159 - Patient Pioneers in Gene Therapy with AVROBIO

<p>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.</p> <p>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.</p> <p>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!</p> <p style="font-weight: 400;">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. </p> <p style="font-weight: 400;">For additional information, visit <a href="http://www.avrobio.com/" target="_blank" rel= "noopener" data-saferedirecturl= "https://www.google.com/url?q=http://www.avrobio.com&source=gmail&ust=1632781690597000&usg=AFQjCNHAaSRjFFt7a1e_LyVuoJCVZd8Nmw">www.avrobio.com</a> </p>