In a first-of-its kind conference, the Cystic Fibrosis Foundation invited more than 100 scientists to Savannah, Ga., from June 26-29 to discuss how recent advancements in the treatment of
The main goals of the New Technologies Advancing Toward a One-time Cure conference were to encourage scientists -- from graduate students to senior-level researchers in
This was the first time that researchers and companies in these non-CF disciplines were collectively invited to participate in the annual conference and marks a commitment to developing new technologies that could repair or replace the defective CF gene.
"We are now sitting at the precipice of a tremendous opportunity in genetic technology," said William R. Skach, M.D., senior vice president for research affairs at the CF Foundation. "We need to convince researchers to focus on unlocking this potential for cystic fibrosis."
Katherine High, a pioneer in the field of gene therapy, opened the conference by providing insight into the process of developing and evaluating gene therapy, a
As the Foundation begins to explore these new therapeutic options for CF, designing ways to test their effectiveness in people is just one of the challenges that scientists at the conference identified. Researchers also need to know which cells to target, how to reach those cells efficiently and decide whether to repair or replace the
"The CF Foundation will be inviting smaller groups of researchers to work on finding solutions to problems identified during the meeting," said Katherine Tuggle, director of research for the CF Foundation. She said the Foundation will also use knowledge gained at the conference to decide where to devote future resources.
Dr. Skach said that the conference reminded him of those in the late 1990s when researchers gathered to talk about the challenges and opportunities of developing CFTR modulators. Those discussions led to the development of ivacaftor (Kalydeco®) and lumacaftor/ivacaftor (Orkambi®).