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 ...

Sarepta's cost-saving moves, FDA surprises, and pipeline potential position SRPT as a unique opportunity. Read here for an ...

The FDA appears to have won its staring contest with Sarepta over shipments of Elevidys, after the company blinked and agreed ...

Detailed price information for Generation Bio Co. (GBIO-Q) from The Globe and Mail including charting and trades.