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.

The sweeping workforce reduction that began Tuesday was made worse by mistakes and poor communication, including directions for some CMS employees to contact a director who died last year.

Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

Clearance of Vykat unlocks what analysts anticipate will be a lucrative market opportunity for Soleno Therapeutics.

Amvuttra’s clearance in a rare form of cardiomyopathy could help Alnylam turn a regular profit — if it can wrest control of a competitive and fast-changing market.

Optum, the pharmacy benefit manager owned by UnitedHealth Group, placed Journavx on some of its commercial formularies in an interim decision while a formal review continues.

Striking study results last year indicated a new type of medicine may improve on Merck’s dominant immunotherapy, spurring a wave of research practically overnight.