Gene therapies are in the process of redefining the treatment of rare diseases. However, a set of complex and unique challenges must be overcome for these therapeutics to achieve their commercial potential.
The 3rd annual Gene Therapy for Rare Disorders Europe takes place from October 15th to 17th in London and is dedicated solely to tackling late-stage regulatory, reimbursement, clinical and manufacturing challenges. It is your comprehensive guide to gene therapy commercialization!
Download the official event guide to learn what the 20+ expert speakers will discuss, including:
- How the National Institute for Clinical Excellence (NICE) assesses gene therapies, including insights into case histories to gain useful knowledge into their decision making processes on health technologies, policies, and clinical guidelines
- A discussion by the European Medicines Agency (EMA) on how and when to most effectively engage with regulators to get a clearer understanding of the gene therapy regulatory landscape in Europe
- How to handle strategic manufacturing with Vivet Therapeutics, including internal facility building, partnering with CMOs and others to overcome manufacturing capacity bottlenecks with creative solutions.
- Insights into the potential of lentiviral vectors with Orchard Therapeutics, to understand that there’s more to gene therapy than AAV.
- How to speed up gene therapy development with Sangamo Therapeutics, by incorporating patient perspective into study designs and key documentation, to enable maximal efficiency while keeping patients at the center of gene therapy development
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