Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Ultragenyx Pharmaceutical (RARE) has completed the rolling submission of its U.S. marketing application for its gene therapy ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College ...

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, used in the investigational gene therapy SRP-9003. This therapy is being ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College of Medicine and the UMass Chan Medical School have achieved ...

Efficient viral vector production is essential for the cost-effective manufacture of cell and gene therapies. Many of the current production workflows are reliant on basal media in batch-mode, which ...

Minaris will move its center for viral vector innovation to occupy space within CGT Catapult's collaborator laboratories in London and will work with CGT Catapult on the further development of ...

Patients with hemophilia B continued to experience low annualized bleeding rates and stable endogenous factor IX expression 5 years after a single infusion of etranacogene dezaparvovec (Hemgenix), the ...

Authors explore complexities in scaling gene-therapy production with special attention is to monitoring and enhancing ...