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Colleen Kelly: Bridging the Access Gap in Gene Therapy for Sickle Cell Disease
Colleen Kelly/ LinkedIn
Jul 27, 2026, 08:10

Colleen Kelly: Bridging the Access Gap in Gene Therapy for Sickle Cell Disease

Colleen Kelly, Attending Physician and Pediatric Hematologist/Oncologist at Dana-Farber Cancer Institute and Boston Children’s Hospital, shared a post on LinkedIn about a recent article she and her colleagues co-authored, published in Pediatric Blood and Cancer, adding:

“Our new paper is out in Pediatric Blood and Cancer.

Gene therapy can be a transformative treatment for sickle cell disease. But its resource- and time-intensive nature may limit who can actually access it.

In our study, 60% of families with a child with sickle cell disease faced material hardship — food, housing, utility, or transportation insecurity — in the last year.

Only 40% of parents felt they could access gene therapy with their current resources. And families facing material hardship were far less likely to report being able to access this treatment, with 95% lower odds after adjustment.

A transformative therapy is only effective if families can reach it. As these life-changing but resource-intensive therapies expand, access must be part of the design.”

Title: Out of Reach: Understanding Resource Barriers to Gene Therapy for Pediatric Patients With Sickle Cell Disease

Authors: Colleen A. Kelly, Morgan A. Paul, McKenzie Griffin, Sunyu Kang, Rahela Aziz-Bose, Leanne Duhaney, Callie Fry, Puja J. Umaretiya, Kristine Karvonen, Erica Esrick, Sharl Azar, Natasha M. Archer, Kira Bona

Colleen Kelly: Bridging the Access Gap in Gene Therapy for Sickle Cell Disease

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Callie Fry Colleen A. Kelly Erica Esrick Health Hematology Hemostasis Hemostasis News Hemostasis Today Kira Bona Kristine Karvonen Leanne Duhaney McKenzie Griffin Medicine Morgan A. Paul Natasha M. Archer Pediatric Blood and Cancer Puja J. Umaretiya Rahela Aziz-Bose Sharl Azar Sickle Cell Disease Sunyu Kang
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