Gene Therapy
Center of Excellence
Gene Therapy
Nationwide Children’s Hospital is home to one of the most advanced cell and gene therapy programs of its kind. Our expertise spans the full spectrum of discovery, development and delivery, offering life-changing solutions to children with some of the world’s rarest and most complex diseases.
The Gene Therapy Center of Excellence is comprised of physician leaders, researchers, dedicated clinical staff, pharmacy experts and administrative expertise. Our multidisciplinary approach to each patient treated through the Center of Excellence is intended to provide a comprehensive patient and family experience throughout the entire gene therapy journey.
From groundbreaking clinical trials to FDA-approved gene therapies, our teams have provided successful outcomes for patients with genetic, neuromuscular, immunologic, hematologic and oncologic conditions. Through deep collaboration between clinicians, researchers and pharmaceutical partners, we’re not only delivering curative therapies today, but we’re also accelerating the future of treatment for children everywhere.
Kevin M. Flanigan
Kevin M. Flanigan, MD, is an attending neurologist at Nationwide Children's and director of the Jerry R. Mendell Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children's Hospital.
Diana X Bharucha-Goebel
Diana Bharucha-Goebel, MD, is Medical Director of the Gene Therapy Center of Excellence and principal investigator in the Jerry R. Mendell Center for Gene Therapy.
The Jerry R. Mendell Center for Gene Therapy aims to investigate and employ the use of gene and cell-based therapeutics for prevention and treatment of human diseases. Nationwide Children's has over 25 open and enrolling trials for cell and gene therapies. See the links below for the latest studies and eligibility.
The Gene Therapy Center of Excellence has dosed over 100 therapies since it’s inception. We are proud to have been the leading institution to discover both Elevidys and Zolgensma. Nationwide Children’s Hospital Gene Therapy Center of Excellence is currently a treatment center for the following therapies:
- Casgevy (Vertex Pharmaceuticals & CRISPR Therapeutics) is a groundbreaking CRISPR-based gene-editing therapy approved for individuals with sickle cell disease or transfusion-dependent beta-thalassemia. It works by editing the patient’s own stem cells to produce healthy red blood cells. The therapy involves collecting stem cells, modifying them in a lab and re-infusing them after the patient undergoes conditioning chemotherapy.
- Elevidys (Sarepta Therapeutics) was delveloped by researchers and scientists at Nationwide Children's for children with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. The therapy delivers a shortened version of the dystrophin gene to muscle cells via a single intravenous (IV) infusion, aiming to help slow disease progression and improve muscle function.
- Zolgensma (Novartis Gene Therapies) is a one-time gene therapy developed by researchers and scientists at Nationwide Children's to treat spinal muscular atrophy (SMA) in children under age 2. It works by replacing the missing or nonfunctioning SMN1 gene with a functional copy, delivered through a single IV infusion. This early intervention helps preserve motor function and prevent progression of the disease.
- Hemgenix (CSL Behring) Hemgenix is a gene therapy for adults with hemophilia B that aims to reduce or eliminate the need for regular factor IX infusions. It uses an adeno-associated virus (AAV) vector to deliver a working gene to the liver, allowing the body to produce its own clotting factor. Administered as a single intravenous infusion, Hemgenix offers long-term potential to manage bleeding episodes.
- Kymriah (Novartis) Kymriah is a CAR T-cell therapy designed for children and young adults with certain types of relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). It involves collecting a patient’s T cells, genetically modifying them to attack cancer cells, and then reinfusing them. This highly personalized immunotherapy can provide a new option when other treatments have failed.
- Roctavian (BioMarin Pharmaceutical) Roctavian is a gene therapy approved for adults with severe hemophilia A who do not have antibodies against the therapy’s viral vector. Delivered through a single IV infusion, it introduces a functioning gene that enables the body to produce factor VIII, significantly reducing bleeding episodes and the need for routine infusions.
- Zynteglo (Bluebird Bio) is a gene therapy for people with transfusion-dependent beta-thalassemia (TDT). It works by inserting a functional copy of the beta-globin gene into the patient’s stem cells, allowing them to produce healthy hemoglobin. After chemotherapy, the modified cells are infused back into the patient to reduce or eliminate the need for chronic blood transfusions.
- Lyfgenia (Bluebird Bio) is a gene therapy for sickle cell disease in patients ages 12 and older with a history of vaso-occlusive events. It uses a lentiviral vector to insert a modified gene into the patient’s stem cells, which are then returned to the body after conditioning. The therapy helps the body produce functional hemoglobin, aiming to reduce painful crises and long-term complications.
A Dedicated Team
At Nationwide Children’s Hospital, gene and cell therapy isn’t an add-on service; it is a specialty focus just like cancer, neuromuscular or hematology. Families come here because of our team whose entire focus is just successfully delivering gene and cell therapy, from the very first appointment through long-term follow-up. That means your child is supported by specialists who dedicate their careers to this field, providing the depth of experience and attention you won’t find everywhere else.
For questions please call our team at (614) 938-1107
