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.