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PTC Withdraws Translarna FDA Bid – Data Gaps Cited
PTC Therapeutics said it will stop pursuing FDA approval for Translarna (ataluren) after regulators indicated the submitted data are unlikely to meet the agency’s threshold for substantial...
Australia Launches First Pediatric mRNA Brain‑Cancer Trial
Australia opened Paedneo‑Vax, the world’s first multisite pediatric trial testing individualized mRNA vaccines for aggressive brain tumors, funded by Providence Therapeutics, the Australian...
Anterior Raises $40M to Automate Care Approvals with AI
Anterior closed a $40 million financing to scale an AI platform that automates administrative care-authorization work for health plans, MedCity News reported. The company says its system can...
Agios Advances Mitapivat, Tebapivat in Sickle‑Cell After Thalassemia Win
After a regulatory success in thalassemia, Agios is preparing to present mixed Phase III Rise Up data for mitapivat and tebapivat to the FDA as it seeks full approval in sickle‑cell disease, the...
NIH’s All of Us Tops 1 Million — Representative Genomic Resource Achieved
The NIH announced that its All of Us research program has reached 1 million participants, meeting its goal of creating a diverse, population‑representative dataset of genomic and health data. The...
NIAID Restructures: Biodefense and Pandemic Prep Deprioritized, Emails Show
Nature reported that staff at the National Institute of Allergy and Infectious Diseases were directed to remove references to 'biodefense' and 'pandemic preparedness' from the institute’s web...
FDA Rejects Disc Medicine’s Bitopertin—Biomarker Link Deemed Uncertain
The FDA has rejected Disc Medicine’s bitopertin, the agency saying uncertainties remain about the surrogate blood biomarker used to support efficacy. The decision, reported by STAT, halted the...
Moderna’s Flu Review Stalls... Regulatory Pushback Ripples Through Biotech
The FDA declined to review Moderna’s mRNA flu vaccine application, a move that has clouded the company’s cash‑flow guidance and reignited debate over regulatory expectations for next‑generation...
Exact Sciences Q4 Revenue Jumps 23% — Shareholders to Vote on $23B Abbott Deal
Exact Sciences reported a 23% year‑over‑year revenue increase for Q4 2025 and beat consensus on screening volumes, lifting momentum ahead of a special shareholders’ meeting to approve Abbott’s...
Bio‑Rad’s ddPCR Lift—Product Momentum Meets Technology Adoption
Bio‑Rad reported modest overall revenue growth driven in part by expansion of its droplet digital PCR (ddPCR) business and stronger consumables pull‑through, the company said on its Q4 call....
Ultragenyx Cuts 10% of Workforce as FDA Flags Gene‑Therapy Gaps
Ultragenyx announced a restructuring that will eliminate roughly 130 roles—about 10% of headcount—citing a push toward profitability in 2027 and recent regulatory setbacks. The company said its...
Lyell Launches First‑of‑Its‑Kind CAR‑T Trial—Direct Test vs Marketed Products
Lyell Immunopharma has initiated a head‑to‑head CAR‑T clinical trial that will test its experimental cell therapy directly against marketed CAR‑T products. The design aims to generate comparative...
Anterior Raises $40M—AI Platform to Slash Care‑Approval Times
Anterior closed a $40 million financing to scale an AI platform that automates administrative clinical workflows for health plans, the company said. The platform is designed to accelerate...
Australia Launches World‑First Pediatric mRNA Brain‑Cancer Trial
Australia has launched Paedneo‑Vax, the first multisite pediatric trial testing individualized mRNA vaccines for aggressive childhood brain tumors. Funded by Providence Therapeutics with public...
Vertex’s CRISPR Therapy Rebounds—Casgevy Sales Triple in Latest Quarter
Vertex reported a significant sales increase for Casgevy, its CRISPR‑based gene therapy developed with CRISPR Therapeutics, with quarterly sales more than tripling versus the prior quarter....
VCs Launch European Life Science Coalition: €24B to Mobilize Investment
A consortium of venture capital firms and research institutions has formed the European Life Science Coalition to boost private and public investment into Europe’s biotech sector. The...
FDA rejects Disc therapy — expedited voucher route falters
The FDA rejected Disc Medicine’s bitopertin application, marking a high-profile rebuke of a rare-disease submission that had been expedited under a new priority-review voucher program. STAT...
NIAID reshapes priorities: biodefense and pandemic preparedness deprioritized
Internal directives at NIAID instructed staff to remove the terms “biodefense” and “pandemic preparedness” from public materials as the institute refocuses funding toward basic immunology and...
BridgeBio’s oral FGFR3 matches Voxzogo on height — adds pill option
BridgeBio reported that infigratinib, an oral FGFR3 inhibitor, produced height gains comparable to Voxzogo in trials for achondroplasia while offering an oral dosing option. BioCentury’s reporting...
Lyell launches head-to-head CAR-T trial: experimental therapy to face rivals
Lyell Immunopharma initiated a first-of-its-kind clinical trial testing its experimental CAR-T therapy directly against marketed CAR-T products, the company said. The trial will evaluate...