Advanced Therapies Rare Disease 2020



With over 90% of known rare diseases lacking a targeted treatment or cure, advanced therapies such as cell and gene based, have been proving to be a source of hope to patients suffering from these indications with high unmet medical need.

While recent approvals, and advances in R&D pipelines for these types of therapies have shown their potential, multiple challenges emerged from developing, manufacturing and commercializing cell and gene therapies.

As the largest rare disease conference globally, the World Orphan Drug Congress USA has become the perfect opportunity for a multi-stakeholder discussion and gathering to address how to bring these advanced therapies to patients faster!



Featured Advanced Therapies Speakers Include: