The Bio Report

RNA therapies and vaccines have already shown their power. Nutcracker Therapeutics is taking a nod from the high-tech sector to harness semiconductor-like biochips and microfluidic engineering to create a fully automated platform to increase the speed and scale at which RNA therapies can be discovered, developed, and manufactured. It is developing multimodal RNA therapies which serves as an RNA cocktail to attack multiple disease mechanisms at once. We spoke to Geoff Nosrati, chief business officer of Nutcracker, about the company’s platform technology, its lead program targeting HPV-driven tumors, and its focus on multimodal RNA therapies.

Immune checkpoint inhibitors are transforming cancer care, but still have low response rates in most solid tumors. When cancer spreads to the liver, it can render immunotherapies ineffective because of the large number of immune-suppressive macrophages that are present there. Teclison has developed platform technology that chokes off tumors and activates the immune system against the cancer. The company said its technology can significantly enhances the therapeutic benefit of immunotherapies and can be leveraged to treat nearly all solid tumors. We spoke to Ray Lee, founder and chief medical officer of Teclison, about the company’s platform technology, it’s lead therapeutic candidate, and how it can trigger a systemic immune response.

Inspirna is putting RNA biology to work to address difficult to treat cancers that affect large numbers of people, such as colorectal cancer, small cell lung cancer, and non-small cell lung cancer. Its platform technology is being used to discover the underlying drivers of cancer that can be drugged by both small molecules and biologics to address RNA dysregulation. We spoke to Masoud Tavazoie, co-founder and CEO of Inspirna, about the role RNA dysregulation plays in certain cancers, Inspirna’s platform technology, and its clinical pipeline in development.

Kinase inhibitors have become staples in the arsenal to battle cancer. These medicines can disrupt the cell signaling that drives the growth and spread of tumors. There are more than 30 approved kinase inhibitors today, but they often have dose limiting toxicities, in part because they often lack specificity and can have activity against multiple kinases at once. Blueprint Medicines is seeking to develop next-generation kinase inhibitors that are highly selective to treat genomically-defined cancers and rare diseases. We spoke to Fouad Namouni, president of research and development for Blueprint, about the company’s discovery platform, the large unexplored territory of kinases, and what makes Blueprint’s kinase medicines next-generation.

Cell and gene therapies offer great promise for treating, preventing, or curing serious health issues like cancer, genetic disorders, immunodeficiencies, and rare diseases. Nevertheless, the complexity and costs of producing and delivering these personalized medicines creates barriers to patients benefiting from their potential. Orgenesis is seeking to change the business model around cell and gene therapies through its Point-of-Care Platform, which it says can lower the cost, accelerate development, and expand access for patients. We spoke to Vered Caplan, CEO of Orgenesis, about the challenges of cell and gene therapy, how the company’s Point-of-Care Platform technology addresses those, and its network approach to building a pipeline of therapies across a wide range of indications.

Radiopharmaceuticals have long been shown to be effective at killing cancers, but often damage healthy cells because they are so toxic. Convergent Therapeutics has developed platform technology that takes advantage of the power of radiopharmaceuticals, but links them to targeting mechanisms, such as monoclonal antibodies and ligands, to deliver them in a precise and highly targeted ways. Its lead experimental therapy is an actinium-linked monoclonal antibody that targets prostate-specific membrane antigen to treat prostate cancer. The ability to use dual targeting mechanisms offers the potential for powerful combinations of radiopharmaceuticals while limiting their toxicities. We spoke to Philip Kantoff, co-founder and CEO of Convergent, about the company’s platform technology for developing next-generation radiopharmaceuticals, how the company targets tumor surface molecules, and how its approach minimizes the toxicity of these therapies.

Whether it’s capturing the imagination of a venture capitalist, enticing a potential partner, or attracting talent, CEOs need to be able to tell their company’s story effectively. In his new book “Winners Have a Story,” Giuseppe Marzio discusses how CEOs can turn complex science and technology into compelling tales. Marzio, who worked as a scientist in biopharma before launching a communications agency, comes to his interest in storytelling honestly. Growing up with a love for the cinema, he had dreams of becoming a filmmaker and has studied the structure of stories. We spoke to Marzio, founder of the communications agency Chiaro, about his new book “Winners Have a Story,” why a good pitch shares a common structure with novels and movies, and why a company’s strategy and story are inextricably linked.

It’s been understood for more than 60 years that cancer cells overproduce sugars on their surface. More recently it’s been discovered that these sugars can help cancer cells evade the immune system. Palleon Pharmaceuticals is developing immunotherapies that work by targeting these cell surface sugars and make them vulnerable to attack. We spoke to David Feltquate, chief medical officer of Palleon, about the potential to harness glycobiology to treat cancer, how the company is working to modulate cell-surface sugars, and its emerging pipeline of glyco-immuno therapies.

Current treatment of acute myeloid leukemia generally consists of chemotherapy followed by a hematopoietic stem cell transplant to eliminate any remaining cancer cells from the bone marrow. Even though outcomes remain poor with around 40 percent of transplanted patients experiencing cancer relapse, and patients who do relapse having two-year survival rates of less than 20 percent, the approach has changed little in 40 years. One reason is that newly transplanted patients are unable to receive targeted cancer therapies since those therapies would be toxic to the fragile transplanted stem cells. Vor Bio’s solution is to edit the stem cells prior to transplanting them so they lack the receptors targeted therapies attack. We spoke to Robert Ang, president and CEO of Vor Bio, about the company’s platform technology, how it works, and why it has the potential to change outcomes for patients with AML and other hematologic malignancies.

