Ultragenyx

Ultragenyx Pharmaceutical Inc. is a United States-based biopharmaceutical company focused on rare and ultra-rare genetic diseases, particularly conditions in which treatment options are limited or absent and the underlying biology creates substantial unmet medical need. The company develops and commercializes medicines across several therapeutic areas, including metabolic, bone, endocrine, muscle and central nervous system disorders. Its research and development model spans multiple modalities rather than relying on a single technology platform: biologic medicines, small molecules, gene therapies, antisense oligonucleotides and messenger-RNA approaches have all formed part of its portfolio. The company was founded in 2010 by Emil Kakkis, a physician and rare-disease drug developer whose earlier work included research and clinical development at Harbor-UCLA Medical Center, leadership as chief medical officer of BioMarin and the creation of the EveryLife Foundation. Kakkis established Ultragenyx around the idea that rare diseases could be addressed through focused development programs, specialized regulatory strategies and a willingness to build commercial infrastructure for patient populations too small for many conventional pharmaceutical models. He became the company's president and chief executive officer. Ultragenyx became a public company in 2014 through an initial public offering that raised $126 million. Its commercial portfolio subsequently expanded through internally developed programs, collaborations, licensing arrangements and acquisitions. The company acquired Dimension Therapeutics in 2017, adding adeno-associated-virus gene-therapy manufacturing capabilities and several development programs. It also completed the acquisition of GeneTx Biotherapeutics in 2022 after exercising an option to purchase the company and its lead antisense oligonucleotide program for Angelman syndrome. The company's marketed medicines include Mepsevii, or vestronidase alfa, for mucopolysaccharidosis type VII; Crysvita, or burosumab, for X-linked hypophosphatemia and, in certain markets, tumor-induced osteomalacia; and Dojolvi, or triheptanoin, for long-chain fatty-acid oxidation disorders. Ultragenyx also holds commercial and development rights outside the United States for Regeneron's evinacumab-dgnb, marketed as Evkeeza, for homozygous familial hypercholesterolemia. These products cover enzyme replacement, antibody-based treatment and specialized metabolic nutrition, illustrating the company's strategy of matching therapeutic modality to disease biology. Beyond marketed products, Ultragenyx has maintained a broad clinical pipeline. Programs described in reference materials include setrusumab for osteogenesis imperfecta; several gene-therapy candidates for glycogen storage disease type 1a, ornithine transcarbamylase deficiency, hemophilia A, Sanfilippo syndrome, Wilson disease and Duchenne muscular dystrophy; and investigational antisense or messenger-RNA programs for Angelman syndrome and glycogen storage disease type III. The company has also worked with partners including GeneTx, Kyowa Hakko Kirin, Mereo Biopharma, Daiichi Sankyo, Arcturus Therapeutics, Bayer and Regeneron. Ultragenyx is headquartered in Novato, California, with an additional California presence in Brisbane and operations connected to the Boston area. A gene-therapy manufacturing plant near Boston was announced in 2020 and was under construction in the reference period. The company also maintains a Latin American headquarters in Miami. Its corporate positioning combines specialist rare-disease expertise with an increasingly diversified development and manufacturing platform. Recognition cited in the reference material includes inclusion among Deloitte's fastest-growing North American technology and life-sciences companies in 2020 and 2021, recognition by BioSpace as a best company to work for in 2021, and the 2021 California Life Sciences Pantheon Leadership Award received by Kakkis.

