Speakers: Ian Blair, Jaya Goyal, Cindy Xia, Tricia Thornton-Wells, Thomas Roddy, Rachel Church, Brett Howell, Collin Brown, Rodney Rouse, Mithun Khatta, Brian Schmidt, Dong Geng, Paul McDonagh, Rangaraj Narayanan, Hoa Nguyen, Saeho Chong
Organizers: Jaya Goyal (Wave Life Sciences), Karen Hongmei Cao (Alnylam), Chandra Prakash (Agios), Pratap Singh (Alexion), Haojing Rong (Kymera), and Jing-Tao Wu (Alnylam)
Date: 2018-12-03- 12/4/2018
Time: 8:45-16:30 Eastern Time
Registration fee: (USD): Regular: $300; Regular - Day 1 only: $200; Regular - Day 2 only: $200; For Academic / Unemployed: $200
Location: Hyatt Regency Boston, One Avenue de Lafayette, Boston, MA, 02111
Major Sponsor: (2)Applied BioMath, LLC; Worldwide Clinical Trials;(Major Sponsorship - Day 2 Morning Session still open)
Vendor show vendors registered to date: (11)BioDuro; BioreclamationIVT; Charles River Laboratories; Frontage; Medpace; Absorption Systems; Alturas Analytics, Inc.; Biomere; MedChem Partners; MicroConstants; QPS
Registration: http://www.PBSS.org
Registration deadline:2018-12-02  (it will close sooner if the seating cap is reached)

About the Topic

Fundamentals and Emerging Trends in Translational Research: Identifying Right Target, Right Drug, Right Dose and Right Patient

Date/Time:        December 3 and 4, 2018, 8:45 am - 4:30 pm

Fees:                    Regular $300; Unemployed & Academic $200; Major Sponsor $2000; Vendor Show $500

Venue:                Hyatt Regency Boston, One Avenue de Lafayette, Boston, MA 02111

Registration:      www.PBSS.org

Workshop Introduction

Conventionally, medicines were based on chemical entities that demonstrated their safety and efficacy in patients, but the fundamental structure of drugs has evolved over the years to include a much wider array of options. Typical product portfolio and clinical pipelines currently contain multiple distinct modalities, as a result, pre-clinical Investigative and bioanalytical tools need to be refined or established to improve the translation of data/information from in vitro assays to in vivo readouts and PK/pharmacology/safety data from animals to humans which would ultimately lead to a higher success rate in drug development. Building a consensus on what screening/ tests are appropriate at the discovery and preclinical stage for these molecules is important. This workshop/symposium will bring experts in the field who are applying drug discovery and development experience to support the new therapeutic modalities developed for targeted or rare populations.

Day 1 (Dec. 3) Agenda:

Plenary Lecture: Proteomics data advancing translational research: Ian Blair, A. N. Richards Professor, Vice-Chair, Department of Systems Pharmacology and Translational Therapeutics, U Penn. (8:45am to 9:30am)

Morning Session: Fundamentals of Translational Research

1. Precision Medicine Oncology and beyond: Jaya Goyal, Wavelife Sciences. (9:30 to 10:10am)
2. Break and vendor show: (10:10 to10:30am)
3. The Evolving Role of ADME in Translational Research, Cindy Xia, Takeda, (10:30 to 11:10am)
4. Using Genetic, Physiological and Neuroimaging Biomarkers to Select, Stratify or Interpret Patient Subgroups in Clinical Trials: Tricia Thornton-Wells, Vertex Pharmaceuticals. (11:10 to 11:50pm)
5. Major Sponsor presentation: Applied BioMath (11:50 to 12:05pm)
6. Lunch and network break (12:05 to 1:00pm)

Afternoon Session: Emerging Tools in Translational Research

1. Integrating a Mass Spec Lab for Multi-omic Characterization of Biomarkers and Drug Targets: Thomas Roddy, Agios.(1:00 to 1:40pm)
2. Candidate Biomarkers of Drug-Induced Liver Injury: Performance in Healthy Volunteers and Patients: Rachel Church, University of North Carolina (1:40 to 2:20pm)
3. Break and vendor show: (2:20 to 2:40PM)
4. A Quantitative Systems Toxicology Modeling Tool of Drug-Induced Liver Injury (DILI), to Assess DILI Risk in Drug Development: Brett Howell, DILIsym (2:40 – 3:20pm)
5. In vitro platforms for de-risking nephrotoxicity: Collin Brown (3:20 -4:00pm)
6. Panel discussion (4:00-4:30 PM)

Day 2 (Dec. 4) Agenda:

Plenary Lecture: Regulatory Science – A Vital Component of Translational Research: Rodney Rouse, Associate Director, Division of Applied Regulatory Science, Office of Clinical Pharmacology, Office of Translational Science, Center for Drug Evaluation & Research, U.S. Food and Drug Administration (8:45 to 9:30am)

Morning Session: Translational Strategies for Immuno/ Oncology (I/O)

1. Considerations for Non-clinical Evaluation of I/O Products and the Translational Role of Syngeneic and Genetically Engineered Mouse Models: Mithun Khatta, Takeda (9:30 to 10:10am)
2. Break and vendor show: (10:10 to 10:30am)
3. Mechanistic Modeling to Support Clinical Development and Dose Finding for I/O Agents and Their Combinations: Brian Schmidt, BMS (10:30 to 11:10am)
4. Translational bioanalytical assay strategies for CAR-T therapy: Dong Geng, Legend Biotech (11:10 to 11:50am)
5. Major Sponsor Presentation: Worldwide Clinical Trials (11:50 to 12:05pm)
6. Lunch and network break (12:05 to 1:00pm) 

Afternoon session: Rare Diseases and Oligonucleotide Therapies

1. Translating sequencing and computing to the hospital: How to diagnose babies with genetic diseases in 19 hours and why it's important: Paul McDonagh, Alexion Pharmaceuticals (1:00 to 1:40pm)
2. QSP tools for rare disease in discovery and development: Rangaraj Narayanan/Hoa Nguyen,Shire (1:40 to 2:20pm)
3. Break and vendor show: (2:20 to 2:40PM)
4. In Vivo Fate of siRNA-GalNac Conjugates: Saeho Chong, Alnylam (2:40 to 3:20pm)
5. Panel Discussion: (3:20 to 3:50pm)
6. Closing Remarks: (3:50 to 4:00pm)

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