Pariglasgene brecaparvovec is a Gene Therapy owned by Ultragenyx Pharmaceutical, and is involved in 3 clinical trials, of which 1 was completed, and 2 are ongoing.
DTX-401 activates the G6pase enzyme in the body. The inherited gene mutation resulting in altered glycogen metabolism and glucose homeostasis ultimately leads to the glycogen storage disease. The therapy restores the production of the glucose-6-phosphatase enzyme. The absence of G6pase activity in liver cells leads to the inability to convert glycogen to glucose leading to a deficiency in normal metabolic function and hypoglycemia. The therapy enhances the activity of G6pase and suppress the disease severity.
The revenue for Pariglasgene brecaparvovec is expected to reach a total of $1.3bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Pariglasgene brecaparvovec NPV Report.
Pariglasgene brecaparvovec Overview
DTX-401 is under development for the treatment of glycogen storage disease type 1a. It is administered through intravenous route as infusion. The therapeutic comprises of adeno-associated virus vector (AAV) type 8 encoding G6PC gene. It is developed based on NAV vector technology.
Ultragenyx Pharmaceutical Overview
Ultragenyx Pharmaceutical (Ultragenyx) develops novel medicines for rare and ultra-rare genetic diseases. It has three marketed products, Mepsevii (vestronidase alfa) for the treatment of Mucopolysaccharidosis (Sly syndrome); Dojolvi (triheptanoin), a highly purified, synthetic, 7-carbon fatty acid triglyceride for long-chain fatty acid oxidation disorders (LC-FAOD) and Crysvita (burosumab), a fully human monoclonal antibody for the treatment of X-linked hypophosphatemia (XLH). Its pipeline includes DTX401 for the treatment of glycogen storage disease type Ia, or GSDIa; UX701 for Wilson disease; UX053 for glycogen storage disease type III; and GTX-102 for Angelman syndrome in partnership with GeneTx. It works in partnership with various academic institutes and biotechnology companies to advance its product candidates. Ultragenyx is headquartered in Novato, California, the US.
The company reported revenues of (US Dollars) US$351.4 million for the fiscal year ended December 2021 (FY2021), an increase of 29.7% over FY2020. The operating loss of the company was US$381.7 million in FY2021, compared to an operating loss of US$330.1 million in FY2020. The net loss of the company was US$454 million in FY2021, compared to a net loss of US$186.6 million in FY2020. The company reported revenues of US$90.7 million for the third quarter ended September 2022, an increase of 1.5% over the previous quarter.
Quick View – Pariglasgene brecaparvovec
|Highest Development Stage|