The Bio Report

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.

Small molecule drugs and monoclonal antibodies often fall short at addressing severe inflammatory and immune‑mediated diseases. Mesoblast has spent more than 15 years industrializing mesenchymal stromal cell therapies to treat these conditions. In late 2024, it won U.S. Food and Drug Administration approval for Ryoncil, the first mesenchymal stromal cell therapy approved in the United States. Ryoncil is approved to treat steroid‑refractory acute graft‑versus‑host disease in pediatric patients 2 months of age and older. Mesoblast founder and CEO Silviu Itescu discusses how this class of therapy can help dampen cytokine‑driven hyperinflammation, how the company is extending its platform into other high‑burden conditions, and what distinguishes its second‑generation cell therapy now in development from its first.

Most existing therapies for multiple sclerosis do a good job of reducing relapses and inflammatory activity, but they largely fail to stop the slow neurodegeneration that drives long-term disability, especially in progressive forms of the disease. Immunic Therapeutics is trying to reshape the treatment landscape for multiple sclerosis with its experimental once-daily oral therapy, designed not only to curb inflammation and relapses but also to tackle the neurodegeneration that silently drives disability progression in both relapsing and progressive forms of MS. Daniel Vitt, CEO of Immunic, discusses how the company’s experimental MS therapy works, how it may protect neurons from cell death, and the potential for its dual mechanism of action to change the treatment landscape.

The treatment of inflammatory bowel disease currently relies on immunosuppressive therapies that often lose effectiveness, carry infection risks, and drive high treatment cycling. Abivax is betting that fine-tuning, rather than suppressing, the immune system can reshape the treatment paradigm in IBD. Marc de Garidel, CEO of Abivax, discusses how a once-failed HIV candidate evolved into a late‑stage oral IBD therapy that may deliver durable remission, how it acts upstream of key inflammatory pathways, and its potential in a crowded but still underserved IBD market.

Many people with the genetic condition Lynch syndrome live with the near‑certainty that they will one day develop cancer and have few options beyond constant screening and, in some cases, preventive surgery. Nouscom is trying to change that by training the immune system to spot and destroy cancer cells before tumors ever form. We spoke to Marina Udier, CEO of Nouscom, about the company’s experimental cancer vaccines, how they work, and the broader potential for the company’s off‑the‑shelf cancer therapies.

Alzheimer’s disease drug development has long focused on slowing memory loss, but for many families, the tipping point that makes home care impossible is not cognition—it is psychosis. Hallucinations and delusions in Alzheimer’s are a distinct, prevalent, and under-recognized target for therapy. We spoke to Elizabeth Thompson, executive vice president and head of R&D at Acadia Pharmaceuticals, about the biology behind psychosis in dementia, the company’s experimental therapy to treat the condition in people with Alzheimer’s disease, and the forces reshaping the drug development landscape to enable the development of such treatments.

RICO, the Racketeer Influenced and Corrupt Organizations Act, was originally designed to prosecute organized crime. Today, it sits at the center of a landmark class action against two of the world’s largest pharmaceutical companies over the diabetes drug Actos. Attorney Harrison James of Wisner Baum discusses Painters and Allied Trades District Council 82 Health Care Fund v. Takeda, a national civil RICO case alleging that Takeda and Eli Lilly carried out a coordinated, years-long scheme to downplay known bladder cancer risks. The complaint asserts that regulators, physicians, and third-party payers were misled, leading to billions of dollars in reimbursements for the drug. James discusses how RICO’s legal framework applies in the pharmaceutical context, what it took to secure class certification where similar efforts have failed, and the broader implications this case may hold for the industry.

Pancreatic cancer remains one of oncology’s deadliest diagnoses, with standard treatments often offering only transient tumor shrinkage at the cost of grueling side effects and rapid resistance. Immuneering is using transcriptomic and informatics tools to design a MEK inhibitor dosed in intense daily pulses rather than continuously. This approach aims to restore a more normal signaling rhythm in healthy cells while repeatedly ambushing tumors. Ben Zeskind, CEO of Immuneering, discusses how the company is using its informatics-driven dosing regimen to re-engineer targeted cancer therapy so it extends survival, delays resistance, and is better tolerated.

