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Hematologists Consider Where New Therapies Fit In Sickle Cell Treatment Paradigm

Potential Cures May Not Be Available To All

 
• By Mandy Jackson

The first gene editing medicine and gene therapy for sickle cell disease were approved as the ASH meeting got under way, but novel oral drugs may be key to making treatment more accessible.

Normal red blood cell and sickle cell flow inside in artery
Hematologists considered use of new sickle cell treatments at ASH • Source: Shutterstock

The US Food and Drug Administration approvals of Vertex Pharmaceuticals Incorporated/CCRISPR Therapeutics AG’s gene editing medicine Casgevy (exagamglogene-autotemcel, exa-cel) and bluebird bio inc.’s gene therapy Lyfgenia (lovotibeglogene autotemcel, lovo-cel) gave doctors two new options to consider for patients with sickle cell disease (SCD) and a lot to discuss about how these novel treatments will be used going forward during the recent American Society of Hematology (ASH) annual meeting.

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