National Alliance of Sickle Cell Disease Consensus Recommendations for Gene Therapy Implementation

Gene therapy for sickle cell disease (SCD) is rapidly advancing, with approved products and others in development, creating an urgent need to define how treatment centers can safely and effectively deliver this care. The National Alliance of Sickle Cell Centers (NASCC) conducted a modified Delphi consensus process involving 64 SCD centers across the United States with expertise in SCD care to identify essential elements required for gene therapy delivery and longitudinal patient management. Participants evaluated proposed elements of care delivery and center infrastructure, with consensus defined a priori as ³70% agreement on categorization. Twenty-four elements were identified as essential, and four as recommended for optimal implementation. These elements span clinical infrastructure, multidisciplinary care, treatment delivery, and longitudinal follow-up. The resulting framework provides a practical foundation to guide centers in developing and standardizing gene therapy programs for SCD. These consensus recommendations support safe, consistent, and high-quality care as access to gene therapy expands and may inform future clinical practice, program development, and research in this evolving field.