Community Lunch & Learns

Recently Approved Therapies

February 23, 2023 | 12:00 PM CT

This session will go in depth about some of the gene therapy approvals in 2022 including Skysona (for CALD), Zynteglo (for beta thalassemia), and Hemgenix (for hemophilia B). Attendees will hear from doctors and researchers about the pathway to these therapies, and the outlook on access for those within the various patient populations. 


Moderator: Rachel Bailey, PhD, assistant professor, UT Southwestern Medical Center  


12-12:05 p.m. 

Recently Approved Therapies  

12:05-12:45 p.m. 


  • Amy Waldman, MD, medical director of the Leukodystrophy Center and pediatric neurologist at Children’s Hospital of Philadelphia 

  • Alexis Thompson, MD, MPH, chief of the division of hematology at Children's Hospital of Philadelphia 


12:45-1 p.m.  

Thank You to Our Sponsors

Learn about ways to support this program


Register for the ASGCT Policy Summit

September 23-24, 2024 | Washington, D.C.

This site uses cookies to offer you a better user experience and to analyze site traffic. By continuing to use this website, you accept our use of cookies.