Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical is a biopharmaceutical company based in the United States that specializes in developing therapeutics for rare and ultra-rare diseases, particularly serious genetic disorders. The company focuses on identifying and commercializing innovative treatments that address significant unmet medical needs. Its product portfolio includes Crysvita, which is used to treat X-linked hypophosphatemia, and Mepsevii, indicated for Mucopolysaccharidosis VII. By leveraging existing scientific knowledge and emphasizing clear mechanisms of action, Ultragenyx aims to create impactful therapies for conditions that currently lack effective treatments. The company prioritizes efficient development strategies in a field characterized by limited information and established precedents, drawing on the management team's expertise to advance its mission of improving patient outcomes in rare disease populations.

Howard Horn

Executive Vice President and CFO

Emil D. Kakkis

CEO and President

4 past transactions

Amlogenyx

Seed Round in 2024
Amlogenyx is a novel gene therapeutics company that focuses on developing lysosome-based treatments for Alzheimer's disease. It is a spin-off from Ultragenyx Pharmaceutical.

GeneTx Biotherapeutics

Acquisition in 2022
GeneTx Biotherapeutics is a biotechnology company focused on developing innovative therapeutics for Angelman syndrome, a rare genetic disorder. Established in 2017 and headquartered in Downers Grove, Illinois, the company specializes in antisense therapy, which aims to silence gene expression by binding to the RNA produced by the affected gene. This approach is designed to provide patients with access to advanced treatment options for Angelman syndrome, addressing the unmet medical needs associated with this condition.

Solid Biosciences

Post in 2020
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Dimension Therapeutics

Acquisition in 2017
Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.
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