The Bio Report

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 Clinic that showed that regenerative benefits from treatments stemmed from exosome-mediated biological signaling that promoted healing rather than from transplanted stem cells themselves. We spoke to Atta Behfar, co-founder and CEO of RION, about how the company’s purified exosome products work, their potential as scalable, cost-effective regenerative therapies, and how they avoid the immune issues that have long hampered cell-based approaches.

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 platform to advance the discovery and development of precision medicines. We spoke to Jain, chief scientific officer of Sapient, about how its technology bridges the gap from discovery to clinical development, accelerates drug development timelines, and expands opportunities to drug previously undruggable targets.

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 early promise. Results suggest it not only suppresses tumor growth but also activates the immune system to combat cancer. We spoke to Dan Schmitt, president and CEO of Actuate Therapeutics, about elraglusib’s potential to overcome chemoresistance, its recent clinical successes in metastatic pancreatic cancer, and the drug’s unique multimodal mechanism.

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, about why the hub-and-spoke model is well-suited for the development of ophthalmic therapies, its initial assets in-licensed from the Singapore Eye Research Institute, and how it identifies early-stage candidates with high potential.

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 spoke to ​Brian Weitzner, director of computational and structural biology at Outpace Bio and co-founder of OpenFold, about the creation of the collaborative effort, how its open licensing model ensures broad accessibility, and how it stacks up against AlphaFold.

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-founder and CEO of Junevity, about the role of transcription factors in healthy biology and aging-related disease, the company’s AI-driven platform for identifying dysregulated transcription factors, and its lead programs targeting type 2 diabetes and obesity.

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 2 study demonstrated that the experimental therapy, when combined with the standard of care, provided a 13-month survival benefit compared to women receiving only the standard of care. We spoke to Stacy Lindborg, CEO of Imunon, about the company’s DNA-mediated immunotherapy, how it avoids the systemic toxicities that undermined earlier IL-12 approaches, and how it could change the treatment landscape for the disease.

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 and senior fellow of the Hoover Institution at Stanford University, about China’s all-of-nation strategy, how the United States has misallocated research dollars, and why a cultural embrace of biotechnology will be critical for the success of either country.

In this enlightening discussion, Hani Goodarzi, a core investigator at the ARC Institute, shares insights on the Virtual Cell Atlas, a groundbreaking initiative that aggregates single-cell data. Joining him are Gilad Almogy, CEO of Ultima Genomics, who highlights their cost-effective sequencing technology, and Serge Saxonov, CEO of 10x Genomics, explaining how scalable sequencing enables extensive data generation. Together, they explore how this innovative platform aims to revolutionize drug discovery, offering models that predict cellular responses and enhance understanding of disease mechanisms.

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 class of biologics for respiratory and inflammatory diseases, starting with bronchopulmonary dysplasia. We spoke to Marc Salzberg, CEO of Airway Therapeutics, about the company’s experimental therapy zelpultide alfa, why it’s a pipeline-in-a-product, and its development path forward.