2019 AGENDA — Day One

07:00 - 08:0060 mins

Networking

Continental Breakfast

07:00 - 08:0060 mins

Main Agenda

Conference Registration Open

08:00 - 08:1515 mins

Main Agenda

Conference Chair’s Opening Remarks

Robert Metz - Senior VP, Business Operations & External Affairs, Horizon Pharma

08:15 - 15:15420 mins

Partnering Meetings

One-to-one meetings

08:15 - 09:0045 mins

Keynotes

Patient and Caregiver Perspectives Keynote Panel

Nicole Boice - Founder, Global Genes
Jocelyn Duff - Founder & CEO, Cure CMT4J
Keisha Greaves - Founder & Owner, Girls Chronically Rock, and State Ambassador, Muscular Dystrophy Association
Bo Bigelow - Chairman, Foundation for USP7-Related Diseases
Glenn O’Neill - President, Cure Sanfilippo Foundation

09:00 - 09:4545 mins

Keynotes

Science Luminaries Keynote

Lyndsey Scull - Vice President, Communications, Alliance for Regenerative Medicine
Alan Beggs, PhD - Director, The Manton Center for Orphan Disease Research, Division of Genetics & Genomics, Boston Children’s Hospital
Sam Wadsworth - Chief Scientific Officer, Ultragenyx Gene Therapy
Richard Horgan - Founder & President, Cure Rare Disease

09:45 - 10:3045 mins

Keynotes

Regulatory and Policy Update Address Keynote

John Hallinan - Chief Business Officer, Massachusetts Biotechnology Council
Janet Maynard, MD, MHS - Director, Office of Orphan Products Development, Food and Drug Administration
Samir Shaikh - Deputy Director, Patient Affairs Staff, Office of the Commissioner, U.S. Food and Drug Administration
Martine Zimmermann - Global Head, Regulatory Affairs, Alexion
Lucas Kempf, MD - Acting Associate Director, Rare Diseases Program, OND, CDER, FDA

10:30 - 11:0030 mins

Networking

Networking and Refreshment Break

11:00 - 11:4545 mins

Keynotes

Focus on Funding Keynote Panel

Jeremy Springhorn - Chief Business Officer, Syros Pharmaceuticals
Phil Ross - Vice Chairman, Investment Banking, JP Morgan
Chris Garabedian - Chairman and CEO, Xontogeny
Neil Littman - Vice President, Business Development, Notable
Yury Kukushkin - Principal, 4BIO Capital

11:45 - 12:3045 mins

Keynotes

Technology Spotlight Keynote

Jen Beachell - President & Rare Disease Executive, Beachell Consulting LLC
John Reynders - Vice President, R&D Strategy, Program Management & Data Sciences, Alexion Pharmaceuticals, Inc.
Tim Considine - SVP, Strategic Development, Recursion Pharmaceutical
Niven Narain, PhD - Co-Founder, President & CEO, Berg Health
Tiffany Freitas - Chief Business Officer, PathAI

12:30 - 12:4515 mins

Main Agenda

Massachusetts Legislative Update — An Act to Create a Rare Disease Advisory Council

Joseph McKenna - State Representative, 18th Worcester District, Massachusetts House of Representatives

12:45 - 13:4560 mins

Networking

Networking Luncheon

13:45 - 14:0015 mins

New Launch & Commercialization

Track Chair's Opening Remarks

Robert Metz - Senior VP, Business Operations & External Affairs, Horizon Pharma

13:45 - 14:0015 mins

Partnering & Investment

Track Chair's Opening Remarks

Chris Garabedian - Chairman and CEO, Xontogeny

13:45 - 14:0015 mins

Reimbursement & Access

Track Chair's Opening Remarks

Mary Roberts - Divisional Vice President, Business Development, Envoy Health

13:45 - 14:0015 mins

Patient-Driven Progress

Track Chairs' Opening Remarks

Jocelyn Duff - Founder & CEO, Cure CMT4J
Kristin Anthony - President, PTEN Hamartoma Tumor Syndrome Foundation

14:00 - 14:4545 mins

Partnering & Investment

Investing Today in Tomorrow’s Life-Transforming Treatments — Opportunities for Venture Capitalists to Move Science to Medicine

