Texas Children’s Researchers Outline Path to Broader Sickle Cell Gene Therapy Access

A new commentary in The Lancet Haematology by Alexander I. Ngwube (top right) and Cecile L. Karsenty (top left) outlines a path for moving sickle cell gene therapy from scientific breakthrough to equitable patient access. 

Their message is clear: these therapies can transform lives, but realizing their potential will require system-wide changes that better equip clinicians, coordinate care and support patients and families from referral through long-term follow-up.

Dr. Ngwube and Karsenty’s commentary builds on the team’s national survey in Blood Immunology & Cellular Therapy. Among 138 pediatric hematology-oncology physicians surveyed, 97.1% were likely to discuss gene therapy when a family expressed interest. However, referral practices, counseling confidence and prereferral testing varied, highlighting gaps that could affect how consistently patients and families learn about and access these therapies.

To address these gaps, the authors call for coordinated referral pathways, focused clinician education, shared-care networks and robust long-term surveillance. Implementing these recommendations could help more children and adolescents with sickle cell disease benefit from gene therapy through more consistent, coordinated and equitable care, regardless of where they receive it.

This national research effort brought together experts from Texas Children’s, Baylor College of Medicine, Children’s National Hospital and University of Rochester Medical Center/Golisano Children’s Hospital. Additional survey authors included Drs. Anil George, Khaled Yassine, Duc T. Nguyen, Titilope A. Fasipe, Helen E. Heslop, Venée N. Tubman, Allistair Abraham and John T. Horan. The work received internal research funding from Texas Children’s Hospital.

Read the commentary:
https://doi.org/10.1016/S2352-3026(26)00216-4 

Read the national survey:
https://doi.org/10.1016/j.bict.2026.100074