CSL Behring Agrees to Acquire Novel Late-Stage Gene Therapy Candidate for Hemophilia B Patients from uniQure
KING of PRUSSIA, Pa. ? Unique gene therapy has the potential to be one of the first to market treatments to provide potentially long-term benefits with only one dose ? CSL Behring builds on legacy of delivering innovative treatment options for people with Hemophilia B
According to CSL Behring?s Executive Vice President, Head of Research and Development and Chief Medical Officer Bill Mezzanotte, ?We are exceedingly encouraged by the data we?ve seen on AMT-061. Not only has the treatment option demonstrated robust clinically meaningful responses in FIX activity, but it has also exhibited excellent safety over multiple years of observation.?Expanding our gene therapy portfolio to treat hemophilia B, a disease state well known to CSL Behring, exemplifies how we are strategically aligning our rare and serious disease focus and our targeted therapeutic area focus with our core scientific platforms to transform the lives of patients.?
This acquisition will also enhance CSL Behring?s capabilities in its growing gene therapy portfolio. The company is currently developing a stem cell gene therapy (CSL200) for the treatment of sickle cell disease and has recently established an alliance with Seattle Children?s Research Institute to develop a stem cell gene therapy for primary immunodeficiency diseases -- another rare disease area where CSL Behring has leading capabilities.
Perreault added, ?Upon approval, this next-generation therapy will be highly complementary to our existing best-in-class hemophilia B product portfolio with an?alternate best-in-class treatment option. With the license to AMT-061, we are building on our legacy of delivering lifesaving innovations in hematology where, today, we offer a market leading product for hemophilia B and we are a leader in therapies for treating hemophilia A, von Willebrand disease, thrombosis, and other life-threatening?conditions.?
CSL Behring has put patients first by addressing the world?s most serious, complicated and rare diseases for over 100 years. The company is now bringing that same commitment to gene therapy; its mission is to address unmet patient needs and enable patients to get the very most out of life. About Etranacogene Dezaparvovec (AMT-061) Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with uniQure?s AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates. About CSL Behring CSL Behring?is a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients? needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, respiratory disease, and neurological disorders. The company?s products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn. CSL Behring operates one of the world?s largest plasma collection networks, CSL Plasma. The parent company,?CSL Limited?(ASX:CSL;USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 26,000 people,?and?delivers its life-saving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit Vita?CSLBehring.com/vita?and follow us on?Twitter.com/CSLBehring.