Gene Therapies Pave the Way to a Potential Cure for Sickle Cell Disease


Cell and gene therapies (CGT) present unparalleled opportunities to cure diseases with high unmet need.  One such underserved patient population showing great promise from these medical advancements is sickle cell disease (SCD).  While new advancements appear very promising, innovators in the overall gene therapy space also face unprecedented challenges across their commercialization journey – from market access, coverage and reimbursement issues to contracting and real-world evidence requirements that do not follow the norm.
In Xcenda’s most recent CGT white paper, we first review progress specifically in the SCD space. The piece also examines special considerations manufacturers need to make for successful launch of these therapies, whether in SCD or in other therapeutic areas pursuing cures through CGT.  It is imperative that stakeholders across the commercialization continuum work together to ensure that patients awaiting these cures can access them and benefit from their clinical advancement. 

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