The Cystic Fibrosis Foundation hosted more than 100 researchers from various fields at its “New Technologies Advancing Toward a One-time Cure” conference last month to debate how new research and treatments for genetic diseases across the board could lead to a cure for cystic fibrosis (CF), an inherited condition in which the lungs and digestive system can become clogged with thick, sticky mucus.
For the first time, graduate students and expert scientists in the fields of stem-cell biology, gene editing and gene therapy, were invited to participate. The move marked the foundation’s commitment to developing new technologies that could repair or replace the defective CF gene – the CFTR gene.
“We are now sitting at the precipice of a tremendous opportunity in genetic technology,” said Dr. William R. Skach, senior vice president for research affairs at the CF Foundation in a feature story posted on the foundation’s website. “We need to convince researchers to focus on unlocking this potential for cystic fibrosis.”
Opening the conference, Katherine High, Director of the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, provided insights into the process of developing and evaluating gene therapy, a therapeutic approach that inserts normal genes into cells to correct genetic (inherited) disorders.
High is a world renowned hematologist who studies the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders. Her pioneering studies of gene therapy for hemophilia have led to a focus on clinical translation of genetic therapies for hemophilia and other inherited disorders like CF.
Scientists at the conference highlighted the need for designing gene therapy trials to test the effectiveness in patients. Scientists need to know which cells to target, how to efficiently target them, and then decide whether to repair or replace the defective gene.
Katherine Tuggle, director of research for the CF Foundation, said the foundation will use information gathered at the conference to decide how to spend its resources.
“The CF Foundation will be inviting smaller groups of researchers to work on finding solutions to problems identified during the meeting,” Tuggle said.