Gene Therapy Center of Excellence (GTCOE)
The Gene Therapy Center of Excellence (GTCOE) at Nationwide Children's works with patients with rare and life-limiting diseases who could be treated with gene therapy.
The center offers a full range of support for patients and families. This includes taking part in clinical trials, working with insurance companies, and delivering approved therapies.
FDA-approved Gene Therapies Currently Available at the Center
- Elevidys® (Duchenne Muscular Dystrophy)
- Zolgensma® (Spinal Muscular Atrophy)
- Roctavian® (Hemophilia A)
- Hemgenix® (Hemophilia B)
- Casgevy® (Sickle Cell and Thalassemia)
- Zynteglo® (Thalassemia)
- Lyfgenia® (Sickle Cell)
Clinical Trials Currently Available at the Center
- Phase I/IIa Intrathecal Gene Delivery Clinical Trial for IGHMBP2-Related Diseases
- An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)
- A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
What Does the Gene Therapy Center of Excellence Offer Physicians and Scientists?
- Delivering FDA-Approved Gene Therapy Products
- The center offers intravenous (IV) gene therapy delivery, adhering to all biosafety and compliance measures to ensure the safe administration of approved therapies to patients.
- The center streamlines processes and creates standardized procedures to address prior authorization needs as well as ordering, receipt and storage of therapies. The result is shorter turnaround times from therapy approval and production to patient delivery.
- Facilitating and Conducting Clinical Studies
- The center is the source for best practices related to investigational gene therapies, including training for clinical researchers.
- Providing Resources for Collaborating With Family Foundations
- Foundations are often created by patient families seeking resources and information about current gene therapy research and open clinical trials. Because physicians and scientists frequently build relationships with these family foundations, the center collaborates with the Nationwide Children's Bespoke/Precision Gene Therapy Feasibility Committee to help guide them through the journey from discovery to clinical study and use of new gene therapies.
Why Choose Us?
The Gene Therapy Center of Excellence is a designated Germain Family Accelerator Program. The Accelerator Program designation provides additional funding to speed the integration of clinical care and research to make the largest impact on child health outcomes.
Nationwide Children's Hospital Is the World's Leader for Gene Therapies
Five-year-old Gideon Griffiths receives Elevidys®, the first FDA-approved gene therapy for Duchenne muscular dystrophy, at Nationwide Children’s Hospital, where the therapy was invented and first tested.