In cancer, the rigorous translational machinery of the human body goes haywire, causing an overproduction of proteins that fuel the growth and spread of tumors, as well as enabling them to evade the immune system. Effector Therapeutics is developing a new class of cancer therapies called selective translation regulator inhibitors, or STRIs, that can inhibit the production of proteins that drive a cancer. We spoke to Steve Worland, president and CEO of Effector, about the company’s new class of therapies, how they target a central node where two major cancer signaling pathways converge, and how they pack the punch of a combination therapy in a single drug.

While cell therapies hold great promise for not only halting the progression of degenerative disease but potentially restoring the body, cost has remained a barrier. Lineage Cell Therapeutics is developing allogenic cell therapies that don’t rely on modifying a patient’s own cells, making them scalable and less costly than autologous ones. At the end of last year, the company entered into a partnership with Genentech to develop and commercialize OpRegen, its experimental cell therapy for dry age-related macular degeneration. We spoke to Brian Culley, CEO of Lineage Cell Therapeutics, about the company’s platform technology for off-the-shelf cell therapies, how its platform works, and what the collaboration with Genentech will do to advance the technology.

A broad range of serious diseases involve chronic inflammation, which causes both pain and progressive damage. Xalud Therapeutics is developing a locally-delivered, non-viral gene therapy that is designed to harness the ability of interleukin-10 to regulate the immune system and restore homeostasis. The company’s lead indication is in osteoarthritis We spoke to Diem Nguyen, CEO of Xalud, about the role inteleukin-10 plays in regulating multiple pathways in the immune system, Xalud’s gene therapy, and the indications the company is pursuing.

Traditional antibody discovery depends, in part, on a bit of good fortune that banks on the hope that either screening antibody libraries or exposing an organism to an antigen will result in the discovery of a compelling therapy. Yanay Ofran, CEO of Biolojic Design, says the problem with this approach is that it ignores many of the performance-related aspects of an antibody beyond its ability to bind to a target. His company’s AI platform for antibody discovery seeks to capitalize on the full capabilities of antibodies to develop smarter therapies that can serve as molecular switches and act conditionally. We spoke to Ofran about Biolojic’s platform technology, how it mimics the way the immune system makes antibodies, and why its approach will lead to smarter therapies.

The rise of antibiotic resistance and the threat of emerging viral pathogens have created global public health threats. Recce Pharmaceuticals is developing a new class of synthetic anti-infectives that it says can overcome the hyper-cellular mutation of bacteria and viruses. What’s more, the company says their potency doesn’t diminish even with repeated use. We spoke to James Graham, CEO of Recce, about its synthetic anti-infectives, how they work, and why they can be used repeatedly against a broad range of bacteria and viruses.

Whether in an operating room or in a trauma situation, the ability to quickly stop bleeding represents a critical need in healthcare. At the end of December, Cresilon filed for U.S. Food and Drug Administration approval to market its hemostatic gel to rapidly stop bleeding. The filing follows the launch of Vitigel, Cresilon’s hemostatic gel for the animal health market. We spoke to Joe Landolina, co-founder and CEO of Cresilon, about how he developed the plant-derived gel, the significant need he is seeking to address, and how the gel is able to halt bleeding in a matter of seconds.

In cancer, synthetic lethality refers to a state in which two genetic mutations that alone may allow a cancer cell to survive will kill it when they exist simultaneously. Cyteir Therapeutics is seeking to exploit that strategy with what it calls next-generation synthetically lethal therapies to treat a wide range of cancers. We spoke to Markus Renschler, president and CEO of Cyteir therapeutics, about synthetic lethality, Cyteir’s pipeline, and life as a small public biotech in 2022.

AI Dynamics is seeking to make AI accessible. Though its NeoPulse platform can be used across industries, the life sciences is one of the key markets the company is targeting with its technology being used to do everything from target identification for drug development to diagnosing and triaging TB patients by the sounds of their cough. We spoke to Rajeev Dutt, CEO and president of AI Dynamics, about the company’s core AI technology, how it seeks to make AI accessible, and why he believes it can transform drug development and healthcare by moving the needle on costs.

Social anxiety disorder can cause debilitating physical and emotional manifestations when a person is doing such things as meeting new people, speaking in public, or interviewing for a job. It can cause sweating, a rapid heartbeat, shortness of breath, and dizziness. The fear of embarrassment and humiliation can lead to depression and even addiction. VistaGen Therapeutics is developing intranasally delivered therapies for CNS disorders that can provide rapid relief at the onset of an episode much like someone with asthma might use a rescue inhaler. We spoke to Shawn Singh, CEO of VistaGen, about social anxiety disorder, the need for innovative approaches to treat that and other CNS conditions, and the potential for rapid onset therapies to treat a range of mental health conditions.

Humacyte’s human acellular vessels are experimental, engineered, off-the-shelf replacement vessels that are being developed for vascular repair, reconstruction, and replacement. The vessels are designed to overcome long-standing limitations in vessel tissue repair and replacement. Though not yet approved, the manufactured vessels are getting a real-world test as the company has been providing them to hospitals in Ukraine to treat wounded civilians and soldiers with vascular injuries. We spoke to Laura Niklason, CEO of Humacyte, about the company’s human acellular vessels, how they are produced, and the potential needs they can address.

The emergence of tyrosine kinase inhibitors represented a major advance in the fight against cancer, but the ability of tumors to mutate and develop resistance to these therapies remains a challenge. Theseus Pharmaceuticals is developing what it calls “pan-variant” kinase inhibitors in the hopes of outsmarting tumors by anticipating the range of kinases that may drive their spread and growth as they change. We spoke to Tim Clackson, president and CEO of Theseus Pharmaceuticals, about its structural-based approach to drug development, how it determines the appropriate targets for its pan-variant kinase inhibitors, and why it believes its next-generation TKIs can overcome the challenge of drug resistance.