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The First CRISPR-Armed Virus Was Just Tested in Humans. It Worked Safely.
For the first time ever, a CRISPR-armed bacteriophage has been tested in humans and the safety results, published in The Lancet Microbe, are remarkably clean. SNIPR Biome's therapy sends engineered viruses into the gut to hunt E. coli (including antibiotic-resistant strains), then uses CRISPR to shred the bacteria's DNA from within. In 36 healthy volunteers, the treatment caused no serious side effects, stayed confined to the gut, and at the highest dose significantly reduced E. coli levels. The company is now testing it in cancer patients whose weakened immune systems make gut infections potentially lethal.
Why it matters: Antibiotic-resistant infections killed 1.14 million people in 2021, with deaths projected to hit 1.91 million annually by 2050. This trial represents the first convergence of phage therapy and gene editing in humans, potentially opening an entirely new front in the war against superbugs.
Read more →Clinical and Regulatory
The FDA Wants Fake Brain Surgery Before Approving This Huntington's Gene Therapy
UniQure's stock cratered over 40% after the FDA demanded a sham brain surgery trial before it will approve AMT-130 for Huntington's disease. The gene therapy slowed disease progression by 75% in Phase 1/2 data, but the agency insists on a placebo arm where patients get their skulls drilled open without receiving treatment. The resulting ethical showdown could set the precedent for every surgical gene therapy targeting the brain.
Read more →AbbVie's Skyrizi Delivers for Crohn's Patients Other Drugs Couldn't Help
Sixty-five percent of patients in AbbVie's Phase 3 AFFIRM trial had already failed at least one advanced therapy. Skyrizi still moved the needle: 55% hit clinical remission at week 12 versus 30% on placebo. The trial also tested an all-subcutaneous regimen that could eliminate IV infusions entirely, a major convenience upgrade for a franchise on pace to hit $17.5 billion in 2025 revenue.
Read more →BMS's Sotyktu Faces March 6 FDA Verdict on Psoriatic Arthritis Expansion
Bristol Myers Squibb's oral TYK2 inhibitor Sotyktu faces a March 6 FDA decision on expanding into psoriatic arthritis. The drug's unique mechanism offers cleaner safety than rival JAK inhibitors (which carry boxed warnings), and its Phase 3 trials showed significant symptom improvement. An approval would push the drug into a much larger market.
Read more →Winners and Losers
Theravance Bet Everything on One Drug. It Just Lost.
Theravance slashed 50% of its workforce and killed its entire R&D operation after ampreloxetine failed Phase 3 for the second time. The company is now a royalty holding company sitting on roughly $400 million in cash, exploring strategic alternatives. Its stock dropped roughly 41% in a single session.
Read more →Biohaven Was Supposed to Be Cutting. Now It's Accelerating.
Four months after slashing R&D spending by 60%, Biohaven reversed course and is now accelerating two molecular degrader programs. The reason: early data showing 87% target protein reduction in Graves' disease and promising biomarker activity in IgA nephropathy. If pivotal trials succeed, it would validate extracellular protein degradation as a therapeutic strategy.
Read more →MASH Claims Another Victim as Boehringer Kills OSE Partnership
Boehringer Ingelheim terminated its MASH partnership with OSE Immunotherapeutics after an efficacy failure, adding to the graveyard of drugs that couldn't crack this massive-market liver disease. The collapse underscores just how stubbornly difficult MASH remains, even with big pharma resources behind the effort.
Read more →Funding and Financings
The Monthly Weight-Loss Shot That Just Raised $73 Million
Beijing-based QL Biopharm closed a $72.9 million Series C led by OrbiMed to develop what could be the world's first once-monthly GLP-1 injection for weight loss. Its lead drug zovaglutide is already in Phase III trials, and its core R&D team includes members from Novo Nordisk's R&D center. In a market where weekly shots are already old news, 12 injections a year instead of 52 is a compelling pitch.
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