Cellular and Gene Therapy Program
Cellular and Gene Therapy Program
From answers to action
Changing lives through advanced gene therapies
Gene therapy is creating new possibilities for children with genetic conditions and cancer. At Children’s Mercy, we bring genomic discovery and advanced treatment together to help care teams better understand a child’s condition and identify therapies designed around their individual needs.
What is gene therapy?
Gene therapy is a medical treatment that uses genetic material to correct, replace, silence or modify genes that are causing disease. Gene therapy can be delivered in different ways. In some treatments, a patient's cells are collected, corrected in a laboratory and returned. In others, the therapy is delivered directly to cells in the body. Both approaches aim to help cells follow healthier genetic instructions.
Why does gene therapy matter?
Depending on the condition and treatment, gene therapy may:
- Treat disease at its source.
- Improve quality of life.
- Reduce the need for ongoing therapies.
- In some cases, provide long-lasting or life-changing benefits.
Gene therapy options at Children’s Mercy
We offer gene therapy options for several conditions through approved treatments and clinical trials. Availability and eligibility depend on each child’s diagnosis and individual needs.
Current gene therapies
- Casgevy: Sickle cell disease and beta thalassemia
- Lyfgenia: Sickle cell disease
- Zynteglo: Beta thalassemia
- Itvisma: Spinal muscular atrophy
- Zolgensma: Spinal muscular atrophy
- Elevidys: Duchenne muscular dystrophy
Upcoming gene therapies
- Fayuvi: Sanfilippo syndrome, also called mucopolysaccharidosis type IIIA
- Genglycos: Glycogen storage disease type Ia
- Hemgenix: Hemophilia B
Why choose Children’s Mercy for gene therapy?
Gene therapy is complex, and every child’s needs are different. At Children’s Mercy, specialists, researchers and multidisciplinary care teams work together to help families understand their options and determine an appropriate path forward.
From evaluating eligibility to coordinating treatment and follow-up care, we support children and families through each step with expertise, compassion and a focus on safety.
The Children’s Mercy Genomic Medicine Center is is a world leader in uncovering the genetic causes of complex conditions. By combining those discoveries with advances in gene therapy, we can help identify more personalized treatment options for children. Together, these innovations are creating new possibilities for earlier diagnoses, more targeted therapies and brighter futures for children and families.
In the news
From breakthrough to bedside: collaboration with Basepath Health to expand delivery of life-changing cell and gene therapies
Children’s Mercy recently announced a major expansion of its cell and gene therapy program, reinforcing our commitment to delivering the most advanced, life-changing treatments for children with complex and rare conditions.
Gene therapy and sickle cell disease: Tim's story
After a lifetime of battling the pain of sickle cell disease, Tim is now making history as the first Children’s Mercy patient to receive gene therapy for the disease.
