The evolution of AAVs in cell and gene therapy
AAV development for cell and gene therapy in 2023 is being impacted by manufacturing and regulation challenges, however advancing technologies offer opportunity, according to leaders in the field.
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AAV development for cell and gene therapy in 2023 is being impacted by manufacturing and regulation challenges, however advancing technologies offer opportunity, according to leaders in the field.
The first approved gene therapy for haemophilia A reduced the mean annualised bleeding rate from 5.4 bleeds to 2.6 bleeds per year in a clinical study.
Medicines which represented significant progress in their therapeutic areas have been listed in the European Medicines Agency's Human Medicines Highlights 2022 report.
Lecanemab for Alzheimer's and the first potential gene therapy primed for US launch for haemophilia A are two potential blockbuster Drugs to Watch™, says a 2023 report.
European approval of BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec) one-time gene therapy represents a breakthrough in the treatment of adults with severe haemophilia A.
Results from several haemophilia trials have been presented at the International Society on Thrombosis and Haemostasis (ISTH) Annual Congress 2022, here EPR summarises some of the key developments.
Roche shares initiative commitments that will extend prophylactic treatment access to patients with haemophilia, as well as investments to provide testing capabilities for HIV and Tuberculosis in developing countries.
Efanesoctocog alfa significantly and meaningfully reduces annualised bleeding rates in haemophilia A patients, according to new Phase III data.
Giroctocogene fitelparvovec had a mean annualised bleeding rate (ABR) of just 1.4 percent over two years, with no bleeding events in the first year post infusion.
The occurrence of non-fatal thrombotic events in Phase III study participants has called into question the safety of concizumab.
New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018.
New research has found that of the drugs in the pre-registration phase, 10 are expected to become blockbusters over the next six years.
The CMA has cleared the anticipated purchase of gene therapy company Spark Therapeutics by pharmaceutical company Roche Holdings.
Hemlibra has been approved to prevent the frequency of bleeding episodes in patients who have developed FVIII inhibitors...