The field of gene therapy is young and rapidly evolving, with new information discovered every day and an ever-increasing number of novel therapies entering various phases of clinical trials. For over 15 years, Nationwide Children’s Hospital in Columbus, Ohio, has performed as a research leader in the gene therapy field, and Andelyn Biosciences was born of this expertise in 2020. As gene therapy continues to grow, scientists must continue to learn from the rich, albeit recent, history and consider new insights from early developmental research through commercial production of a gene therapy product. There are high-level points and potential challenges to consider, including early development and selection of a viral vector, vector design, strategies for upstream and downstream process and platform development, in-process characterization of a product, and scalability, among others. This overview will cover these topics through the process of bringing a new product to the clinic.
1. Explain what is gene therapy is.
2. Explain what steps are involved for getting a gene therapy product to the clinic.
3. Discuss development and manufacturing processes, analytics, and potential challenges.