The Bio Report

Until recently, treatments for both chronic and acute pain had been an area that went for decades without the development of innovative new treatments. South Rampart Pharma is seeking to develop safer and more effective pain management therapies that avoid the liver toxicity of acetaminophen, the kidney toxicity of NSAIDs, and the abuse potential of opioids. We spoke to Hernan Bazan, co-founder and CEO of South Rampart Pharma, about the need for new pain medicines, the company’s first-in-class experimental analgesic, and how it avoids the problems common with other pain medications.

The Zimin Institute for AI Solutions in Healthcare, a joint initiative between the Zimin Foundation and the Technion – Israel Institute of Technology, is seeking to accelerate the integration of AI into the life sciences. Although it is pursuing a wide range of opportunities from precision medicine to therapeutic discovery, its primary focus is on addressing real-world problems and conducting translational research. We spoke to Shai Shen-Orr, director of the Zimin Institute for AI Solutions, about the challenges the institute seeks to address, how it operates, and how it aims to bridge the gap between academic research and commercial applications in healthcare AI.

The search for non-opioid pain relievers opioids has focused on pharmaceutical alternatives including non-steroidal anti-inflammatories, antidepressants, and anticonvulsants. Brixton Biosciences is developing Neural Ice, an injectable frozen slurry that can degenerate nerves in targeted areas and provide pain relief for extended periods. The discovery capitalizes on unexpected findings about the effects of a fat-reducing cosmetic procedure. We spoke to Sameer Sabir, co-founder and CEO of Brixton Biosciences, about the unmet need for pain management, how Neural Ice works, and the company’s initial focus on osteoarthritis and post-operative knee pain.  

Antibodies have been powerful tools for inhibiting a targeted protein. Abalone Bio is pursuing a new class of antibody therapies called activating antibodies that can regulate cellular processes and restore their balance. One aspect that makes these rare antibodies attractive is that they can target previously undruggable G protein-coupled receptors, allowing them to treat diseases that have been hard to address. We spoke to Richard Yu, co-founder and CEO of Abalone Bio, about activating antibodies, how the company generates massive data sets for its AI-driven platform technology, and how it looks beyond binding to explore the function of these antibodies.

Jorge Goldstein trained for a career in molecular biology and biochemistry before becoming a patent attorney, a background that positioned him to help shape patent law for the biotech industry throughout his 40-year career. In his new book Patenting Life: Tales from the Front Lines of Intellectual Property and the New Biology, Goldstein offers a history of the biotech industry through the lens of the critical patent battles that shaped the landscape. We spoke to Goldstein, founder of the law firm Sterne, Kessler, Goldstein & Fox, about the industry’s critical patent battles, his new book, and how he is working today to use patents as instruments for social and economic justice.

Eosinophilic esophagitis is a progressive allergic disease characterized by difficulty swallowing and gastric reflux. It results from an elevated number of inflammatory immune cells in the walls of the esophagus. If left untreated, it can cause long-term complications, including scarring and difficulty swallowing. Revolo Biotherapeutics is developing a synthetic peptide therapy that can potentially restore immune system homeostasis in EOE and other allergic diseases. We spoke to Woody Bryan, president and CEO of Revolo, about eosinophilic esophagitis, how the company’s experimental therapy restores homeostasis to the immune system, and how people exposed to tuberculosis pointed the way toward the experimental therapy.

NK cells, part of the innate immune system, serve as the body’s first line of defense. These cells can recognize and kill abnormal or infected cells. As therapies, they have the advantage over CAR-T and other cell therapies because they can be used off-the-shelf without undergoing gene editing or other genetic modifications. They also don’t trigger cytokine storms, a common reaction to CAR-T therapies that can cause a systemic inflammatory response that can range from flu-like symptoms to life-threatening complications. The greater safety and lower costs of these NK cell therapies open the door to broader uses beyond cancer to include autoimmune diseases. We spoke to Fred Aslan, president and CEO of Artiva Biotherapeutics, about the company’s off-the-shelf NK cell therapies, the case for pairing them with monoclonal antibodies, and how they can broaden the uses for cell therapies.

More than a decade after the approval of a curative therapy for hepatitis C, hepatitis B has proven more challenging to tackle. Vir Biotechnology, in collaboration with Alnylam Pharmaceuticals, is developing a promising combination approach that marries Vir’s monoclonal antibody tobivibart with Alnylam’s siRNA elebsiran. We spoke to Mark Eisner, chief medical officer of Vir, about hepatitis B, the company’s combination therapy in development with Alnylam, and its platform technologies for developing treatments for infectious disease and oncology.

While GLP-1 agonists have been all the rage in treating obesity, Coya Therapeutics sees potential for these therapies to address inflammatory diseases. In fact, Coya is developing its low-dose interleukin 2 in combination with several different agents. The belief is that its approach will address inflammation by targeting dysfunctional regulatory T cells. The company is pursuing multiple neurodegenerative conditions, as well as autoimmune and metabolic diseases. We spoke to Arun Swaminathan, CEO of Coya Therapeutics, about its pipeline-in-a-product strategy to treat neurodegenerative and other inflammatory diseases, its pursuit of a GLP-1 combination therapy for these conditions, and the challenges of being a newly minted public company in the current financial environment.

Though cancer vaccines have been an area of great promise, in practice they have faced several challenges because of the heterogeneity of tumors, the ability of the tumor microenvironment to suppress the immune system, and the challenges of producing a strong and sustained T-cell response. OSE Immunotherapeutics’ off-the-shelf cancer vaccine Tedopi has shown promising results in a phase 3 study in patients with non-small cell lung cancer and the company is now conducting a confirmatory phase 3 study. We spoke to Nicolas Poirier, CEO of OSE Immunotherapeutics, about the company’s off-the-shelf cancer vaccine, non-small cell lung cancer, and how it is leveraging its immune system expertise through partnerships with leading pharmaceutical companies.

