This workshop will bring together cross-disciplinary investigators to discuss the potential for human translation of next-generation circuit therapies for disorders of the central nervous system. Please join us on June 29-30!
Beginning tomorrow, this virtual workshop will focus on the use of innovative and novel tools, including optogenetics, chemogenetics, and gene editing, as well as cutting-edge approaches to neuromodulation. Speakers will discuss existing approaches to circuit manipulation, advances in gene therapy approaches, and how they may inform future central nervous system (CNS) circuit therapies in humans. We invite those from a diversity of perspectives and backgrounds to attend, including stakeholders in academic research and industry; clinicians; representatives from scientific societies, funding bodies, and advocacy organizations; and interested members of the public.
The workshop will take place on June 29 and 30, 2021 from 10:00 AM – 3:00 PM (EST). The event is open to anyone and will be hosted on Zoom and NIH videocast.
Day 1: Tuesday, June 29, 2021
Session 1: Circuit tools case studies
This session will highlight various existing approaches to manipulating circuits in humans. Speakers will explore clinical case studies of genetic, device, and optogenetic-based tools currently used to treat disorders of vision. Presentations will be followed by a panel discussion on lessons learned from these approaches and future developments. Speakers include Drs. Deniz Dalkara, Bob Greenberg, and Botond Roska.
Session 2: From gene therapy to circuit therapy
The next session will explore recent advances in gene therapy and editing approaches and learn how they may inform future CNS circuit therapies. Presentations will be followed by a panel discussion on lessons learned from these fields and future developments. Speakers include Drs. Paul Heppenstall, Viviana Gradinaru, Alex Mas Monteys, Flavia Vitale, and Ying Kai Chan.
Day 2: Wednesday, June 30, 2021
Session 3: CNS translational hurdles
This session will identify and discuss major hurdles impeding the translation of circuit therapies from basic science to therapeutic tools. Speakers will highlight safe delivery and validation in humans and animal models, as well as avenues to overcome translational hurdles. Speakers include Drs. Petra Kaufmann, Kaitlyn Sadtler, Bosiljka Tasic, Federico Mingozzi, and Jim Pickel.
Session 4: CNS translational environment
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 (i.e., through the Food and Drug Administration) and successes necessary to demonstrate viability and de-risk for funders (e.g., venture capitalists, government partners, and public-private partnerships). Attendees will also hear from the patient perspective. Speakers include Drs. Feorillo Galivo and Scott Sternson, as well as Bill Podd and Racheal Salzman.