Gene therapy is a way to treat a disease by changing gene expression. That can be accomplished by correcting the mutation in the gene, providing a replacement copy of the mutated gene, introducing a surrogate (substitute) gene that produces a protein that can perform a similar role to the non-functional protein, or supplying genes that treat other symptoms of the disease. Gene therapy holds incredible promise in Duchenne and this panel will feature companies exploring various gene therapy approaches to treat Duchenne.
Gene Therapy Introduction by Tim Cripe, MD, PhD, FAAP (Nationwide Children’s Hospital)
Sarepta (Louise Rodino-Klapac, PhD, Senior Vice President, Gene Therapy)
Pfizer (Beth Belluscio, MD-PhD, Global Clinical Lead)
Audentes (Fulvio Mavilio, PhD, Senior Vice President, Translational Science)
Solid (Joel Schneider, Ph.D., Chief Technology Officer)
Followed by discussion and Q & A