A bespoke CRISPR therapy suggests a blueprint for treating ‘N-of-1’ diseases

A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design and test a new CRISPR medicine in just a few months. 

BioMarin to buy rare disease drugmaker Inozyme for $270M

The deal “fits like a glove” for BioMarin’s business, according to an analyst who also thinks there could be a “valuable” market opportunity for Inozyme’s main drug.

Innovating a healthier tomorrow: Entering a new era of medicine

Regeneron defeats Amgen in PCSK9 case; 2 cell therapy biotechs cut staff

A federal jury found Amgen liable for violating antitrust and tort laws. Elsewhere, Allogene and Kyverna trimmed staff and the FDA scheduled an advisory meeting.

Biotech’s M&A outlook is uncertain. Track the deals that are happening here.

Novartis and BioMarin Pharmaceutical, in striking recent deals to respectively buy biotechs Regulus and Inozyme, both made offers that valued their targets at triple-digit premiums.