Scientists Discuss Challenges to Repair or Replace Genes at CF Research Conference

The Cystic Fibrosis Foundation invited more than 100 scientists to its research conference in Savannah, Ga., last month to discuss recent advancements in gene therapy, gene editing and stem-cell biology and how these new technologies could be applied to finding a one-time cure for cystic fibrosis.

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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 genetic diseases could lead to a one-time cure for cystic fibrosis.

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 gene therapy, gene editing and stem-cell biology -- to focus their research efforts on CF and to build collaborations that will be essential for developing future therapies. This conference is part of the Foundation's overall research strategy to support basic scientific research into the disease and to develop new therapies.

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 therapeutic approach that inserts normal genes into cells to correct genetic disorders. High is the president and chief scientific officer of Spark Therapeutics in Philadelphia, a company that is conducting clinical trials for multiple gene therapy programs.

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 gene.

"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®).

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