The Bio Report

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.