159 - Patient Pioneers in Gene Therapy with AVROBIO

Thanks to the AVROBIO team for a great day of patient advocacy. We feel so honored to be a part of these conferences and always take so much away from them.  And a huge thank you to panelists Jordan, Darren, and Kim. Gene Therapy treatments have the potential to make a profound impact in rare disease. However, these potentially permanent therapies present unique considerations for any participant in clinical trials. When we were presented with the opportunity to moderate a panel of people who either participated in gene therapy studies or had extensive knowledge to share on this topic, we jumped on it! AVROBIO’s vision is to bring personalized gene therapy to the world. They aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle, and bone. AVROBIO’s ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease.  For additional information, visit www.avrobio.com