pharmaphorum

FEBRUARY 22, 2021

That therapy uses an adeno-associated virus (AAV) as a vector rather than a lentivirus, a vector type that features in approved gene therapies such as Roche’s Luxturna and Novartis’ Zolgensma. The case comes just weeks after UniQure halted a trial of its haemophilia B gene therapy AMT-061 after an unexpected case of liver cancer.

Immunization 52

Immunization 52

Pharmaceutical Technology

JUNE 21, 2023

The high price tag may be hard to defend , but with lifetime treatment costs for haemophilia B reaching as high as $23m for some, the single-dose treatment could prove far more cost-effective. Despite the limited market, the price tags of haemophilia make it potentially lucrative. Haemophilia B is a rare condition.

FDA 98

FDA 98

pharmaphorum

AUGUST 26, 2022

Roctavian delivers a working copy of the Factor VIII gene into cells via an adeno-associated virus (AAV) vector, and offers a one-shot, potentially curative therapy for haemophilia A. million spread over five years.

FDA 111

FDA 111