ASGCT Co-Hosts Second Congressional Briefing on Sickle Cell Disease

ASGCT Staff - November 26, 2019

ASGCT co-hosted a briefing on Capitol Hill to hightlight how policymakers can support sickle cell disease (SCD) screening as well as treatment development and access.

<p>(From left) Michael C. Burgess, MD (R-TX) (with Matthew Porteus, M.D., Ph.D., and Jennelle Stephenson), and Danny K. Davis (D-IL), gave remarks emphasizing the importance of supporting sickle cell disease research.</p>

Last week ASGCT co-hosted a briefing on Capitol Hill to highlight how policymakers can support sickle cell disease (SCD) screening, treatment development, and access to new treatments. After co-hosting a high-interest briefing with Senate offices in June, ASGCT hosted this briefing in conjunction with House offices—Representatives Barbara Lee (D-CA), Michael C. Burgess, MD (R-TX), and Danny K. Davis (D-IL). Reps. Burgess and Davis gave remarks emphasizing the importance of supporting SCD research.

In addition to the two representatives, ASGCT member Matthew Porteus, M.D., Ph.D., professor of pediatrics at Stanford University, explained the science behind the gene therapy and gene editing approaches that are currently in development for sickle cell disease. Jennelle Stephenson, who received a gene therapy in a clinical trial two years ago, stated in her presentation that she did not realize what life is like for most people—without recurring debilitating pain crises and hospitalizations—until after she received the therapy.

ASGCT consultant Remy Brim, Ph.D., vice president at BGR Group concluded the program with an overview of how policymakers and advocates can help, by supporting: 

  • Research funding through the National Institutes of Health of basic science and SCD-focused initiatives;
  • Novel payment models for gene therapies for SCD upon their approval that would address their unique nature as potentially one-time treatments;
  • The Newborn Screening Saves Lives Reauthorization Act (HR 2507) by advocating to House leadership that reauthorization of newborn screening be included in the next legislative vehicle that carries authorizations or new policies;
  • House Resolution 606, which stresses the importance of awareness of sickle cell trait status and the development and equitable access to new treatments;
  • Dedicated funding for the Centers for Disease Control and Prevention Sickle Cell Data Collection program.

Slides from the briefing are available here. The briefing was co-hosted by the American Society of Hematology, American Society for Transplantation and Cellular Therapy, the Sickle Cell Disease Association of America, and the Pediatric Hospital Sickle Cell Disease Collaborative.





Related Articles


Advocacy Reflects on Accomplishments and Looks Ahead to Future Efforts

ASGCT Staff - January 20, 2020

ASGCT Policy Summit Highlights Patient and Advocate Perspectives

ASGCT Staff - October 14, 2019

Rhoda Baer acquired from National Cancer Institute


Bridging the Gap Between Scientists And Patients In Gene Therapy Development

David Barrett, J.D. - September 27, 2019

Credit: U.S. Mission Photo by Eric Bridiers,


ASGCT Submits Comments to WHO Advisory Committee

ASGCT Staff - August 22, 2019