CF Foundation Research Overview Genetic Therapies 101

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Genetic therapies hold the potential to treat the underlying cause of the disease for everyone with cystic fibrosis, regardless of mutation. In this webinar, Dr. Deepika Polineni and Dr. Marty Solomon provide an overview of RNA and gene therapy, starting from the basics. • Originally recorded December 14, 2021. • 0:00 | Genetic Therapies 101 • 02:56 | Genetics 101: Understanding the science of the CFTR protein • 18:53 | Status of clinical trials for genetic therapies in cystic fibrosis • 36:08 | When will clinical trials for genetic therapies start? • 37:49 | What are the current barriers to inserting a normal copy of CFTR DNA into lung stem cells? • 40:31 | How close are genetic therapies to being approved for clinical use? • 44:20 | Is there any research happening for people with two rare mutations? • 45:41 | What are the mental consequences of genetic therapies for people with CF? • 48:00 | How far along is the research into whether CRISPR could possibly benefit people with CF? • 49:27 | Could mRNA delivery or gene delivery potentially impact other cells like the liver or pancreas? • 50:39 | Could sweat tests become one of the annual tests to monitor CFTR function? • 51:45 | Some people have not been able to take Trikafta because of the way it affects the liver and kidneys. Are any of these new potential therapies not processed by the liver? • 53:47 | Will the COVID vaccine help with CF mRNA research? • 56:30 | What are you most excited about for CF research in the coming year? • Further reading: • Our research into genetic therapies: https://www.cff.org/research-clinical... • Gene delivery for cystic fibrosis: https://www.cff.org/gene-delivery-cys... • Gene therapy for cystic fibrosis: https://www.cff.org/gene-therapy-cyst... • Gene editing for cystic fibrosis: https://www.cff.org/research-clinical...

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