Human leukocyte antigen, or HLA, genes, help the immune system tell the difference between the body’s own tissues and outside threats. In some people, certain versions of HLA genes mistakenly flag normal proteins as dangerous, which can push immune cells to attack joints, nerves, the gut, or other organs. Many autoimmune diseases are driven by changes in HLA genes. RheumaGen is developing a new kind of gene-editing treatment that aims to cure autoimmune diseases by going after one of their root genetic triggers. Instead of broadly weakening the immune system, the company’s goal is to switch off a single “bad” version of an immune gene while leaving the rest of the body’s defenses intact. We spoke to Richard Freed, CEO of RheumaGen, about the role of HLA genes in autoimmune diseases, how the company’s gene-editing therapies work, and its lead program in rheumatoid arthritis.

Asia is quickly becoming a powerhouse for biopharma innovation, changing ideas about where breakthrough science and fast, cost-efficient drug development happen. A new McKinsey & Company report shows how countries like China, Japan, and India are each building their own strengths across the drug development continuum. We spoke to Fangning Zhang, a partner in McKinsey’s Shanghai office, about what’s driving this shift, how it could make innovation more affordable, and why treating Asia as optional may mean missing the next wave of global R&D.

Leukemia once threatened Aaron Viny’s life, but now it defines his mission. Diagnosed with acute lymphoblastic leukemia as a college student, he survived chemotherapy, central nervous system relapse, and an allogeneic stem cell transplant from his younger brother—an experience that made him aware of both the power and toxicity of conventional cancer care. Today, as a hematologist-oncologist and laboratory researcher at Columbia University, Viny is helping reimagine how we treat blood cancers by shifting from blunt, cell-killing approaches to precision strategies that rewire malignant cells and their ecosystems. We spoke to Viny, assistant professor of medicine at Columbia University Vagelos College of Physician and Surgeons, about the case for thinking of hematological cancers as regulatory problem rather than focusing on genetic mutations, the potential for looking at epigenetic activators and deactivators of genes to treat them, and how he is harnessing new technology to look at cell-surface proteins to dis

When Amy Burroughs stepped in as CEO of Terns Pharmaceuticals, she not only had to fill a void created by the death of her predecessor, but also lead a strategic shift from an increasingly crowded area of metabolic disease to focus on its experimental therapy for chronic myeloid leukemia. The company’s allosteric BCR-ABL inhibitor binds to a different site on the fusion protein than most first- and second-generation tyrosine kinase inhibitors. The data have the company and its investors believing the drug can reset the bar for both efficacy and tolerability in a multibillion-dollar market. We spoke with Burroughs about reinventing the company, the decision to seek partners for non-core assets, and how she is charting a clear path toward a broader oncology future.

A One‑Two Gene Therapy Punch to Non-Muscle Invasive Bladder Cancer Non–muscle invasive bladder cancer is a common, slow-progressing form of bladder cancer that makes up a majority of the roughly half a million new cases diagnosed each year. For decades, doctors have relied on a weakened bacterium called BCG, an intravesical immunotherapy, as a standard treatment for early-stage disease, but it fails in about 30 to 40 percent of patients. EnGene is taking a different approach with detalimogene, an experimental, non-viral gene therapy designed to trigger a powerful but localized immune response right where the cancer lives in the bladder. We spoke with Ron Cooper, CEO of EnGene, about this therapy for non–muscle invasive bladder cancer, how its dual payload is meant to activate both an innate and adaptive immune response in the bladder, and the company’s $130 million financing at the end of 2025.

One of the challenges of treating brain tumors is delivering potent biologic therapies across the blood-brain barrier. Adaptin Bio has developed platform technology that harnesses a patient’s own T cells to transport bispecific therapeutic payloads across the blood-brain barrier and into other targeted tissue with an initial focus on treating glioblastoma. We spoke to Michael Roberts, co-founder and CEO of Adaptin Bio, about the unmet need in glioblastoma, the limitations of current blood-brain barrier–crossing strategies, and how the company’s platform seeks to change the treatment paradigm by using patient-derived T cells as delivery vehicles for targeted biologics.

