NUS Medicine-led study shows extracellular vesicles carrying gene-targeting antisense oligonucleotides (ASOs) and immune ...

The RNA Horizons Therapeutics Symposium2025 convened leading scientists, clinicians, and biotech innovators in Palermo, Sicily, to explore the newest frontiers in RNA biology and medicine. Building on ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

Arrowhead Pharmaceuticals faces key FDA decisions, legal challenges, and trial results. Read the latest analysis on the stock ...

A study has confirmed the effectiveness of a drug that can treat acquired genetic hearing loss. On the 11th, a research team led by Professor Ji Heon-young, Professor Jang Seung-hyun of Yonsei ...

Stoke Therapeutics, Inc. , a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine, and Biogen Inc. today announced the publication of ...

The committee reviewed data from studies showing that the antisense oligonucleotide reduced the monthly mean rate of angioedema attacks.

– Despite treatment with standard-of-care anti-seizure medicines, children with Dravet syndrome experienced high seizure burden and plateaued in neurodevelopment, resulting in a widening gap relative ...

Despite treatment with standard-of-care anti-seizure medicines, children with Dravet syndrome experienced high seizure burden and plateaued in ...

The FDA’s Marty Makary and Vinay Prasad have laid out a path for CRISPR treatments on demand. An expert weighs in on what it ...

There was an increase in the hepatic fat fraction, the long-term consequences of which are unknown, says Robert Rosenson.

The Lysosomal Storage Disease Treatment Market is expanding due to increased diagnosis rates and innovations in enzyme replacement, gene, and RNA-based therapies. Precision medicine and personalized ...