The Bio Report

Earth will not live forever. Whether its destruction comes as the result of cosmic inevitability or human-caused cataclysm, the planet will eventually meet its demise. Though that may be billions of years from now, Chris Mason in his book “The Next 500 Years,” argues humans have a more obligation to do what they can ensure life from Earth can extend beyond the planet. Mason, principal investigator on NASA’s twins study and a professor at Weill Cornell Medicine, discussed the effects of space on the human body, how it may be possible to genetically engineer human and other life forms to thrive in environments less hospitable than Earth through the use of new genetic tools, and the ethical considerations around these issues.

While great strides have been made the fight against cancer with new therapies, dose-limiting toxicities of these agents can have a big impact on the quality of life for patients and lead doctors to alter dosing at the price of optimal outcomes. Rather than treating the symptoms of the side effects of cancer drugs, OnQuality Pharmaceuticals is developing a pipeline of therapies that target the biologic pathways that at their source. It is developing targeted supportive therapies to treat such things as dermatologic conditions and diarrhea caused by cancer medicines. We spoke to Michael McCullar, CEO of OnQuality, about the need his company is addressing, its pipeline, and how he expects others to think about the value of the therapies the company is developing.

While great progress has been made in understanding the human genome, its functional counterpart, the human proteome, remains relatively unexplored. In part, that’s because advances in the tools to identify the far bigger universe of protein variants has been lacking. Seer Bio has developed a platform for large-scale proteomics studies that it says is both unbiased and scalable. We spoke to Omid Farokhzad, chair and CEO of Seer Bio, about the state of proteomics, How Seer’s technology works, and why it can help advance our understanding of health and disease.

The development of mRNA therapies is allowing drug developer to pursue targets once considered undruggable. Anima Biotech has developed a platform that allows it to use small molecule that selectively control mRNA and can decrease or increase the translation of proteins. The approach has broad applicability as evidenced by the company’s pipeline, which includes experimental therapies for oncology, neurology, infectious disease, and fibrosis. We spoke to Yochi Slonim, CEO of Anima, about the company platform technology, how it can modulate the expression of proteins, and how it is leveraging its technology through partnerships with some of the world’s largest pharmaceutical companies.

The biopharmaceutical industry has long struggled with R&D productivity. Long time industry strategic consultant Mike Rea, founder and CEO IDEA Pharma, thinks he’s hit on a possible solution. Taking a cue from the tech industry, Rea in May announced the launch of Protodigm, which he describes as a contract skunkworks company. The approach is intended to allow a multi-disciplinary team work autonomously to take its clients early-stage assets and explore multiple development options at once with the intent of de-risking innovation while saving time and money. We spoke to Rea about the industry’s challenges with R&D productivity, the benefits Protodigm’s skunkworks approach could provide, and how it will work with industry.

While cities across the country have lifted COVID-19 restrictions and public gatherings have returned, the lack of effective treatments for people who develop severe reactions to the virus continues to pose a public health problem. The situation is acute in less developed nations where vaccination levels are low, and hospitals continue to be overrun with patients. Researchers at Mount Sinai earlier this year reported in the journal Cell that a widely available and inexpensive drug targeting inflammatory genes has reduced morbidity and mortality in mice infected with the virus that causes COVID-19. The drug, topotecan, is now in clinical trials in India as a potential treatment for patients who suffer from a hyper-inflammatory response to the virus. We spoke to Ivan Marazzi, senior author of the study and an associate professor of microbiology at the Icahn School of Medicine at Mount Sinai, about topotecan, what makes it compelling as a potential treatment for COVID-19, and why it may be well suited for treating people with the condition across the globe.

Osteoarthritis is a painful, progressive disease of the joints for which there is no cure. The condition affects more than 32.5 million adults in the United State and the incidence is growing as a result of aging, obesity, and sports injuries. Flexion Therapeutics is developing an experimental gene therapy to treat the condition. Rather than correcting an underlying genetic mutation, the experimental therapy delivered into the joint codes for the production of the anti-inflammatory protein interleukin-1 receptor antagonist. It is expected to deliver as-needed anti-inflammatory activity to joint tissues over the long-term. We spoke to Mike Clayman, co-founder and CEO of Flexion Therapeutics, about osteoarthritis, the problem with existing therapeutic approaches, and the company’s efforts to develop a gene therapy to get the body to produce an anti-inflammatory protein as needed.