Despite the emergence of new modalities and drug development technologies, the cost and time to produce new therapies has changed little, and failure rates remain high. Xaira aims to change that with a systematic, AI‑driven approach that tackles three pervasive bottlenecks—choosing the right targets, designing the right molecules, and matching the right patients—by running as much work as possible in silico and using high‑dimensional causal datasets to train “virtual cell” foundation models. The company is initially focusing on high‑value, historically undruggable targets and ultimately on building a pipeline of differentiated biologics. We spoke with Marc Tessier‑Lavigne, co‑founder and CEO of Xaira, about applying end‑to‑end AI across target discovery, molecular design, and patient stratification; the company’s more than $1 billion in funding, and how it seeks to enable a new generation of scientists fluent in both AI and biology.

Finding New Targets on the Surface of Misfolded One of the biggest hurdles in drug development is targeting proteins found in both healthy and diseased cells without triggering toxic side effects. In cancer, this challenge often translates into narrow therapeutic windows, collateral damage to normal tissues, and forced dose reductions that limit efficacy. The result is a crowded field where many companies chase the same well-known targets, leaving vast patient populations without effective options. Immuto Scientific is taking a different approach. The company is redefining how targets are identified—focusing not on genetic sequence, but on disease-specific protein conformations. By studying the structural shapes that proteins take in malignant cells, Immuto aims to distinguish cancerous from healthy tissue, broaden therapeutic windows, and unlock new or previously undruggable targets across oncology and beyond. We spoke with Faraz Choudhury, co-founder and CEO of Immuto Scientific, about the company’s AI-enab

Systemic chemotherapy remains the foundation of cancer treatment, but its widespread toxicity too often cuts short potential therapeutic benefits. NanOlogy is developing a new approach—localized cancer therapy that keeps the drug where it’s needed most. We spoke to Marc Iacobucci, managing director of Nanology about the company’s precision particle engineering platform, how it transforms existing oncology drugs into microparticles optimized for intratumoral delivery; and how this enables sustained, high-concentration dosing inside tumors that destroys cancer cells, stimulates immune responses, and spares patients the debilitating effects of systemic chemotherapy.

One of the challenges in treating the neurodegenerative condition Alzheimer’s disease is intervening early enough in the course of illness to provide meaningful benefit. AltPep is developing therapeutics with companion diagnostics that target toxic α-sheet–containing oligomers, which are thought to form very early in the disease and act as molecular triggers of downstream amyloid pathology. These structures are believed to represent some of the earliest detectable stages of Alzheimer’s and to promote the formation of amyloid plaques, a hallmark feature of the condition.​ We spoke with Valerie Daggett, founder and CEO of AltPep, about the relationship between α-sheet oligomers and the onset of Alzheimer’s disease, how the company’s synthetic peptides are designed to bind and neutralize these pathogenic agents, and the potential for this platform to be extended to a broader set of amyloid diseases.

Biotech stocks staged a dramatic turnaround in 2025, with the XBI well outpacing the S&P 500 despite concerns over leadership changes at the U.S. Food and Drug Administration, the Trump administration’s efforts to put constraints on drug pricing, and its broader cuts to health and science agencies. A pick-up in deal-making, along with falling interest rates, helped buoy the sector with growing anticipation for continued improvement in 2026. We sat down with Adam Feuerstein, senior biotech writer for STAT, for our annual review-preview edition, the noteworthy trends in the biotech sector 2025, and what’s in store at the upcoming JPMorgan Healthcare Conference and beyond in 2026.   

The approach to post-surgical pain relief has relied on short-duration treatments and notably opioids. That’s led to the associated risks of dependence and abuse, prolonged hospitalizations, and slower recoveries. Allay Therapeutics is developing an alternative to opioids to treat post-surgical pain with an initial focus on knee replacement surgeries. Allay’s guitar pick-sized polymer is implanted during surgical procedures and it releases the analgesic bupivacaine as it dissolves over 30 days. We spoke to Adam Gridley, CEO of Allay Therapeutics, about post-surgical pain, the need for alternatives to opioids; and the company’s implanted, extended-release analgesic.