~ Achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit; Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the...
Genezen, a gene therapy CDMO, has agreed to acquire uniQure's commercial gene therapy operations in Lexington, MA, bringing a strategic manufacturing facility and an experienced team of employees to the company.
UniQure, with sale of plant, outsources Hemgenix manufacturing
~ Proposed divestiture streamlines uniQure’s focus and represents an important milestone in its ongoing effort to significantly reduce operating expenses ~ ~ Genezen to manufacture global...
~ Designation based on 24-month interim Phase I/II clinical data for AMT-130 announced in December 2023 ~ ~ AMT-130 is the first therapeutic candidate to receive RMAT Designation for Huntington’s...
uniQure announces first quarter 2024 results
The US Food and Drug Administration (FDA) is proposing the creation of two lists of drugs or drug categories that are difficult to compound under sections 503A and 503B of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as well as criteria for inclusion on the lists.
~ Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington™s disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory...
With its $3.5 million price tag, CSL Behring and uniQure’s one-off hemophilia B gene therapy Hemgenix is one of the world’s most expensive therapies. On Friday, three-year follow-up from its registrational study offers a new look at the drug’s curative potential.
Uniqure Huntington`s gene therapy data `on the favorable side`