uniQure Presents New Preclinical Data in Hemophilia A

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.

uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, presented new preclinical data on its gene therapy candidates AMT-150 for Spinocerebellar Ataxia type 3, AMT-190 for Fabry disease, and AMT-180 for the treatment of hemophilia A. The data are featured in presentations at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. uniQure is delivering a total of 22 data presentations at the meeting, which is taking place virtually from May 12 to May 15.
“Our collective presence at ASGCT showcases the breadth of uniQure’s gene therapy expertise and leadership, including our research capabilities and excellence in developing and manufacturing novel gene therapies,” stated Matt Kapusta, chief executive officer at uniQure. “We are happy to share new preclinical data on our gene therapy candidates, as well as our innovations in technology and manufacturing.”
The uniQure presentations in Spinocerebellar Ataxia type 3 (SCA3) show a continuation of strong proof-of-concept data in mice and other preclinical models, as well as encouraging new data in non-human primates (NHPs). Additionally, new data related to AAV biology show that a single administration of AAV5-hFIX in newborn mice led to stable hFIX expression up to 18 months after dosing.
AMT-180 for Hemophilia A
Hemophilia A is an X-linked bleeding disorder resulting from a deficiency in coagulation Factor VIII that serves as a cofactor for Factor IX in the activation of the coagulation cascade. About 30 percent of the hemophilia A patient population develops inhibitors to Factor VIII over the course of the disease. AMT-180 comprises a recombinant AAV5 vector incorporating a proprietary modified Factor IX gene.
Data from multiple in vivo studies in rodents and NHPs demonstrated that a single administration of AMT-180 was well-tolerated and without increased coagulation activation markers. The preclinical studies were used to predict suitable clinical doses by assessing Factor VIII-independent clotting activity and correlating this to FIX-FIAV protein levels. The studies demonstrated that the dose calculation model in NHPs predicted dose-dependent increases in FVIII-independent activity.
 
Read the press release in its entirety here.