| Friday, June 23, 2023 |
|
| |
|
| 4:00PM - 6:00PM |
Registration |
| |
|
| 6:00PM - 8:00PM |
Welcome reception and dinner |
| |
|
| Saturday, June 24, 2023 |
|
| |
|
| 7:30AM - 8:45AM |
Breakfast and registration |
| |
|
| 9:00AM - 9:30AM |
Welcome and opening remarks |
| |
We'll kick off the day by letting you know what we've been up to, how we're supporting the LCA and rare IRD community, and how to get the most out of your conference. |
| |
- Courtney Coates, Director of Outreach & Development, Hope in Focus
- Laura Manfre, Chair, Board of Directors, Hope in Focus
|
| |
|
| 9:30AM |
Kids and parents leave for the Children's Museum |
| |
|
| 9:45AM - 10:45AM |
Panel: Advocating for Treatments |
| |
What does it mean when industry, FDA, and others talk about the "voice of the patient"? What role can we, as a patient community, play in advancing treatments? Why is it critically important that those living with the disease are included in the drug development process? This session will answer these questions and more! |
| |
- Jonathan Stokes, Abbvie, Moderator
- Jacose Bell, Spark Therapeutics
- Wiley Chambers, Food and Drug Administration
- Jill Dolgin, Beacon Therapeutics
- Laura Manfre, Hope in Focus
- Emily Place, Mass Eye and Ear
|
| |
|
| 10:45AM - 11:00AM |
Morning coffee break sponsored by Janssen |
| |
|
| 11:00AM - 12:00PM |
Panel: Living with LCA |
| |
Hear first-hand from those living with LCA. This panel will be moderated by a father, his two high school-aged children with vision loss due to LCA, a young professional, one of the first brave persons to go through a clinical trial for gene therapy, and a Teacher of the Visually Impaired (TVI). They will discuss and share their experiences on key topics, and they will include time for you to ask questions. |
| |
- Lee St. Arnaud, Moderator
- Beth Borysewicz, Educational Consultant State of Connecticut Department of Aging and Disability Services with Bureau of Education Services for the Blind
- Mohamed Farid, Founder and CEO, MKF Continuity
- Tami Morehouse, RPE65, volunteered for the first clinical trial for gene therapy
- Patrick St. Arnaud, IQCB1 / NPHP5
- Mirielle St. Arnaud, IQCB1 / NPHP5
|
| |
|
| 12:00PM - 12:45PM |
Lunch |
| |
|
| 12:45PM - 1:15PM |
LCA & Drug Development: Setting the Stage |
| |
Our partners at the Foundation Fighting Blindness will talk about the background of LCA, what makes it unique, and how that affects therapy approaches followed by an overview of the drug development process explaining what it takes to turn research into treatments. |
| |
- Chad Jackson, Foundation Fighting Blindness
- Ben Shaberman, Foundation Fighting Blindness
|
| |
|
| 1:15PM - 2:15PM |
Panel: Gene Agnostic Therapies |
| |
During this session we will learn about therapies in development that can benefit multiple gene mutations and its potential impact for the LCA community. |
| |
- Chad Jackson, Moderator
- Huma Qamar, Ocugen
- Eric Daniels, Kiora Pharmaceuticals
- Ben Shaberman, Foundation Fighting Blindness
|
| |
|
| 2:15PM - 2:30PM |
Break - Time to stretch! |
| |
|
| 2:30PM - 3:30PM |
Panel: Gene Therapies for LCA |
| |
This session will provide an overview of gene therapy, the current progress of gene therapies in the pipeline for LCA, and information about how gene therapies translate to the clinic as an approved treatment. |
| |
- Ben Shaberman, Moderator
- Shannon Boye, Atsena Therapeutics
- Ash Jayagopal, Opus Genetics
- Thomas Mendel, The Ohio State University
- Bikash Pattnaik, University of Wisconsin
|
| |
|
| 3:30PM - 3:45PM |
Bringing it All Together |
| |
|
| 3:45PM - 5:30PM |
Kids return from Children's Museum |
| |
Exhibits and Cookies & Cocktails reception |
| |
|
| 6:00PM - 8:00PM |
Party Like a Local Dinner |
| |
Join us for dinner, music, and fun for all ages, including adaptive games and activities for children. |
| |
|
| Sunday, June 25 |
|
| |
|
| Sunday morning, June 25 — We're planning a trip to the Indianapolis Zoo! Sign-up sheet and information available at registration and the Hope in Focus booth. |
