Course Schedule of Activities
Course Offering During the Week of February 8, 2021
Monday, February 8, 2021
11:00 am – 11:05 am
Welcome & Housekeeping
Carolone Foote, ASENT
11:05 am – 11:35 am
Overview: The Neurotherapeutics Discovery and Development Process
Michael A. Rogawski, MD, PhD, University of California, Davis
11:35 am – 11:45 am
Live Discussion
Barbra Slusher, PhD
11:45 am – 12:15 pm
Session 1: Target and Pathway Interrogation
James Inglese, PhD, National Center for Advancing Translational Sciences, NIH
12:15 am – 12:25 am
Live Discussion
Michael A. Rogawski, MD, PhD
12:25 pm – 12:55 pm
Break
12:55 pm – 1:25 pm
Session 2: Biology Basics for Identifying Hits
William Janzen, BS, Ribometrix, Inc.
1:25 am – 1:35 am
Live Discussion
Karl Scheidt, PhD
1:35 pm – 2:05 pm
Session 3: Moving from Hit to Lead to Clinical Candidate
James Barrow, PhD, Johns Hopkins School of Medicine
2:05 am – 2:15 am
Live Discussion
Barbara Slusher, PhD
2:15 noon – 2:30 pm
Break
2:30 pm – 3:00 pm
Session 4: Antibody-Based Therapies
Gai Ayalon, PhD, Ultragenyx Pharmaceuticals
3:00 am – 3:10 am
Live Discussion
Michael A. Rogawski, MD, PhD
3:10 am – 3:15 am
Introduction to Meet with Mentors
3:15 pm – 4:00 pm
Meet with Mentors Live Breakout
4:00 pm – 4:45 pm
Office Hours with Course Directors and Faculty
Please drop by if you have questions for them.
Tuesday, February 9, 2021
11:00 am – 11:05 am
Welcome & Overview of the Day
Barbra Slusher, PhD
11:05 am – 11:35 pm
Session 5: Intellectual Property
Jeffrey W. Childers, JD, PhD, Casimir Jones S.C.
11:35 am – 11:45 am
Live Discussion
Barbra Slusher, PhD
11:45 am – 11:50 am
Introduction of Invited Guest Speaker
Michael A. Rogawski, MD, PhD
11:50 am – 12:20 pm
Invited Guest Speaker: The Story of Risdiplam/Everysdi
Alexander Stephan, PhD, Roche Pharma Research
12:20 am – 12:30 am
Live Discussion
Michael Rogawski, MD, PhD
12:30 pm – 1:00 pm
Break
1:00 pm – 1:30 am
Session 6: ADME
Kennan Marsh, PhD, AbbVie
1:30 pm – 1:40 pm
Discussion
Barbara Slusher, PhD
1:40 pm – 2:10 pm
Session 7: Vignette - A story of "Bench to (Almost) Bedside"
Barbara Slusher, PhD, Johns Hopkins University
2:10 pm – 2:20 pm
Live Discussion
Karl Scheidt, PhD
2:20 pm – 2:35 pm
Break
2:35 pm – 3:05 pm
Session 8: Alternative Approaches to Lead Generation
Sam Enna, PhD, University of Kansas
3:05 pm – 3:15 am
Live Discussion
Michael A. Rogawski, MD, PhD
3:15 pm – 3:20 pm
Introduction to Round Robins
3:20 pm – 4:15 pm
Engagement Exercise: Round Robin Day 1
4:15 pm – 4:20 pm
Introduction to Office Hours
4:20 pm – 5:00 pm
Office Hours with Course Directors and Faculty
Please drop by if you have questions for them.
Wednesday, February 10, 2021
11:00 am – 11:05 am
Welcome & Overview of the Day
Karl Scheidt, PhD
11:05 am – 11:25 am
Session 9A: Formulation and Routes of Administration
Maureen Donovan, PhD, University of Iowa
11:25 am – 11:45 am
Session 9B: Alternative Routes of Administration
James Cloyd III, PharmD, University of Minnesota
11:45 pm – 12:00 am
Live Discussion
Michael A. Rogawski, MD, PhD
12:00 pm – 12:30 pm
Session 10: Toxicology
Adaline Smith, PhD, DABT Adagen Preclinical Solutions
11:45 pm – 12:00 am
Live Discussion
Barbara Slusher, PhD
12:40 pm – 1:10 pm
Break
1:10 pm – 1:40 pm
Session 11: IND Enabling Studies and Preparation of the IND
Edward (Ted) Spack, PhD, Medared
1:40 pm – 1:50 pm
Discussion
Karl Scheidt, PhD
1:50 pm – 2:20 pm
Session 12: Rigor in Translational Drug Discovery and Development
Shai Silberberg, PhD, NINDS
2:20 pm – 2:30 am
Discussion
Michael A. Rogawski, MD, PhD
2:30 pm – 2:45 pm
Break
2:45 pm – 2:50 pm
Introduction to Round Robins
2:50 pm – 4:15 pm
Engagement Exercise 2: Round Robin Day 2
4:15 pm – 4:20 pm
Introduction to Office Hours
4:20 pm – 5:00 pm
Office Hours with Course Directors and Faculty
Please drop by if you have questions for them.