Sumitomo Dainippon Pharma’s $3 billion strategic transaction with Roivant Sciences that gave rise to Sumitovant, represents an effort by the Japanese drug company to make the drug development process faster and more efficient. A critical piece of this effort is steps the company has taken to harness information technology to capitalize on existing knowledge to inform decision making. We spoke to Bill McMahon, chief algorithmic analytics officer of Sumitovant, about the approach Sumitovant is taking, the way uses information technology to improve the drug development process, and how the subsidiary may be influencing the way its parent company operates.

The ability to identify diseases at their earliest stages through the detection of minute levels of biomarkers offers the potential to make interventions before the outward physical manifestations of illnesses can bloom and long-term damage can occur. Quanterix is developing a platform of ultra-sensitive biomarker tests to give researchers greater insight into the transition from health to disease and the ability to detect diseases at an earlier point in their development. We spoke to Kevin Hrusovsky, chairman, president, and CEO of Quanterix, about the company’s ability to detect minute amounts of biomarkers, the implications this has for drug development and diagnostics, and how this can help drive a future of precision health.

The marriage of synthetic biology to mRNA is creating the potential for a range of programmable therapeutics that can provide new ways of treating deadly and chronic diseases. Strand Therapeutics is creating a platform for developing these long-acting mRNA drugs that it says can be precise, multi-functional, and deliver potentially curative treatments with a single dose. We spoke to Jake Becraft, co-founder and CEO of Strand, about the company’s programmable mRNA therapeutic platform, how it works, and the indications it is pursuing.

Cardiometabolic conditions, such as type 2 diabetes and heart disease, have long been treated with pharmacologic interventions. Better Therapeutics believes cognitive behavioral therapy delivered through its prescription digital therapeutics platform, will provide better outcomes at a lower cost. We spoke to David Perry, co-founder and executive chairman of Better Therapeutics, about the company’s prescription digital therapeutics platform, the case for personalized cognitive behavior therapy delivered this way, and what it will take to get doctor to prescribe it and payers to reimburse for it.

The side effects of opioids and the potential for addiction underlies the need for new approaches to the treatment of pain. SiteOne therapeutics is developing a pipeline of therapies that target sodium ion channels to block the electric signals sent from the site of pain. Unlike other similar drugs, SiteOne is able to target these sodium channels with great specificity to avoid the types of side effect that have limited the value of drugs that target these proteins. We spoke to John Mulcahy, president and CEO of SiteOne, about the company’s approach to pain, its pipeline, and how its therapies are able to hit their targets with precision. Special offer to listeners of The Bio Report: With the Digital Library from DeepDyve, you can search 100 million scientific papers with full access to 20 million articles, annotate them, and share with colleagues. It’s one-stop, affordable research. Try one month of DeepDyve’s enterprise service for free. Go to deepdyve.com/podcast and use the code BIOREPORT.

Earlier this year NovelluxDx rebranded itself as Fore Biotherapeutics signaling a shift from being a diagnostics company to its new life as a precision cancer therapeutics drug developer. Building on its functional genomics platform, the company is in-licensing small molecule drug candidates it sees having potential to treat hyper-targeted patient populations. We spoke to Usama Malik, CEO of Fore Biotherapeutics, about the move from diagnostics to drug development, the approach Fore is taking, and why it thinks it can see value in molecules that others may miss.

When variants of the COVID-19 virus emerged in late 2020, it raised concerns about whether people already infected with the original virus, or vaccinated against it, would be able to generate a protective immune response that would confer protection against these new strains. Researchers at the National Institutes of Health’s National Institute of Allergy and Infectious Diseases and Johns Hopkins University recently reported encouraging findings from a study that took a detailed look at the antibodies in the blood from COVID-19 patients. To do this, they used a deep immunomics technology platform developed by ImmunoScape. We spoke to Brian Abel, senior director of business development at ImmunoScape and lead of the company’s COVID-19 Vaccine Task Force, about the COVID-19 study, the company’s technology, and how it is being used to develop the next generation of immunotherapies.

Hypoxia, a potentially fatal shortage of oxygen in tissues, is a complicating factor in a variety of serious illnesses including cancer, cardiovascular disease, and respiratory conditions. Diffusion Pharmaceuticals lead experimental candidate, TSC, enhances the ability of the body to deliver oxygen to where it is needed most. We spoke to Bob Cobuzzi, CEO of Diffusion Pharmaceuticals, about hypoxia, the company’s lead therapeutic candidate to address it in a broad range of conditions, and its clinical development strategy for the drug.

