The chief of hematology at Children’s Hospital of Philadelphia discussed dual approval of the 2 gene therapies that coincided with ASH’s 2023 conference.
“Both of the agents were approved down to age 12. My hope is that we continue to gather information to determine whether or not one or both of these approaches are safe and effective in children under the age of 12. That remains to be seen, but I think that certainly it will be very important because I think one could make the case that there would likely be benefit, if safe, for children who are younger.
December 8, 2023, saw the simultaneous approval by the FDA of Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel), marketed under the name Casgevy, for the treatment of severe sickle cell disease (SCD), and bluebird bio’s lovotibeglogene autotemcel (lovo-cel), marketed as Lyfgenia, also for SCD.1,2 Notably, these approvals, both of which carried indications for patients aged 12 years and older, occurred in the immediate lead-up to the the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California, at which new research relevant to both of the newly approved gene therapies was presented.
In an interview with CGTLive™ at the ASH 2023 meeting, Alexis Thompson, MD, MPH, the chief of hematology at Children’s Hospital of Philadelphia, gave her thoughts on these landmark approvals. Thompson emphasized the importance of the moment for hematology, and characterized the results seen with both gene therapies during clinical trials as “impressive”. Thompson spoke on the need for continued research on exa-cel and lovo-cel, specifically with regard to determining whether an expanded indication for patients under the age of 12 years could be safe and effective. She also highlighted the need to take measures to enable patients to access these high-cost therapeutics. Thompson also spoke about other research she was excited to see at ASH’s 2023 conference, such as that on investigational gene-editing treatments for SCD like Editas Medicine’s AsCas12a-edited cell therapy EDIT-301 and Beam Therapeutics’ CRISPR base-editing therapy BEAM-101.