Wednesday, March 7, 2018
7:45 am – 8:30 am
Check-in and Continental Breakfast
8:30 am – 9:00 am
Overview: The Neurotherapeutics Discovery and Development Process
Michael A. Rogawski, MD, PhD, University of California, Davis
9:00 am – 9:50 am
Session 1: Target and Pathway Interrogation
James Inglese, PhD, National Center for Advancing Translational Sciences, NIH
9:50 am – 10:15 am
Break
10:15 am – 11:05 am
Session 2: Biology Basics for Identifying Hits
William Janzen, BS, Epizyme, Inc.
11:05 am – 11:55 am
Session 3: Moving from Hit to Lead to Clinical Candidate
James Barrow, PhD, Johns Hopkins School of Medicine
11:55 am – 12:45 am
Session 4: Antibody-Based Therapies
Gai Ayalon, PhD, Genentech
12:45 pm – 1:55 pm
Lunch
Students paired with mentors
1:55 pm – 2:45 pm
Session 5: Assessing Unmet Needs and Advancing Early Stage Medical Innovations
in Multiple Domains to Attract Resources and Progress to Clinical Impact
Steven Schachter, MD, Harvard Medical School
Mike Dempsey (The Innovation Cycle in Defining Clinical Needs) and John Collins, PhD [Putting Together an Action Plan to Patient: The Healthcare Innovation Cycle Guidance and Impact and Tracking System (GAITS)], CIMIT, Massachusetts General Hospital
2:45 pm – 3:35 pm
Session 6: Intellectual Property
Jeffrey W. Childers, PhD, Michael Best & Friedrich LLP
3:35 pm – 3:55 pm
Break
3:55 pm – 5:30 pm
Engagement Exercise 1: Clarify vision statement and begin to develop the components of presentation
5:30 pm – 6:15 pm
Networking Reception
6:15 pm – 8:15 pm
Dinner
Thursday, March 8, 2017
7:15 am – 8:00 am
Continental Breakfast
8:00 am – 8:50 am
Session 7: Alternative Approaches to Lead Generation
Sam J. Enna, PhD, The University of Kansas
8:50 am – 9:40 am
Session 8: ADME
Kennan Marsh, PhD, AbbVie
9:40 am – 10:00 am
Break
10:00 am – 10:50 am
Session 9: Preclinical Models of CNS Disease: Efficacy and Target Engagement
Matthew Kennedy, PhD, Merck
10:50 am – 12:30 pm
Session 10: Rigor in Translational Drug Discovery and Development
Shai Silberberg, PhD, NIH/NINDS
12:30 pm – 1:45 pm
Lunch
Responsible Conduct of Research Exercise
1:45 pm – 5:30 pm
ASENT Pipeline Presentations
5:30 pm – 6:00 pm
Break
6:00 pm – 8:30 pm
Course Dinner and After Dinner Talk
Development of SPINRAZA™ (nusinersen) for Spinal Muscular Atrophy
C. Frank Bennett, PhD, Ionis Pharmaceuticals, Inc.
Friday, March 9, 2017
7:15 am – 8:00 am
Continental Breakfast
8:00 am – 8:50 am
Session 11: Toxicology
Adaline Smith, PhD, DABT, Ironwood Pharmaceuticals
8:50 am –9:40 am
Session 12: IND Enabling Studies and Preparation of the IND
Edward Spack, PhD, Medared
9:40 am – 10:00 am
Break
10:00 am – 11:00 pm
Session 13: Formulation and Route of Administration
James Cloyd III, PharmD, University of Minnesota
11:00 am – 10:20 am
Break
11:20 am – 12:15 pm
Session 14: Gene-base Therapies
Michael G. Kaplitt, MD, PhD, Weill Cornell Medical College
12:15 pm – 1:30 pm
Lunch
1:30 pm – 5:30 pm
Engagement Exercise 2: Round Robin
5:45 pm – 8:30 pm
Course Dinner
Hilton Washington DC/Rockville Hotel & Executive Meeting Center
Saturday, March 10, 2017
7:00 am – 7:40 am
Continental Breakfast
7:40 – 8:30 am
Session 15: Rethinking How to Treat Neurodegenerative Disease
Lee L. Rubin, PhD, Harvard University
8:30 am – 9:20 pm
Session 16: Funding Academic Drug Discovery Research
Charles L. Cywin, PhD, NIH/NINDS
9:20 am – 10:30 am
Engagement Exercise 3: Student Presentations
10:30 am – 10:50 am
Break
10:50 am – 12:00 am
Engagement Exercise 3: Student Presentations (continued)
12:00 pm – 12:15 pm
Conclusion: Where do we go from here? Presentation of plan for continued mentoring, engagement and course evaluation over two-year period
Course Directors
12:15 pm
Adjourn
7:45 am – 8:30 am
Check-in and Continental Breakfast
8:30 am – 9:00 am
Overview: The Neurotherapeutics Discovery and Development Process
Michael A. Rogawski, MD, PhD, University of California, Davis
9:00 am – 9:50 am
Session 1: Target and Pathway Interrogation
James Inglese, PhD, National Center for Advancing Translational Sciences, NIH
9:50 am – 10:15 am
Break
10:15 am – 11:05 am
Session 2: Biology Basics for Identifying Hits
William Janzen, BS, Epizyme, Inc.
