beat · 11 stories
Talus Bio's Ptarmigan-1, a structure-free AI model, predicts binding sites on proteins that lack a fixed 3D structure — the class industry shorthand calls 'undruggable' — and is now accessible through a public portal at ptarmigan.app.
The strongest known genetic risk factor for Alzheimer's pushes the cells that brace the brain's smallest blood vessels into scar-formers, thickening vessel walls, weakening the blood-brain barrier, and trapping amyloid around vessels.
Lab dishes and patient samples from marginal zone lymphoma, a slow-growing B-cell non-Hodgkin lymphoma, showed that pairing a drug that physically destroys the BTK (Bruton's tyrosine kinase) survival protein with a next-generation BCL2 (B-cell
Huntington's has no cure. Twelve patients who received a one-time gene therapy into the brain still outpace untreated peers at year four, by 44%, down from 80% at year three.
VYD2311, a monoclonal antibody rather than a vaccine, posted a quieter side-effect profile than Pfizer's Comirnaty; Invivyd plans to seek accelerated FDA approval.
A 25% mean body-weight drop is a category, not just a number. The same trial was designed to also test whether that magnitude moves knee pain and sleep apnea; the subgroup readouts are the next thing to watch.
ARPA-H, HHS's health-research arm, wants to replace rigid Phase 1/2/3 trials with SURPASS — Simulation-augmented, Real-time Platform Adaptive Seamless Trials — a continuous, platform-adaptive design that reweights dosing in real time.
A 28,000-person Nature study maps a second, independent route for reactivating fetal hemoglobin, the oxygen-carrying protein babies make in the womb, beyond Casgevy's gene-editing approach.
DTU-led team shows a soft polymer implant that records neurons, stimulates them, and delivers drugs at different brain depths, tested only in mice.
ICH E21, a draft international pregnancy-inclusion guideline, would push drug developers from exclusion to inclusion. The US registry has no field to show it.
In two rare genetic disorders — Ataxia-Telangiectasia and Bloom syndrome, which break DNA repair and shorten life — an antiviral sensor called cGAS mistakes the body's own damaged DNA for an infection, and the resulting inflammation may accelerate