AGENDA | FAQ | BACKGROUND | ADDITIONAL INFO
Parent Project Muscular Dystrophy (PPMD) fights to end Duchenne Muscular Dystrophy. We accelerate research, raise our voices to impact policy, demand optimal care for every single family, and strive to ensure access to approved therapies.
I will be attending the annual PPMD Conference this Sunday March 3, 2019 through next Tuesday March 5, 2019. Here is the Agenda…
2019 Advocacy Conference
3 Day Agenda
DAY 1 – SUNDAY, MARCH 3rd
Advocate Training
Mayflower Hotel, 1127 Connecticut Ave, NW, Washington, DC 20036
Room: State Room
2:15pm Registration opens
3:00 – 3:15 Welcome Address & Review of Conference Activities
Pat Furlong, Annie Kennedy, Ryan Fischer
3:15 – 3:35 Congressional Environment – What are we walking into
Nick Manetto, Faegre BD
3:35 – 4:00 Perspective from a former House member
Representative Luke Messer
4:00 – 4:40 Duchenne Advocacy – Making an Impact
Annie Kennedy and Ryan Fischer
4:40 – 4:50 Why Advocacy Matters
Joel Wood
4:50 – 5:30 Advocacy Agenda – This year’s “ask”
Preparing for your meetings
Annie Kennedy & Ryan Fischer
5:30 – 6:00 Soapbox Consulting – Meeting schedules
6:00 – 7:00 Dinner served and break into state groups
DAY 2 – MONDAY, MARCH 4th
Duchenne Gene Therapy Policy Forum
Mayflower Hotel – State Room ¨ 9:00am – 3:30pm
8:15am Registration opens (Breakfast served 8am)
9:00 – 9:10 Welcome and Overview of Forum
Abby Bronson, SVP Research Strategies, PPMD
Session 1 – Where We Are Today
9:10 – 9:40 CBER Update: Agency update on new guidances and where we are since 2016
Celia Witten, MD, PhD, CBER
9:40 – 10:25 Presentations by Companies (15 minutes each)
Pfizer, Sarepta, Solid
Each company will provide update of current gene therapy program
10:25 – 10:40 Q&A from Audience and Discussion with Three Companies
(Moderated by Tim Franson, MD, YourEncore)
Session 2 – AAV in Gene Therapy
10:40 – 11:00 AAV in Duchenne
Lee Sweeney, PhD, Paul Wellstone MD Cooperative Center (UF)
The use AAV in Gene Therapy, what we know from animal models, and work done leading up to human trials.
11:00 – 11:20 Overview of AAV and Use of AAV in Other Diseases
Tim Cripe, MD, Nationwide Children’s Hospital
Overview of what we know in humans affected by other diseases
Session 3 – Gene Therapy Hurdles
11:20 – 11:40 Titers, Testing, and Screening
Barry Byrne, MD, University of Florida
A discussion about prescreening for antibodies and current questions community is raising around screening and titer testing. What are the implications for getting a private test done?
Shouldn’t families know if they are “out of the game” so they can make other choices?
11:40 – 12:20 Getting Around AAV Issues in Gene Therapy and the Potential for Redosing.
(Moderated by Abby Bronson, SVP Research Strategies, PPMD)
Panelists: Tim Cripe, MD, Nationwide Childrens; Joe Kornegay Texas A&M;
Dongshen Duan, PhD, University of Missouri; Olivier Danos, PhD, REGENXBIO
Panel Topics include:
• What are ‘neutralizing antibodies’?
• Those who test positive during screening – what next?
• Plasmaphereses – level setting on where this is in development and if it is a plausible next step strategy in Duchenne gene therapy
• Preparing for screening
• How/can we get to re-dosing?
12:20 – 12:40 Audience Q&A and discussion
(Moderated by Abby Bronson, SVP Research Strategies, PPMD)
12:40 – 1:30 Lunch
Session 4 – The Patient Voice
1:30 – 1:55 Results of PPMD Preference Studies on GT
Ryan Fischer, SVP Community Engagement, PPMD
1:55 – 2:40 Patient Voice Panel – panel of families discussing GT
Moderated by Pat Furlong, Founding President & CEO, PPMD
Session 5 – Looking Toward Future Strategies in Gene Therapy
2:40 – 3:00 What We are Learning About Immunology and the Future for GT
Carrie Miceli, PhD, UCLA
3:00 – 3:15 Audience Q&A and Discussion (Moderated by Abby Bronson, SVP Research Strategies, PPMD)
3:15 – 3:30 Closing Thoughts and Next Steps
Tim Franson, MD, YourEncore
DAY 3 – TUESDAY, MARCH 5th
Advocates head to the hill
No breakfast served on Tuesday am
Advocates head to Capitol Hill for House and Senate Meetings.
Transportation instructions via metro or cab can be found in advocate packets.
All Advocates will be given schedules for meetings via email and on Sunday.
12 Noon – 1pm: PPMD Congressional Hill Briefing
Realizing the Promise of Patient-Focused Drug Development. (Box lunch will be served.)
Where: Room – Senate Visitors Center 202-203. (The Capitol Visitor Center, the main entrance to the U.S. Capitol, is located beneath the East Front plaza of the U.S. Capitol at First Street and East Capitol Street, across from the Supreme Court. Please be in the line for Security by 11:15 AM)
All advocates invited to attend as long as it doesn’t conflict with a Hill meeting.