The Bio Report

Gain Therapeutics' platform technology Magellan leverages AI, structural biology, and physics-based models to identify novel binding sites on otherwise undruggable proteins implicated in diseases. The company’s experimental Parkinson’s disease therapy has the potential to slow or stop progression of the neurodegenerative condition by stabilizing a lysosomal enzyme implicated in the disease. We spoke to Gene Mack, interim CEO of Gain, about the company’s platform technology, how its experimental therapy for Parkinson’s disease works, and what other conditions might be good candidates for its platform technology to target.

We continue our holiday tradition by welcoming STAT News Senior Biotech Writer Adam Feuerstein for our annual look back at the year that was in biotech and what’s ahead for the industry with the JPMorgan Healthcare conference and beyond in 2025. Feuerstein offers his view on finance and dealmaking in 2024, new drug approvals, and his annual take on the best and worst CEOs of the year. We also discuss what Trump 2.0 may look like for the industry, changes coming to the FDA and other agencies, and what hot technologies to watch in the year ahead. 

Prolific Machines uses light to precisely control virtually any function in any cell to transform what is possible with biomanufacturing. In combination with optogenetics and AI, the technology has the potential to impact a wide range of industries, from food production to pharmaceuticals, by enabling new capabilities, reducing costs, and improving sustainability. We spoke to Deniz Kent, co-founder and CEO of Prolific Machines, about the company’s photomolecular platform technology, the benefits it provides over traditional biomanufacturing methods, and how it could be used to not just cultivate meat but make a steak.

ImpriMed is working to deliver on the promise of precision medicine by using a patient’s live cancer cells to see how they respond to different treatment options and artificial intelligence to predict which medicines will work best. The company has had impressive results with its customers to date, but the catch is that it has initially targeted its service to the veterinary market, and its dogs and cats have benefited from it. The company is now working to bring its offering to two-legged patients. We spoke to Sungwon Lim, CEO of ImpriMed, about its functional precision medicine and AI platform to match cancer patients to the best available therapy for them, its decision to roll out the service first to the veterinary market, and what it is doing to bring its service to humans.

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

One of the promises of AI is to redefine what’s possible by enabling the discovery of compounds that exist in a much larger chemical space than scientists have previously been able to explore. Deepcure is using AI and physics to discover small molecule therapies that can bind to difficult to target proteins. We spoke to Kfir Schreiber, co-founder and CEO of Deepcure, about the company’s AI technology platform, its focus on autoimmune diseases, and why he believes its technology will allow it to develop small molecule drugs that can provide alternatives to biologics to treat these conditions.

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.  

Hibernation is not just a matter of deep sleep. Animals that hibernate are able to do so without suffering damage to tissue and muscle. Understanding the biology of hibernation can unlock potential insights into obesity, heart attack and stroke, muscle atrophy, neuroprotection, and longevity. Fauna Bio is studying genomic data from so-called "extreme mammals" and applying its proprietary AI platform to perform comparative genetic analysis to find gene-disease links and leverage millions of years of evolutionary adaptations to identify new therapeutic opportunities for human health. We spoke to Ashley Zehnder, founder and CEO of Fauna Bio, about the types of insights that can be gleaned from the genetics of hibernating animals, the wide range of human health conditions that might be addressed with such an approach, and the company’s deal with Eli Lilly to apply its AI platform to discover new obesity drugs.

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.

The DDR pathway plays a critical role in repairing DNA damage in healthy cells that would otherwise cause mutations or cell death. When this pathway is altered and becomes unable to repair this damage, mutated cells can grow out of control and become cancerous. They can also be more resistant to standard chemotherapies and radiation. Aprea Therapeutics is developing therapies to target mutations in genes in this pathway to treat certain cancers.  We spoke to Oren Gilad, president and CEO of Aprea Therapeutics about the DDR pathway, the use of so-called synthetic lethality to treat these cancers, and why Aprea’s approach may result in safer and more targeted therapies. Editor's note: We are have deleted out the original post because there were problems with the file and have reposted the interview here.

Nearly 70 percent of patients with chronic conditions fail to take their medicines as prescribed. This can lead to poor outcomes and greater healthcare costs. Vivani Medical, which has developed implantable drug delivery technology, said it can provide steady drug release for up to six months with a single implant. This not only can address the problem of non-compliance, but can also reduce side effects associated with fluctuating drug release from conventional delivery methods. We spoke to Adam Mendelsohn, CEO of Vivani Medical, about its implantable drug delivery technology, how it works, and why its lead indication is a GLP-1 implant for weight loss and diabetes.

One of the key obstacles to developing new drugs and diagnostics is the lack of access to longitudinal biological and clinical patient data. Culmination Bio, a spin-out of Intermountain Health, is building the largest patient data platform with more than 40 years of de-identified patient electronic health records and biospecimen data. We spoke to Lincoln Nadauld, president and CEO of Culmination Bio, about the data driving the company’s platform, how partners are using its data today, and how it has the potential to accelerate the development of new diagnostics and therapies.

NAMPT is a critical enzyme that regulates how cells use energy. It can also play an important role in the ability of cancer cells to survive, grow, and spread. While it’s been recognized as a potential target to treat certain cancers, developing NAMPT inhibitors have been challenging because they can disrupt energy metabolism in healthy cells. Remedy Plan Therapeutics believes it’s overcome the challenges of NAMPT inhibition by a unique mechanism of action that can avoid the problem of toxicity. We spoke to Greg Crimmins, founder and CEO of Remedy Plan Therapeutics, about the role of NAMPT in cancer, the challenges of developing NAMPT inhibitors without toxicity, and why he believes the company has solved the problem.

The emergence of CRISPR, a powerful tool for gene editing, promises to correct devastating genetic diseases, but it also raises concerns about how it will be used and who will make those decisions. Neal Baer, in his new book “The Promise and Peril of CRISPR,” brings together a series of essays by bioethicists, geneticists, and others who explore ethical and policy considerations raised by the technology. We spoke to Baer, co-director of the master’s degree program in Media, Medicine, and Health at Harvard Medical School; about whether there are unique questions raised by CRISPR as a dual-use technology, the book’s recasting of the case of Chinese scientist He Jiankui, and why he thinks there needs to be public debate now over the use of the technology to make heritable changes to the genome.

Speeding Protein Engineering with Whether it’s the development of new biotherapeutics, or replacing hydrocarbons from their role in industrial manufacturing, engineering proteins suited for a task is a time consuming and expensive process. Cradle Bio has developed generative AI technology to accelerate protein engineering by reducing the number of experiments needed to arrive at a product candidate and enable the optimization of multiple properties at once. We spoke to Elise de Reus, co-founder of Cradle, about the company's generative AI technology, how it works, and how it’s changing the process of protein engineering.