1. Scrip
  2. >>Therapy Areas
  3. >>Blood and Clotting

UniQure Has High HOPE For Hemophilia B Gene Therapy

 
• By Kevin Grogan

The Dutch company's update on AMT-061 and the initiation of a pivotal trial called HOPE-B of around 50 patients could put the product in pole position to be the first gene therapy on the market for the severe orphan blood clotting disorder.

Racing cars
Uniqure in pole position for hemophilia gene therapy? • Source: Shutterstock

uniQure NV has presented promising updated results from a Phase IIb trial of its gene therapy AMT-061 for hemophilia B which the Netherlands-headquartered firm believes could be best in class and first to market ahead of Spark Therapeutics Inc. and Pfizer Inc.'s rival candidate.

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Blood and Clotting

Sanofi Set To Shake Up Crowded Hemophilia Space After Qfitlia Approval

 
• By Kevin Grogan

The small interference RNA therapeutic can be used by patients regardless of inhibitor status.

With Phase III Success, PharmaEssentia To Seek Besremi Label Expansion (Update)

 
• By Joseph Haas

Taiwan-based PharmaEssentia hopes to add essential thrombocytopenia to the label for its pegylated interferon product, already approved to treat polycythemia vera.

Regeneron Will Move Two Factor XI Agents Into Phase III

 
• By Joseph Haas

With successful Phase II data versus active comparators for both REGN7508 and REGN9933, Regeneron hopes to position the candidates to compete against existing factor X products.

ASH: Pfizer’s Hemophilia A Gene Therapy May Have Slight Liver Safety Advantage

 
• By Mandy Jackson

Hematologists’ concerns about hepatoxicity with hemophilia A gene therapies may limit use, but Phase III data for Pfizer’s candidate at ASH show a lower rate of ALT increases than seen with BioMarin’s Roctavian.

More from Therapy Areas

In Brief: BriaCell’s Breast Cancer Cell Therapy Boosts Survival In Phase II

 
• By Sushmita Panda

BriaCell announced its cell therapy Bria-IMT plus checkpoint inhibitors bested Gilead’s Trodelvy in a Phase II trial in HR+ breast cancer; a Phase III trial is enrolling.

Telix Moves TLX101 Forward On Encouraging Early Brain Cancer Data

 
• By Alaric DeArment

The company is planning to start a registrational program on its home turf in Australia and is planning to bring the therapy to the US as well.

Leqembi Launch Set For Germany And Austria As EU Approval Finally Comes

 
• By Neena Brizmohun

Eisai is working on securing reimbursement across the EU for Leqembi now that the Alzheimer’s disease treatment has secured marketing approval from the European Commission.