Discovery & Development

How antisense oligonucleotides open up targeted approaches to rare disease treatment.

Long-acting injectables help with adherence, but what if an unfolding pill could provide an oral option?

Researchers develop a new platform to improve delivery of oligonucleotide therapies to muscle.

And getting the right people involved from the start. Here’s why collaboration is key for successful clinical development.

Why researchers are excited about the potential of extracellular vesicles from umbilical cord-derived stem cells.

We asked three rare disease drug developers about their work, the challenges facing the field, and what they would do with unlimited funds.

A new study reports targeted CRISPR delivery in retinal and neurological disease models – without viral vectors.

How connecting the data dots can lead to better development and clinical trals. 

A Lassa fever vaccine candidate shows promise in animal studies, with guinea pigs 100 percent protected from lethal virus exposure.

How precision analytics is driving the future of pharmaceuticals, from small molecules to CRISPR gene editing.