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.

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

10:30-11:00 am Keynote Speaker: Phil Santangelo, Winship Cancer Institute:

Session 1: Viral Delivery

11:05-11:10 am Session Overview: Martin Mense, Cystic Fibrosis Foundation

11:10-11:25 am Jennifer Hamilton, Stealth Startup: Cell Type-Programmable Delivery to Enable Genome Engineering in Vivo

11:30-11:45 am Melissa Kotterman, Iris Medicine: Capsid Engineering to Improve Lung Delivery & Development of a Gene Therapy for CF

11:50 am-12:05 pm Trevor Parry, Krystal Biotech: KB407 Developmental Overview

12:10-12:25 pm Clodagh O’Shea, IconOVir Bio:

12:30-1:05 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

1:55-2:10 pm Derfogail Delcassian, UC Berkeley

2:15-2:30 pm Niren Murthy, UC Berkeley: Acid Degradable Lipid Nanoparticles Enhance the Delivery of mRNA in Vivo in Multiple Organs.

2:35-2:50 pm Warren Chan, University of Toronto: Molecular Strategies to Control Nanoparticle Delivery in the Body

2:55-3:30 pm Panel Discussion moderated by Bill Skach, Cystic Fibrosis Foundation

3:35-3:55 pm Break

 

Session 3: Funding Opportunities

3:55-4:10 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