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 developed by researchers and scientists at Nationwide Children's for children with Duchenne muscular dystrophy (DMD) who have a genetically confirmed disease causingvariantsin 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 infants and children under age 2 years. 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. With early in life treatment, patients with SMA can achieve motor development and milestones beyond what was ever observed in the natural course of the disease. Treatment with Zolgensmahas led to ventilator free survival, especially for those who have a predicted severe form of disease, and is truly lifesaving and transforming health and development.

Hemgenix (CSL Behring) 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) 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) 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 conditioning, 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 years 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 chemotherapy conditioning. The therapy helps the body produce functional hemoglobin, aiming to reduce painful crises and long-term complications.