User:Grmike/sandbox

Ultragenyx is a biopharmaceutical company focused on the development of novel products for rare and ultra-rare life-threatening diseases. The drugs are both treatment options and therapeutic in nature. Some of its drugs are being developed in collaboration with Genetex, Mereo Biopharma and Daiichi Sankyo.

Notable Products

 * GTX-102 - Indicated as a possible treatment option for angelman syndrome, a rare, neurogenetic disorder. Cleared by the FDA in January of 2020 to enter into the clinical study phase. >
 * Setrusumab - Has the ability to improve bone production and density leading to greater bone strength. Osteogenesis imperfecta is a rare and devastating genetic disease, with currently no approved therapies.
 * Triheptanoin a purified medium-chain triglyceride, is a source of calories and fatty acids for patients with long-chain fatty acid oxidation disorders, in which the body is unable to produce energy from fat. If left untreated, patients can have severe complications, including early death.

Gene Therapy Technology

 * The company's research in gene therapy has yielded successful results in the sphere of biotech. In mid 2020 Daiichi Sankyo chose Ultragenyx’s proprietary adeno associated virus (AAV)-based gene therapy manufacturing technology to be used in the manufacture of investigational drugs for AAV gene therapy.