Deliver The Cure: Challenges in Delivering Genetic Medicines for Cystic Fibrosis
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Background
Cystic fibrosis is a rare, progressive, genetic disease that affects the lungs, pancreas, and other organs. It affects nearly 40,000 people in the U.S. and an estimated 105,000 people have been diagnosed with CF across 94 countries. Approximately 1,000 new cases of CF are diagnosed each year. Cystic fibrosis can affect people of any racial or ethnic background.
Today, there are treatments that correct the dysfunctional CFTR protein for many people with cystic fibrosis. However, some people cannot take these treatments because side effects are too great, or their CFTR mutation does not respond to those treatments.
The Cystic Fibrosis Foundation believes that genetic medicines hold the greatest promise in providing transformative therapies, and ultimately a cure, for people living with cystic fibrosis. CF is a model indication for genetic medicines because the disease stems from mutations in a single gene. Precision delivery of DNA, RNA, and ribonucleoproteins (RNPs) to address CFTR mutations are a top priority for scientists pursuing therapies for cystic fibrosis.
To discuss and stimulate advances in therapeutic delivery, the Cystic Fibrosis Foundation, Bakar Labs, and QB3 are hosting a collaborative symposium focused on innovation in nucleic acid and RNP delivery methods. The symposium is planned in three segments:
- Viral methods for nucleic acid delivery
- Non-viral methods for nucleic acid delivery
- Funding opportunities and flash startup showcase
The event will feature companies and entrepreneurial faculty from the Bakar Labs and UC Berkeley ecosystem. Full agenda is below.
Goal
The goal of the symposium is to forge connections that accelerate the discovery, development, and commercialization of genetic medicines for cystic fibrosis.
Audience
Investors, academic and industry scientists, graduate students, and entrepreneurs are invited to attend the symposium.
Venue
All sessions will be held in the auditorium at Bakar Labs, located at 2625 Durant Ave., Berkeley, CA 94720.
Agenda
Welcome
9:00-9:30 am Check-in and breakfast
9:30-9:40 am Welcome address
9:40-10:30 am Intro to CF and CFTR Protein: Martin Mense & Kara Foshay, Cystic Fibrosis Foundation
10:30-11:05 am Keynote Speaker: Phil Santangelo, Winship Cancer Institute
Session 1: Viral Delivery
11:05-11:10 am Session Overview: Martin Mense, Cystic Fibrosis Foundation
11:10-11:30 am Jennifer Hamilton, Stealth Startup
11:30-11:50 am Melissa Kotterman, Iris Medicine
11:50 am-12:10 pm Trevor Parry, Krystal Biotech
12:10-12:30 pm Clodagh O’Shea, IconOVir Bio
12:30-1:10 pm Panel Discussion moderated by Bill Skach, Cystic Fibrosis Foundation
1:10-1:50pm Lunch
Session 2: Non-Viral Delivery
1:50-1:55 pm Session Overview: Chris Penland, Cystic Fibrosis Foundation
1:55-2:15 pm Derfogail Delcassian, UC Berkeley
2:15-2:35 pm Niren Murthy, UC Berkeley
2:35-2:55 pm Warren Chan, University of Toronto
2:55-3:55 pm Panel Discussion moderated by Bill Skach, Cystic Fibrosis Foundation
3:35-3:55 pm Break
Session 3: Funding Opportunities
3:55-4:15 pm Funding Mechanisms: Carlos Perez-Garcia & John Sheridan, Cystic Fibrosis Foundation
4:15-4:35 Flash Company Showcase
Networking Reception
4:35-5:35 Networking Reception
Organizers
The symposium is being organized by Bakar Labs and QB3 in concert with the media and communications team at the CF Foundation. For questions about the event, please contact Riley Hummel, deputy director of outreach programs at QB3 and Bakar Labs.
Speakers
