The Bio Report

A Pipeline in a Product that Reimagines Control of Inflammation

Plasma gelsolin is an abundant, endogenous regulator of inflammation that is consumed during severe inflammatory insults. When levels fall too low, patients are at higher risk of organ damage and death, particularly in settings like acute respiratory distress syndrome where a dysregulated inflammatory response floods the lungs with fluid and leaves patients dependent on ventilatory support with no approved therapies today. BioAegis Therapeutics is working to turn recombinant human plasma gelsolin into a pipeline-in-a-product. Susan Levinson, CEO of BioAegis, discusses recombinant human plasma gelsolin as a potential first-in-class treatment for ARDS and other inflammasome-driven conditions, how it modulates cytokine storms without suppressing the immune system, and its potential in other conditions including neurodegenerative diseases.

Building a Genetics Engine to Crack the Target Bottleneck

A chronic shortage of high‑quality targets remains one of the biggest constraints in drug discovery, even as therapeutic tools become more powerful and diverse. Regeneron is tackling that problem with its Regeneron Genetics Center, which has built a genetics‑driven discovery engine that integrates human genetics with rich clinical data, large‑scale proteomics, and AI‑driven analytics. Aris Baras, head of the Regeneron Genetics Center, discusses how proteomics is reshaping RGC’s view of risk prediction, how AI helps his team sift through hundreds of millions of variants, and what it really takes to scale this kind of effort and translate it into more successful, transformative therapies for patients.

Stopping Shape-Shifting Tumors with a First-in-Class Epigenetic Drug

Epigenetics, the layer of chemical switches that controls how genes are turned on and off, can act like cancer’s operating system when a single epigenetic enzyme becomes essential for a tumor to survive. K36 Therapeutics is developing first‑in‑class medicines that block an epigenetic enzyme that helps certain multiple myeloma cells grow, change identity to escape treatment, and become resistant to today’s drugs. Terry Connolly, CEO of K36, discusses a new way to fight cancer by changing how cancer cells read their DNA instead of chasing one mutation at a time, how K36’s experimental therapies aim to re‑sensitize tumors to existing treatments, and the potential to create new options for people whose cancers have stopped responding.

Rewriting the Rules of Antibody Drug Design

Most marketed antibodies work as antagonists, simply shutting off a receptor, even though many immune, metabolic, and cancer pathways require more nuanced control. Metaphore Biotechnologies' function‑first platform combines live-cell experiments with machine learning to read how receptors and binding partners behave in living systems, distill those complex dynamics into the key functional features, and then design functional antibodies that agonize, bias, or multi-target pathways from the outset. Metaphore CEO Angela Hwang discusses how the company is using this approach to open up difficult or previously undruggable targets, generate medicines with better profiles, and give drug developers greater control over complex signaling than traditional drug development approaches allow.

Mapping Cellular Stress Biology to Tackle Undruggable Targets

Cells continuously sense their environment and in response to stressors, adapt, recover, or die. Soley Therapeutics uses its AI platform to capture thousands of intracellular features and map how cells sense, interpret, and respond to stress. The approach gives Soley the ability to pursue previously undruggable targets. It has generated more than 10 novel oncology programs in less than two years and advanced two first‑in‑class experimental small molecule therapies toward the clinic. Yerem Yeghiazarians, co-founder and CEO of Soley, discusses the science behind the company’s first-in-class lead candidate, the applicability of Soley’s platform to a broad set of diseases, and the capital efficiency of the company’s approach to drug development.

Turning Abandoned Drugs into Breakthroughs

Promising drugs can become abandoned or underused because of tolerability issues, poor drug‑like properties, or other fixable limitations, even when there is already compelling human evidence that they work. PureTech Health starts with an unmet need and human pharmacology, then systematically dissects and solves the specific liabilities of discontinued drugs to unlock breakthroughs in an approach that has proved to be a highly efficient means of value creation. Eric Elenko, president and co‑founder of PureTech, discusses the company’s disciplined approach to drug innovation around rescuing promising but discontinued therapeutics, its hub‑and‑spoke structure, and how this model can neutralize emotional bias, enforce clear success criteria, and turn partially derisked assets into commercial successes.

Targeting Cancer Survival Genes in Solid Tumors

Most cancer therapies hit one or a few pathways that tumors can escape by mutating, activating alternative survival routes, or pumping drugs out, leading to relapse and poor survival in indications such as liver, ovarian, and prostate cancer. Nuago is developing single-construct short RNAs that simultaneously silence many survival genes in cancer cells to achieve durable tumor cell killing with minimal toxicity to normal tissue. Nuago CEO Robert Schickel discusses the biology behind toxic RNA seeds and tumor-suppressive microRNAs; the company’s lead programs in liver, ovarian, and prostate cancer; and the implications if its cancer-agnostic RNA platform can live up to its preclinical promise.

Addressing Treatment Gaps in Gout

Gout may be one of the oldest known forms of arthritis, but it remains widely misunderstood, undertreated, and a source of silent suffering for millions of people who are often blamed for their disease rather than offered effective care. Current therapies to lower urate levels suffer from limitations and safety challenges. Crystalys Therapeutics is in late-stage development of a next‑generation urate inhibitor that is already approved in Japan and China. Crystalys CEO James Mackay discusses the biology of gout, why standard therapies often fail to get uric acid to target levels, and how the company’s next‑generation URAT1 inhibitor may fill the treatment gap and change daily life for patients living with moderate to severe gout.