**This workshop has ended - Recording now available!**
Workshop Agenda
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10:00 am ET
David McMullen, MD; National Institute of Mental Health
John Ngai, PhD; NIH BRAIN Initiative
10:10 am ET
SESSION 1: CIRCUIT TOOLS CASE STUDIES
Session 1 will highlight various existing approaches to manipulating circuits in humans. Clinical case studies of genetic, device, and optogenetic-based tools currently used to treat disorders of vision will be explored. How lessons learned from these approaches can inform circuit manipulation tool development for other indications and circuits will be the focus of the following panel discussion.
10:10 am ET
Deniz Dalkara, PhD; Institut de la Vision
10:30 am ET
Bob Greenberg, MD, PhD; Alfred E. Mann Foundation
10:50 am ET
Botond Roska, MD, PhD; Institute of Molecular and Clinical Ophthalmology Basel
11:10 am ET
Moderator: Beverly Davidson, PhD; Children’s Hospital of Philadelphia
- Jessica Morgan, PhD; University of Pennsylvania
- Sheila Nirenberg, PhD; Cornell University
- Alberto Auricchio, MD; Telethon Institute of Genetics and Medicine
- Kafui Dzirasa, MD, PhD; Duke University
- Susanne Ahmari, MD, PhD; University of Pittsburgh
- Deniz Dalkara, PhD; Institut de la Vision
- Bob Greenberg, MD, PhD; Alfred E. Mann Foundation
- Botond Roska, MD, PhD; Institute of Molecular and Clinical Ophthalmology Basel
12:00 pm ET
BREAK
12:30 pm ET
SESSION 2: FROM GENE THERAPY TO CIRCUIT THERAPY
Session 2 will explore recent advances in gene therapy and editing approaches and learn how they may inform future CNS circuit therapies. The speakers and subsequent panel will probe how lessons from these fields can be used for circuit manipulation now as well as how it can guide future developments.
12:30 pm ET
Paul Heppenstall, PhD; Scuola Internazionale Superiore di Study Avanzati
12:45 pm ET
Viviana Gradinaru, PhD; California Institute of Technology
1:00 pm ET
Alex Mas Monteys, PhD; Children’s Hospital of Philadelphia
1:15 pm ET
Flavia Vitale, PhD; University of Pennsylvania
1:30 pm ET
1:45 pm ET
Moderator: Viviana Gradinaru, PhD; California Institute of Technology
- Holly Tabor, PhD; Stanford University
- Nicole Paulk, PhD; University of California San Francisco
- Mark Budde, PhD; California Institute of Technology
- Timothy Yu, MD, PhD; Boston Children’s Hospital
- Tobias Moser, MD; Georg-August-Universität Göttingen
- Paul Heppenstall, PhD; Scuola Internazionale Superiore di Study Avanzati
- Alex Mas Monteys, PhD; Children’s Hospital of Philadelphia
- Flavia Vitale, PhD; University of Pennsylvania
- Ying Kai Chan, PhD; Harvard University
2:45 pm ET
Closing Remarks
Doug Meinecke, PhD; National Institute of Mental Health
3:00 pm ET
END OF DAY 1
10:00 am ET
10:10 am ET
Session 3 will identify and discuss major hurdles impeding the translation of circuit therapies from basic science to therapeutic tools. Safely delivering, and validating in humans and animal models, transgene constructs to specific cell-types and circuits poses many difficulties. The speakers for this session will both highlight these difficulties and discuss potential avenues to overcome these hurdles.
10:10 am ET
Petra Kaufmann, MD; Affinia Therapeutics
10:25 am ET
Kaitlyn Sadtler, PhD; National Institute of Biomedical Imaging and Bioengineering
10:40 am ET
Bosiljka Tasic, PhD; Allen Institute
10:55 am ET
Federico Mingozzi, PhD; Spark Therapeutics
11:10 am ET
Jim Pickel, PhD; National Institutes of Health
11:30 am ET
Moderator: David McMullen, MD; National Institute of Mental Health
- Kathy Ferrara, PhD; Stanford University
- Gordon Fishell, PhD; Harvard University
- Adam Hantman, PhD; Janelia Research Campus
- Clive Svendsen, PhD; Ceders-Sinai Medical Center
- Sergiu Pasca, MD; Stanford University
- Petra Kaufmann, MD; Affinia Therapeutics
- Kaitlyn Sadtler, PhD; National Institute of Biomedical Imaging and Bioengineering
- Bosiljka Tasic, PhD; Allen Institute
- Frederico Mingozzi, PhD; Spark Therapeutics
- Jim Pickel, PhD; National Institutes of Health
12:30 pm ET
BREAK
1:00 pm ET
This final session will bring in speakers from the wider regulatory and commercialization vantages to provide perspective on requisite steps to begin testing in humans (FDA) and successes necessary to demonstrate viability and de-risk for funders (VCs, government partners, and public-private partnerships). We will also hear from the patient perspective to better understand how the translational community and patient groups can work together to advance new therapies.
1:00 pm ET
Feorillo Galivo, MD, PhD; Food and Drug Administration/CBER
1:15 pm ET
Scott Sternson, PhD; Janelia Research Campus
1:30 pm ET
Bill Podd, JD; Landmark Capital
1:45 pm ET
Rachel Salzman, DVM; Alcyone Therapeutics
2:00 pm ET
Moderator: Ryan Richardson, NINDS/OBD
- Allan Basbaum, PhD; University of California San Francisco
- Jose-Alain Sahel, MD; University of Pittsburgh
- Tracy Dixon-Salazar, PhD; Lennox-Gastaut Syndrome Foundation
- Clare Ozawa, PhD; Versant Ventures
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- Feorillo Galivo, MD, PhD; Food and Drug Administration/CBER
- Scott Sternson, PhD; Janelia Research Campus
- Bill Podd, JD; Landmark Capital
- Rachel Salzman, DVM; Alcyone Therapeutics
2:50 pm ET
Closing Remarks
David McMullen, MD; National Institute of Mental Health
3:00 pm ET
ADJOURN