uniQure Announces License Agreement with CSL Behring to Commercialize Hemophilia B Gene Therapy - Seite 3

uniQure plans to continue to leverage its leading gene therapy platform, including the Company’s deep expertise with AAV5, to develop potentially best-in-class gene therapies. AAV5-based gene therapies have been demonstrated to be safe and well tolerated in a multitude of clinical trials, including uniQure trials conducted in hemophilia B and other indications. No patient treated in clinical trials with uniQure’s AAV5 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 viable treatments in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment.  uniQure also may seek to in-license or acquire additional product candidates that align with its research and development strategy.  

In addition, uniQure plans to further strengthen its proprietary gene therapy platform by expanding its manufacturing capacity to support a broad pipeline, including product candidates for diseases with larger prevalence, as well as investing further in new technologies to improve the efficacy, safety and applicability of its gene therapies to patients.

As part of uniQure’s effort to focus on those gene therapy programs that have the greatest potential to improve patients’ lives and generate long-term value for shareholders, uniQure plans to de-prioritize its research program of AMT-180 for patients with hemophilia A.    

Moelis & Company acted as a financial advisor to uniQure in this transaction.

Conference Call Today at 5:30 p.m. EDT

uniQure will host a conference call today, June 24, 2020, at 5:30 p.m. Eastern Daylight Time. The conference call may be accessed by dialing [phone] for domestic callers and [phone] for international callers. The passcode for the call is 9499239. Please specify to the operator that you would like to join the "uniQure Conference Call." The conference call will be webcast live under the investor relations section of uniQure’s website at www.uniQure.com and will be archived there following the call for 90 days.