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New LRRK2 variant P1446L drives neurodegeneration via microglial inflammation
Investigators identified a novel LRRK2 mutation (P1446L) that promotes dopaminergic neuron loss through microglia‑driven inflammation and apoptosis, according to a paper in npj Parkinson’s...
MINFLUX maps cardiac ryanodine receptor in 3D in living cells
Researchers applied MINFLUX super‑resolution microscopy to resolve the subunit architecture and three‑dimensional orientation of cardiac ryanodine receptors (RyR) inside living cells. The study...
Autophagy sorts mitochondria to define CD8+ T cell fate and division
Teams at the Max Delbrück Center and University of Oxford reported in Nature Cell Biology that autophagy regulates mitochondrial inheritance during asymmetric CD8+ T cell division, directing...
Antibiotic exposure associated with altered breast cancer survival in population study
A large population‑based cohort study from England and Wales, published in Nature Communications, found associations between antibiotic use and breast cancer survival outcomes. The analysis...
Cytokinetics lands first FDA approval – Myqorzo cleared for obstructive HCM
The FDA approved Cytokinetics’ Myqorzo, giving the company its first U.S. drug authorization after a 27-year run. Regulators cleared the therapy to treat obstructive hypertrophic cardiomyopathy...
BioMarin to buy Amicus for $4.8B – Fabry and Pompe assets added
BioMarin agreed to acquire Amicus Therapeutics for $4.8 billion, adding marketed treatments for Fabry and Pompe diseases to its rare-disease portfolio. The transaction accelerates BioMarin’s...
Insmed stock tumbles after Brinsupri clinical setback
Insmed reported a clinical setback for Brinsupri that triggered an immediate market-cap reduction, according to BioCentury’s clinical report. The failure or delay in Brinsupri’s development...
Nonprofit brings abandoned rare-disease gene therapy to market – scalability questioned
A nonprofit organization succeeded in taking an abandoned rare-disease gene therapy to market, reporting a path to patient access where commercial sponsors had exited. The group partnered with...
U.S. research funding frozen – NIH grant reviews and operations curtailed
The U.S. government enacted operational freezes across the NIH in 2025, halting grant reviews, external communications, travel, and hiring—moves that cost the research community more than $1.5...
U.S. vaccine schedule overhaul proposed — experts warn of risks
Reporting indicates U.S. officials are considering aligning the childhood vaccine schedule more closely with Denmark by removing or reducing routine immunizations for diseases such as rotavirus,...
JAX completes NYSCF acquisition: unified nonprofit aims to accelerate translational genetics
The Jackson Laboratory finalized its acquisition of the New York Stem Cell Foundation, launching a unified nonprofit that combines JAX’s mouse genetics with NYSCF’s automated human stem-cell...
Antisense oligonucleotides reverse SMA defects in organoid models – Nature Commun.
A Nature Communications study showed targeted antisense oligonucleotide (ASO) therapy could rescue developmental defects in spinal muscular atrophy (SMA) organoids. The team demonstrated...
Fruquintinib plus sintilimab shows activity in pMMR advanced endometrial cancer
A multicenter Phase Ib/II trial reported clinically meaningful responses combining fruquintinib, a VEGFR inhibitor, with sintilimab, an anti‑PD‑1 antibody, in patients with...
Oral antivirals tied to lower post‑COVID cardiovascular risk – target trial emulation
A Nature Communications target trial emulation found that nirmatrelvir/ritonavir (Paxlovid) and molnupiravir reduced the risk of cardiovascular events after COVID‑19 infection. The study used...
FDA clears Cytokinetics heart drug: first U.S. approval
The FDA approved Cytokinetics’ Myqorzo to treat obstructive hypertrophic cardiomyopathy, marking the company’s first U.S. drug approval after a multi-decade effort. The clearance positions...
BioMarin buys Amicus for $4.8B – rare‑disease footprint expands
BioMarin agreed to acquire Amicus Therapeutics for $4.8 billion, adding two approved enzyme-replacement therapies for Fabry and Pompe diseases and strengthening BioMarin’s rare-disease commercial...
Partial hold on Merck/Daiichi ADC trial after patient deaths
Regulators and sponsors paused parts of a phase III antibody‑drug conjugate (ADC) program after an unexpected cluster of fatal interstitial lung disease (ILD) events. The FDA placed a partial hold...
White House unveils nine most‑favored‑nation drug deals — broad pharma list
The White House announced a set of agreements with nine major drugmakers designed to lower U.S. drug prices by aligning some Medicare/Medicaid prices with peer‑nation levels. Companies named in...
Galux and Boehringer join on AI protein design — targeted therapeutics push
South Korean AI‑driven protein discovery firm Galux signed a research agreement with Boehringer Ingelheim to explore AI‑guided, purpose‑specific protein molecule design. Galux will validate...
Genomics partnerships scale single‑cell and methylation assays
Two commercial collaborations aim to expand high‑throughput functional genomics and epigenomics. Parse Biosciences struck a deal with Codebreaker Labs to combine engineered variant libraries with...