Drug Discovery

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

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

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.

Bluebird bio’s financial situation is a stark reminder of the challenges of developing advanced therapies for rare diseases.

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

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

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

The Access to Medicine Foundation has developed the 2026 AMR Benchmark to recognize and promote urgency in the ongoing AMR challenge.

Early work from Scripps researchers uses a virtual library to synthesize plant-based metabolites: picrotoxanes.