Ultragenyx Pharmaceutical Inc. was established in 2010 by Emil Kakkis, a physician and rare-disease specialist whose experience included work at Harbor-UCLA Medical Center, service as chief medical officer of BioMarin and founding the EveryLife Foundation. The company was created to address inherited diseases affecting relatively small patient populations, especially conditions for which no approved therapy existed or for which available treatment was inadequate. Kakkis assumed the roles of president and chief executive officer and shaped the company around focused clinical development, regulatory specialization and the use of several therapeutic technologies. A major early corporate milestone came in 2014, when Ultragenyx completed an initial public offering in the United States that raised $126 million. Public-market financing supported the expansion of its research organization and the advancement of rare-disease candidates. In 2015, the company entered a collaboration with Arcturus Therapeutics to develop messenger-RNA products, signaling an interest in emerging modalities alongside its work in biologics and small molecules. Ultragenyx broadened its gene-therapy capabilities in 2017 through the acquisition of Dimension Therapeutics. The transaction provided AAV-based manufacturing technology and added several development assets, including a program partnered with Bayer for hemophilia A. The same year also brought a clinical setback: the company discontinued Ace-ER development for GNE myopathy after a study failed to meet its primary endpoint, which measured improvement in arm strength, as well as three secondary measures involving physical function and lower-limb strength. The company began building its commercial presence through approvals for products aimed at highly specialized patient groups. Vestronidase alfa, marketed as Mepsevii, was approved in November 2017 for mucopolysaccharidosis type VII, a rare inherited metabolic disorder also called Sly syndrome. Burosumab, marketed as Crysvita, received United States approval in 2018 for X-linked hypophosphatemia. In 2020, burosumab was also approved for tumor-induced osteomalacia. Triheptanoin, marketed as Dojolvi, was approved in 2020 for long-chain fatty-acid oxidation disorders. The compound is a purified medium-chain triglyceride whose metabolites can help replenish intermediates in energy metabolism. A separate development effort in glucose transporter type 1 deficiency was halted in 2017 after a mid-stage study failed. Ultragenyx continued to invest in infrastructure and geographic reach. In 2020 it announced a gene-therapy manufacturing plant near Boston; the facility was under construction in the period covered by the reference material. The company has a principal base in Novato, California, another California location in Brisbane, a presence in the Boston area and a Latin American headquarters in Miami. It has also developed or commercialized therapies in the United States, Canada, Latin America, Europe and Japan. The portfolio expanded through external partnerships and rights agreements. Ultragenyx has worked with GeneTx, Kyowa Hakko Kirin, Mereo Biopharma, Daiichi Sankyo, Bayer, Arcturus Therapeutics and Regeneron. In 2022, it exercised an option to acquire GeneTx Biotherapeutics, agreeing to an upfront payment of $75 million and potential milestone payments of up to $115 million. The transaction closed in August 2022 and added GTX-102, an antisense oligonucleotide in early development for Angelman syndrome. Ultragenyx also obtained rights outside the United States to develop and commercialize Regeneron's evinacumab-dgnb, marketed as Evkeeza, for homozygous familial hypercholesterolemia. By the period represented in the source material, Ultragenyx had three products with United States Food and Drug Administration approval—Mepsevii, Crysvita and Dojolvi—alongside additional products approved in other regions and a pipeline spanning biologics, gene therapy, antisense oligonucleotides and mRNA. Investigational programs included setrusumab for osteogenesis imperfecta, gene therapies for several metabolic and neuromuscular diseases, and programs for Angelman syndrome and glycogen storage disease type III. The company received external recognition in 2020 and 2021 as one of Deloitte's fastest-growing North American technology and life-sciences companies and was named by BioSpace among the best companies to work for in 2021. In 2021, Kakkis received the California Life Sciences Pantheon Leadership Award.

• 2022

  GeneTx acquisition completed

  [1]Ultragenyxen.wikipedia.org

  Ultragenyx completes its acquisition of GeneTx and adds the GTX-102 Angelman syndrome program.

• 2020

  Dojolvi approval

  [1]Ultragenyxen.wikipedia.org

  Triheptanoin is approved for long-chain fatty-acid oxidation disorders.

• 2018

  Crysvita approval

  [1]Ultragenyxen.wikipedia.org

  Burosumab is approved in the United States for X-linked hypophosphatemia.

