Ultragenyx

Ultragenyx Pharmaceutical Inc. was founded in 2010 by Emil Kakkis because of his interest in therapies for rare genetic diseases. He became the Chief executive officer and President and focused the company to "meeting the need for treatments for ultra-rare diseases" — understood as affecting fewer than one patient per 50,000 people.[4][5] In 2014 the USA-based company went public with an IPO that raised $126 million.[6] At that time it had four treatments in clinical trials.

• Burosumab (KRN-23; brandname Crysvita) has been approved as a therapy for X-linked hypophosphatemia.[7][8] It works by raising the levels of phosphate in the blood thereby making it possible for faster healing of bone lesions in osteomalacia.[9]
• Triheptanoin, a purified medium-chain triglyceride, has been approved for the treatment of long-chain fatty acid oxidation disorders, in which the body is unable to produce energy from fat.[10] Due to its odd-chain properties, Triheptanoin is broken down into metabolites that replace deficient intermediates in the Citric Acid Cycle.[11]
• Vestronidase alfa was approved in the United States in November 2017 to treat children and adults with the inherited metabolic condition mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.[12]

• GTX-102 - under investigation as a possible treatment option for Angelman syndrome, a rare, neurogenetic disorder.[13][14][15]
• Setrusumab - under investigation for osteogenesis imperfecta, a rare genetic disease with no approved therapies.[16]

Since 2015 Ultragenix has been collaborating with Arcturus Therapeutics to develop mRNA products. Arcturus' clinical programs also include a potential covid-19 vaccine candidate.[17]

• LUNAR-GSD - under development for glycogen storage disease type III.[18]
• LUNAR-Rare - therapeutic candidates for unspecified disease targets.

Ultragenyx acquired Dimension Therapeutics in 2017[19] to obtain adeno-associated virus (AAV)-based gene therapy manufacturing technology.[20]

The company's recent research and development collaborations with competitors has followed failures with several in-house products. The company has been accused of making rushed and incomplete regulatory applications.[21] Investment banks Evercore and Jefferies noted that several potential products have "meaningful and underappreciated clinical risk."[22] [23]

• Ace-ER's - Possible treatment for GNE myopathy was discontinued after failing to meet three key secondary endpoints that evaluated patients' physical functions, leg muscle strength and knee extension force.[22] Previous trials in mice had shown efficacy. [24]
• UX007 - Stage 3 trials continued despite failing a mid stage study.[22]