NIST – FDA GENOME EDITING WORKSHOP

APRIL 23-24, 2018

NIST, 100 BUREAU DRIVE, GAITHERSBURG, MD 20899

BLDG. 101, GREEN AUDITORIUM

AGENDA

DAY 1 – Monday, April 23

8:00 am Check-in at registration desk

9:00 am Opening Remarks

Sheng Lin-Gibson, Ph.D., Acting Division Chief, Biosystems and Biomaterials Division, NIST

Raj Puri, Ph.D., Division Director, Cellular and Gene Therapies Division, FDA

9:10 am Introduction to NIST Genome Editing Consortium

Samantha Maragh, Ph.D., Leader, NIST Genome Editing Program

9:25 am NIST Measurement Assurance Perspective

Anne Plant, Ph.D., NIST Fellow

9:45 am FDA Regulatory Perspective

Anna Kwilas, Ph.D., Biologist, Center for Biologics Evaluation and Research (CBER)

Stella Lee, Ph.D., Biologist, Center for Veterinary Medicine (CVM)

Zivana Tezak, Ph.D., Associate Director for Science and Technology, Center for Devices and 

Radiological Health (CDRH)

10:30 am NIH Somatic Cell Genome Editing Program

Colin Fletcher, Ph.D., Program Director, NIH/NHGRI

10:40 am BREAK

10:50 am Keynote: Reading an Edited Genome

Fyodor Urnov, Ph.D., Deputy Director, Altius Institute for Biomedical Sciences

11:30 am Panel Short Talks

Matt Porteus, M.D., Ph.D., Assoc. Professor, Dept. Pediatrics, Stanford University

Edward Rebar, Ph.D., VP Technology, Sangamo Therapeutics

Tom Barnes, Ph.D., Senior VP Innovation, Intellia Therapeutics

Will Eyestone, Ph.D., Senior Project Manager, Xeno R&D, Revivicor Inc.

Craig Mickanin, Ph.D., Director-Chemical Biology & Therapeutics, Novartis Institutes for 

Biomedical Research

12:20 pm Panel Discussion

12:40 pm LUNCH BREAK (for purchase at NIST cafeteria)

1:30 pm Lexicon

Brett Robb, Ph.D., Scientific Director, RNA & Genome Editing, New England Biolabs
1:50 pm Assays Qualification

Samantha Maragh, Ph.D., Leader, NIST Genome Editing Program

Shengdar Tsai, Ph.D., Assistant Member, Dept. of Hematology St. Jude Children's Hospital 

Kevin Holden, Ph.D., Head of Synthetic Biology, Synthego Corporation

2:25 pm Data & Metadata

Kendell Clement, Ph.D., Research Fellow, MGH/Harvard Medical School 

2:45 pm BREAK

3:00 pm Discussion of key questions

3:30 pm

Breakout Session Round 1 

Assay Qualification: Green Auditorium; Data & Metadata: Portrait Room; Lexicon: LR C

4:20 pm Breakout Session Round 2 

Assay Qualification: Green Auditorium; Data & Metadata: Portrait Room; Lexicon: LR C

5:00 pm Wrap up of Day 1

5:15 pm Adjourn

5:30 pm Shuttle departure from NIST to Hilton Gaithersburg 

DAY 2 – Tuesday, April 24

8:30 am Check-in at registration desk

9:00 am Welcome Day 2

9:10 am Tech Talk 1: Developing Meganucleases for Clinical Applications

Derek Jantz Ph.D., CSO, Precision Biosciences

9:25 am Tech Talk 2: Methods for Gene Editing Measurement and Off-Target Discovery

Christopher Wilson Ph.D., Sr. Director, Lead Discovery, Editas Medicine

9:40 am Tech Talk 3: dGH Analysis Tools for Gene Editing

Christopher Tompkins Ph.D., President & General Manager, KromaTiD Inc.

9:55 am Tech Talk 4: Fiber Vision Molecular Combing Platform

Aaron Bensimon Ph.D., CEO, Genomic Vision

10:10 am BREAK

10:30 am Lexicon breakout sessions report with discussion

Brett Robb, Ph.D., Scientific Director, RNA & Genome Editing, New England Biolabs

11:00 am Assay Qualification breakout session report with discussion

Samantha Maragh, Ph.D., Leader, NIST Genome Editing Program

11:30 am Data & Metadata breakout session report with discussion

Kendell Clement, Ph.D., Research Fellow, MGH/Harvard Medical School

12:00 pm Summary and closing remarks

12:30 pm Adjourn

1:30 – 3:30 pm Closed NIST Genome Editing Consortium Meeting (Portrait Room)

3:45 pm Shuttle departure from NIST to Hilton Gaithersburg