Pfizer: begins human trials of hemophilia A gene therapy
(CercleFinance.com) - Pfizer and Sangamo said on Wednesday that a first participant has been dosed in a phase 3 trial on their investigational gene therapy for haemophilia A patients.
The study will evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult (aged 18-64 years) male participants with moderately severe to severe haemophilia A.
The primary endpoint is impact on annual bleed rate (ABR) through 12 months following treatment, with participants to be analysed throughout a five-year study period following the infusion.
Under the terms of the collaboration agreement, Pfizer's partner Sangamo - a genomic medicines firm - has now earned a 30 million dollar milestone payment.
For the record, Pfizer is now operationally and financially responsible for research, development, manufacturing and commercialisation activities for giroctocogene fitelparvovec.
Copyright (c) 2020 CercleFinance.com. All rights reserved.
The study will evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult (aged 18-64 years) male participants with moderately severe to severe haemophilia A.
The primary endpoint is impact on annual bleed rate (ABR) through 12 months following treatment, with participants to be analysed throughout a five-year study period following the infusion.
Under the terms of the collaboration agreement, Pfizer's partner Sangamo - a genomic medicines firm - has now earned a 30 million dollar milestone payment.
For the record, Pfizer is now operationally and financially responsible for research, development, manufacturing and commercialisation activities for giroctocogene fitelparvovec.
Copyright (c) 2020 CercleFinance.com. All rights reserved.