Thursday, February 11, 2021
11:00 am – 11:05 am
Welcome & Overview of the Day
Michael A. Rogawski, MD, PhD
11:05 am – 11:35 pm
Session 13: Antisense Oglionucleotides
Gene Liau, PhD, Stoke Therapeutics
11:35 am – 11:45 pm
Discussion
Karl Scheidt, PhD
11:45 am – 12:15 pm
Session 14: Clinical Considerations in the Non-Clinical Phase of Drug Discovery and Development
James J. Vornov, MD, PhD, Johns Hopkins School of Medicine and Medpace
12:15 pm – 12:25 pm
Discussion
Barbara Slusher, PhD
12:25 pm – 1:00 pm
Break
1:00 pm – 1:30 pm
Session 15: Funding Academic Drug Discovery Research
Charles L. Cywin, PhD, NIH/NINDS
1:30 pm – 1:40 pm
Discussion
Michael A. Rogawski, MD, PhD
1:40 pm – 1:45 pm
Introduction to Round Robin
1:45 pm – 2:45 pm
Engagement Exercise: Round Robin 3
2:45 pm – 2:50 pm
Introduction to Office Office Hours
2:50 pm – 3:15 pm
Office Hours with Course Directors and Faculty
Please drop by if you have questions for them.
Students Work on Presentations
Friday, February 12, 2021
11:00 am – 11:05 am
Welcome & Overview of the Day
Barbara Slusher, PhD
11:05 am – 1:30 pm
Engagement Exercise: Student Presentations
1:30 pm – 2:00 pm
Conclusion: Where do we go from here? Course evaluation and survey
Course Directors
2:00 pm
Adjourn
11:00 am – 11:05 am
Welcome & Housekeeping
Carolone Foote, ASENT
11:05 am – 11:35 am
Overview: The Neurotherapeutics Discovery and Development Process
Michael A. Rogawski, MD, PhD, University of California, Davis
11:35 am – 11:45 am
Live Discussion
Barbra Slusher, PhD
11:45 am – 12:15 pm
Session 1: Target and Pathway Interrogation
James Inglese, PhD, National Center for Advancing Translational Sciences, NIH
12:15 am – 12:25 am
Live Discussion
Michael A. Rogawski, MD, PhD
12:25 pm – 12:55 pm
Break
12:55 pm – 1:25 pm
Session 2: Biology Basics for Identifying Hits
William Janzen, BS, Ribometrix, Inc.
1:25 am – 1:35 am
Live Discussion
Karl Scheidt, PhD
1:35 pm – 2:05 pm
Session 3: Moving from Hit to Lead to Clinical Candidate
James Barrow, PhD, Johns Hopkins School of Medicine
2:05 am – 2:15 am
Live Discussion
Barbara Slusher, PhD
2:15 noon – 2:30 pm
Break
2:30 pm – 3:00 pm
Session 4: Antibody-Based Therapies
Gai Ayalon, PhD, Ultragenyx Pharmaceuticals
3:00 am – 3:10 am
Live Discussion
Michael A. Rogawski, MD, PhD
3:10 am – 3:15 am
Introduction to Meet with Mentors
3:15 pm – 4:00 pm
Meet with Mentors Live Breakout
4:00 pm – 4:45 pm
Office Hours with Course Directors and Faculty
Please drop by if you have questions for them.
Tuesday, February 9, 2021
11:00 am – 11:05 am
Welcome & Overview of the Day
Barbra Slusher, PhD
11:05 am – 11:35 pm
Session 5: Intellectual Property
Jeffrey W. Childers, JD, PhD, Casimir Jones S.C.
