register

News & Trends - Biotechnology

UniQure’s gene therapy potential in haemophilia B

Health Industry Hub | December 13, 2019 |

uniQure, a leading gene therapy company announced updated clinical data on the three patients treated in uniQure’s ongoing Phase IIb study of etranacogene dezaparvovec, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B.

In addition, uniQure presented up to 4 years of follow-up data on the 10 patients in the Phase I/II trial of AMT-060, its first-generation gene therapy for the treatment of hemophilia B. These clinical data are being presented this weekend in poster presentations at the 61st Annual Meeting of the American Society of Hematology (ASH), taking place in Orlando, Florida.

Key Outcomes

  • Sustained FIX Activity at Therapeutic Levels Up to 50% of Normal at One Year After Administration of Etranacogene Dezaparvovec in Phase IIb Study
  • Zero Bleeds and No Requirement for Immunosuppression in One Year Following Dosing with Etranacogene Dezaparvovec
  • Long-term Clinical Benefit and Tolerability of AMT-060 Maintained in All Patients Through up to 4 Years of Follow-Up

“We are very pleased with these latest results, and continue to believe that etranacogene dezaparvovec has the potential to be the first- and best-in-class gene therapy for patients with hemophilia B,” stated Robert Gut, M.D., Ph.D., chief medical officer of uniQure. “We remain focused on dosing all patients in our ongoing, fully-enrolled HOPE-B pivotal trial, and expect to announce top-line data on our primary endpoint of Factor IX activity by the end of 2020.”

Differentiate your business, build thought leadership and boost network growth by reaching the Australian Pharma, MedTech and Biotech industry professionals. Health Industry Hub offers solutions to vendors and suppliers servicing the industry.

Register FREE to receive the latest news, innovations and insights from Health Industry Hub; the only one-stop-hub connecting Australia’s Pharma, MedTech and Biotech industry professionals and its key stakeholders.


Medical and Science

Government fails diabetes patients with no implementation or funding plan

Government fails diabetes patients with no implementation or funding plan

Health Industry Hub | November 5, 2024 |

Medical & Science: Ahead of World Diabetes Day on 14 November, A/Professor Sof Andrikopoulos, CEO of the Australian Diabetes Society […]

More


News & Trends - Pharmaceuticals

Government under fire as Senator Ruston slams decision to defer access to new medicines

Government under fire as Senator Ruston slams decision to defer access to new medicines

Health Industry Hub | November 5, 2024 |

Pharma News: Revelations that as many as 44 new medicines are facing months-long delays under the Albanese Government have sparked […]

More


News & Trends - Pharmaceuticals

Patients call for greater transparency of Australia's most common osteoporosis therapy

Patients call for greater transparency of Australia’s most common osteoporosis therapy

Health Industry Hub | November 5, 2024 |

Pharma News: Consumers should be fully informed about the potential side effects of Australia’s most common osteoporosis treatment, according to […]

More


Medical and Science

Digital solutions: Bridging pharma gaps to deliver real impact for patients and clinicians

Digital solutions: Bridging pharma gaps to deliver real impact for patients and clinicians

Health Industry Hub | November 4, 2024 |

Medical & Science: In today’s era of rapid technological advancements, the integration of digital solutions in healthcare offers both transformative […]

More


This content is copyright protected. Please subscribe to gain access.