hemophilia A

Takeda broadens gene therapy scope, turns to Poseida for non-viral approach

The progress of gene therapies continues to be stymied by safety risks associated with the viruses used to deliver them. Takeda Pharmaceutical is partnering with Poseida Therapeutics in a bet that the biotech’s non-viral technologies could offer a safer alternative.

Roche to Present New data of Hemlibra (emicizumab) in P-IIIb STASEY Study for Hemophilia A at ISTH 2021

Shots: The company will present data from the final analysis of P-IIIb STASEY study of Hemlibra (emicizumab) reinforcing the safety & efficacy of Hemlibra in people with hemophilia A with factor VIII inhibitors Spark will share data from the P-I/II study of SPK-8011, demonstrating that hepatocyte expression of factor VIII can be stable and durable …

Roche to Present New data of Hemlibra (emicizumab) in P-IIIb STASEY Study for Hemophilia A at ISTH 2021 Read More »

BioMarin Reports Results of Valoctocogene Roxaparvovec in P-III GENEr8-1 Study for Hemophilia A

Shots: The ongoing P-III GENEr8-1 study involves assessing of valoctocogene roxaparvovec (6e13 vg/kg dose) in 134 patients with with severe hemophilia A. The patients received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up The study met 1EPs & 2EPs i.e. reduction in ABR by 84% demonstrating superiority to factor …

BioMarin Reports Results of Valoctocogene Roxaparvovec in P-III GENEr8-1 Study for Hemophilia A Read More »

FDA rejects BioMarin’s hemophilia A gene therapy in ‘massive surprise’

In a CRL issued for valoctocogene roxaparvovec, the FDA told BioMarin that differences between its Phase I/II and Phase III studies limited the ability to rely on the earlier trial to support durability of effect, which an analyst said likely referred to a product comparability issue.