies, how its technology works, and how it is working to cut the cost, and speed the delivery of these immunotherapies to patients.

The administration of biologics can be challenging because of the sheer volume of product needed to deliver a dose to a patient. It is for this reason that many of these therapies must be infused into a patient, a process that is disruptive, costly, and can take several hours. Halozyme’s Enhanze drug delivery technology has been used by a number of biologics producers to take a therapy that would otherwise need to be infused to allow it to be administered with a subcutaneous injection. We spoke to Renee Tannenbaum, vice president of global partnering for Halozyme , about the company’s Enhanze drug delivery technology, how it works, and the company’s reorganization to focus on partnerships around it.

NASH, a form of fatty liver disease, causes progressive damage and can lead to inflammation, fibrosis, and the development of cancer. It has become an increasing health concern as the Western diet has spread throughout the globe. Today it is estimated that as much as 5 percent of the world’s population has NASH with a greater percent of people with the condition in the United States. There is no simple blood test to diagnose NASH, which means it often progresses to a fibrotic stage before it is detected. There is also no approved therapy to treat it. We spoke to Robert Foster, CEO of Hepion, about the company’s experimental therapy for NASH, how it works, and why it may also have utility as a treatment for COVID-19.

The complexity of neurodegenerative diseases has made it a challenging area for drug developers. Gene Kinney, CEO of Prothena, has long been involved in the pursuit of therapies for diseases such as Alzheimer’s and believes there has been great progress in understanding and targeting these conditions. In fact, he says we are entering a golden age of neuroscience. We spoke to Kinney about the state of drug development for neurodegenerative conditions, Prothena’s pipeline, and its effort to target protein dysregulation in these diseases.

The U.S. Food and Drug Administration delivered a blow to Pharnext in 2019 when it told the company it should conduct an additional late-stage study of its experimental therapy for the rare neurological condition Charcot-Marie Tooth disease. A manufacturing problem that affected the dose delivered to some patients invalidated one the arms of its study. The company uses artificial intelligence and network pharmacology to identify molecules that may be involved in modulating a disease and searches for synergistic combinations of both existing and novel therapies to treat conditions. Since it’s clinical setback, the company named David Horn Solomon as its new CEO, sharpened its focus, and moved forward with an additional study. We spoke to Solomon about the company’s approach to therapeutic development, its focus on neurological conditions, and the case for using combination therapies as the best way to tackle the diseases it targets.

MindMotion GO is a first-of-its-kind mobile neurorehabilitation therapy system that helps people regain motor and task functions through a gamified approach. While the game playing aspects of the technology may help engage patients in activities designed to help them recover from brain injury, the technology is validated by clinical studies and has won regulatory clearance. It should not be confused with conventional video games. We spoke to John Krakauer, chief medical advisor for MindMaze, about neurorehabilitation, how MindMotion Go works, and the benefits of being able to move neurorehabilitation out of the medical centers to treat patients in their homes, particularly during a pandemic.

People with the rare and fatal genetic disease spinal muscular atrophy in recent years have seen the approval of an antisense therapy as well as a gene therapy. Genentech has now won U.S. Food and Drug Administration approval for Evrysdi, the first oral, at-home treatment for the condition. We spoke to Levi Garraway, chief medical officer and head of global product development at Genentech, about Evrysdi, how it works, and how it fits into the choices physicians and patients have when it comes to treating spinal muscular atrophy.

While the revolution in genomics has led to rapid improvements in the cost and speed of sequencing and created new insights into the genetic drivers of health and wellness, proteomics has lagged behind. Being able to capture a comprehensive view of the changing level of proteins in an individual could play a significant role in bringing about an era of precision medicine. SomaLogic is providing a push in that direction with its SomaScan Discovery platform, which can read 5,000 protein measurements in the blood through a single assay. We spoke to Roy Smythe, CEO of SomaLogic, about the role proteins play in health and wellness, the way the company’s technology works, and the opportunity for it to help advance the area of precision medicine.

The problem of drug resistance isn’t limited to bacteria. It is also a growing concern with fungal species that is causing an increasing need for new agents to combat these microbes. Scynexis is developing the experimental therapy ibrexafungerp, the first of a new class of therapies for serious fungal infections. We spoke to Marco Taglietti, president and CEO of Scynexis, about the problem of drug resistant fungal infections, the company’s experimental therapy ibrexafungerp, and why it may provide a new way of treating a range of serious fungal infections.

Colorectal cancer is the third most common form of the cancer. While screening is an effective means of preventing it, many people fail to get a colonoscopy because of the invasive nature of the procedure and the preparation and sedation that goes with it. Check-Cap is a clinical-stage company developing C-Scan, the first capsule-based system for preparation-free, colorectal cancer screening. The capsule uses ultra-low dose X-ray and wireless communication technologies to generate information on the contours of the inside of the colon as it passes through it. This creates a 3D map that allows physicians to look for polyps and other abnormalities. We spoke to Alex Ovadia, CEO of Check-Cap, about colorectal cancer, why people avoid being screened as they should, and how the company’s C-scan capsule-based system works.

To see the affects of aging all you have to do is look in a mirror and watch the changes over time. Fountain Therapeutics is training its artificial intelligence platform to look at individual cells to detect changes that occur as cells get older and discover therapeutics that target underlying mechanisms of aging. The company believes this will provide new ways to target therapies to treat a range of diseases associated with aging. We spoke to John Dimos, CEO of Fountain, about the company’s AI platform, it’s approach to understanding aging at a cellular level, and how it provides new ways of discovering and developing therapeutics for diseases of aging.

The emergence of immunotherapies has represented a powerful addition to the cancer arsenal, but frequently they fail to deliver benefits to patients. Understanding what therapies will benefit which patients remains a challenge because of the complexity of the immune system. Immunai is applying artificial intelligence to map the immune system and understand its complexities at a granular level to better understand its role in health and disease. It is applying what it learns to avoid clinical trial failures, improve combinations of immunotherapies, and guiding future therapeutic development in cancer and a broad range of other conditions. We spoke to Danny Wells, scientific founder of Immunai, about the company’s efforts to map the immune system, the challenges in doing so, and how this has the potential to improve drug development.

Artificial intelligence is working its way into all aspects of pharmaceutical companies’ operations. While much attention has been given to the role these systems can play in drug discovery, IQVIA sees a significant opportunity to use them to transform the area of regulatory compliance. We spoke to Ronan Brown, senior vice president and head of IQVIA Integrated Global Compliance, about the role AI system can play in improving flagging returns on investment in R&D by allowing regulatory departments to operate more efficiently, breakdown data silos within pharmaceutical companies that hamper performance, and allow companies to focus less on rote work and more on regulatory strategies.