• 2017

  Dimension Therapeutics acquisition

  [1]Ultragenyxen.wikipedia.org

  Ultragenyx acquires Dimension Therapeutics and adds AAV gene-therapy manufacturing technology and development programs.

• 2017

  Mepsevii approval

  [1]Ultragenyxen.wikipedia.org

  Vestronidase alfa is approved for mucopolysaccharidosis type VII.

• 2014

  Initial public offering

  [1]Ultragenyxen.wikipedia.org

  The company becomes publicly traded in an offering reported to have raised $126 million.

• 2010

  Ultragenyx is founded

  [1]Ultragenyxen.wikipedia.org

  Emil Kakkis establishes Ultragenyx as a rare-disease biopharmaceutical company.

A rare-disease specialist focused on developing and commercializing therapies for genetically defined conditions with high unmet medical need, using a multi-modality platform and targeted partnerships.

• Biologic antibody · 2018

  Crysvita

  Crysvita is the brand name for burosumab, an antibody therapy developed for disorders involving abnormal phosphate regulation. It received United States approval in 2018 for X-linked hypophosphatemia, a genetic condition characterized by inadequate phosphate levels and impaired bone mineralization. In 2020, the product also received approval for tumor-induced osteomalacia. Ultragenyx developed and commercialized the product through collaboration with Kyowa Hakko Kirin.

• Specialized medical nutrition · 2020

  Dojolvi

  Dojolvi is purified triheptanoin, a medium-chain triglyceride used in the dietary management of long-chain fatty-acid oxidation disorders. Its odd-chain structure produces metabolites that can help replace deficient intermediates in the citric acid cycle, supporting energy production when patients cannot effectively use long-chain fats. The product received approval in 2020. A separate investigation in glucose transporter type 1 deficiency was discontinued after a mid-stage clinical study did not succeed.

• Enzyme replacement therapy · 2017

  Mepsevii

  Mepsevii is the brand name for vestronidase alfa, an enzyme replacement therapy for mucopolysaccharidosis type VII, also known as Sly syndrome. The inherited metabolic disease affects children and adults and is associated with deficient breakdown of specific complex molecules. Mepsevii received United States approval in November 2017 and is one of Ultragenyx's foundational commercial rare-disease products.

• Monoclonal antibody · 2022

  Evkeeza

  Evkeeza is the brand name for evinacumab-dgnb, a medicine developed by Regeneron for homozygous familial hypercholesterolemia. Ultragenyx obtained development and commercial rights in markets outside the United States in 2022. The arrangement extends Ultragenyx's portfolio into a severe inherited lipid disorder while preserving its broader strategy of using external partnerships and rights agreements to supplement internal programs.

• Biologic

  Setrusumab (UX143)

  Setrusumab, identified in the Ultragenyx pipeline as UX143, is an investigational biologic for osteogenesis imperfecta. The condition is a rare genetic disorder affecting bone strength and is described in the reference material as having no approved therapies at the relevant time. The program represents Ultragenyx's focus on genetically defined skeletal diseases beyond its marketed phosphate-regulation treatment.

• Antisense oligonucleotide

  GTX-102

  GTX-102 is an investigational antisense oligonucleotide for Angelman syndrome, a rare neurogenetic disorder. Ultragenyx added the program through its 2022 acquisition of GeneTx Biotherapeutics. The asset illustrates the company's practice of combining acquisitions with internal research to build a pipeline across multiple molecular modalities.

• Gene therapy

  DTX401

  DTX401 is an investigational gene-therapy program for glycogen storage disease type 1a. It entered Ultragenyx's broader gene-therapy portfolio through the company's development activities and its acquisition of Dimension Therapeutics. The program is intended for a rare inherited metabolic disease involving impaired handling of glucose and glycogen.

• Gene therapy

  DTX301

  DTX301 is an investigational gene-therapy candidate for ornithine transcarbamylase deficiency, an inherited metabolic disorder. It is part of Ultragenyx's gene-therapy pipeline and reflects the company's emphasis on addressing diseases caused by defined genetic defects rather than pursuing broad primary-care indications.

