Biotechnology
ARPA-H's $125 million GIVE program aims to revolutionize RNA medicine manufacturing through distributed, automated platforms that can produce personalized therapies on demand near patients.
Pharmaceuticals
GSK is advancing an mRNA flu vaccine that targets both hemagglutinin and neuraminidase into Phase 3, a more ambitious dual-antigen approach than Moderna's recently approved mFlusiva. Here is what the science means and why it matters.
Biotechnology
Novartis and Bristol Myers Squibb paused their autoimmune CAR-T programs on the same day, forcing the field to reckon with safety challenges that may be inherent to translating oncology science into autoimmune disease.
Pharmaceuticals
The FDA's approval of BESREMi marks the first new therapy for essential thrombocythemia in nearly 30 years, signaling a shift toward disease-modifying treatments that target the molecular drivers of this rare blood cancer.
Pharmaceuticals
Novartis, Amgen, and Eli Lilly are racing to address Lp(a), a genetic cardiovascular risk factor affecting 1 in 5 people with no approved treatment. The first pivotal outcomes data could arrive within weeks.
Biotechnology
The FDA's approval of Rasonque marks a watershed moment in oncology—the first therapy to directly target RAS, ending three decades of failed attempts to drug one of cancer's most intractable targets.
Pharmaceuticals
Takeda's FDA approval of Orzeyful marks a paradigm shift in narcolepsy treatment—the first medicine to directly target orexin deficiency rather than manage symptoms.
Biotechnology
Roche commits $2.3 billion to Hanmi's UCN2 obesity drug candidate, signaling a strategic shift toward muscle-preserving mechanisms beyond GLP-1 agonists.
Pharmaceuticals
The FDA approved a subcutaneous starting dose of lecanemab, allowing Alzheimer's patients to begin treatment at home via weekly self-injection—removing a major access barrier to this disease-modifying therapy.
Biotechnology
The FDA's accelerated approval of Ultragenyx's Genglycos marks the first gene therapy for glycogen storage disease type Ia, signaling a new era in treating rare metabolic diseases at their genetic root.
Wall Street Wire
Moderna's cancer vaccine with Keytruda just worked. That's huge: the stock nearly tripled, and the whole biotech index moved. As attention around oncology innovators increases, several other companies are doing exciting things that may be worth watching.
Biotechnology
Moderna and Merck's personalized mRNA cancer vaccine intismeran autogene has succeeded in its first Phase 3 trial in melanoma, marking the first late-stage win for any mRNA cancer vaccine and raising profound questions about the future of oncology and the mRNA platform itself.