The Bio Report

While cell therapies have been advancing rapidly, therapies that rely on taking a patient’s own cells, altering them, and reinfusing them back into a patient are costly. Part of the reason why is that the process for doing this is labor intensive. Cellino has developed a platform for producing personalized autologous cell therapies in an automated and scalable fashion. We spoke to Marinna Madrid, co-founder and vice president of product, for Cellino, about the company’s platform technology for cell-based therapies, how it works, and why it may help enable a new era of personalized regenerative therapies.

Integrins are a diverse family of proteins that play an essential role in many cellular biological processes. They also have been implicated a number of autoimmune, cardiovascular, and metabolic diseases, as well as fibrosis and cancer. While a number of biologics have come to market that target integrins, drug developers have been stymied in efforts to develop oral therapies that can target these proteins. Morphic Therapeutic is developing a new generation of oral integrin drugs it believes can transform the treatment model for a range of serious medical conditions. We spoke to Praveen Tipirneni, president and CEO of Morphic Therapeutic, about intgerins, the challenges of using small molecule therapies to target them, and why this can have a dramatic effect on how patients with a range of conditions get treated.

As the treatment of cancers has moved toward an increasing emphasis on the role the immune system can play in fighting tumors, a range of new ways to enlist and train the immune system have emerged. Candel Therapeutics is developing oncolytic viral immunotherapies, which it says combines anti-tumor activity while also stimulating the immune system. We spoke to Paul Peter Tak, president and CEO of Candel Therapeutics, oncolytic viral immunotherapies, how they work, and why they may be able to bring benefits to the treatment of a range of solid tumors.

The discovery and cell line development of biotherapeutics has been traditionally a distinct process. Absci is taking what it describes as a more wholistic approach by collapsing the process down and addressing the functionality and manufacturability of therapeutic candidates simultaneously. We spoke to Sean McClain, founder and CEO of AbSci, about how it is using AI and synthetic biology to reinvent the discovery process, how this is expanding the therapeutic potential of proteins, and how it translates into time and cost benefits.

While CAR-T cell therapies have been a promising new area of cancer treatments, they are costly to produce, have had limited success in treating solid tumors, and can carry sometimes serious side effects. Shoreline Biosciences is developing off-the-shelf natural killer and macrophage cellular immunotherapies derived from induced pluripotent stem cells for cancer, inflammatory, and genetic diseases. The company programs these cells to target and kill tumors, as well as repair tissue homeostasis. The company said its approach allows for the creation of a streamlined, affordable, and scalable manufacturing process that can deliver cell therapy treatments to patients in a faster and more cost-effective manner. We spoke to Kleanthis Xanthopoulos, co-founder and CEO of Shoreline Biosciences, about the company’s off-the-shelf cell therapies, its focus on the innate immune system, and how it’s leveraging its technology through recent partnerships with BeiGene and Kite Pharma.

The ability to discover new drugs can be limited by the tools and technologies small companies can access. Alloy Therapeutics says it democratizing access to a set of technologies and capabilities that are foundational for discovering and developing biologics. We spoke to Errik Anderson, CEO of Alloy Therapeutics, discusses the challenges of drug discovery the company is seeking to address, its business model, and the dealmaking it has done to build beyond platform technologies to become involved in the discovery and development of therapeutic candidates.

One of the consequences of the proliferation of genetic sequencing has been a shortage of genetic counselors. It’s left existing counselors to manage a heavy load as its increased the demands on onboarding patients, consenting them, educating them and interpreting results. The digital health company Igentify is helping providers and genetic counselors scale their services with its AI-based platform that extends through the entire genetic testing process. We spoke to Doron Behar, co-founder and CEO of Igentify, about its platform technology, how it works, and why it will allow genetic counselors to handle a much larger volume of patients.

