The Bio Report

Targeting a Regulator of Inflammation to Treat Alzheimer’s Disease

A complex of proteins that play an essential role in regulating the innate immune system known as the NLRP3 inflammasome is becoming a growing target of interest among drug developers to disrupt immune cell signaling. Halia Therapeutics is developing a pipeline of therapies that target the NLRP3 inflammasome to address not only inflammatory disorders like psoriasis and colitis, but neurologic conditions such as Alzheimer’s disease. We spoke to David Bearss, president and CEO of Halia, about the NLRP3 inflammasome, the role it plays in Alzheimer’s and other neurologic conditions, and the case for this therapeutic approach.

Enabling Regenerative Therapies without Immunosuppression

Though the transplantation of insulin-producing islet cells has been used to treat people with type 1 diabetes, such procedures require the use of immunosuppressive drugs to prevent the immune system from rejecting the cells. iTolerance is developing platform technology that can be used with tissue, organoid, and cell therapies without the need for life-long immunosuppression. We spoke to Anthony Japour, president and CEO of iTolerance, about the company’s platform technology that eliminates the need for immunosuppressive drugs with tissue and cell transplantation, the company’s lead experimental cell therapy for type 1 diabetes, and the broad range of regenerative therapies that could benefit from the technology.

Making Vaccine and Biotherapeutic Production Fast and Affordable

Dyadic is working to bridge the gap between high-yield, low-cost, and large-scale industrial biotechnology, and low-yield, high-cost, small-scale biopharmaceuticals. It’s C1 technology, which is a fungal expression system, can efficiently produce enzymes and other proteins. Earlier this year, the company achieved a milestone when it began dosing patients in a phase 1 clinical trial in South Africa of its COVID-19 booster vaccine. The company expects the first-in-human trial to accelerate the adoption of the C-1 production platform for vaccine and therapeutic candidates. We spoke to Mark Emalfarb, president and CEO of Dyadic, about the company’s fungal-based manufacturing platform, how it can product large volumes of enzymes and other proteins in a fast and cost-effective manner, and the potential this has to change the way biologics are manufactured.    

Forcing Cancers to Reveal Themselves

Sam Gambhir understood the ravages of cancer. His wife developed breast cancer and survived. His son later developed brain cancer at the age 15 and died. And in 2020, Gambhir himself succumbed to cancer. Before he died, though, Gambhir, who served as division chief of the Canary Center for Early Cancer Detection and Molecular Imaging at Stanford University, hit upon an idea. Rather than hunt for cancers in the hopes of making an early diagnosis, he devised a way for them to produce synthetic biomarkers to cause them to reveal themselves. He co-founded Earli, which seeks to enable the diagnosis cancers when they are most treatable. We spoke to David Suhy, co-founder and chief scientific officer of Earli, about the company’s synthetic biomarker technology that makes cancers visible with a PET scan, how it works, and how this has the potential to alter outcomes for patients by enabling treatments of patients before their disease progresses.

Making Clinical Trials Diverse and Accessible

Finding diverse groups of qualified participants for clinical studies can slow the development of needed medicines. The difficulty in attracting racial and ethnic minorities, women, and the elderly also creates concerns that trial results will fail to reflect what would happen in the real world. Some 80 percent of clinical trials fail to meet enrollment deadlines and an average of 30 percent of participants drop out in part because of the location and duration of these studies. CVS Health in 2021 launched CVS Clinical Trial Services, which capitalizes on CVS’ rich database of patients and its large footprint that puts a CVS location within 10 miles of 85 percent of the U.S. population. We spoke to Owen Garrick, chief medical officer of Clinical Trial Services at CVS Health, about the company’s push into clinical trials, the need to expand the diversity of participants in these studies, and how CVS is leveraging its resources to increase access to and participation in clinical trials.

Mining Bugs for Drugs

The microbial world has been a rich source of medicines, but our ability to explore the full potential of the microbes both in us and around us has been limited by technology and the difficulty of culturing most microbes in a lab. Biosortia Microbiomics has developed a platform for finding, amplifying, and screening microbes as a potential source of novel, small molecule drugs. We spoke to Ross Youngs, CEO and founder of Biosortia, about the case for exploring various microbiomes to discover new small molecule drugs, how the company’s platform technology enables it to investigate a much broader range of microbes, and its business model for capitalizing on the discoveries it makes.

A Company That Bets Its Therapies Will Click with Patients

One of the underlying limitations of most cancer therapies is the fact that only about 1 to 2 percent of the drug that’s administered ever reaches tumors. That limits the dose because of the potential for unwanted side effects, and limits the efficacy by delivering less than ideal amounts of drug to tumors. Shasqi is developing cancer therapies that use click chemistry to activate agents at the site of tumors to reduce systemic toxicity and increase the anti-tumor activity of its medicines. We spoke to José Mejía Oneto, founder and CEO of Shasqi, about the company platform technology, how it works, and why this can lead to safer and more powerful cancer therapies.

A Company Where It takes Guts and Brains to Develop Drugs

The gut is the body’s largest sensory organ with a surface area 100 times that of the skin. It contains more nerve cells than the spinal cord, most of the immune system, and 95 percent of the body’s serotonin. Kallyope has assembled a discovery and translation platform to developed therapies to treat metabolic, gastrointestinal, and neurologic conditions by targeting the gut-brain connection. We spoke to Nancy Thornberry, chair of R&D for Kallyope, about the gut-brain connection, the company’s platform technology, and the case for targeting these interactions to treat a wide range of conditions.