gene therapy

Gene Therapy for the Masses?
Gene Therapy for the Masses? 1024 683 Katie Brind'Amour, PhD, MS, CHES

Long-lived financial and logistical hurdles make bringing new gene therapy products to market a major challenge. To help bring more of these medical miracles to fruition, experts across industry, regulatory review, science and medicine have begun to problem solve together.  With the approval of the gene therapy Kymriah® (tisagenlecleucel) in 2017, the Food and Drug…

Innovative Gene Therapy Approach for Treating Patients with Tuberous Sclerosis Type 2
Innovative Gene Therapy Approach for Treating Patients with Tuberous Sclerosis Type 2 150 150 Lauren Dembeck

Tuberous sclerosis complex (TSC) is a devastating genetic disease that affects nearly 1 in every 5,500 newborns and approximately 2 million people worldwide. The disease is characterized by the formation of non-malignant tumors throughout multiple organs, including the kidney, lungs, eyes, and heart, but predominantly the brain. It is typically diagnosed in infants and young…

Going Viral: The AAV Approach to Curing Cancer
Going Viral: The AAV Approach to Curing Cancer 1024 256 Emily Siebenmorgen

According to Timothy Cripe, MD, PhD, chief of the Division of Hematology and Oncology at Nationwide Children’s Hospital, it’s an incredible time to be working on cancer treatment – and now, targeted cancer prevention. “There’s so much going on in the cancer world these days,” says Dr. Cripe, who is also a principal investigator in…

FDA Approves Micro-dystrophin: A Small Gene With Big Promise
FDA Approves Micro-dystrophin: A Small Gene With Big Promise 1024 632 Abbie Miller

UPDATE: On June 22, 2023, the FDA announced accelerated approval for SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in the Center for Gene Therapy at Nationwide Children’s Hospital. The FDA approved the treatment for pediatric patients 4-5 years old with DMD. Jerry Mendell, MD, principal investigator in the Center for Gene Therapy…

A Single-Dose Cancer Immunotherapy via Gene Transfer
A Single-Dose Cancer Immunotherapy via Gene Transfer 150 150 Mary Bates, PhD

Researchers at Nationwide Children’s Hospital report proof-of-principle results for a new gene therapy cancer treatment. The off-the-shelf, single-dose immunotherapy serves as an alternative to CAR-T therapy and can be engineered to be on-demand. T cells redirected to cancer cells either via a chimeric antigen receptor (CAR-T) or a bispecific molecule have been game-changers in treating…