Search Results

Cure Rare Disease, a clinical-stage nonprofit biotechnology organization, announces the addition of a Chief Advancement Officer to its leadership team and welcomes three new members to its board of directors.

A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications.

In exchange for its investigational gene therapies, Regenxbio will receive $110 million upfront and up to $700 million in milestones. After hitting an all-time low of $6.95 at close of business yesterday, the stock surged on the news by nearly 20% before markets opened Tuesday.

Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, announced updates from their rare endocrine disease portfolio with two late-breaking poster presentations and one oral presentation during the Endocrine Society’s ENDO 2024 Meeting that was held June 1-4, 2024, in Boston, Massachusetts.