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Tuesday, June 18 • 10:00am - 11:00am
#220: Targeting Shared Molecular Etiologies to Accelerate Drug Development for Rare Diseases

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Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-571-L04-P; CME 1.00; RN 1.00

Many rare diseases share the same underlying molecular etiology that are therapeutically targetable. Basket trial design based on molecular etiology enables more patients to gain access to clinical trials and accelerate identification of new therpies

Learning Objectives

Recognize the potential of basket trial design in rare disease; Apply basket trial design in rare diseases with share molecular etiologies.

Chair

David Andrew Pearce, PhD

Speaker

Shared Platforms Approaches/Technologies for Rare Diseases
Philip (P.J.) Brooks, PhD

Targeting Shared Molecular Etiologies to Accelerate Drug Development for Rare Diseases
Christina Waters, PhD, MBA


Speakers
avatar for David Pearce

David Pearce

President of Innovation and Research/Chair International Rare Disease Research, Sanford Health, United States
David Pearce is President of Innovation & Research for Sanford Health. He completed his undergraduate Bachelor of Science Degree with honors in biological sciences at Wolverhampton Polytechnic in 1986. He gained his PhD in 1990 at the University of Bath, UK Dr. Pearce has been researching... Read More →
avatar for Philip (P.J.) Brooks

Philip (P.J.) Brooks

Deputy Director, Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences (NCATS), NIH, United States
Philip J. (P.J.) Brooks is the deputy director of NCATS’ Division of Rare Diseases Research Innovation. Brooks represents NCATS in the NIH-wide Gene Therapy Working Group, the Regenerative Medicine Innovation Project and the International Rare Diseases Research Consortium (IRDiRC... Read More →
CW

Christina Waters

Chief Executive Officer and Founder, RARE Science, Inc., United States
Dr. Christina Waters has been responsible for leading medical research in a broad range of organizations ranging from academia and disease – focused non-profits to biotech and large pharmaceutical companies. Her broad range of experience in basic, translational and clinical research... Read More →

Tuesday June 18, 2024 10:00am - 11:00am PDT
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  02: ClinTrials -ClinOps, Forum