Biotech & Life Sciences

A teenager in Berlin was near palliative care. After seven years on sildenafil — the active ingredient in Viagra — he is still alive. Six patients with Leigh syndrome, a fatal mitochondrial disease, have now been treated off-label, with mixed but striking results. A larger trial is coming.

Kenneth Shock enrolled in Neuralinks trial the day the registry opened. Eight weeks later he said his first decoded words. The technology is real. The gap between the demo and what patients actually need is real too.

AstraZeneca called it a success. The adult HPP trial missed its goal—but a subgroup of patients who developed the disease as children did show benefit. The problem: 80 percent of HPP patients are adults.

Two tiny genes nobody bothered to study may account for hundreds of thousands of unexplained neurodevelopmental cases. Now there is a path to therapy.

"We now have the ability to design organisms from scratch that have never existed in nature," researchers wrote. Then came the benchmark data.

Novo Nordisk’s new Wegovy subscription saves patients up to $1,200 a year. The harder problem: roughly 65% of obesity patients quit GLP-1 drugs within 12 months.

Eli Lilly's oral GLP-1 could win FDA approval within weeks. Ambrosia Biosciences has never published a single animal experiment showing its rival pill even works in animals.

When Astellas walked away from a $2.18B deal, AviadoBio had a trial but no path forward — until two nonprofits with $1M/year decided to fill the gap themselves.

Baby KJ's bespoke CRISPR therapy worked. The company built to replicate it collapsed nine days after the FDA's new pathway was announced — a sign the science is ahead of any business that could deliver it at scale.

In mouse models, the therapy shrank ovarian, lung, and pancreatic tumors — with durable remission in the ovarian model, where surviving mice resisted new tumors when researchers reintroduced cancer cells weeks later.

OpenEvidence just deployed its AI across the full clinical care team at Mount Sinai — physicians, nurses, and pharmacists across a 48,000-person health system. It is the first enterprise rollout aimed at the entire care team, and the company calls it the new baseline for what a modern hospital lo...