Name
Plenary 2: Overview of Genetic Therapies
Speakers
Joseph Pilewski - University of Pittsburgh Medical Center
Anthony Durmowicz - Cystic Fibrosis Foundation
Anthony Durmowicz - Cystic Fibrosis Foundation


Time
8:45 AM - 9:45 AM (EDT)
Description
The first CF genetic therapy trials in the 1990’s were an exciting milestone for the CF Community. While the outcome of these trials were not what we had hoped, with none of these genetic therapies moving forward to approval, much was learned from these early trials. Additionally, as the genetic therapy field has advanced over the last two decades, we can also look to other rare diseases to learn from their experience and successes. This session will explore the lessons learned from the early CF genetic therapy trials as well as what we can learn from genetic therapy approvals in other diseases like Spinal Muscular Atrophy and apply to our CF trials.