On a quest to bring new a new hemophilia gene therapy to the clinic, one research team leapt over hurdle after hurdle to produce enough viral therapeutics to treat a large number of patients.
Gene therapy promises the possibility of replacing damaged and faulty disease-causing genes with functional copies, essentially rescuing phenotypes and restoring individuals to health. At the heart of this technology are viral vectors, little vessels that leverage the evolutionary power of viruses to penetrate and shuttle genetic information into cells.
Manipulating viruses to carry genetically-modified gene copies and then producing large quantities of these viral carriers for gene therapy is not easy. Before any gene therapy can make it to the clinic, its feasibility and success largely hinges on critical early steps in viral vector optimization and production. In this episode, we will explore the hurdles that researchers face in viral vector development and how one team overcame these challenges to produce a groundbreaking gene therapy for hemophilia.
Bryan Piras, PhD
Therapeutics Production and Quality
St. Jude Children’s Research Hospital
Drug Discovery News Talks Science is a podcast where we discuss the latest news in preclinical and translational research. Behind every medical and scientific advancement lies a harrowing story of mystery and discovery. Come with us as we share these stories and connect you to the scientific minds behind them.
This podcast episode is sponsored by Mirus Bio. Mirus Bio revolutionized transfection more than 25 years ago with the introduction of its flagship product, TransIT®-LT1. Pioneering new delivery solutions, such as TransIT-X2®, Mirus is a critical supplier of novel transfection reagents. With the introduction of TransIT-VirusGEN®, Mirus expanded expertise to virus manufacturing, supporting researchers from discovery to clinic.