Ultragenyx is an American

biopharmaceutical A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. Different from totally synthesized pharmaceuticals, th ...

company involved in the Research and Development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases. Ultragenyx is based in Novato, CA and Brisbane, CA and has a presence in the Boston area, including a gene therapy plant under construction as of 2021. The company’s Latin American headquarters is located in Miami. Ultragenyx collaborates on product development with other companies including GeneTX,

Kyowa Hakko Kirin is a Japanese pharmaceutical and biotechnology company under the Kirin Holdings, and is among the 40 largest in the world by revenue. The company is headquartered in Chiyoda-ku, Tokyo and is a member of the Nikkei 225 stock index. History On ...

, Mereo Biopharma and Daiichi Sankyo. Ultragenyx has three products

Burosumab Burosumab, sold under the brand name Crysvita, is a human monoclonal antibody medication approved 2018 for the treatment of X-linked hypophosphatemia and tumor-induced osteomalacia. Medical uses In the European Union and the United States, b ...

Triheptanoin Triheptanoin, sold under the brand name Dojolvi, is a medication for the treatment of children and adults with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). The most common adverse reactions include abdominal pain, d ...

and

Vestronidase alfa Vestronidase alfa, sold under brand name Mepsevii, is a drug for the treatment of Sly syndrome. It is a recombinant form of the human enzyme beta-glucuronidase. It was approved in the United States in November 2017, to treat children and adul ...

that have received FDA approval and several others currently in clinical trials. The company also holds the non-US commercial rights to

Regeneron Regeneron Pharmaceuticals, Inc. is an American biotechnology company headquartered in Westchester County, New York. The company was founded in 1988. Originally focused on neurotrophic factors and their regenerative capabilities, giving rise to i ...

’s evinacumab-dgnb, which is approved by the FDA and EMA. The company also has therapies approved outside the U.S. in Canada, Latin America, Europe, and Japan. In 2020 and 2021, Ultragenyx was named one of Deloitte’s fasting growing technology and life sciences companies in North America and one of the best companies to work for by BioSpace. Also in 2021, the company’s CEO,

Emil Kakkis Emil Kakkis (born 1960) is an American medical geneticist known for his work to develop treatments for ultra rare disorders. He is the Founder of the Everylife Foundation for Rare Disease and Founder, CEO and President of Ultragenyx Pharmaceut ...

, was awarded the California Life Sciences Pantheon Leadership award.

History

Ultragenyx Pharmaceutical Inc. was founded in 2010 by

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 A chief executive officer (CEO), also known as a central executive officer (CEO), chief administrator officer (CAO) or just chief executive (CE), is one of a number of corporate executives charged with the management of an organization especially ...

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 An initial public offering (IPO) or stock launch is a public offering in which shares of a company are sold to institutional investors and usually also to retail (individual) investors. An IPO is typically underwritten by one or more investment ...

that raised $126 million. In 2015 Ultragenyx began collaborating with

Arcturus Therapeutics Arcturus Therapeutics is an American RNA medicines biotechnology company focused on the discovery, development and commercialization of therapeutics for rare diseases and infectious diseases. Arcturus has developed a novel, potent, and safe RNA t ...

to develop mRNA products. Ultragenyx acquired Dimension Therapeutics in 2017 to obtain

adeno-associated virus Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus ''Dependoparvovirus'', which in turn belongs to the family '' Parvoviridae''. They are small (approximately 26 nm i ...

(AAV)-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.

Products

(brand name Mepsevii) was approved in November 2017 to treat children and adults with the inherited metabolic condition mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. *

(KRN-23; brand name Crysvita) was approved in 2018 by the FDA to treat

X-linked hypophosphatemia X-linked hypophosphatemia (XLH) is an X-linked dominant form of rickets (or osteomalacia) that differs from most cases of dietary deficiency rickets in that vitamin D supplementation does not cure it. It can cause bone deformity including shor ...

. In 2020 the drug was approved to treat

tumor-induced osteomalacia Oncogenic osteomalacia, also known as oncogenic hypophosphatemic osteomalacia, is an uncommon disorder resulting in increased renal phosphate excretion, hypophosphatemia and osteomalacia. It may be caused by a phosphaturic mesenchymal tumor. Sign ...

. *

(brand name Dojolvi) a purified medium-chain triglyceride, was approved in 2020 for the treatment of long-chain fatty acid oxidation disorders in which the body is unable to produce energy from fat. Due to its odd-chain properties, Triheptanoin is broken down into metabolites that replace deficient intermediates in the

Citric Acid Cycle The citric acid cycle (CAC)—also known as the Krebs cycle or the TCA cycle (tricarboxylic acid cycle)—is a series of chemical reactions to release stored energy through the oxidation of acetyl-CoA derived from carbohydrates, fats, and protei ...

. Trials on this drug as treatment for a different condition, Glut1 deficiency, a seizure disorder, were halted in 2017 due to a failure of a mid stage study. * Evinacumab-dgnb (brand name Evkeeza) was approved in February 2021 for the treatment of homozygous familial hypercholesterolemia. Developed by

, Ultragenyx acquired the sales and development rights in 2022 in markets outside the United States.

Pipeline

Biologic

* UX143 (Setrusumab) - under investigation for

osteogenesis imperfecta Osteogenesis imperfecta (; OI), colloquially known as brittle bone disease, is a group of genetic disorders that all result in bones that break easily. The range of symptoms—on the skeleton as well as on the body's other organs—may be ...

, a rare genetic disease with no approved therapies.

Gene Therapy

* DTX401 - in clinical development for glycogen storage disease type 1s (GSD1a). * DTX301 - in clinical development for treatment of ornithine transcarbamylase (OTC) deficiency. * DTX201/BAY 2599023 - in clinical development for treatment of

hemophilia A Haemophilia A (or hemophilia A) is a genetic deficiency in clotting factor VIII, which causes increased bleeding and usually affects males. In the majority of cases it is inherited as an X-linked recessive trait, though there are cases which arise ...

. * UX701 - in clinical development for treatment of

Wilson Disease Wilson's disease is a genetic disorder in which excess copper builds up in the body. Symptoms are typically related to the brain and liver. Liver-related symptoms include vomiting, weakness, fluid build up in the abdomen, swelling of the legs, ...

. * UX810 - in clinical development for treatment of Duchenne Muscular Dystrophy (DMD).

ASO/mRNA

*GTX-102 - in clinical development as a possible treatment option for

Angelman syndrome Angelman syndrome or Angelman's syndrome (AS) is a genetic disorder that mainly affects the nervous system. Symptoms include a small head and a specific facial appearance, severe intellectual disability, developmental disability, limited to no ...

, a rare, neurogenetic disorder. *UX053 - in clinical development for glycogen storage disease type III (GSDIII).

References

{{Pharmaceutical companies of the United States Biotechnology companies of the United States Companies listed on the Nasdaq Companies based in Marin County, California Pharmaceutical companies of the United States