Wet age-related macular degeneration is a progressive disease and a leading cause of vision loss in patients over the age of 60. Vision loss is caused by the leakage of blood and other fluid from abnormal blood vessel growth underneath and into the retina. Though the condition is treated by a class of therapies known as VEGF inhibitors, these biologics need to be repeatedly injected into patients’ eyes and for a variety of reasons patients’ real-world experience with the drugs don’t match clinical trial outcomes. Adverum Biotechnologies is developing a gene therapy to treat the condition. Rather than addressing an underlying genetic cause though, the gene therapy carries a sequence that causes the eye to produce aflibercept, the VEGF inhibitor marketed as Eyelea and Zaltrap. We spoke to Laurent Fischer, CEO of Adverum, about the company’s gene therapy, how it works, and why the one-and-done approach could translate into better results for patients.

TNF inhibitors, such as Humira and Remicade, have been a great biotech success story. These multi-billion franchises treat a range of autoimmune conditions including rheumatoid arthritis, psoriasis, and Crohn’s disease. Jim Woody, who led Centocor’s development team for Remicade, the first of the TNF-inhibitors, is today in pursuit of new uses for these therapies. Now CEO of 180 Life Sciences, Woody and his team are pushing TNF inhibitors into new indications for inflammatory and fibrotic conditions. We spoke to Woody about the role of TNF in the inflammatory process, the indications his company is pursuing, and why these well-established drugs have gone untapped for these purposes. The Bio Report welcomes DeepDyve as a sponsor. Search 100 million scientific papers with full access to 20 million articles. It’s one-stop, affordable research. Try the enterprise version of the service free for one month. Go to deepdyve.com/podcast and use the code BIOREPORT

The plant world has been a ready source of bioactive compounds that can improve human health. There are more than 10 million natural compounds in the plant world, but less than 0.1 percent of these compounds have been explored. Brightseed’s Forager is an artificial intelligence discovery platform that is being used to map millions of bioactive natural compounds to identify ones with potential to address human health needs. We spoke to Jim Flatt, co-founder and CEO of Brightseed, about the company’s discovery platform, its business model, and why it’s pushing beyond nutrition and supplement companies to forge partnerships with drug developers.

TFF Pharmaceuticals thin film freezing technology allows it to take vaccines, small molecule drugs, and biologics and turn them into a fine powder. The process not only can be used to eliminate the need for low temperature storage of certain medicines, but also improve the of water solubility of drugs and allow them to be delivered as inhaled therapies. We spoke to Glenn Mattes, president and CEO of TFF Pharmaceuticals, about its platform technology, the company’s efforts to build a pipeline by reformulating existing therapeutics, and its plans to use partnerships to realize the full potential of the technology.

The gene editing technology CRISPR-Cas9 is being harnessed to alter DNA, but Locanabio is using it to create a new class of genetic medicines that can precisely target and modify dysfunctional RNA. The company says that its approach will allow it to produce in vivo medicines that combine the specificity of RNA-binding proteins with the effect of a one-time gene therapy. We spoke to Jim Burns, CEO of Locanabio, about its platform technology, the benefits of using CRISPR to target disease-causing RNA, and why this approach could be applied to a broad range of genetic diseases.

In January 2020, antisense pioneer and Ionis Pharmaceuticals founder Stanley Crooke launched a nonprofit to design and deliver custom RNA-targeted therapies free of charge for individual patients with ultra-rare diseases. The organization, n-Lorem Foundation, leverages Ionis’ technology platform to speed the discovery and development of custom antisense oligonucleotides. More than a year later, the work of the foundation is well underway with a number of therapies in development to treat individual patients. We spoke to Crooke, CEO of the n-Lorem Foundation, about the need the foundation is addressing, why antisense oligonucleotides are well suited to the task, and what challenges it faces in scaling the operations to address the needs of a greater number of patients.

Octave Bioscience is seeking to transform the way neurodegenerative diseases are managed by providing new insights into the severity of a patient’s condition, its progression, and using evidence-based insights to improve outcomes. The company, which recently raised $32 million, is completing development of its care management platform for neurodegenerative disease. We spoke to Bill Hagstrom, CEO of Octave, about the company’s efforts to harness novel measurements of disease to individualize care, the case it’s making with payers, and why the company is starting with MS.