11:05 am – 11:55 am
Session 3: Moving from Hit to Lead to Clinical Candidate
James Barrow, PhD, Johns Hopkins School of Medicine
11:55 am – 12:45 am
Session 4: Antibody-Based Therapies
Gai Ayalon, PhD, Genentech
12:45 pm – 1:55 pm
Lunch
Students paired with mentors
1:55 pm – 2:45 pm
Session 5: Assessing Unmet Needs and Advancing Early Stage Medical Innovations
in Multiple Domains to Attract Resources and Progress to Clinical Impact
Steven Schachter, MD, Harvard Medical School
Mike Dempsey (The Innovation Cycle in Defining Clinical Needs) and John Collins, PhD [Putting Together an Action Plan to Patient: The Healthcare Innovation Cycle Guidance and Impact and Tracking System (GAITS)], CIMIT, Massachusetts General Hospital
2:45 pm – 3:35 pm
Session 6: Intellectual Property
Jeffrey W. Childers, PhD, Michael Best & Friedrich LLP
3:35 pm – 3:55 pm
Break
3:55 pm – 5:30 pm
Engagement Exercise 1: Clarify vision statement and begin to develop the components of presentation
5:30 pm – 6:15 pm
Networking Reception
6:15 pm – 8:15 pm
Dinner
Thursday, March 8, 2017
7:15 am – 8:00 am
Continental Breakfast
8:00 am – 8:50 am
Session 7: Alternative Approaches to Lead Generation
Sam J. Enna, PhD, The University of Kansas
8:50 am – 9:40 am
Session 8: ADME
Kennan Marsh, PhD, AbbVie
9:40 am – 10:00 am
Break
10:00 am – 10:50 am
Session 9: Preclinical Models of CNS Disease: Efficacy and Target Engagement
Matthew Kennedy, PhD, Merck
10:50 am – 12:30 pm
Session 10: Rigor in Translational Drug Discovery and Development
Shai Silberberg, PhD, NIH/NINDS
12:30 pm – 1:45 pm
Lunch
Responsible Conduct of Research Exercise
1:45 pm – 5:30 pm
ASENT Pipeline Presentations
5:30 pm – 6:00 pm
Break
6:00 pm – 8:30 pm
Course Dinner and After Dinner Talk
Development of SPINRAZA™ (nusinersen) for Spinal Muscular Atrophy
C. Frank Bennett, PhD, Ionis Pharmaceuticals, Inc.
Friday, March 9, 2017
7:15 am – 8:00 am
Continental Breakfast
8:00 am – 8:50 am
Session 11: Toxicology
Adaline Smith, PhD, DABT, Ironwood Pharmaceuticals
8:50 am –9:40 am
Session 12: IND Enabling Studies and Preparation of the IND
Edward Spack, PhD, Medared
9:40 am – 10:00 am
Break
10:00 am – 11:00 pm
Session 13: Formulation and Route of Administration
James Cloyd III, PharmD, University of Minnesota
11:00 am – 10:20 am
Break
11:20 am – 12:15 pm
Session 14: Gene-base Therapies
Michael G. Kaplitt, MD, PhD, Weill Cornell Medical College
12:15 pm – 1:30 pm
Lunch
1:30 pm – 5:30 pm
Engagement Exercise 2: Round Robin
5:45 pm – 8:30 pm
Course Dinner
Hilton Washington DC/Rockville Hotel & Executive Meeting Center
Saturday, March 10, 2017
7:00 am – 7:40 am
Continental Breakfast
7:40 – 8:30 am
Session 15: Rethinking How to Treat Neurodegenerative Disease
Lee L. Rubin, PhD, Harvard University
8:30 am – 9:20 pm
Session 16: Funding Academic Drug Discovery Research
Charles L. Cywin, PhD, NIH/NINDS
9:20 am – 10:30 am
Engagement Exercise 3: Student Presentations
10:30 am – 10:50 am
Break
10:50 am – 12:00 am
Engagement Exercise 3: Student Presentations (continued)
12:00 pm – 12:15 pm
Conclusion: Where do we go from here? Presentation of plan for continued mentoring, engagement and course evaluation over two-year period
Course Directors
12:15 pm
Adjourn
