Skip navigation
Agenda

Mapping Pre-Clinical and Clinical Timelines to CMC and CMC Regulatory Strategies

November 14-15, 2018
  • Washington, DC

Agenda

Want more agenda details? Download the brochure.

DAY ONE Wednesday, November 14, 2018

7:30

Registration and Continental Breakfast

8:30

Chairperson’s Welcome and Opening Remarks

Manal Morsy, MD, PhD, MBA
Senior Vice President and Head of Global Regulatory Affairs
Athersys, Inc.

Regulatory Perspectives on Emerging
Cell and Gene-Based Therapies

8:45

Understand the Current Regulatory Landscape of Cell and Gene-Based Therapies

Tejashri Purohit-Sheth
Director/DCEPT, Office of Tissues and Advanced Therapies, CBER
U.S. Food and Drug Administration

PANEL RMAT Submissions for Regenerative Medicine —
Enhanced FDA Engagement and Expedited Approval Paths

MODERATOR:

Manal Morsy, MD, PhD, MBA
Senior Vice President and Head of Global Regulatory Affairs
Athersys, Inc.

PANELISTS:

Tejashri Purohit-Sheth
Director/DCEPT, Office of Tissues and Advanced Therapies, CBER
U.S. Food and Drug Administration

Kaye Spratt, PhD
Senior Vice President, Regulatory Affairs
Abeona Therapeutics, Inc.

Anne-Virginie Eggimann
Vice President, Regulatory Science
bluebird bio, Inc.

10:15

Networking and Refreshment Break

10:45

Explore Industry Perspectives on Current and Incoming Regulations

Kaye Spratt, PhD
Senior Vice President, Regulatory Affairs
Abeona Therapeutics, Inc.

11:30

Learn Strategies for Successful BLA Submissions and Success

Scott Burger
Principal
Advanced Cell & Gene Therapy, LLC

12:15

Networking Luncheon

CMC Regulatory Considerations for
Biologics Development

1:30

Examine Analytical Methods for Biotechnology Comparability

Alicia Gilbert
Director of Regulatory Affairs – CMC
REGENXBIO Inc.

2:15

Strategies for Rapid Scale-Up of CMC Operations Without Compromising Regulatory Success

Alan Smith
Executive Vice President, Technical Operations
Bellicum Pharmaceuticals

3:00

Networking and Refreshment Break

3:30

PANEL Explore Differences between FDA and EMA’s Approach to CMC and Opportunities for Synergy

MODERATOR:

Mehrshid Alai
Head of Global CMC
Kite Pharma, a Gilead Company

PANELISTS:

Alan Smith
Executive Vice President, Technical Operations
Bellicum Pharmaceuticals

Scott Burger
Principal
Advanced Cell & Gene Therapy, LLC

Aby J. Mathew
Senior Vice President and Chief Technology Officer
BioLife Solutions Inc.

4:15

Spotlight on Ancillary Materials Navigating Ancillary Materials Across Regulatory Jurisdictions

Aby J. Mathew
Senior Vice President and Chief Technology Officer
BioLife Solutions Inc.

5:15

Close of Day One

Networking, Wine and Cheese Reception immediately following the final session on day one

DAY TWO Thursday, November 15, 2018

8:00

Continental Breakfast

8:30

Chairperson’s Review of Day One

Manal Morsy, MD, PhD, MBA
Senior Vice President and Head of Global Regulatory Affairs
Athersys, Inc.

Understand Pre-Clinical Success

8:45

Explore Regulatory Requirements Related to Product/Process Changes for Cell and Gene Therapies

Lisa Taylor
Gene and Cell Therapy Consultant,
Former Vice President – Head of Healthcare Law and Compliance
Juno Therapeutics

Elizabeth Smith
Senior Strategic Development Advisor – Retired
Juno Therapeutics

9:30

Understand the Linkages Between CMC and Pre-Clinical Work in Gene and Cell Therapy Programs

Speaker TBA

10:15

Networking and Refreshment Break

Optimize Clinical Trial Design for
Cell and Gene-Based Therapy

10:45

Clinical Trial Design for Expedited Pathway Success

Lisa Kadyk Ph.D
Associate Director, Therapeutics
California Institute for Regenerative Medicine

11:30

Learn to Select Alternative Endpoints to Measure Clinical Trial Success

Anthony Davies
Founder & CEO
Dark Horse Consulting Inc.

