The Bio Report

CytoMed, a spin out of A*STAR—Singapore’s Agency for Science, Technology, and Research—is developing novel cell-based immunotherapies that can go beyond the limitations of existing CAR T therapies. The company is focused on the development of off-the-shelf therapies derived from the delta gamma T cells from healthy donors. We spoke to Wee Kiat Tan, chief operating officer of CytoMed, about the company’s platform technology for off-the-shelf CAR T therapies, the advantages they provide over existing autologous CAR T therapies, and why he believes they will be successful at treating solid tumors.

For academic scientists who make groundbreaking discoveries that can lead to new medicines, bridging the so-called valley of death—the gulf between the lab and having a validated candidate that is ready to begin human clinical trials—continues to be daunting. Foundery Innovations, which bills itself as a venture studio, sees a big opportunity in partnering with academic researchers to carry promising new immunotherapies through to the clinic. Its team has deep experience in immunotherapy development and brings a wide range of skills and cutting-edge platform technologies to de-risk development and accelerate the time to the clinic. We spoke to Max Krummel, founder and managing member of Foundery Innovations, about the venture studio’s business model, the resources it brings to bear, and how it works with academic researchers and universities.

Vericel, which acquired Sanofi’s cell therapy and regenerative medicine unit, markets advanced cell therapy products for sports medicine and severe burn care. The company’s MACI is an autologous cellularized scaffold used in the repair of cartilage defects of the knee. Epicel, its cultured epidermal autografts, are a permanent skin replacement for the treatment of patients with deep dermal or full thickness burns greater than or equal to 30 percent of total body surface area. We spoke to Nick Colangelo, president and CEO of Vericel, about the markets the company is targeting, how its products are changing outcomes for patients, and the company’s plans for growth.

Understanding the physical relationship of cells and how they interact can provide new understanding of diseases and help improve drug development and clinical treatment decisions. Nucleai uses its AI-powered spatial biology platform to transform pathology data into insights. The digital assays it develops enhances drug development, supports treatment decisions, and helps stratify patients to improve care. We spoke to Ken Bloom, head of pathology for Nucleai, about the company’s AI spatial biology platform, how it works, and the potential it has to advance precision medicine.

Vaccitech first gained attention as co-inventor of the Covid-19 vaccine licensed to AstraZeneca. The University of Oxford spin-out is developing a pipeline of T cell immunotherapies to treat and cure chronic infectious diseases, autoimmune diseases, and cancers. The company’s lead experimental therapy is a potentially curative monotherapy for chronic hepatitis B viral infection. We spoke to Bill Enright, CEO of Vaccitech, about the company’s platform technologies, how they work, and how they enable the development of more effective immunotherapies.

One reason that CAR T therapies haven’t been more effective at treating solid tumors is their reliance on targeting antigens on the surface of cells, which can often be present on healthy cells as well. Affini-T Therapeutics is addressing this weakness in current cell therapies by using its platform technology to develop T cell receptor engineered T cells. Its so-called TCR-T cells are able to recognize intracellular targets and attack the drivers of mutations that are inaccessible to CAR-T therapies. We spoke to Jak Knowles, CEO of Affini-T Therapeutics, about the company’s TCR-T cell therapies, how they are engineered, and why they have the potential to be effective at treating solid tumors that have evaded the power of CAR T therapies.

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.