One of the challenges in treating brain cancer and other diseases of the central nervous system is delivering therapeutics beyond the blood-brain barrier. NeOnc Technologies using a natural compound derived from essential oils in plants that not only can kill cancer cells, but can cross the blood-brain barrier. What’s more, it can transport other therapies as well. We spoke to Thomas Chen, founder and CEO of NeOnc, about brain cancer, how the blood-brain barrier complicates the delivery of therapies to treat the condition, and how its experimental candidate that is delivered intranasally works.

Some 70 percent of potential therapeutic targets are believed to be beyond the reach of conventional small molecule therapies or biologics. Macrocyclic peptides offer a way to get at elusive targets while providing desirable characteristics of both small molecule drugs and biologics. They offer oral bioavailability, can permeate cells, and engage complex targets with specificity. Unnatural Products is harnessing AI to create synthetic macrocyclic peptides to pursue previously undruggable targets. We spoke to Cameron Pye, CEO and co-founder of Unnatural Products, about how these peptides can target proteins that traditional therapies cannot reach, the engineering process for the company’s synthetic macrocyclic peptides, and their potential to lead to new innovative therapies for a range of conditions from cancer to obesity.

Through the creation of a chemical programming language, Chemify said it has been able to expand the chemical space it can explore. Chemify is combining this unique programming language with robotics and AI to digitize chemistry. The company, though, has much grander ambitions than being a drug developer. It wants to be the infrastructure for the industry. We spoke to Lee Cronin, founder and CEO of Chemify, about the company’s platform technology for digitizing chemistry, how its chemical programming language expands the chemical space it can explore, and how the integration of robotics into its platform accelerates drug discovery.

Prescription digital therapies, the use of software to treat disease, are growing in number. Already there have been 140 prescription digital therapies that have been granted market access through national regulatory and reimbursement pathways. That represents a five-fold increase in the since 2021, according to the IQVIA Digital Health Trends 2024 report. We spoke to Murray Aitken, executive director of the IQVIA Institute for Human Data Science, about the report, why digital health companies faced challenging times in recent years, and why your next prescription may be in the form of an app. 

There has been growing interest in the potential of psychedelics to treat a variety of mental health conditions. While some focus has been put on synthesizing compounds that can target the receptors psychedelics bind to without inducing their hallucinatory effects, Filament Health is taking a notably different approach. The company, co-founded by Ben Lightburn who previously ran a firm specializing in natural extraction technologies, is focusing on developing pharmaceutical grade botanical drugs that may benefit from multiple active compounds rather than an isolated active ingredient. We spoke to Ben Lightburn, founder and CEO of Filament Health, about the company’s approach to psychedelic medicine, the case for natural botanical extracts rather than synthetic compounds, and its lead experimental therapy that targets methamphetamine use disorder. 

Gain Therapeutics' platform technology Magellan leverages AI, structural biology, and physics-based models to identify novel binding sites on otherwise undruggable proteins implicated in diseases. The company’s experimental Parkinson’s disease therapy has the potential to slow or stop progression of the neurodegenerative condition by stabilizing a lysosomal enzyme implicated in the disease. We spoke to Gene Mack, interim CEO of Gain, about the company’s platform technology, how its experimental therapy for Parkinson’s disease works, and what other conditions might be good candidates for its platform technology to target.

We continue our holiday tradition by welcoming STAT News Senior Biotech Writer Adam Feuerstein for our annual look back at the year that was in biotech and what’s ahead for the industry with the JPMorgan Healthcare conference and beyond in 2025. Feuerstein offers his view on finance and dealmaking in 2024, new drug approvals, and his annual take on the best and worst CEOs of the year. We also discuss what Trump 2.0 may look like for the industry, changes coming to the FDA and other agencies, and what hot technologies to watch in the year ahead. 

Prolific Machines uses light to precisely control virtually any function in any cell to transform what is possible with biomanufacturing. In combination with optogenetics and AI, the technology has the potential to impact a wide range of industries, from food production to pharmaceuticals, by enabling new capabilities, reducing costs, and improving sustainability. We spoke to Deniz Kent, co-founder and CEO of Prolific Machines, about the company’s photomolecular platform technology, the benefits it provides over traditional biomanufacturing methods, and how it could be used to not just cultivate meat but make a steak.

ImpriMed is working to deliver on the promise of precision medicine by using a patient’s live cancer cells to see how they respond to different treatment options and artificial intelligence to predict which medicines will work best. The company has had impressive results with its customers to date, but the catch is that it has initially targeted its service to the veterinary market, and its dogs and cats have benefited from it. The company is now working to bring its offering to two-legged patients. We spoke to Sungwon Lim, CEO of ImpriMed, about its functional precision medicine and AI platform to match cancer patients to the best available therapy for them, its decision to roll out the service first to the veterinary market, and what it is doing to bring its service to humans.

One of the limitations of small molecule drugs and monoclonal antibodies is the difficulty they face in binding to a large number of proteins that could prove to be critical targets in combating various diseases. Aikium is harnessing the power of artificial intelligence and synthetic biology to create a new class of protein biologics called SeqRs that are designed to bind to disordered regions of proteins that have long been beyond the reach of traditional therapeutics. We spoke to Eswar Iyer, co-founder and CEO of Aikium, about the novel class of SeqR proteins the company is developing, how they can bind to targets that traditional medicines can’t, and the potential to transform drug development by expanding the world of druggable targets.