The Bio Report

One of the limitations of small molecule drugs and monoclonal antibodies is the difficulty they face in binding to a large number of proteins that could prove to be critical targets in combating various diseases. Aikium is harnessing the power of artificial intelligence and synthetic biology to create a new class of protein biologics called SeqRs that are designed to bind to disordered regions of proteins that have long been beyond the reach of traditional therapeutics. We spoke to Eswar Iyer, co-founder and CEO of Aikium, about the novel class of SeqR proteins the company is developing, how they can bind to targets that traditional medicines can’t, and the potential to transform drug development by expanding the world of druggable targets.

Esophageal cancer is a growing healthcare concern with a steady increase in the number of cases in the last four decades, a development that runs counter to what’s been seen in other major types of cancer. Some 22,000 people are diagnosed each year in the United States with the disease and it is responsible for 16,000 deaths annually. Now, though, Lishan Aklog, chairman and CEO of Lucid Diagnostics, says today esophageal cancer is preventable through early detection in the pre-cancer stage. We spoke to Aklog about Lucid’s test to detect esophageal cancer, how it works, and its potential to change outcomes for people with the condition by catching it before it turns into a deadly cancer.

While existing immunotherapies have changed cancer care, there are several types of cancer where they have limited or no efficacy. Pheast Therapeutics is addressing that by looking to macrophages, part of the innate immune system. These white blood cells gobble up pathogens, cancer cells, and other foreign substances. Certain tumors, though, can evade their attack by expressing checkpoints that serve as “don’t eat me” signals. Pheast is developing macrophage checkpoint inhibitors to block these signals and enlist macrophages in the fight against cancers. We spoke to Roy Maute, cofounder and CEO of Pheast Therapeutics, about how tumors evade the innate immune system, the company’s experimental macrophage checkpoint inhibitor, and its initial focus on ovarian and triple negative breast cancer.

About 98 percent of the the human genome consists of non-protein coding regions known as the “dark genome.” Once derided as “junk DNA,” these regions are increasingly understood to play a critical role in the regulation of the genome and offer a novel means of targeting diseases. Haya Therapeutics is exploring long non-coding RNAs as potential therapies to treat a range of diseases. We spoke to Samir Ounzain, co-founder and CEO of Haya, about the dark genome, the potential to use lncRNAs to treat diseases, and its recently announced collaboration with Eli Lily to use Haya’s platform technology to discover therapies for obesity and related metabolic conditions

Kfir Schreiber, Co-founder and CEO of Deepcure, specializes in AI-driven small molecule therapies for autoimmune diseases. He discusses how AI is revolutionizing drug discovery by exploring previously unreachable chemical spaces. The conversation delves into the shift from biologics to small molecules, emphasizing their advantages in treatment. Kfir also highlights Deepcure's innovative techniques in generating effective inhibitors, showcasing the potential to turn challenging targets into viable therapies while reducing toxicity and improving patient outcomes.

The BET family of proteins regulates gene expression. Their overexpression has been implicated in both cancer and inflammatory diseases. Vyne Therapeutics is developing oral and topical BET inhibitors that treat inflammatory conditions ranging from rheumatoid arthritis to the skin condition vitiligo. We spoke to Vyne CEO David Domzalski and Vyne Chief Scientific Officer Iain Stuart, about the role BET plays in autoimmune diseases, its platform technology, and its efforts to develop BET inhibitors that are both potent and specific.

In August, the U.S. Food and Drug Administration approved Adaptimmune’s Tecelra, the first engineered cell therapy to treat a solid tumor. The T cell receptor gene therapy is approved to treat synovial sarcoma, a rare soft tissue cancer that most often affects young adults. We spoke to Adrian Rawcliffe, CEO of Adaptimmune, about the approval, how TCR therapies differ from CAR T therapies, and what other cancers might benefit from such an approach.  

Ashley Zehnder, the founder and CEO of Fauna Bio, dives into the fascinating world of hibernation and its secrets for human health. She discusses how the genomic data from hibernating animals can unlock potential treatments for obesity, heart attacks, and more. With her company's use of AI, they're identifying gene-disease links that could lead to groundbreaking drug therapies. The partnership with Eli Lilly highlights the innovative strategies being used in drug discovery, particularly in tackling obesity and enhancing our understanding of tissue regeneration.

Senescent cells, ones that no longer divide but are metabolically active, are associated with aging. They are also implicated in a broad range of aging-related diseases including cancer, diabetes, and neurodegenerative conditions. In the case of cancer, these cells can help protect tumors from a person’s immune system. Immorta Bio is seeking to address aging-related diseases by targeting senescent cells and killing them. We spoke to Thomas Ichim, president and chief scientific officer of Immorta Bio, about aging-related diseases, the role senescent cells play in these conditions, and why the company’s therapeutic approach may also have promise of addressing aging itself and extending healthy years of life.

From cell to cell within a given tissue, variability exists. Single-cell sequencing technologies from 10X Genomics is helping researchers and drug developers understand cellular diversity in tissue once thought to be made up of homogenous populations. This has the potential to lead to new understandings of diseases, open up new targets for drug developers, and provide for more tailored approaches to the treatment of diseases. We spoke to Michael Schnall-Levin, chief technology officer and founding scientist of 10X Genomics, about the company’s platform technologies, the diversity of the cellular populations within the body, and how this is changing our understanding of health and disease.