Typewriter is betting that a jumping gene borrowed from evolution can insert therapeutic DNA more safely than a virus — delivered by the same lipid nanoparticles that carried the COVID vaccines.
Two compounding facilities are closing, and with them a product premature babies need within hours of birth. The FDA responded with immediately-effective guidance loosening the rules on who may compound it.
GTX-102 was carrying $1.8 billion in peak sales expectations and the hopes of families with no approved treatment. “No differences between the treated and control groups that could support efficacy” ends both.
Twenty years of repeat brain scans in healthy adults show the cortex thinning at least seven years before amyloid becomes visible on PET. Either the plaques are late, or something other than amyloid starts the damage.
A $10 billion contract is now $27 billion, the committee found out from the press, and Oracle declined to appear. The vote to subpoena Larry Ellison and Mike Sicilia was 19–0.
Nearly $95M from Paul Allen’s estate, split across three Seattle institutions, to build proteins and genes evolution never made — deliberately using many small task-specific models rather than one giant one.
Most babies in wealthy countries carry C. difficile without symptoms, and medicine has long treated that as harmless. A study in Science asks whether asymptomatic really means inconsequential — and answers no.
The FDA approved a breast cancer drug to be started when a blood test detects resistance — before any scan shows the cancer growing. That sequencing, not the molecule, is the precedent.
Genetics said Lp(a) causes heart disease. Pelacarsen lowered it convincingly in 8,300 patients. Cardiovascular events did not move — and the field now has to work out which part of that chain was wrong.
The FDA removed a boxed warning that had suppressed hormone therapy for two decades. Demand rose, and the patches — harder to manufacture than pills — ran short.
HMPL-A830 is not quite an ADC. Its antibody blocks EGFR while carrying a KRAS inhibitor to the tumor — a single molecule aimed at the escape route that has undermined KRAS drugs in the clinic.
The fear was that a pig kidney would sensitise the immune system and cost a patient their place in line for a human organ. Two recipients have now received human kidneys without antibody complications.