Monday, March 25, 2019
7:15 am – 8:00 am
Check-in and Continental Breakfast
8:00 am – 8:20 am
Overview: The Neurotherapeutics Discovery and Development Process
Michael A. Rogawski, MD, PhD, University of California, Davis
8:20 am – 9:10 am
Session 1: Target and Pathway Interrogation
James Inglese, PhD, National Center for Advancing Translational Sciences, NIH
9:10 am – 10:00 am
Session 2: Biology Basics for Identifying Hits
William Janzen, BS, Ribometrix, Inc.
10:00 am – 10:20 am
Break
10:20 am – 11:10 am
Session 3: Moving from Hit to Lead to Clinical Candidate
James Barrow, PhD, Johns Hopkins School of Medicine
11:10 pm – 12:00 pm
Session 4: Antibody-Based Therapies
Gai Ayalon, PhD, Ultragenyx
12:00 noon – 1:00 pm
Lunch
Students paired with mentors
Vignettes: Success Stories in Neurotherapeutics Discovery
1:00 pm – 1:30 pm
Gene Therapy Approaches for the Treatment of Neurodegenerative Lysosomal Storage Diseases
Miguel Sena-Esteves, PhD, University of Massachusetts
1:30 pm – 2:00 pm
The Development of Aimovig for the Preventive Treatment of Migraine
Gopi Shanker, PhD, Novartis
Break-out 1 with Vignette Speakers
2:00 pm – 2:30 pm
Group 1: Miguel Sena-Esteves, PhD
Group 2: Gopi Shanker, PhD
2:30 pm – 3:00 pm
Discovery of an Important Treatment for Epilepsy and Neuropathic Pain
Richard B. Silverman, PhD, Northwestern University
3:00 pm – 3:30 pm
Q&A with Vignette Speakers Richard B. Silverman
Break-out 2 with Vignette Speakers
3:30 pm – 4:00 pm
Group 1: Gopi Shankar, PhD
Group 2: Miguel Sena-Esteves, PhD
4:00 pm – 4:15 pm
Break
4:15 pm – 5:05 pm
Session 5: Intellectual Property
Jeffrey W. Childers, PhD, Casimir Jones S.C.
5:05 pm – 5:30 pm
Break
5:30 pm – 6:15 pm
Networking Reception
6:15 pm – 8:15 pm
Dinner
ASENT Careers Program
Tuesday, March 26, 2019
7:15 am – 8:00 am
Continental Breakfast
8:00 am – 8:50 am
Session 6: Alternative Approaches to Lead Generation
Sam J. Enna, PhD, The University of Kansas
8:50 am – 9:40 am
Session 7: ADME
Kennan Marsh, PhD, AbbVie
9:40 am – 10:00 am
Break
10:00 am – 10:50 am
Session 8: Preclinical Models of CNS Disease: Efficacy and Target Engagement
Matthew Kennedy, PhD, Merck
10:50 pm – 11:40 pm
Session 9: Rigor in Translational Drug Discovery and Development
Shai Silberberg, PhD, NIH/NINDS
11:40 am – 1:55 pm
Lunch
Responsible Conduct of Research Exercise
1:55 pm – 2:45 pm
Session 10: Antisense
Toby Ferguson, MD, PhD, Biogen
2:45 pm – 3:35 pm
Session 11: Gene Therapy with Viral Vectors
Edgardo Rodríguez-Lebrón, PhD, Lacerta Therapeutics
3:35 pm – 5:35 pm
Engagement Exercise 1: Round Robin Day 1
5:35 pm – 6:00 pm
Break
6:00 pm – 8:30 pm
Course Dinner and After Dinner Talk
Development of LUXTURNA™ (voretigene neparvovec) for Retinal Dystrophy,
the First FDA-approved Adeno-Associated Virus Vector Gene Therapy Approved in the U.S.
Katherine A. High, MD, Spark Therapeutics and Perelman School of Medicine at the University of Pennsylvania
Wednesday, March 27, 2019
7:15 am – 8:00 am
Continental Breakfast
8:00 am – 8:50 am
Session 12: Toxicology
Adaline Smith, PhD, DABT, Ironwood Pharmaceuticals
8:50 am –9:40 am
Session 13: IND Enabling Studies and Preparation of the IND
Edward Spack, PhD, Medared
9:40 am – 10:00 am
Break
10:00 am – 10:50 am
Session 14: Formulation and Route of Administration
James Cloyd III, PharmD, University of Minnesota
10:50 am – 11:40 am
Session 15: Clinical Considerations in the Non-Clinical Phase of Drug Discovery and Development
James J. Vornov, MD, PhD, Johns Hopkins School of Medicine and Medpace
12:00 pm – 1:30 pm
Lunch
1:30 pm – 2:20 pm
Session 16: Funding Academic Drug Discovery Research
Charles L. Cywin, PhD, NIH/NINDS
2:20 pm – 2:40 pm
Break
2:40 pm – 5:10 pm
Engagement Exercise 2: Round Robin
5:10 pm – 6:00 pm
Break
6:00 pm – 8:30 pm
Course Dinner
Seasons 52
Thursday, March 28, 2019
7:15 am – 8:30 am
Continental Breakfast
8:30 am – 11:45 am
Engagement Exercise 3: Student Presentations
11:45 am – 12:00 pm
Conclusion: Where do we go from here? Course evaluation
Course Directors
12:00 pm
Adjourn
7:15 am – 8:00 am
Check-in and Continental Breakfast
8:00 am – 8:20 am
Overview: The Neurotherapeutics Discovery and Development Process
Michael A. Rogawski, MD, PhD, University of California, Davis
8:20 am – 9:10 am
Session 1: Target and Pathway Interrogation
James Inglese, PhD, National Center for Advancing Translational Sciences, NIH
9:10 am – 10:00 am
Session 2: Biology Basics for Identifying Hits
William Janzen, BS, Ribometrix, Inc.
