Wellstone-Workshops Agenda

Eric P Hoffman, PhD. Director, Wellstone-DC, Research Center for Genetic Medicine
Carrie Miceli, PhD. UCLA.
Diana Escolar, MD. Co-Director, Wellstone-DC, Medical Director CINRG
John McCall, PhD, PharMac
Robert Damoiseaux, PhD, Director, UCLA HTS Facility.

Monday 17 April 2006

Morning

7:45 AM

Bus leaves from Marriott Residence Inn to CNMC

8:00 - 8:30

Continental Breakfast (provided at the workshop)

Overview

8:30 AM

John McCall, PhD, PharMac
High Throughput Screening: A Key Part of Modern Drug Discovery

8:50

Discussion

HTS cell-based assay development and use.

9:00 AM

Doug Auld, PhD, NIH Chemical Genomics Center
The NIH Molecular Libraries Initiative: Chemical Tools for the Genome Era

9:20

Discussion

9:30 AM

Jon Tinsley, PhD, VASTox, Oxford
HTS for Duchenne muscular dystrophy.

9:50

Discussion

10:00 AM

John Babiak, PhD, PTC Therapeutics
PTC's HTS technology

10:20

Discussion

10:30 - 11:00 BREAK

11:00 AM

H Lee Sweeney, PhD, University of Pennsylvania
Muscular dystrophy targets

11:20

Discussion

11:30 AM

Carrie Miceli, PhD, UCLA
HTS screening project in the dystrophies.

11:50AM

Discussion

12:00 - 1:00 LUNCH (provided at the workshop)

HTS cell-based assay development and use (continued).

1:00 PM

Scott Diamond, PhD, University of Pennsylvania
HTS screens in neurological disease.

1:20

Discussion

1:30 PM

Marko Pregel, PhD, Genzyme
Genzyme drug discovery for the muscular dystrophies.

1:50

Discussion

2:00 PM

Jill Panetta, PhD, InnoCentive, Inc Innovative assay platforms for HTS.

2:20

Discussion

Drug libraries and screening methods

2:30 PM

Tom Fletcher, PhD, Southern Research Institute
The NINDS HTS drug screening core.

2:50

Discussion

3:00 - 3:30 BREAK

3:30 PM

Robert Damoiseaux, PhD, UCLA
The UCLA HTS facility

3:50

Discussion

4:00 PM

Achim Brinker, PhD, Novartis
Small molecule screens at the GNF

4:20

Discussion

4:30 PM

Loren Miraglia, Novartis
HTS using shRNA

4:50

Discussion

5:15PM

Bus leaves from CNMC to Marriott Residence Inn

6:15

Bus leaves from Marriott Residence Inn to Tosca (10 block walk)

6:30PM

Reception and Dinner (with workshop participants)

Keynote address:	Tools of the Modern Medicinal Chemist Artie Romero, Pfizer
Location:	Ristorante Tosca 1112 F Street, NW , Washington , DC 20004 202.367.1990

Participants will take a taxi or walk to Marriott Residence from Tosca (10 block walk)

Tuesday 18 April 2006

Morning
7:45 AM	Bus leaves from Marriott Residence Inn to CNMC
8:00 - 8:30	Continental Breakfast (provided at the workshop)

Drug libraries and screening methods (concluded)

8:30 AM	Daniel Scherman, PhD, AFM. AFM HTS effort
8:50	Discussion

Post-hit: In vivo models/cores (human, mouse, dog)

9:00 AM	Qi Long Lu, PhD, Carolinas Medical Center Oligonucleotide therapeutics in DMD.
9:20	Discussion
9:30 AM	Kanneboyina Nagaraju, DVM, PhD, Wellstone-DC The DoD muscular dystrophy murine drug screening facility.
9:50	Discussion

10:00 - 10:30 BREAK

10:30 AM	Diana Escolar, MD, Wellstone-DC, CINRG The CINRG international clinical trial network for muscular dystrophy.
10:50	Discussion
11:00 AM	Shin'ichi Takeda, MD, PhD, NCNP Kodaira , Japan The canine muscular dystrophy testing facility
11:20	Discussion

Prioritization of assays, drug libraries, and animal testing
11:30 AM	Prioritization of assays, screens, pre-clinical and clinical targets Kanneboyina Nagaraju, DVM, PhD
11:50	Discussion

12:00 - 1:00 LUNCH (provided at the workshop)

1:00PM	Transportation departs from CNMC to National and Marriott

Attendees: NIH and DoD Program Representatives
Glen Nuckolls, PhD	Muscle Disorders and Therapies Program Director, NIAMS, NIH
John Porter, PhD	Program Director for Neuromuscular Disease, NINDS, NIH
Jim Hanson, MD	Center for Developmental Biology & Perinatal Medicine, NICHD, NIH
Jill Heemskerk, PhD	Program Director, Technology Development, NINDS, NIH
Julie Wilberding, PhD	Congressionally Directed Medical Research Programs, USAMRMC
E. Melissa Kaime, MD	Congressionally Directed Medical Research Programs, USAMRMC

Stakeholders

Joel Wood	Foundation to Eradicate Duchenne
David Heil	Foundation to Eradicate Duchenne
Sharon Hesterlee, PhD	Muscular Dystrophy Association
Pat Furlong, PhD	Parent Project Muscular Dystrophy
Benjamin Seckler, MD	Charley's Fund
Tom Wicka	Bash for Nash
Debra Miller	Cure Duchenne Muscular Dystrophy
Jeffrey Rosenfeld, MD, PhD	Carolinas Medical Center
Stan Nelson, MD	UCLA

Wellstone Muscular Dystrophy Center Representatives

Kevin Campbell, PhD	University of Iowa Button
Charles Thornton, MD	University of Rochester
Paul Robbins, PhD	University of Pittsburgh