The Bio Report

Sepsis remains one of medicine’s most intractable and costly problems, arising when an infection triggers a runaway immune response that damages organs long after a pathogen is controlled. It accounts for an estimated $53 billion in Medicare spending alone, yet more than a hundred drug trials have failed to yield a single approved drug that directly treats the underlying immune dysfunction. Inflam­matix, which grew out of work at S...

Rion is developing platelet-derived exosome therapeutics as off-the-shelf, room-temperature-stable alternatives to traditional cell therapies. The company’s lead program is advancing toward phase 3 trials in diabetic foot ulcers, and it is building a broader pipeline across musculoskeletal, cardiovascular, pulmonary, dermatologic, and women’s health indications. The company is built on stem cell research conducted at the Mayo Clini...

The genomics revolution promised to unravel diseases and lead to treatments that addressed their root causes. In reality, says Mo Jain, your zip code remains a better predictor for how healthy you will be over the course of your life than your genetic code does. That’s because, except for monogenic diseases, etiology tends to be far more complex than the identification of a single gene. Sapient is using its AI-driven, multi-omics p...

The enzyme GSK3β, in healthy cells, is involved in glucose metabolism. In cancer cells, though, it serves as a master regulator of tumor growth, progression, and cell survival. While GSK3β has long been an attractive target in cancer therapy, it has been difficult to inhibit due to the poor pharmaceutical characteristics and adverse effects of therapeutic candidates. Actuate Therapeutics’ experimental therapy elraglusib has shown e...

Hub-and-spoke business models—the use of a central core of business functions with pipeline assets spun out into subsidiary companies—have gained traction for the benefits they can provide in terms of capital efficiency, diversification of risks, and improved access to capital. Eyexora is applying that business model to accelerate the development of therapies for ophthalmic indications. We spoke to Theresa Heah, CEO of Eyexora, abo...

OpenFold, an open-source, collaborative initiative founded in 2022 to address the challenges of protein structure prediction and design using artificial intelligence, emerged as a response to the restricted commercial access to DeepMind’s AlphaFold platform. Leveraging public datasets and using a pre-competitive consortium model, OpenFold seeks to democratize cutting-edge protein engineering tools for both industry and academia. We...

Transcription factors control the genetic programs that maintain cellular balance, but while they have been seen as compelling targets for aging-related disease, they have long been considered “undruggable.” Junevity’s RESET platform leverages large-scale human omics data and AI models to pinpoint key transcriptional drivers of disease and to design siRNA therapies that restore healthy gene expression.​ We spoke to John Hoekman, co...

Ovarian cancer remains one of the deadliest cancers affecting women, and it is expected to claim nearly 13,000 lives in the United States in 2025. Despite progress in survival rates, nearly 80 percent of patients are still diagnosed at advanced stages, when the disease has already spread and is difficult to treat. Imunon’s experimental DNA-mediated immunotherapy is designed to deliver interleukin-12 directly into the tumor. A phase...

China’s emergence as a biotech superpower may have appeared to happen suddenly, but it reflected a long-term vision and policies over many years that enabled its success. As biotechnology transforms not only medicine but also industries such as food, fuel, and materials, the competition between China and the United States to shape the future bioeconomy is intensifying. We spoke to Drew Endy, associate professor of bioengineering an...

The ARC Virtual Cell Atlas uses high-throughput single-cell genomics, artificial intelligence, and open science to understand the complexities of cellular behavior. Developed through a partnership between the ARC Institute, 10x Genomics, and Ultima Genomics, the public domain resource integrates data from hundreds of millions of cells. By curating and harmonizing vast amounts of single-cell measurements, the Atlas paves the way for...

Bronchopulmonary dysplasia is a condition that affects preterm infants, especially those born with underdeveloped lungs who require prolonged oxygen therapy or mechanical ventilation to help them breathe after birth. It is characterized by damage and abnormal development of the lung tissue and airways, often resulting from life-saving interventions necessary for babies born very prematurely. Airway Therapeutics is developing a new ...

MASH, a chronic and progressive form of fatty liver disease that until recently was known as NASH, affects millions of people in the United States, and its incidence continues to rise. In fact, MASH is now among the leading causes of liver transplantation in the United States. 89bio is developing an experimental therapy to target multiple disease mechanisms of the condition. We spoke to Rohan Palekar, CEO of 89bio, about what’s dri...

The average kidney transplant recipient experiences kidney failure within 10 to 12 years after a transplant, putting them on a cycle that ends with kidney failure and a need for a new transplant. This cycle adds to strains on transplant recipients, payers, providers, and the healthcare system and taxes the limited supply of organs for transplantation. Eledon Pharmaceuticals is seeking to extend the functional life of transplanted o...

Antimicrobial resistance is projected to kill up to 10 million people a year by 2050. One particular area of concern is drug-resistant gonorrhea, where existing therapies are being exhausted. Taxis Pharmaceuticals is developing therapies to combat evolving superbugs by targeting their cellular infrastructure and disrupting the mechanisms that fuel antimicrobial resistance. We spoke to Greg Mario, president and CEO of Taxis, about t...

Brain tumors are difficult to treat in part because of the blood-brain barrier, the need to protect healthy and sensitive tissue surrounding tumors, and the limits of existing therapies. While there have been great advances in other types of cancer, there’s been relatively little progress in treating these CNS tumors. Plus Therapeutics is developing targeted radiotherapies that provide high doses of radiation directly into the tumo...

Macrocycles are complex compounds that can interact with targets that are often unreachable with traditional small molecules. Orbis Medicines is addressing the challenge with its nCycles, synthetic macrocycle drugs that are orally available but hit targets that would otherwise require biologic therapies. We spoke to Morten Graugaard, CEO of Orbis Medicines, about its class of synthetic macrocycles called nCycles, its platform techn...

Animal testing for experimental therapies is slow, expensive, and an imperfect predictor for how a drug will act in humans. The Foundation for the National Institutes of Health in July announced a program to advance innovative laboratory technologies that model human biology, enabling faster, less expensive testing. We spoke to Stacey Adam, vice president of scientific partnerships for the FNIH and leader of the public-private part...

Many cancer patients are prescribed opioids to manage pain associated with their disease, but studies have shown that the use of these pain killers naturally mutes the immune response and can reduce the efficacy of immunotherapies. Glycyx Therapeutics is developing a drug it believes can allow opioids to relieve pain while preventing them from working against immunotherapies. We spoke to Lorin Johnson, chief scientific officer of G...

When Takeda in 2023 paid Nimbus Therapeutics $4 billion upfront and the potential for two additional $1 billion milestone payments for its experimental TYK2 inhibitor, the deal was an eye-popping validation of Nimbus’ approach. The company, an early innovator in a computational chemistry, has now integrated AI into its approach to drug discovery. And though it’s been an innovator in technology, it’s also been an innovator in its po...