Ultragenyx

Ultragenyx was founded in 2010 by current CEO Emil Kakkis. Emil's past research into ultra-rare diseases steered the company's focus to "meeting the need for treatments for ultra-rare diseases"—understood as affecting fewer than one patient per 50,000 people.[4][5] Four years later on February 4, 2014 it went public with an IPO that raised $126 million.[6] At the time it had four drugs 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 It was approved in the United States in November 2017, to treat children and adults with an inherited metabolic condition called 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 disorde related to autism.[13][14][15]
• Setrusumab - under investigation for osteogenesis imperfecta, a rare genetic disease with no approved therapies.[16]

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

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

Research into new ways of using biotech appears to have caught the company's attention. Ultragenyx's adeno-associated virus (AAV)-based gene therapy manufacturing technology is being used to mass produce investigational drugs for AAV gene therapy.[19] This technology was acquired via a 2017 acquisition of Dimension Therapeutics.[20]

The company's recent string of collaborations with competitors on product research and development has come after a number of its own product failures. Investment banks Evercore and Jefferies have said that a number of items in the product stream have "meaningful and underappreciated, clinical risk."[21] [22] The company has been accused of rushing through market application submissions irrespective of their completeness.[23]

• Ace-ER's - Possible treatment for GNE Myopahy was terminated after failing to meet three key secondary endpoints that evaluated patients' physical functions, leg muscle strength and knee extension force.[21] It was previously shown to be effective in mice. [24]
• UX007 - Despite failing a mid stage study, Ultragenyx continued forward with stage 3.[21]