Ultragenyx

Ultragenyx Pharmaceutical Inc. was founded in 2010 by Emil Kakkis based on his history of developing therapies for rare disease starting at Harbor-UCLA Medical Center, continuing through his role as Chief Medical Officer of BioMarin and as founder of the EveryLife Foundation. He became the Chief executive officer and President, focusing the company to meet the treatment needs for rare diseases understood as affecting fewer than 200,000 people in the U.S.. In 2014 the USA-based company went public with an IPO that raised $126 million. In 2015 Ultragenyx began collaborating with Arcturus Therapeutics to develop mRNA products. Ultragenyx acquired Dimension Therapeutics in 2017 to obtain adeno-associated virus -based gene therapy manufacturing technology as well as three internal candidates and one partnered candidate with Bayer for Hemophilia A.In 2017, the company discontinued development of Ace-ER's treatment for GNE myopathy after the product didn’t meet the study’s primary endpoint which was significant improvement in arm strength. It also failed to meet three key secondary endpoints that evaluated patients' physical functions, leg muscle strength and knee extension force. Previous trials in mice had shown efficacy. In 2020 Ultragenyx announced a new Gene Therapy Plant being built near Boston.In July 2022 Ultragenyx announced that it would exercise its option to acquire GeneTx Biotherapeutics for an up-front cash payment of $75 million, plus another $115 million in potential milestone-dependent payments. Ultragenyx will add the antisense oligonucleotide therapy GTX-102 to its broad pipeline of therapies indicated for various rare diseases. GTX-102 is currently in early-stage development for Angelman syndrome. The deal was completed in August 2022.