11:35 am – 11:45 am
Live Discussion
Barbra Slusher, PhD
11:45 am – 11:50 am
Introduction of Invited Guest Speaker
Michael A. Rogawski, MD, PhD
11:50 am – 12:20 pm
Invited Guest Speaker: The Story of Risdiplam/Everysdi
Alexander Stephan, PhD, Roche Pharma Research
12:20 am – 12:30 am
Live Discussion
Michael Rogawski, MD, PhD
12:30 pm – 1:00 pm
Break
1:00 pm – 1:30 am
Session 6: ADME
Kennan Marsh, PhD, AbbVie
1:30 pm – 1:40 pm
Discussion
Barbara Slusher, PhD
1:40 pm – 2:10 pm
Session 7: Vignette - A story of "Bench to (Almost) Bedside"
Barbara Slusher, PhD, Johns Hopkins University
2:10 pm – 2:20 pm
Live Discussion
Karl Scheidt, PhD
2:20 pm – 2:35 pm
Break
2:35 pm – 3:05 pm
Session 8: Alternative Approaches to Lead Generation
Sam Enna, PhD, University of Kansas
3:05 pm – 3:15 am
Live Discussion
Michael A. Rogawski, MD, PhD
3:15 pm – 3:20 pm
Introduction to Round Robins
3:20 pm – 4:15 pm
Engagement Exercise: Round Robin Day 1
4:15 pm – 4:20 pm
Introduction to Office Hours
4:20 pm – 5:00 pm
Office Hours with Course Directors and Faculty
Please drop by if you have questions for them.
Wednesday, February 10, 2021
11:00 am – 11:05 am
Welcome & Overview of the Day
Karl Scheidt, PhD
11:05 am – 11:25 am
Session 9A: Formulation and Routes of Administration
Maureen Donovan, PhD, University of Iowa
11:25 am – 11:45 am
Session 9B: Alternative Routes of Administration
James Cloyd III, PharmD, University of Minnesota
11:45 pm – 12:00 am
Live Discussion
Michael A. Rogawski, MD, PhD
12:00 pm – 12:30 pm
Session 10: Toxicology
Adaline Smith, PhD, DABT Adagen Preclinical Solutions
11:45 pm – 12:00 am
Live Discussion
Barbara Slusher, PhD
12:40 pm – 1:10 pm
Break
1:10 pm – 1:40 pm
Session 11: IND Enabling Studies and Preparation of the IND
Edward (Ted) Spack, PhD, Medared
1:40 pm – 1:50 pm
Discussion
Karl Scheidt, PhD
1:50 pm – 2:20 pm
Session 12: Rigor in Translational Drug Discovery and Development
Shai Silberberg, PhD, NINDS
2:20 pm – 2:30 am
Discussion
Michael A. Rogawski, MD, PhD
2:30 pm – 2:45 pm
Break
2:45 pm – 2:50 pm
Introduction to Round Robins
2:50 pm – 4:15 pm
Engagement Exercise 2: Round Robin Day 2
4:15 pm – 4:20 pm
Introduction to Office Hours
4:20 pm – 5:00 pm
Office Hours with Course Directors and Faculty
Please drop by if you have questions for them.
Thursday, February 11, 2021
11:00 am – 11:05 am
Welcome & Overview of the Day
Michael A. Rogawski, MD, PhD
11:05 am – 11:35 pm
Session 13: Antisense Oglionucleotides
Gene Liau, PhD, Stoke Therapeutics
11:35 am – 11:45 pm
Discussion
Karl Scheidt, PhD
11:45 am – 12:15 pm
Session 14: Clinical Considerations in the Non-Clinical Phase of Drug Discovery and Development
James J. Vornov, MD, PhD, Johns Hopkins School of Medicine and Medpace
12:15 pm – 12:25 pm
Discussion
Barbara Slusher, PhD
12:25 pm – 1:00 pm
Break
1:00 pm – 1:30 pm
Session 15: Funding Academic Drug Discovery Research
Charles L. Cywin, PhD, NIH/NINDS
1:30 pm – 1:40 pm
Discussion
Michael A. Rogawski, MD, PhD
1:40 pm – 1:45 pm
Introduction to Round Robin
1:45 pm – 2:45 pm
Engagement Exercise: Round Robin 3
2:45 pm – 2:50 pm
Introduction to Office Office Hours
2:50 pm – 3:15 pm
Office Hours with Course Directors and Faculty
Please drop by if you have questions for them.
Students Work on Presentations
Friday, February 12, 2021
11:00 am – 11:05 am
Welcome & Overview of the Day
Barbara Slusher, PhD
11:05 am – 1:30 pm
Engagement Exercise: Student Presentations
1:30 pm – 2:00 pm
Conclusion: Where do we go from here? Course evaluation and survey
Course Directors
2:00 pm
Adjourn
