The Bio Report

The case for precision medicine to treat cancer has been clear. Understanding the molecular underpinnings of a patient’s cancer can allow doctors to use targeted therapies that can best address their particular tumor type. The people behind One Health believe it should be no different for dogs. The company, which bills itself as the world’s first canine cancer care organization, is seeking to bring precision cancer care to our four-legged friends. We spoke to Christina Lopes, founder and CEO of One Health, about the state of canine cancer care, the company’s FidoCure service and the market for state-of-the art medicine for dogs.

As potential vaccines for COVID-19 approached approval, the focus has been on the safety and efficacy of candidates. Now that vaccines have been approved, attention has been shifting to the complex logistical challenges of manufacturing, distributing, and delivering vaccines to patients. The process has opened up visibility into many aspects of the supply chain that are usual taken for granted. One of those aspects are the vials used to store that vaccines and the threat that a shortage of glass bottles could cripple distribution efforts. SiO2 Material Science, which won a $143 million U.S. government contract for vials and syringes, is applying semiconductor technology to create plastic containers with a nano coating of glass inside. We spoke to Christopher Weikart, head of scientific affairs and chief scientist for SiO2 Material Science, the considerations that go into a vaccine vial, the technology SiO2 is using, and why it offers advantages over traditional glass and plastic.

There are many approved or experimental therapies in development for a range of indications that, because of their mechanisms of action, have been pursued as potential therapies to treat patients with COVID-19. One of the most advanced candidates in this category is lenzilumab, an experimental monoclonal antibody that’s in development for certain cancers and other conditions. The antibody has the potential to neutralize a cytokine known as GM-CSF, which can trigger a severe immune reaction and cause hyperinflammation as a result of a cytokine storm. It is this immune response that underlies the most serious cases of COVID-19 virus. We spoke to Cameron Durrant, chairman and CEO of Humanigen, about lenzilumab, how it was recognized as a potential treatment for COVID-19, and the path forward for the therapy.

One of the challenges of conducting clinical trials is finding enough patient to include in a control arm of a study. This can slow the pace of drug development and increase its costs. Unlearn.AI is seeking to change that by using its artificial intelligence platform to create digital twins of trial participants that can serve as control arms in studies. We spoke to Charles Fisher, founder and CEO of Unlearn, about the concept of digital twins, the potential to accelerate clinical trials with their use, and why the company is focusing on the area of complex neurologic diseases.

Traditional efforts to treat autoimmune conditions have focused on inhibiting proteins involved in the immune response. Abivax is developing therapies that modulate RNA to enhance the body’s natural machinery to disrupt the production of cytokines, the proteins that signal the immune system to mount an attack in the first place. The company’s approach to modulating RNA may not only have utility in autoimmune conditions like inflammatory bowel disease, but in other indications including infectious disease and cancer. We spoke to Hartmut Ehrlich, CEO of Abivax, about the company’s lead therapeutic, how it works, and why the approach can have implications in a broad range of diseases.

Jo Bhakdi wants to build a future where people are protected against most diseases through early detection. His company Quantgene, which marries artificial intelligence with the ability to detect cell-free DNA in the blood with great sensitivity, believes it can help extend the lives of its customers by ten years within ten years. Quantgene is offering both its Serenity subscription service to consumers, as well as other offerings to therapeutic developers to support R&D using its technology. We spoke to Bhakdi, founder and CEO of Quantgene, about his unusual path to becoming a precision medicine CEO, how he expects technology to transform healthcare, and why he believes the company will be able to extend the lives of its customers through early detection of disease.

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.