New Frameworks for Agile Innovation of Gene Editing Therapies

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Gene editing technologies have advanced dramatically over the past decade, offering curative potential for heritable diseases once deemed untreatable. Their precision and versatility are reshaping therapeutic possibilities across a wide range of genetic conditions. This session convenes government leaders alongside industry and academic experts to craft a forward-looking regulatory and reimbursement framework that can keep pace with these agile innovations. Speakers will discuss the areas where balanced flexibility is most needed—from IND applications to regulatory reviews to unpredictable payer landscapes—and show how curative innovations can move faster and cost less while adding value to society. Panelists will also explore the pace of technological change in gene editing and its implications for patient access.

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    Speakers

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    Rebecca Ahrens-Nicklas

    Director, Gene Therapy for Inherited Metabolic Disorders Frontier Program, The Children's Hospital of Philadelphia
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    Rosa Canet-Aviles

    Chief Science Officer, California Institute for Regenerative Medicine
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    Gilmore O'Neill

    CEO, Editas Medicine
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    Andrew Rosen

    CEO, National Ataxia Foundation

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