Venetoclax, already used for blood cancers, pushed latently infected cells to die and cut the intact viral reservoir in SIV-infected macaques, with effects lasting months.
GTX-102 missed all its goals in a Phase 3 study for the rare disorder, wiping out a would-be blockbuster and pushing the company toward significant cost cuts.
Emerging from stealth, the startup uses a “jumping gene” and lipid nanoparticles to insert therapeutic genes inside the body — a non-viral bet amid mounting safety worries about viral vectors.
With two suppliers shutting down, the FDA is deploying regulatory tools to prevent a shortage of the intravenous nutrition that premature and critically ill babies depend on.
Tracking healthy adults with MRI for nearly two decades, researchers found structural brain changes at least seven years before amyloid plaques became visible on scans.
Backed by Paul Allen’s estate, the Allen Institute, UW and Fred Hutch are building open AI models to invent new biological parts — a bet on narrow, purpose-built AI over one-size-fits-all systems.
Novartis and Ionis’s pelacarsen dramatically lowered lipoprotein(a), a common inherited cardiovascular risk, yet failed to cut heart attacks, strokes or cardiac deaths in its pivotal trial.
The Department of Veterans Affairs’ electronic health-record overhaul has ballooned from $10 billion to $27 billion — and lawmakers say they learned of it from the news, not the company.
Camizestrant is the first cancer therapy cleared based on spotting a resistance mutation in circulating tumor DNA — letting doctors switch treatment before scans show the cancer is growing again.
Most infants carry C. difficile without obvious symptoms, but new research finds it can remodel the developing gut and immune system — with effects that may last into adulthood.
In mice, harmful T cells attacking the brain in tau-driven neurodegeneration were activated in lymph nodes — and blocking that step preserved memory, all without a drug crossing into the brain.
Ionis’s Zanvastro, an antisense drug that dials down a toxic protein, stabilized walking in patients with the ultra-rare, progressive neurological disorder — which until now had no disease-modifying therapy.