Ultragenyx Pharmaceutical Inc.
NEWS
When Sekar Kathiresan’s brother unexpectedly died from a heart attack, he changed the course of his life to found a company with a novel medicine that may prevent them.
Several CEOs have stood out from their peers throughout the past year, winning recognition from the Healthcare Technology Report. BioSpace looks at five of these leaders.
The Glycogen Storage Disease Program at Connecticut Children’s and UConn Health reported success in a Phase I/II trial of a gene therapy for GSD-Ia.
GeneTx Biotherapeutics and Ultragenyx Pharmaceutical’s jointly prepared treatment for Angelman syndrome have been provided a green single by DSMB recently.
Collaboration is key to faster breakthroughs. The NIH, FDA, and 15 private organizations have announced they are joining forces for the sake of the 30 million Americans suffering from a rare disease.
It was yet another busy week for clinical trial news. Here’s a look.
Redosing is a large and growing concern for gene therapy developers and regulators.
Several biotech and biopharmaceutical companies have announced new wins with the U.S. Food and Drug Administration (FDA), with these wins ranging from Investigational New Drug (IND) application approvals for cancer treatment candidates to Fast Track Designations for investigational agents aimed toward opioid use disorder (OUD).
JOBS
IN THE PRESS