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CF Foundation | Research Overview: Genetic Therapies in Cystic Fibrosis

Genetic therapies, including RNA therapy and gene therapy, could potentially work for any person with cystic fibrosis, including people with nonsense and rare mutations. During our webinar, CF Foundation staff Kara Foshay, PhD, senior director of genetic therapy programs, and Katie Howe, MS, CCRC, clinical trial navigator, share an overview of genetic therapies, starting from the basics. Please excuse that the Spanish interpretation at the beginning of the video. It is resolved within the first two minutes. Originally recorded February 21, 2024. 0:00:00 | Research Update: Genetic Therapies for Cystic Fibrosis 03:01 | How do mutations in the CFTR gene cause cystic fibrosis? 05:47 | Why do only certain CFTR mutations respond to CFTR modulators? 08:09 | How are genetic therapies different from CFTR modulators? 10:45 | What is the timeline and process for developing a genetic therapy? 12:30 | How would gene editing work for CF? 15:43 | How would gene therapy, also called gene replacement, work for CF? 18:30 | How would RNA therapy work for CF? 21:10 | What will a clinical trial for a CF genetic therapy look like? 27:51 | What resources are available to help find clinical trial opportunities? 30:15 | What are the phases of clinical trials when developing a new drug? 32:05 | What are the logistics of participating in a genetic therapy clinical trial, and how is participants’ safety protected? 35:45 | What are the CF community’s key needs and challenges around genetic therapies? 36:31 | How can I get involved in genetic therapy research? 38:32 | What is in the pipeline for those who can’t take CFTR modulators? 39:43 | How will genetic therapies impact people who are post-transplant? 40:26 | What is a realistic timeframe for genetic therapies to be available to people with CF? 41:11 | Will clinical trials for genetic therapies accept participants with lower lung function? 42:02 | Why are trials for gene editing in CF still so many years away? 43:14 | What is the status of clinical trials of ASO therapy for splice mutations? 44:28 | Are researchers using artificial intelligence to shorten the drug development timeline? 45:17 | How effective would RNA or gene therapy be compared to Trikafta? 46:32 | When would genetic therapies be available for compassionate use? 47:15 | Can you summarize the available results from phase 1 trials of gene therapy and RNA therapy in CF? 48:13 | Would some participants receive a placebo in a genetic therapy trial? 49:07 | How many people with CF are currently ineligible for CFTR modulators? 49:54 | What mutations are eligible for the currently enrolling genetic therapy trials? 50:40 | What are the lessons learned from previous attempts at gene therapy in CF? 52:02 | What are the most important concepts for people with CF to understand when discussing genetic therapies? 52:54 | Are there any known side effects of genetic therapies? 53:39 | How is Prime Medicine’s research different from other genetic therapy programs in CF? 55:11 | Could a Priority Review Voucher through the FDA help speed up approval of genetic therapies? 55:43 | Could genetic therapies be effective for symptomatic holders of a single CFTR mutation? 56:33 | How hopeful are you about finding a cure for CF in your lifetime? Additional resources: Clinical Trial Finder | https://apps.cff.org/trials/finder Research Into Genetic Therapies | https://www.cff.org/research-clinical... Patient Safety in CF Clinical Trials | https://www.cff.org/research-clinical... Join Community Voice | https://www.cff.org/get-involved/comm... Explorando tratamientos para mutaciones raras y sin sentido: https://www.cff.org/es/explorando-tra...