The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial. Shortly after the regulatory hold was implemented, Intellia disclosed that ...

Intrapleural enzyme therapy (IET) improves outcomes in adult inpatients with complicated parapneumonic pleural effusion or empyema.

"Life is taken away from these children before they even have a chance to live it," says Megan Kempf of her two children, who ...

Regenxbio Inc. faces pivotal catalysts in 2026, notably RGX-121's FDA decision and RGX-202 Duchenne data, potentially ...

Sharp Therapeutics Corp. ("Sharp" or the "Company") (TSX-V: SHRX) (OTCQB: SHRXF), a biotechnology company developing small-molecule therapeutics for genetic diseases, announced today the appointment ...

Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) ...

PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...

Tirzepatide users on hormone therapy had added improvements in weight, cardiometabolic mar ...

Chronic pain affects more than 50 million Americans, yet for decades, treatment options for pain that persists in the absence ...

Unique patent policy, co-funding, and industry mentorship help move discoveries at Rutgers across the “valley of death.” ...

ClearPoint Neuro is positioned for robust growth in 2026, driven by IRRAS, the PRISM 1.5T system clearance, and the company's new CRO facility. While the immediate future of uniQure's AMT-130 remains ...

AI‑driven protein design creates potent anti‑CRISPR inhibitors that block Cas13 activity, offering a new potential tool for safer, more controlled gene editing.