“Probably the most exciting thing I've seen in gene therapy over the last ten years is we now have a lot of tools for selective delivery, which will hopefully make treatments more safe and a lot more successful,” says Dr. Jessica Duis, a geneticist and pediatrician focused on the management of individuals with complex, rare disorders. Dr. Duis, who has worked on several gene therapies that are now approved or progressing through the accelerated approval pathway, is currently VP of Clinical Development at GondolaBio, a clinical-stage biopharmaceutical company focused on developing therapeutics for genetic diseases. 

As you’ll learn in this Year of the Zebra episode with host Lindsey Smith, Dr. Duis is encouraged by other recent advances in genetic technology as well, and thinks momentum will grow as breakthrough treatments emerge. “I think we're hopefully going to continue to see companies that are working in rare disease be more successful and really drive how regulators think about making decisions in terms of bringing treatments to patients. I think we’re at the tip of the iceberg in terms of the future of truly transformational therapies.”  This wide ranging conversation also explores Dr. Duis’ team approach to patient care, her work on clinical endpoints, the importance of patient communities, and her book series, Rare Siblings Stories.

Mentioned in this episode:

GondolaBio

RareDiseaseDoc

Elsevier Healthcare Hub on Rare Diseases

Rare Sibling Stories

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