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Our ability to treat diseases previously untreatable through the delivery of adeno-associated virus (AAV)-based gene therapies has been a tremendous advancement in pharmaceutical development…
Genome editing is a tool that enables a new generation of medicines by directly targeting with high precision the genetic cause of a disease. It also allows for immune evasion and prevents microenvironment responses: concepts vital to more potent, off the shelf, cell, and gene therapies…
The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) brings together regulatory authorities and representatives from the pharmaceutical industry from across the globe to discuss a range of technical and scientific considerations for medicinal…