Gene therapies are a promising area of research to treat cystic fibrosis, but the concepts can be overwhelming. During ResearchCon 2024, Daniel Rorgan, MD, Mike Harnish, PhD, Elyse Jupiter, and Kathryn Singh explore types of genetic therapies, dispel common misconceptions, and disucss efficacy in other diseases.
Originally recorded April 30, 2024.
0:00:00 | Landscape of Genetic Therapies for Cystic Fibrosis
0:25:00 | Introductions
0:01:18 | Elyse shared her son, Tobias’s, story
0:04:12 | Introduction to genetic therapies in CF
0:04:30 | The basics: What is the CF gene?
0:04:50 | The flow of genetic information
0:09:55 | Access to modulator therapy is racially inequitable
0:11:35 | Why is research involving genetic therapies unique?
0:13:30 | Mike Harnish’s story
0:14:16 | The ‘normal’ CF process
0:15:03 | What genetic methods can we use to treat CF?
0:17:02 | mRNA therapies: one-time instructions for cells
0:19:05 | Gene delivery: New, proper instructions
0:20:08 | Gene editing: editing what’s already there
0:21:04 | Where are these therapies already in use?
0:22:00 | Would genetic therapies impact fertility?
0:24:45 | Common concerns about genetic-based therapies
0:26:00 | Who monitors for safety?
0:30:54 | What is done to protect safety?
0:33:30 | Drug development pipeline — genetic therapies
0:34:22 | What side effects have occurred so far?
0:35:18 | What can we learn from research for other medical conditions?
0:35:52 | Why are genetic-based therapies sometime packaged in a virus?
0:37:30 | Will genetic-based therapies impact fertility or children of people with CF?
0:38:02 | What other unique challenges are there for genetic-based therapies?
0:39:34 | What will genetic-based therapies cost? Will everyone have access to them?
0:42:13 | How do we navigate our CF journey?
0:47:20 | What are some key concepts patients should understand before deciding about gene therapy?
0:51:04 | Can you talk more about the SpliceSense therapy as it seems the furthest along?
0:53:23 | What have we learned from the research and approval of gene therapy for sickle cell disease?
0:55:20 | Is anything known about gene therapy and the impact on mental health?
0:57:20 | What are your thoughts on a timeline for a cure?
0:59:17 | Would any genetic therapies be applicable for lung transplant recipients?
1:00:32 | Is there any research to repair the reproductive system of men with CF?
1:01:58 | How is CF genetic therapy research supporting research for other genetic conditions?
1:03:42 | What research is underway for people with very rare mutations?
1:05:02 | Is there any research into whether CF carriers could benefit from genetic therapies?
1:06:10 | Would genetic therapies work for people with advanced lung disease?
