Uploaded September 2024 | Updated September 2026, 7 hours ago
Gene therapy for sickle cell disease is available to you through FDA-approved therapies (lovotibeglogene autotemcel and exagamglogene autotemcel), and through clinical trials. While there are a number of clinical trials for sickle cell gene therapy, the journey can be similar across all treatments.
Nicole Farmer, M.D., Staff Scientist at the NIH Clinical Center, explains that seeking the right therapy is a long process and how it is important to understand what happens at each step. Talk with your healthcare team before, during and after each step.
About the: Sickle cell disease gene therapy education project: genome.gov/research-at-nhgri/Projects/Democratizing-Education
Lovotibeglogene autotemcel Injection: medlineplus.gov/druginfo/meds/a624020.html
Exagamglogene autotemcel: medlineplus.gov/druginfo/meds/a624020.html
Gene Therapy for Sickle Cell Disease through Clinical Trials: clinicaltrials.gov/search?term=%22gene%20therapy%22&cond=Sickle%20Cell%20Disease&country=United%20States&locStr=United%20States&viewType=Table#classicRedirect
Gene therapy for sickle cell disease is available to you through FDA-approved therapies (lovotibeglogene autotemcel and exagamglogene autotemcel), and through clinical trials. While there are a number of clinical trials for sickle cell gene therapy, the journey can be similar across all treatments.
Nicole Farmer, M.D., Staff Scientist at the NIH Clinical Center, explains that seeking the right therapy is a long process and how it is important to understand what happens at each step. Talk with your healthcare team before, during and after each step.
About the: Sickle cell disease gene therapy education project: genome.gov/research-at-nhgri/Projects/Democratizing-Education
Lovotibeglogene autotemcel Injection: medlineplus.gov/druginfo/meds/a624020.html
Exagamglogene autotemcel: medlineplus.gov/druginfo/meds/a624020.html
Gene Therapy for Sickle Cell Disease through Clinical Trials: clinicaltrials.gov/search?term=%22gene%20therapy%22&cond=Sickle%20Cell%20Disease&country=United%20States&locStr=United%20States&viewType=Table#classicRedirect
