The Bio Report

More than 138,000 people in the United States and 1.4 million people worldwide are struggling with malignant brain tumors. The average five-year survival rate of people with the most common malignant brain tumor—glioblastoma multiforme—is less than 5 percent and there have been no notable improvements in the last three decades. The Ivy Brain Tumor Center is using phase 0 clinical trials to provide individualized treatment to patients with brain tumors. The U.S. Food and Drug Administration introduced phase 0 trials in 2004 to address concerns about the slow pace and high cost of drug development. The phase 0 trial is used to quickly identify how a drug works in a patient and whether it should be fast-tracked for further development. We spoke to Nader Sanai, director of the Ivy Brain Tumor Center and chief of neurosurgical oncology at the Barrow Neurological Institute, about phase 0 clinical trials, how they work, and how they are allowing the center to take a precision medicine approach to treating patients w

Traumatic injuries and surgical procedures can damage peripheral nerves and cause the loss of muscle or organ function, pain, and the loss of sensation. Axogen has a portfolio of regenerative medicine products to enable surgeons to repair peripheral nerves without the need to harvest nerves from a patient’s own body. We spoke to Karen Zaderej, CEO of Axogen, about peripheral nerve injuries, the company’s portfolio of products, and how it is changing the way surgeons can repair and regenerate damaged nerves.

About 20 years ago, the number of people dying from chronic diseases surpassed the number of people dying from infectious ones. Though vaccines have contributed to this shift, they’ve generally not been harnessed to address chronic conditions. Vaxxinity is seeking to change that by using vaccine technology to address the growing burden of chronic diseases. It’s advancing a new class of synthetic, peptide-based vaccines designed to activate the body’s immune system to produce antibodies to neurodegenerative, cardiovascular and other chronic diseases. The company’s pipeline includes experimental therapies for Alzheimer’s disease, Parkinson’s disease, and hypercholesterolemia. We spoke to Mei Mei Hu, CEO of Vaxxinity, about the case for using vaccines to address chronic diseases, the benefits this approach provides, and how its platform technology activates the immune system to battle non-communicable diseases.      

Developing therapies that work by degrading disease-causing proteins has shown promise, but first-generation approaches have been limited to targeting intracellular proteins and can’t reach membrane and extracellular proteins that represent about 40 percent of the proteome. EpiBiologics is working to expand protein degradation therapies to include membrane and extracellular targets with the goal of eliminating disease-causing proteins that were previously not addressable by traditional therapeutic approaches. We spoke to Rami Hannoush co-founder and interim CEO and president of EpiBiologics, about the company’s platform technology, how its protein degradation approach expands the potential targets for its therapies; and how its built an atlas of tissue-specific degrader antibodies to target proteins involved in cancer, immunologic-, and neurologic-related conditions.

While many people think of biotechnology in terms of its impact on medicine, it’s expected to transform the economy as its power reaches across industries. As biology increasingly becomes an engineering discipline, it is not only reshaping the way we produce food, but a wide range of industrial products as well. Bio-based processes are replacing petroleum-based ones and giving life to new biomaterials, bioplastics, and biofuels. As the SynBioBeta Global Synthetic Biology Conference returns to Oakland, California May 23 to May 25, we spoke to SynBioBeta Founder and CEO John Cumbers, about the state of the emerging bioeconomy, how biotechnology is being embraced across industries, and the unexpected places biotechnology is already showing up.

One reason that cancers can be difficult to treat is that the tumor microenvironment can hamper the ability of drugs to penetrate the tumor and facilitate the development of resistance to medicines. AUM Biosciences is developing targeted therapies that address genetic drivers of a cancer and can disable defenses that hide tumors from the immune system. The company’s lead experimental therapy is being tested in combination with immunotherapies to treat colorectal cancer and other solid tumors. We spoke to Vishal Doshi, chairman and CEO of AUM, about the development of resistance in cancers, the case of combining precision therapies with immunotherapies, and why he believes we can transform cancer from a deadly condition to a chronic disease.

While there has been great interest in harvesting and targeting the microbiome to treat disease, Federation Bio believes for durable benefits its necessary to provide a rich ecosystem of microbes in a single therapy. The company has developed a platform for producing synthetic microbial cell therapies to treat a range of diseases from metabolic disorders to metastatic cancers. Unlike earlier approaches, the company said it is generating potent, reproducible, and complete microbial consortia that stably engraft to provide predictable and durable responses. We spoke to Emily Drabant Conley, CEO of Federation Bio, about its platform technology, how it determines what to include or exclude in a given therapy, and what makes a disease a good candidate for its living therapeutics approach.

One of the challenges of treating traumatic injuries and degenerative conditions is that while there are therapeutic proteins that can promote repair, they can be difficult to deliver to the site of damage and get them to stay there long enough to provide benefit. Therapdaptive has developed platform technology that enables it to produce variants of recombinant proteins that can bind to the surface of implants, devices, and injectable carriers to allow for precision delivery anywhere in the body. We spoke to Luis Alvarez, CEO of Theradaptive, about the company’s platform technology to produce recombinant proteins that bind to materials, the broad applications for the technology, and how the roots of the company go back to a need he saw during his service in the Iraq war.

In 2022, investment in biopharmaceutical companies, pipeline activity, and the launch of novel medicines all dropped from the previous year. A new report from IQVIA’s Institute for Human Data Sciences argues the downturn, after two record-setting years, is a post pandemic return to longer-term trends. We spoke to Murray Aitken, executive director of the IQVIA Institute for Human Data Science, about what happened to the biopharmaceutical sector in 2022, how to make sense of the data, and the shifting landscape in therapeutic and geographic investments.

The aging process is associated with the development of diseases. BioAge Labs is seeking to expand healthy lifespans by developing therapies for aging-related diseases that target key pathways involved in the aging process. The company has built a platform that combines systems biology and AI to leverage extensive data sets to uncover molecular drivers of aging-related diseases. We spoke to Kristen Fortney, CEO of BioAge, about the company's approach to identifying targets for aging-related diseases, its therapeutic pipeline, and whether it's pursuing any novel targets yet.

Cell therapies are revolutionizing the way cancers are treated, but existing cell therapies have their limits. They have been more successful at treating hematologic tumors than solid tumors, and they can sometimes cause serious side effects, such as the destruction of antibodies or what’s known a cytokine storms in which the immune system gets over-revved and attacks healthy cells. Triumvira Immunologics is developing autologous and allogenic T cell therapies that it believes can address the limitations of existing cell therapies and be used to treat both liquid and solid tumors. We spoke to Paul Lammers, CEO of Triumvira, about the company’s platform technology, why it’s robust and versatile, and why its lead indication is for a cancer where effective treatments already exist.

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.

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.

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.    

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.

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.

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.

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.

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.

Providers hold vast amounts of health data that can be harnessed to gain better insights into diseases, improve outcomes for patients, and help bring about an era of precision medicine. Despite advances in artificial intelligence to capitalize on these stores of data, much of it has remained siloed and out of the reach of researchers and drug developers who could put it to use. Last year, a group of 14 health providers caring for tens of millions of patients through thousands of care facilities across the country formed Truveta, a company that has built an AI platform to make structured and de-identified patient data available for research. We spoke to Terry Myerson, CEO of Truveta, about its platform technology, the work it is enabling today, and how AI and the availability of real-world data is changing how biomedical research is conducted.