WVE-006, the leading GalNAc-RNA editing oligonucleotide designed to correct AAT protein and address both lung and liver ...

Molecular Instruments® (MI), the inventor of the HCR™ platform, today announced key enhancements to its HCR™ Gold and HCR™ Pro product lines. These upgrades include next-generation chemistry for ...

Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...

Researchers have found in ALPACA trial that Lepodisiran demonstrated a substantial reduction in lipoprotein(a) levels, ...

Nucleic acid therapies offer targeted treatment for genetic diseases, but delivery remains a key hurdle. Explore how advances ...

A WEHI-led study reveals that blocking minor splicing – an RNA process critical for cell division – reduces tumour growth in ...

In a statement, the Cambridge, Massachusetts-based company said that shipments of Elevidys (delandistrogene moxeparvovec) will restart while it continues dialogue with the FDA "on next steps in the ...

Two months after laying off 20% of its staff, Korro Bio says it has not retreated from its “3-2-1” corporate strategy as it prepares to release interim clinical data on its lead candidate KRRO-110 in ...

UMass President Marty Meehan and healthcare leaders emphasize UMass Chan’s future success depends on its next chancellor’s ability to cultivate talent, face federal challenges head-on, invest in ...

The therapies, which are just starting to enter clinical trials, will need to prove clear clinical and quality-of-life ...

Humanin represents a fascinating intersection of mitochondrial biology, cellular signaling, and organismal physiology. Its ...