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April 30 – May 2, 2018

Boston, MA
The Countdown Is On – 1 Week To Go!

Speakers

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Michael Binks
VP, Rare Disease & Clinical Research
Pfizer

Day One

Tuesday, May 1, 2018

14.15 | Defining Robust Clinical Development Strategies to Deal With the Unique Considerations of Rare Disorders

Scott Burger
Principal
Advanced Cell & Gene Therapy

Vivian Choi
Head of Gene Therapy Research
Shire

Day Two

Wednesday May 2, 2018

12.00 | Establishing Robust Approaches to Quantify AAV Vectors and Build a Robust Path to the Clinic

Olivier Danos
CSO
REGENXBIO

Day Two

Wednesday May 2, 2018

11.30 | The NAV Platform of AAV Vectors for Gene Therapy: Current Applications & Beyond

Mary Dunkle
VP, Educational Initiatives
NORD

Day Two

Wednesday May 2, 2018

13.30 | The Patient Perspective – Understanding Common Misconceptions & Frequently Asked Questions About Gene Therapy

Richard Emmons
Partner
Burns & Levinson

Francois Gianelli
Senior Scientist, Regulatory
Voisin Consulting

Michael Gladstone
Principal
Atlas Ventures

Day Two

Wednesday May 2, 2018

16.00 | Panel Discussion: The Investor’s Perspective – Evaluating How VCs View the Gene Therapy Field

Philip Gregory
CSO
Bluebird Bio

Day One

Tuesday, May 1, 2018

08.30 | Cross-Industry Perspectives on Establishing Gene Therapies as Commercially Viable Therapeutics

Al Hawkins
Founder & CEO
Milo Biotechnology

Sharon Hesterlee
Research Project Lead, Gene Therapy
Bamboo Therapeutics, A Pfizer Company

Day Two

Wednesday May 2, 2018

14.00 | Using Feedback from Patient Groups to Shape Gene Therapy Protocols

Katherine High
President & Head of R&D
Spark Therapeutics

Day One

Tuesday, May 1, 2018

08.30 | Cross-Industry Perspectives on Establishing Gene Therapies as Commercially Viable Therapeutics

08.00 | Keynote: Delivering on the Promise of Gene Therapies: Navigating Key Decision Points in the Path to a Groundbreaking Approval

Sven Kili
VP, Head of Cell & Gene Therapy Development
GSK

Day One

Tuesday, May 1, 2018

11.20 | Establishing Effective Manufacturing & Regulatory Standards

Day Two

Wednesday May 2, 2018

10.00 | Panel Discussion: Global Commercialization Considerations

09.00 | Establishing Value for Gene Therapy Products by Implementing Effective Reimbursement Strategies

Jason Mallory
Director, Gene Therapy Clinical Development
GSK

Day One

Tuesday, May 1, 2018

16.15 | Navigating the Challenges of Gene Therapy Drug Development for Ultra-Rare Conditions

Chris Mason
CSO
AvroBio

Day One

Tuesday, May 1, 2018

17.45 | Chair’s Closing Remarks

07.50 | Chair’s Opening Remarks

Day Two

Wednesday May 2, 2018

16.30 | Chair’s Closing Remarks

08.50 | Chair’s Opening Remarks

Alison McVie-Wylie
Senior Director, Gene Therapy and Biologics Pharmacology, Rare Diseases
Sanofi

Day One

Tuesday, May 1, 2018

13.45 | Developing Robust Preclinical Development Strategies to Deal With the Unique Considerations of Gene Therapies

Gopalan Narayanan
VP, Disruptive Biologics
Voisin Consulting

Steven Paul
President & CEO
Voyager Therapeutics

Day One

Tuesday, May 1, 2018

08.30 | Cross-Industry Perspectives on Establishing Gene Therapies as Commercially Viable Therapeutics

Markus Peters
CCO
Agilis Biotherapeutics

Day Two

Wednesday May 2, 2018

10.00 | Panel Discussion: Global Commercialization Considerations

09.30 | Evaluating Potential Pricing Strategies for Gene Therapies

Patrick Rivers
SVP, Research
Aquilo Capital Management

Day Two

Wednesday May 2, 2018

16.00 | Panel Discussion: The Investor’s Perspective – Evaluating How VCs View the Gene Therapy Field

Jude Samulski
VP, Gene Therapy
Pfizer

Day One

Tuesday, May 1, 2018

11.35 | Comparing Manufacturing Methods for their Suitability for Long-Term Commercial Manufacturing

Athena Countouriotis
CMO
Adverum

Day Two

Wednesday May 2, 2018

15.30 | Advancing Gene Therapies to Patients by Forging Mutually Beneficial Alliances

Jonathan Schwartz
CMO
Rocket Pharmaceuticals

Day Two

Wednesday May 2, 2018

14.30 | Gene Therapy Clinical Trials: Can They be Compelling for Patients/Families, Investigators and Health Authorities?

Kaye Spratt
VP, Regulatory
Abeona Therapeutics

Day One

Tuesday, May 1, 2018

16.45 | Assessing the Key Advantages and Disadvantages of Accelerated Approval Pathways

David Williams
SVP & CSO
Boston Children’s Hospital

Day One

Tuesday, May 1, 2018

14.45 | Investigating the Key Factors Influencing the Clinical Development of Gene Therapies

Steven Zelenkofske
CMO
uniQure

Day One

Tuesday, May 1, 2018

09.30 | Evolving a Strategy to Narrow Down the Potential Scope of Development in the Gene Therapy Space

Casey Maguire
Assistant Professor of Neurology
Harvard Medical School

Alvaro Amorrortu
COO
Solid Biosciences

Day One

Tuesday, May 1, 2018

12.05 | Strategic Decision Making For Scaling Gene Therapy Manufacturing

Gumei Liu
Reviewer & Project Officer, Office of Orphan Products Development
FDA

Day One

Tuesday, May 1, 2018

17.15 | FDA Office of Orphan Products Development: Core Programs to Facilitate Gene Therapy Development for Rare Diseases

Manuel Duval
Director, Data Science & Bioinformatics
Scientist.com

Day One

Tuesday, May 1, 2018

12.35 | Assembling the Range of Resources Required for Rare Disease R&D Organizations

Katherine Stueland
CCO
Invitae

Day One

Tuesday, May 1, 2018

15.15 | Talk Details to be Confirmed