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Congressional Briefing on Progress in Sickle Cell Disease Treatment & Policy Implications

June 18, 2019 @ 2:30 pm - 3:30 pm UTC

TUESDAY, JUNE 18, 2019 | 2:30 – 3:30 PM
RSVP: Betsy Foss-Campbell, bfoss@asgct.org

Join us for updates on Gene therapy approaches presented by prominent scientists in the field
How policymakers can support the development of treatment options for sickle cell disease


Tim Scott, United States Senator

Francesca Cook, MPH, Government Relations Committee member, ASGCT; Senior Director, Pricing and Market Access, REGENXBIO

Julie Kanter, MD, Director of Adult Sickle Cell Program; Associate Professor of Hematology and Oncology, University of Alabama at Birmingham

Punam Malik, MD, Professor of Pediatrics, Marjorie Johnson Chair of Gene and Cell Therapy, Director, Cincinnati Comprehensive Sickle Cell Center; Cancer and Blood Disease Institute, Cincinnati Children’s Hospital

Rodrick Murray, Patient advocate providing a personal story on gene therapy’s value for treating sickle cell disease

Matthew Porteus, MD, PhD, Professor of Pediatrics (Stem Cell Transplantation), Stanford University

David Williams, MD, Senior Vice President and Chief Scientific Officer, Boston Children’s Hospital; Chief, Hematology/Oncology, Boston Children’s Hospital; President, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center; Leland Fikes Professor of Pediatrics, Harvard Medical School

How can policymakers help support the development of gene therapies for sickle cell disease?

  • Support the need for robust NIH research funding
  • Support appropriations to the sickle cell disease prevention and treatment program and implementation of new surveillance and screening authorities passed in 2018
  • Enable novel payment models for approved gene therapies

Hosted in partnership with the Pediatric Hospital Sickle Cell Disease Collaborative and


June 18, 2019
2:30 pm - 3:30 pm
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