While CAR-T cell therapies have emerged as promising anti-cancer agents, their success have been focused on hematologic cancers. ArsenalBio is working to develop more robust cell therapies capable to treating a broader range of cancers by using synthetic biology to make them programable. We spoke to Ken Drazen, CEO of ArsenalBio, about the company’s next-generation T cell therapies, how it is able to program functions into these cells to overcome solid tumor defenses, and the company’s collaboration with Bristol-Myers Squibb announced at the start of the year.

Liquid biopsies have become associated with the area of oncology as they hold the promise of using fragments of DNA circulating in the blood that have been shed by tumors to provide early indications of recurrence and customize treatment strategies. The same approach, though, can be used for infectious diseases. Karius has developed a liquid biopsy test to provide rapid and non-invasive detections of more than 1000 pathogens from a single blood draw. The Karius test helps clinicians avoid invasive, low-yield, and sequential diagnostic tests that can delay treatment for the most vulnerable hospitalized patients. We spoke to Alec Ford, CEO of Karius, about the company’s liquid biopsy test, how it works, and how it can change the treatment of immunocompromised patients.

The World Health Organization estimates that 270 million people worldwide suffer from chronic hepatitis B virus infections, making it a global epidemic that affects more than twice the number of people with hepatitis C and HIV combined. HBV is a leading cause of chronic liver disease and the need for liver transplantation, with up to 1 million people worldwide dying from HBV-related causes each year. While current treatments reduce the viral load, they don’t eliminate it. Assembly Bio is developing a pipeline of therapies aimed at curing HBV. We spoke to John McHutchison, president and CEO of Assembly Bio, about HBV, the company’s efforts to develop a new class of drugs to treat the condition, and what he’s learned from his work at Gilead successfully developing and commercializing therapies for HCV. A note to listeners: As we were publishing this episode Assembly Bio announced plans to discontinue development of its phase 2 candidate ABI-H2158 because safety concerns. The company said it will focus on advanc

Psychedelics have become an area of increasing investment and drug development activities as an emerging group of companies is working to develop these compounds as treatments for neurologic and psychiatric conditions. That’s because of the ability of these drugs to target serotonin, which plays a critical role in the regulation of cognitive function, emotions, memory, and the sleep-wake cycle. Bright Minds Biosciences is developing a pipeline of psychedelics as the next generation of serotonin modulators to treat neuropsychiatric, seizure, and pain disorders by restoring serotonin activity to normal levels. We spoke to Gideon Shapiro, vice president of discovery at Bright Minds Biosciences, about the role serotonin plays in the health of the brain, why psychedelics have emerged as compelling therapeutics for a range of conditions, and the company’s pipeline of drugs in development.

Type 2 diabetes affects nearly 500 million people worldwide and more than 34 million people in the United States. The disease carries an elevated risks of heart attack, stroke, and other serious complications. Fractyl Health believes the approach to treating diabetes by controlling blood sugar levels and other symptoms has been faulty. Research has implicated a critical role the first section of intestine, known as the duodenum, plays in the condition and the company has developed a minimally invasive endoscopic procedure that it believes can correct the problem. We spoke to Harith Rajagopalan, CEO of Fractyl Health, about the company’s experimental procedural therapy to target the root cause of diabetes, how it works, and the path forward to commercialization.

The efforts to develop drugs for neurologic and psychiatric conditions has been plagued with failure. Herophilus is combining organoids derived from patient stem cells along with machine learning to gain new insight into the biology underlying these diseases and discover and develop more effective drugs. We spoke to Saul Kato, co-founder and CEO of Herophilus, about the drug development challenges for these diseases, how his company is using organoids and machine learning to better understand how to target them, and its growing pipeline therapeutic candidates.

While the class of immunotherapies known as checkpoint inhibitors has brought a promising new approach to treating cancer, the development of resistance to these therapies limits the number of patients they benefit. Portage Biotech is building a set of platform technologies and a pipeline of immuno-oncology therapeutic candidates to address this problem. We spoke to Ian Walters, CEO of Portage about the problem of resistance, the company’s pipeline of candidates, and its portfolio-based business model.

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 treati