Workshop Logistics & Agenda Monday, Nov 18 at 7:00 PM - Wednesday, Nov 20 at 5 PM
We are looking forward to seeing you at our upcoming Gene Therapy for A-T workshop! Please find some more detailed information about the workshop here.
Dates
Monday, November 18 at 7 PM - Wednesday, November 20 at 5 PM
Location
Hyatt Regency Boston Harbor (at Boston Logan Airport)
101 Harborside Drive
Boston, Massachusetts 02128, USA
All Meals: Skyline Ballroom on the Second Floor
All Sessions: Harborside Ballroom on the First Floor
Transportation
Shuttle Information – If you're flying into Logan Airport, the Hyatt Regency Boston Harbor offers a free shuttle service. When ready to leave the airport, call the hotel at +1-617-568-1234 to request pickup, and they’ll direct you to the pickup location based on your arrival terminal. Return shuttles to the airport depart the hotel every 20 minutes.
Parking Information – The hotel offers both self-parking and valet options, with parking validation available for both.
Here is the hotel’s transportation page if you need more information:
Co-Chairs
- PJ Brooks, PhD – DRDRI, NCATS, National Institutes of Health
- Brad Margus – A-T Children’s Project
- Peter McKinnon, PhD - St. Jude Children's Research Hospital
- Timothy Yu, MD, PhD - Boston Children's Hospital
Agenda
This meeting will be mostly discussion-based. Prior to the meeting, we will be contacting some of you separately to discuss your participation on a panel. If you are part of a panel discussion, you may be asked to speak for about 5 minutes at the beginning of the session to describe your work as a way to jump start the discussion. No formal slide presentations will be made during the panels, but occasionally, a participant may show just one or two slides to make a point with data.
Note that everyone in the room will be encouraged to contribute to discussions, even if they are not on the panel.
Monday, November 18
- 6:30 pm – Registration
- 7:00 pm – Welcome Dinner
- 9:00pm - ? Lobby Bar
Tuesday, November 19
- 7:30 am – Breakfast
- 8:30 am – Co-Chairs' Welcome and Goals for the Meeting
- 8:35 am - Ataxia-Telangiectasia and the ATM Gene – including clinical presentation and disease course; neuropathology; cellular phenotype, ATM gene and protein product; current landscape of ATM mutations
- 10:15 am – Panel 1 - Potential ATM Gene Replacement Strategies
- 12:00 noon – Lunch
- 1:30 pm – Panel 2 - Potential Gene Editing Strategies
- 3:15 pm – The Atipeksen Story
- 3:45 pm - Panel 3 - Antisense Oligonucleotide Strategies Going Forward
- 6:00 pm – Social Hour (starting right when the last panel ends)
- 7:00 pm – Traditional New England Dinner
- 9:00 pm – ? Late Night Drinks
Wednesday, November 20
- 7:30 am – Breakfast
- 8:30 am - Review of Previous Day’s Highlights and Open Questions
- 9:00 am – Panel 4 - Other Genetic Strategies
- 10:15 am – Tools and Datasets
- 12 noon – Lunch
- 1:30 pm – Tools and Datasets (continued)
- 3:00 pm – Setting Priorities: Where Do We Go From Here
- 5:00 pm - Adjourn
Preparing for the Meeting
If you'd like to do some reading in advance of the workshop, we can suggest the two following articles:
Kim et al. (2023). A framework for individualized splice-switching oligonucleotide therapy. Nature, 619(7971), 828 836. https://pmc.ncbi.nlm.nih.gov/articles/PMC10371869
Rothblum-Oviatt et al. (2016). Ataxia telangiectasia: A review. Orphanet Journal of Rare Diseases, 11(1), Article 159. https://pmc.ncbi.nlm.nih.gov/articles/PMC5123280/
Contact Information
If you have any questions, please reach out to: Jennifer Thornton, Executive Director
Cell +1-561-306-2826 or jennifer@atcp.org.