trastuzumab or both together [TSID11781] Technology appraisal guidance UX111 for treating mucopolysaccharidosis type IIIA [ID6540] Technology appraisal guidance Valoctocogene roxaparvovec for treating ...

UX111 for treating mucopolysaccharidosis type IIIA [ID6540] Technology appraisal guidance Valoctocogene roxaparvovec for treating severe haemophilia A [ID3806] Technology appraisal guidance Venetoclax ...

For 2024, the first full-year revenue numbers for hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec) totaled $26 million. In March 2025, Pfizer Inc. said it was terminating development ...

The CHMP backed a conditional marketing authorisation for Roctavian (valoctocogene roxaparvovec or valrox) for severe haemophilia A in adults without inhibitors – antibodies that make standard ...

The US biotech claimed conditional marketing approval from the European Commission for Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe haemophilia A, who don’t have ...

For instance, BioMarin’s US Food and Drug Administration (FDA)-approved haemophilia A gene therapy, Roctavian (valoctocogene roxaparvovec-rvox), launched in 2023 with a $2.9m price point ...

This new service delivers clinical-grade plasmid DNA production from cell bank to batch release in three months. This can help accelerate the development of gene and cell therapies. Located to serve ...

5. Department of Paediatrics and Adolescent Medicine, Clinical and Molecular Haemostasis, Goethe University, University Hospital, Frankfurt, Germany 6. Department of Internal Medicine, Vascular ...

Introduction: Goat-derived adeno-associated virus (AAV) vectors, such as AAV Go.1, represent a novel platform for gene therapy due to their unique origin and potential advantages in transduction ...