10:00 am – 10:20 am
Break
10:20 am – 11:10 am
Session 3: Moving from Hit to Lead to Clinical Candidate
James Barrow, PhD, Johns Hopkins School of Medicine
11:10 pm – 12:00 pm
Session 4: Antibody-Based Therapies
Gai Ayalon, PhD, Ultragenyx
12:00 noon – 1:00 pm
Lunch
Students paired with mentors
Vignettes: Success Stories in Neurotherapeutics Discovery
1:00 pm – 1:30 pm
Gene Therapy Approaches for the Treatment of Neurodegenerative Lysosomal Storage Diseases
Miguel Sena-Esteves, PhD, University of Massachusetts
1:30 pm – 2:00 pm
The Development of Aimovig for the Preventive Treatment of Migraine
Gopi Shanker, PhD, Novartis
Break-out 1 with Vignette Speakers
2:00 pm – 2:30 pm
Group 1: Miguel Sena-Esteves, PhD
Group 2: Gopi Shanker, PhD
2:30 pm – 3:00 pm
Discovery of an Important Treatment for Epilepsy and Neuropathic Pain
Richard B. Silverman, PhD, Northwestern University
3:00 pm – 3:30 pm
Q&A with Vignette Speakers Richard B. Silverman
Break-out 2 with Vignette Speakers
3:30 pm – 4:00 pm
Group 1: Gopi Shankar, PhD
Group 2: Miguel Sena-Esteves, PhD
4:00 pm – 4:15 pm
Break
4:15 pm – 5:05 pm
Session 5: Intellectual Property
Jeffrey W. Childers, PhD, Casimir Jones S.C.
5:05 pm – 5:30 pm
Break
5:30 pm – 6:15 pm
Networking Reception
6:15 pm – 8:15 pm
Dinner
ASENT Careers Program
Tuesday, March 26, 2019
7:15 am – 8:00 am
Continental Breakfast
8:00 am – 8:50 am
Session 6: Alternative Approaches to Lead Generation
Sam J. Enna, PhD, The University of Kansas
8:50 am – 9:40 am
Session 7: ADME
Kennan Marsh, PhD, AbbVie
9:40 am – 10:00 am
Break
10:00 am – 10:50 am
Session 8: Preclinical Models of CNS Disease: Efficacy and Target Engagement
Matthew Kennedy, PhD, Merck
10:50 pm – 11:40 pm
Session 9: Rigor in Translational Drug Discovery and Development
Shai Silberberg, PhD, NIH/NINDS
11:40 am – 1:55 pm
Lunch
Responsible Conduct of Research Exercise
1:55 pm – 2:45 pm
Session 10: Antisense
Toby Ferguson, MD, PhD, Biogen
2:45 pm – 3:35 pm
Session 11: Gene Therapy with Viral Vectors
Edgardo Rodríguez-Lebrón, PhD, Lacerta Therapeutics
3:35 pm – 5:35 pm
Engagement Exercise 1: Round Robin Day 1
5:35 pm – 6:00 pm
Break
6:00 pm – 8:30 pm
Course Dinner and After Dinner Talk
Development of LUXTURNA™ (voretigene neparvovec) for Retinal Dystrophy,
the First FDA-approved Adeno-Associated Virus Vector Gene Therapy Approved in the U.S.
Katherine A. High, MD, Spark Therapeutics and Perelman School of Medicine at the University of Pennsylvania
Wednesday, March 27, 2019
7:15 am – 8:00 am
Continental Breakfast
8:00 am – 8:50 am
Session 12: Toxicology
Adaline Smith, PhD, DABT, Ironwood Pharmaceuticals
8:50 am –9:40 am
Session 13: IND Enabling Studies and Preparation of the IND
Edward Spack, PhD, Medared
9:40 am – 10:00 am
Break
10:00 am – 10:50 am
Session 14: Formulation and Route of Administration
James Cloyd III, PharmD, University of Minnesota
10:50 am – 11:40 am
Session 15: Clinical Considerations in the Non-Clinical Phase of Drug Discovery and Development
James J. Vornov, MD, PhD, Johns Hopkins School of Medicine and Medpace
12:00 pm – 1:30 pm
Lunch
1:30 pm – 2:20 pm
Session 16: Funding Academic Drug Discovery Research
Charles L. Cywin, PhD, NIH/NINDS
2:20 pm – 2:40 pm
Break
2:40 pm – 5:10 pm
Engagement Exercise 2: Round Robin
5:10 pm – 6:00 pm
Break
6:00 pm – 8:30 pm
Course Dinner
Seasons 52
Thursday, March 28, 2019
7:15 am – 8:30 am
Continental Breakfast
8:30 am – 11:45 am
Engagement Exercise 3: Student Presentations
11:45 am – 12:00 pm
Conclusion: Where do we go from here? Course evaluation
Course Directors
12:00 pm
Adjourn