• Gene therapy

  DTX201 / BAY 2599023

  DTX201, also identified as BAY 2599023, is an investigational gene-therapy program for hemophilia A. The program is associated with a collaboration involving Bayer and was included among the assets connected to Ultragenyx's gene-therapy development activities.

• Gene therapy

  UX111

  UX111 is an investigational gene-therapy candidate for Sanfilippo syndrome. It is directed at a rare inherited disorder and forms part of Ultragenyx's effort to apply AAV and related gene-delivery capabilities to diseases with limited therapeutic options.

• Gene therapy

  UX701

  UX701 is an investigational gene-therapy program for Wilson disease. The candidate is part of the company's metabolic-disease pipeline and reflects its continued expansion into genetic liver and systemic disorders.

• Gene therapy

  UX810

  UX810 is an investigational gene-therapy candidate for Duchenne muscular dystrophy. The program extends Ultragenyx's development activities into severe inherited muscle disease and is one of several gene-therapy candidates described in the company's pipeline.

• Antisense oligonucleotide or messenger RNA program

  UX053

  UX053 is an investigational program for glycogen storage disease type III. The reference material places it within Ultragenyx's ASO and mRNA development activities, although the supplied information does not specify the final clinical modality or development stage in greater detail.

• 2022

  Acquisition of GeneTx BiotherapeuticsM&A

  Background

  GeneTx's lead program, GTX-102, was an early-stage antisense oligonucleotide candidate for Angelman syndrome.

  Action taken

  Ultragenyx exercised its option to acquire GeneTx for an upfront cash payment of $75 million and potential milestone payments of up to $115 million. The transaction closed in August 2022.

  Aftermath

  GTX-102 became part of Ultragenyx's pipeline and strengthened its presence in antisense-based rare-disease development.

  Acquisition consideration. $75 million upfront, plus up to $115 million in potential milestone payments (2022)

  Source

• 2020

  Construction of a Boston-area gene-therapy plantStrategy

  Background

  Ultragenyx was expanding its gene-therapy pipeline and had acquired AAV-related manufacturing capabilities through Dimension Therapeutics.

  Action taken

  The company announced a new gene-therapy manufacturing facility near Boston.

  Aftermath

  The planned facility was intended to strengthen internal manufacturing capacity for the company's growing gene-therapy portfolio; it was under construction in the period described by the reference material.

  Source

• 2017

  Discontinuation of Ace-ER for GNE myopathyOther

  Background

  The investigational Ace-ER treatment was being studied for GNE myopathy, a rare muscle disease. The clinical study did not demonstrate the required improvement in arm strength and also failed three secondary measures of physical function.

  Action taken

  Ultragenyx stopped further development of Ace-ER after the study results did not meet the prespecified efficacy objectives.

  Aftermath

  The program was removed from the active development path, while the company continued to pursue other rare-disease and gene-therapy candidates.

  Source

• 2021.06.29

  Reported2021.06.30

  Ultragenyx appoints Corsee Sanders to its board[2]Ultragenyx任命Corsee Sanders加入董事会biospace.com

  Ultragenyx appointed Corsee Sanders, Ph.D., to its Board of Directors, effective June 29, 2021, as an independent director.

• 2020.07.30

  Reported2020.07.30

  Ultragenyx reports second-quarter 2020 results[3]Ultragenyx公布2020年第二季度业绩finance.yahoo.com

  Ultragenyx reported second-quarter 2020 total revenue of $61.7 million and maintained its full-year 2020 Crysvita revenue guidance for its territories at $125.0 million to $140.0 million.

Read more

1. 1Ultragenyxen.wikipedia.org
2. 2Ultragenyx任命Corsee Sanders加入董事会biospace.com
3. 3Ultragenyx公布2020年第二季度业绩finance.yahoo.com