Shionogi launches global Phase 2 trial for late-onset Pompe disease treatment

Esprit is a multicenter, randomized, placebo-controlled, double-blind study assessing the safety, pharmacodynamics, and preliminary efficacy of S-606001 as a substrate reduction therapy

FDA nod to first treatment for rare genetic brain disorder

The approval comes after a systematic review of published literature

UK pushes space-made medicines from orbit to patients with new support package

The initiative provides clearer regulatory guidance and a defined path from research in space to patient access on Earth

Lonza expands manufacturing pact with Genetix to meet rising demand for gene therapy ZYNTEGLO

Merck delivers resilient 2025 performance despite global headwinds

Strong performances in its Life Science, Healthcare and Electronics divisions — particularly Process Solutions, Cardiovascular treatments and Semiconductor Materials — powered the company’s results

FDA okays JUXTAPID for kids with ultra-rare genetic cholesterol disorder

Moderna strikes $950 million global settlement with Arbutus & Genevant, clears legal hurdles for vaccine pipeline

Moderna will appeal to the Federal Circuit to assert that its government-contractor immunity limits liability under federal law

Theravance Biopharma winds down Ampreloxetine programme after Phase 3 miss

Merck restructures Human Health division for future growth

Merck is currently conducting approximately 80 Phase 3 studies, and the company expects more than 20 new growth drivers over next several years, almost all with blockbuster potential

Axol Bioscience acquires ophthalmology business from Newcells Biotech

Acquisition expands Axol’s portfolio of iPSC-derived retinal organoid and retinal pigment epithelium (RPE) models