{"id":5201,"date":"2023-05-04T18:56:22","date_gmt":"2023-05-04T18:56:22","guid":{"rendered":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/?page_id=5201"},"modified":"2024-06-19T21:08:39","modified_gmt":"2024-06-19T21:08:39","slug":"agenda","status":"publish","type":"page","link":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/agenda\/","title":{"rendered":"Agenda"},"content":{"rendered":"

Agenda
<\/span><\/b><\/b>June 20 (Day 1)<\/b><\/p>\n

View\/download meeting agenda as PDF<\/span><\/span><\/a><\/p>\n<\/div>

All times are in EDT.<\/b><\/p>\n<\/div><\/div><\/div><\/div><\/div>

\n
\n\n<\/thead>\n\n\n\n\n
9:00 AM – 9:15 AM<\/b><\/td>\nIntroduction and Welcome
\n<\/b>Walter Koroshetz, MD<\/em><\/strong>
\nDirector, NIH\/NINDS<\/em><\/strong><\/td>\n<\/tr>\n
9:15 AM – 9:25 AM<\/b><\/td>\nSetting the Stage – Goals of the Workshop
\n<\/b>Vicky Whittemore, PhD<\/em><\/strong>
\nNIH\/NINDS<\/em><\/strong><\/td>\n<\/tr>\n
9:25 AM – 9:50 AM<\/b><\/td>\nAction Potential: An Effort to Develop Genetic Strategies for a Rare Neurological Disease
\n<\/b>Simon Frost<\/em><\/strong>
\nTiber Capital Group<\/em><\/strong><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<\/div>\n<\/div><\/div><\/div><\/div>


\nSession I: State-of-the-art in Genetic Strategies for Treatment<\/span><\/span>
\n<\/span><\/b><\/u>Moderator: Dan Lowenstein, MD; University of California, San Francisco<\/span><\/strong><\/p>\n<\/div><\/div><\/div><\/div><\/div>

\n
\n\n\n\n\n\n\n\n\n\n\n
9:50 AM – 10:00 AM<\/b><\/td>\nIntroduction to Session
<\/b>Dan Lowenstein, MD<\/em><\/td>\n<\/tr>\n
10:00 AM – 10:25 AM<\/b><\/td>\nDeveloping Circuit Based Gene Therapies
<\/b>Beverly Davidson, PhD<\/em>
University of Pennsylvania<\/em><\/td>\n<\/tr>\n
10:25 AM – 10:50 AM<\/b><\/td>\nA Clinically Editable Brain Is Within Sight: Track Record and Trajectory of CRISPR For Neurologic Disease
<\/b>Fyodor Urnov, PhD<\/em>
University of California, Berkeley<\/em><\/td>\n<\/tr>\n
10:50 AM – 11:10 AM<\/b><\/td>\nBreak<\/b><\/td>\n<\/tr>\n
11:10 AM – 11:35 AM<\/b><\/td>\nPrenatal Therapies for Neurologic Diseases<\/b>
Tippi Mackenzie, MD<\/em>
University of California, San Francisco (via Zoom)<\/em><\/td>\n<\/tr>\n
11:35 AM – 12:00 PM<\/b><\/td>\nPanel Discussion<\/b><\/td>\n<\/tr>\n
12:00 PM – 1:00 PM<\/b><\/td>\nLunch<\/b><\/td>\n<\/tr>\n
1:00 PM – 2:00 PM<\/b><\/td>\nPoster Session for Young\/Early Career Investigators<\/b><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<\/div>\n<\/div><\/div><\/div><\/div>


\nSession II: Progress, Pitfalls and Lessons Learned from Neurological and Non-Neurological Trials\/Treatments<\/span>
\n<\/b><\/u>Moderator: Ann Poduri, MD, MPH; Boston Children\u2019s Hospital<\/span><\/span><\/strong><\/p>\n<\/div><\/div><\/div><\/div><\/div>

\n
\n\n\n\n\n\n\n\n\n\n\n\n
2:00 PM – 2:10 PM<\/b><\/td>\nIntroduction to Session
\n<\/b>Ann Poduri, MD, MPH<\/em><\/td>\n<\/tr>\n
2:10 PM – 2:35 PM<\/b><\/td>\nGene Therapy as a Platform: From Giant Axonal Neuropathy to the PaVeGT Project and the Bespoke Gene Therapy Consortium<\/b>
\nCarsten Bonnemann, MD<\/em>
\nNIH\/NINDS<\/em><\/td>\n<\/tr>\n
2:35 PM – 3:00 PM<\/b><\/td>\nUse of ASOs for Genetic Correction of Angelman Syndrome
\n<\/b>Elizabeth Berry Kravis, MD, PhD<\/em>
\nRush University<\/em><\/td>\n<\/tr>\n
3:00 PM – 3:25 PM<\/b><\/td>\nRisks and Treatment Choices for Neurological Diseases<\/b>
\nFlorian Eichler, MD<\/em>
\nMassachusetts General Hospital<\/em><\/td>\n<\/tr>\n
3:25 PM – 3:45 PM<\/b><\/td>\nBreak<\/b><\/td>\n<\/tr>\n
3:45 PM – 4:10 PM<\/b><\/td>\nNew Paths to the Waterfall: Lessons Learned from AAV Product Development<\/b>
\nKathy High, MD<\/em>
\nRockefeller University<\/em><\/td>\n<\/tr>\n
4:10 PM – 4:35 PM<\/b><\/td>\nFrom N of 1 to Population Health to Treat Genetic Epilepsies
\n<\/b>Wendy Chung, MD, PhD<\/em>
\nBoston Children\u2019s Hospital (via Zoom)<\/em><\/td>\n<\/tr>\n
4:35 PM – 5:00 PM<\/b><\/td>\nPanel Discussion<\/b><\/td>\n<\/tr>\n
5:00 PM<\/b><\/td>\nAdjourn<\/b><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<\/div>\n<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div><\/div>