Phosplatin Therapeutics is developing a class of small molecule cancer therapies designed to avoid the problems of drug resistance and toxicity associated with chemotherapies. The company’s lead experimental therapy is a first-in-class small molecule that promotes immunogenic cell death, a type of cell death that elicits an immune response. We spoke to Matthew Price, co-founder, executive vice president, and chief operating officer of Phosplatin, about the company’s lead therapy, its multiple mechanisms of action, and why it may have benefit in a broad range of cancers.

Earlier this year Paige, a company developing artificial-intelligence driven pathology platforms for the diagnosis of cancer, raised $100 million in a series C venture round. The funding came a month after the company, a spin-out of Memorial Sloan Kettering Cancer Center, scored its first two European approvals for its breast cancer and prostate cancer offerings. We spoke to Leo Grady, CEO of Paige, about how the company’s artificial intelligence platform works, why it may lead to the identification of new biomarkers, and the potential for AI to change the way cancer patients are diagnosed.

While cell and gene therapies represent an area of great therapeutic promise, current manufacturing process are expensive and difficult to scale. As a result, this creates bottlenecks that limit patient access to these therapies and hamper the growth of the sector. Ori Biotech has developed an automated and scalable manufacturing platform for cell and gene therapies that seeks to increase capacity and quality while reducing costs. We spoke to Farlan Veraitch, co-founder and chief scientific officer of Ori, about the challenges of manufacturing cell and gene therapies, how the company’s platform addresses those issues, and why it is initially focusing on CAR-T cell therapies.

There’s been a growing industry effort to explore the use of psychedelics for their medicinal benefits. While much of these efforts have looked to these drugs to treat a range of psychiatric conditions, Eleusis sees a broader potential for them. While the company is pursuing psychedelics as potential treatments for major depressive disorder, it also is developing psychedelic candidate in other indications because of their anti-inflammatory properties. We spoke to Shlomi Raz, CEO of Eleusis, about of the case for psychedelics as treatments for inflammatory conditions, how they work, and what challenges the development of these drugs pose.

Regulatory T cell therapies have been pursued as treatment for cancers, but Sonoma Biotherapeutics believes these cells can be harnessed to delivery lasting and potential curative treatments for autoimmune and degenerative diseases. We spoke to Jeff Bluestone, CEO of Sonoma, about regulatory T cell therapies, why they may have broad application in a range of conditions, and what challenges will need to be overcome to usher in an area of cell therapies beyond cancer.

For more than 15 years, Longhorn Vaccines & Diagnostics has been designing and developing products for potential pandemics in developing economies. The recent COVID-19 pandemic, though, put the scalability of the company’s technology to the test as it landed big contracts in the United States for COVID-19 testing. We spoke to Jeff Fischer, president of Longhorn, about the benefits of the company’s technology for gathering and transporting diagnostic samples, how it’s used partnerships to scales it business with speed, and why the pandemic has forever changed the company.

There are many reasons why the sharing of medical data that could be used to gain new insight into diseases can be hampered. Privacy concern, regulatory burdens, and the need to manage security risks are among the significant impediments. Syntegra believes it can solve these problems through its artificial intelligence technology that creates synthetic datasets designed to mirror the statistical properties of real datasets while removing all links to the original patients behind the data. We spoke to Michael Lesh, co-founder and CEO of Syntegra, about the obstacles to data-sharing, how synthetic datasets are developed, and why they might accelerate the pace and lower the cost of research.

While checkpoint inhibitors represent a class of promising new therapies to treat cancer, the efficacy of these immunotherapies have been limited because of the ability of cancers to develop resistance. In part, that’s because of the multiple mechanisms cancers have to evade the immune system. Compugen is using a computational discovery platform to identify proteins and pathways that drive immune resistance mechanisms to checkpoint inhibitors. We spoke to Anat Cohen-Dayag, CEO of Compugen, about the company’s discovery platform, its efforts to develop new treatments that address patients who don’t respond to current checkpoint inhibitors, and its clinical pipeline in development.

The raging pandemic defined 2020, but it was a big year for biotech as the industry saw soaring stocks, record investment, and an impressive number of new drug approvals despite disruptions from COVID-19. We continue our annual tradition of sitting down with Adam Feuerstein, senior writer for Stat and the publication’s national biotech columnist, to discuss the year that was in biotech, the trends that drove the numbers, and what’s ahead in 2021.

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.