Progress in Sickle Cell: Can the Long Biomedical Arc Bend Toward Equity?
Marshall W. Nirenberg Lecture | to
Alexis A. Thompson, M.D., MPH
Chief of the Division of Hematology
Children's Hospital of Philadelphia
Alexis Thompson's career spans bench to beside and reached a historic milestone in 2023, with the approval of the first two gene therapies for sickle cell disease. Now Chief of the Division of Hematology at Children's Hospital of Philadelphia (CHOP), PA, USA, Thompson led clinical research on the lentiviral vector therapy lovotibeglogene autotemcel (lovo-cel) for severe sickle cell disease, approved in the USA for use in adolescents and adults.
Summary
Sickle cell disease has played many substantive roles in advances across biomedical research, yet, until recently, translation of this progress to improvement in patient outcomes have been elusive. This presentation will summarize the accomplishments over time, focusing on newer and potentially transformational therapies, acknowledging continued impediments and exploring opportunities to further make an impact on this condition.
Presentation Objectives
- Review recent scientific advances for sickle cell disease including gene therapy
- Sickle Cell Disease as a study of the complexities of race and science
- Opportunities moving forward
https://videocast.nih.gov/watch=55018
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