The Bio Report

Targeting RNA Dysregulation in Cancer

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.

Building a Pipeline of Next-Generation Kinase Inhibitors

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.

Rethinking How Cell and Gene Therapies Are Made to Improve Accessibility

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.

Developing Next-Generation Radiopharmaceuticals

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.

Why Great Science and Data Aren’t Enough

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.

Targeting Sugars that Let Cancers Evade the Immune System

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.

Engineering Stem Cells to Withstand Targeted 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.

Targeting a Convergence of Signaling Pathways that Drive the Growth and Spread of Cancer

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.