The Bio Report

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.

An Off-the-Shelf Cell Therapy to Calm Cytokine Storms

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.

Slowing Disability in MS

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.

Tuning, Rather than Blocking, Immunity in IBD

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.

Intercepting Cancer When DNA Surveillance Fails

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.

Targeting Psychosis in Alzheimer’s Disease

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.

A Class Action Suits Moves RICO from Mobsters to Medicine

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.