Akshat Jain: The First CRISPR-Cas9 Gene Therapy Approved for Children as Young as 2 with SCD
Akshat Jain, Hematology Pediatric Workforce Task Force at American Society of Hematology, shared a post on LinkedIn:
“Exciting milestone in pediatric hematology!
The U.S. FDA has granted a supplemental approval for Casgevy (exagamglogene autotemcel) — the first CRISPR-Cas9 gene therapy now available for children as young as 2 years old with sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β-thalassemia (TDT).
Previously approved for patients aged 12 years and older, this expansion brings hope to younger patients and their families by enabling earlier intervention.
Clinical evidence from younger patients (extrapolated to patients aged 2 years and older) is comforting to justify the known and unknown cost of this platform for gene editing.
Earlier access can help reduce long-term organ damage, support better growth and development, and improve quality of life for children facing these burdensome conditions.
As a pediatric hematologist and advocate for rare blood disorders, I celebrate this label extension while emphasizing the need for equitable global access, especially in high-burden regions. Continued education, research, and support for patients remain essential.
Congratulations to Vertex Pharmaceuticals , the dedicated researchers, trial participants, and FDA teams for making this possible!”
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