12:15

Close of Conference

Agenda

Want more agenda details? Download the brochure.

Want more agenda details? Download the brochure.

DAY ONE Wednesday, November 14, 2018

7:30

Registration and Continental Breakfast

8:30

Chairperson’s Welcome and Opening Remarks

Manal Morsy, MD, PhD, MBA
Senior Vice President and Head of Global Regulatory Affairs
Athersys, Inc.

Regulatory Perspectives on Emerging
Cell and Gene-Based Therapies

8:45

Understand the Current Regulatory Landscape of Cell and Gene-Based Therapies

Tejashri Purohit-Sheth
Director/DCEPT, Office of Tissues and Advanced Therapies, CBER
U.S. Food and Drug Administration

PANEL RMAT Submissions for Regenerative Medicine —
Enhanced FDA Engagement and Expedited Approval Paths

MODERATOR:

Manal Morsy, MD, PhD, MBA
Senior Vice President and Head of Global Regulatory Affairs
Athersys, Inc.

PANELISTS:

Tejashri Purohit-Sheth
Director/DCEPT, Office of Tissues and Advanced Therapies, CBER
U.S. Food and Drug Administration

Kaye Spratt, PhD
Senior Vice President, Regulatory Affairs
Abeona Therapeutics, Inc.

Anne-Virginie Eggimann
Vice President, Regulatory Science
bluebird bio, Inc.

10:15

Networking and Refreshment Break

10:45

Explore Industry Perspectives on Current and Incoming Regulations

Kaye Spratt, PhD
Senior Vice President, Regulatory Affairs
Abeona Therapeutics, Inc.

11:30

Learn Strategies for Successful BLA Submissions and Success

Scott Burger
Principal
Advanced Cell & Gene Therapy, LLC

12:15

Networking Luncheon

CMC Regulatory Considerations for
Biologics Development

1:30

Examine Analytical Methods for Biotechnology Comparability

Alicia Gilbert
Director of Regulatory Affairs – CMC
REGENXBIO Inc.

2:15

Strategies for Rapid Scale-Up of CMC Operations Without Compromising Regulatory Success

Alan Smith
Executive Vice President, Technical Operations
Bellicum Pharmaceuticals

3:00

Networking and Refreshment Break

3:30

PANEL Explore Differences between FDA and EMA’s Approach to CMC and Opportunities for Synergy

MODERATOR:

Mehrshid Alai
Head of Global CMC
Kite Pharma, a Gilead Company

PANELISTS:

Alan Smith
Executive Vice President, Technical Operations
Bellicum Pharmaceuticals

Scott Burger
Principal
Advanced Cell & Gene Therapy, LLC

Aby J. Mathew
Senior Vice President and Chief Technology Officer
BioLife Solutions Inc.

4:15

Spotlight on Ancillary Materials Navigating Ancillary Materials Across Regulatory Jurisdictions

Aby J. Mathew
Senior Vice President and Chief Technology Officer
BioLife Solutions Inc.

5:15

Close of Day One

Networking, Wine and Cheese Reception immediately following the final session on day one

DAY TWO Thursday, November 15, 2018

8:00

Continental Breakfast

8:30

Chairperson’s Review of Day One

Manal Morsy, MD, PhD, MBA
Senior Vice President and Head of Global Regulatory Affairs
Athersys, Inc.

Understand Pre-Clinical Success

8:45

Explore Regulatory Requirements Related to Product/Process Changes for Cell and Gene Therapies

Lisa Taylor
Gene and Cell Therapy Consultant,
Former Vice President – Head of Healthcare Law and Compliance
Juno Therapeutics

Elizabeth Smith
Senior Strategic Development Advisor – Retired
Juno Therapeutics

9:30

Understand the Linkages Between CMC and Pre-Clinical Work in Gene and Cell Therapy Programs

Speaker TBA

10:15

Networking and Refreshment Break

Optimize Clinical Trial Design for
Cell and Gene-Based Therapy

10:45

Clinical Trial Design for Expedited Pathway Success

Lisa Kadyk Ph.D
Associate Director, Therapeutics
California Institute for Regenerative Medicine

11:30

Learn to Select Alternative Endpoints to Measure Clinical Trial Success

Anthony Davies
Founder & CEO
Dark Horse Consulting Inc.

12:15

Close of Conference