Policy
The FDA launched its Expedited IND Pilot Program on September 15, 2026, as part of Operation TrialBlazer. The ambition is real, but the delays driving research offshore run deeper than the mechanism being deployed to address them.
Biotechnology
GSK's Jideytro demonstrates a 94% response rate in first-line ROS1-positive NSCLC, with a remarkable 15% complete response rate and 100% intracranial response rate in patients with brain metastases.
Biotechnology
Novartis's remibrutinib has achieved what the BTK inhibitor class could not: a Phase 3 win in multiple sclerosis with no liver safety signal. Here's why this matters for the future of MS treatment.
Biotechnology
Amgen's Phase 3 DeLLphi-305 trial delivered a landmark overall survival win for tarlatamab in first-line extensive-stage small cell lung cancer, marking the first time a bispecific T-cell engager has demonstrated this benefit in this setting.
Biotechnology
Novartis's $12 billion acquisition of Avidity Biosciences and the subsequent failure of del-desiran in Phase 3 trials reveals critical lessons about the limits of acquisition-driven drug development in rare disease.
Biotechnology
uniQure has filed the first-ever gene therapy application for Huntington's disease. The science is compelling, the regulatory backstory is dramatic, and the stakes for 30,000 Americans and 200,000 at-risk individuals could not be higher.
Pharmaceuticals
AstraZeneca's Etcamah became the first FDA-approved cancer therapy triggered by a blood test detecting resistance before imaging shows progression. The approval rewrites how oncologists think about treatment switching in HR-positive breast cancer.
Biotechnology
The FDA's approval of Zanvastro marks the first treatment ever approved for Alexander disease, signaling a broader shift in how RNA-targeted therapies can address ultra-rare neurological conditions.
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.