From De-risking to Value Creation – The Evolving Role of Patient Communities in Rare Disease Drug Development and Commercialization

As patient communities and advocacy leaders become more deeply embedded in drug development, especially in rare diseases, it is imperative that organizations understand how patient engagement drives value beyond de-risking. Join Global Genes to hear insights on how patients are changing the landscape for the development of novel therapeutics and how researchers, drug developers and funders can better leverage patient-centric approaches to optimize value throughout development. Delve into how patient advocacy engagement and in drug development can act as a driver of investor value and learn how patient advocates are becoming market makers through their work in catalyzing scientific breakthroughs, data collection and sharing, reshaping clinical research, promoting access and building patient networks.

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EBD Group Inc., 3150 Pio Pico Drive, Suite 201, Carlsbad, CA, 92008

RARE Beyond the Square Partner Plus:

RARE Beyond the Square Partners:

Sara Jane Demy - CEO and Founder, Demy-Colton
Dave Greenwald – VP, Deerfield Management
Rich Horgan – Founder, Cure Rare Disease
Vijay Sappani – Founder and CEO, Ela Capital, CDG Canada
Walt Kowtoniuk – Partner, Third Rock

From de-risking to value creation

Leveraging platform approaches to gene therapies to accelerate therapeutic development for monogenic diseases

Allyson BerentV – CSO and COO, GeneTx Biotherapeutics
P.J. Brooks – Program Director, NIH
Nicole Guadelli – Director and Head of Gene Editing Technologies, Beam Therapeutics
Peter Marks – Director of Center for Biologics Evaluation and Research, U.S. Food and Drug Administration
Nick Leschly – Chief Bluebird, bluebird bio

Advancing patient-centered value frameworks for novel therapeutics in rare disease

Craig Martin – CEO, Global Genes
Diane Berry – Senior VP, Sarepta Therapeutics
Donna Cryer – President and CEO, Global Liver Institute
Jeremy Levin– CEO, Ovid Therapeutics