June 21 (DAY 2)<\/b><\/span><\/p>\n<\/div>

All times are in EDT.<\/span><\/b><\/p>\n

Session III: Considerations for Designing and Implementing Therapy for the Epilepsies<\/span>
\n<\/b><\/u>Moderators: Lori Isom, PhD; University of Michigan & Heather Mefford, MD, PhD; St. Jude\u2019s Children\u2019s Hospital<\/span><\/strong><\/p>\n<\/div><\/div><\/div><\/div><\/div>

\n
\n\n\n\n\n\n\n\n\n
9:00 AM – 9:10 AM<\/b><\/td>\nIntroduction to Session
<\/b>Lori Isom, PhD & Heather Mefford, MD, PhD<\/em><\/td>\n<\/tr>\n
9:10 AM – 9:35 AM<\/b><\/td>\nPredicting Pathogenicity \u2013 Which Variants (Will) Cause Disease? Considering Mechanism \u2013 Approaches for Gain vs Loss of Function
<\/b>Gemma Carvill, PhD<\/em>
<\/b>Northwestern University (via Zoom)<\/em><\/td>\n<\/tr>\n
9:35 AM – 10:00 AM<\/b><\/td>\nTANGO as a Therapeutic Strategy for Epilepsy Design & Implementation: Successes and Challenges
<\/b>Isabel Aznarez, PhD<\/em>
Stoke Therapeutics<\/em><\/td>\n<\/tr>\n
10:00 AM – 10:25 AM<\/b><\/td>\nPinpointing the Perfect Timing: Critical Time Windows for Gene Therapy Delivery
<\/b>Gaia Colasante, PhD<\/em>
San Raffaele Hospital<\/em><\/td>\n<\/tr>\n
10:25 AM – 10:45 AM<\/b><\/td>\nPanel Discussion<\/b><\/td>\n<\/tr>\n
10:45 AM – 11:00 AM<\/b><\/td>\nBreak<\/b><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<\/div>\n<\/div><\/div><\/div><\/div>


\nSession IV: Crucial Issues Beyond the Genetic Strategy<\/span>
\n<\/b><\/u>Moderators: Vicky Whittemore, PhD; NIH\/NINDS & George Umanah, PhD; NIH\/NINDS<\/span><\/strong><\/p>\n<\/div><\/div><\/div><\/div><\/div>

\n
\n\n\n\n\n\n\n\n\n\n\n\n\n
11:00 AM – 11:10 AM<\/b><\/td>\nIntroduction to Session
<\/b>Vicky Whittemore, PhD<\/em><\/td>\n<\/tr>\n
11:10 AM – 11:35 AM<\/b><\/td>\nEnhancing Global Rare Epilepsy Care Through Online Knowledge Resources
<\/b>Dennis Lal, PhD<\/em>
University of Texas – Houston<\/em><\/td>\n<\/tr>\n
11:35 AM – 12:05 PM<\/b><\/td>\nBecoming a Treatable Genetic Condition<\/b>
Mary Ann Meskis<\/em>
Dravet Syndrome Foundation (via Zoom)<\/em><\/td>\n<\/tr>\n
12:05 PM – 1:00 PM<\/b><\/td>\nLunch<\/b><\/td>\n<\/tr>\n
1:00 PM – 1:25 PM<\/b><\/td>\nInformed and Efficient Clinical Trials Design Using Natural History Data and Disease Modeling
<\/b>Melanie Quintana, PhD<\/em>
Berry Consultants<\/em><\/td>\n<\/tr>\n
1:25 PM – 1:50 PM<\/b><\/td>\nConsiderations for Remote Phenotyping in Pediatric Research
<\/b>Heather Hazlett, PhD<\/em>
University of North Carolina <\/em><\/td>\n<\/tr>\n
1:50 PM – 2:15 PM<\/b><\/td>\nEthical Issues Raised in the Development of Genetic Strategies to Treat Rare Neurological Disorders<\/b>
Lainie Ross, MD, PhD<\/em>
University of Rochester<\/em><\/td>\n<\/tr>\n
2:15 PM – 2:35 PM<\/b><\/td>\nPanel Discussion with Discussant
<\/b>Rosa Sherafat, MD
FDA<\/em><\/td>\n<\/tr>\n
2:35 PM – 3:00 PM<\/b><\/td>\nWrap-up Discussion
<\/b>Moderator: Dan Lowenstein, MD<\/em><\/td>\n<\/tr>\n
3:00 PM<\/b><\/td>\nAdjourn<\/b><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<\/div>\n<\/div><\/div><\/div><\/div><\/p>\n","protected":false},"excerpt":{"rendered":"","protected":false},"author":6,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"footnotes":""},"class_list":["post-5201","page","type-page","status-publish","hentry"],"aioseo_notices":[],"_links":{"self":[{"href":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/wp-json\/wp\/v2\/pages\/5201","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/wp-json\/wp\/v2\/users\/6"}],"replies":[{"embeddable":true,"href":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/wp-json\/wp\/v2\/comments?post=5201"}],"version-history":[{"count":93,"href":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/wp-json\/wp\/v2\/pages\/5201\/revisions"}],"predecessor-version":[{"id":5731,"href":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/wp-json\/wp\/v2\/pages\/5201\/revisions\/5731"}],"wp:attachment":[{"href":"https:\/\/event.roseliassociates.com\/ninds-genetic-strategies\/wp-json\/wp\/v2\/media?parent=5201"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}