Michael Pistone - Director, Innovation Acceleration, Innovation Ventures, Cincinnati Children's Hospital
Imran Babar, PhD - Chief Business Officer, Cydan II Inc.
Paul Oppold - Vice President, Investments, Roivant Pharma
Shinchiro Fuse - Managing Director, MPM Capital
Mike Zhao - Vice President, Healthcare, MSQ Ventures

14:00 - 14:4545 mins

Reimbursement & Access

Overcome Critical Challenges in Expanded Access Programs — New Models and Emerging Regulations

Stuart Bell, PhD - Vice President Consulting, Medicines Access, Inceptua

14:00 - 14:4545 mins

New Launch & Commercialization

Build a Cross Functional Foundation for Success in Orphan Product Launch

Richard Williams, RPh - Senior Vice President, Pharmaceutical Innovation and Insights, CSI Group
Rob Roth - Vice President, Marketing & Communication, Albireo
Philina Lee - Vice President, Commercial Strategy & Operations, Blueprint Medicines
Lora Marden - Director, U.S. Gamifant Marketing, Sobi North America

14:00 - 14:4545 mins

Patient-Driven Progress

The Role of Disease Awareness Campaigns for Rare Diseases

Jodie Gillon - Vice President, Patient Advocacy & Clinical Affairs, Abeona Therapeutics
Isabelle Lousada - Founder & CEO, Amyloidosis Research Consortium
Mary McGowan, MHRM - Executive Director, The Myositis Association

14:45 - 15:3045 mins

Reimbursement & Access

Leverage RealWorld Evidence for Improved Outcomes and Access

Steve Silvestri - Director, Public Policy, EveryLife Foundation for Rare Diseases
Nneka Onwudiwe, PharmD, PhD, MBA - Founder and Chief Executive Officer, Pharmacoeconomics Consultants of America (PECA) LLC.
Pat Furlong - Founding President & CEO, Parent Project Muscular Dystrophy
Alexander Cole, DSc, MPH - Executive Director and Global Head Epidemiology, Alexion Pharmaceuticals

14:45 - 15:3045 mins

Partnering & Investment

Partnering and Meeting Time

14:45 - 15:3045 mins

New Launch & Commercialization

Leverage Novel Collaborations in Orphan Drug Commercialization

Neil Littman - Vice President, Business Development, Notable
Robert Metz - Senior VP, Business Operations & External Affairs, Horizon Pharma

14:45 - 15:3045 mins

Patient-Driven Progress

Discuss Opportunities for Patients and Advocates to Engage in Early Research and Scientific Development

Florian Eichler, MD - Director, Center for Rare Neurological Diseases, Massachusetts General Hospital
Patricia Musolino - Assistant Professor of Neurology, Genetic, Vascular & Critical Care, Center for Genomic Medicine, MGH-Harvard Medical School
Kathleen O’Sullivan-Fortin, Esq – ALD/AMN - Advocate, Member Board of Directors, ALD Connect
Robert Burgess, PhD - Professor, JAX Center for Precision Genomics, Jackson Lab
Rebecca Oberman - Executive Director, ML4 Foundation

16:00 - 16:4545 mins

Partnering & Investment

Early-Stage Investing Considerations — Leverage Bio/Pharma’s Expertise and Resources to Find and Build a Patient Network

Yael Weiss - Vice President, Business Development, Ultrageynx
Andy Holt - VP, Business Development & Manufacturing Support, Asklepios BioPharmaceuticals
Eric Waehner - VP, Business Development, Recordati Rare Diseases, Inc.
Debra Miller - CEO and Founder, CureDuchenne
Eric Gomez - Associate Director, Asset Acquisition, BridgeBio

16:00 - 16:4545 mins

Reimbursement & Access

Discuss Innovative Methods for Demonstrating Value to Support Market Access

Clark Paramore - Head of Value Demonstration, bluebird bio
Jen Beachell - President & Rare Disease Executive, Beachell Consulting LLC

16:00 - 16:4545 mins

Patient-Driven Progress

Develop New Models for Patient-Centric Registries

Pat Furlong - Founding President & CEO, Parent Project Muscular Dystrophy

16:00 - 16:4545 mins

New Launch & Commercialization

Employ Optimal Distribution Strategies to Ensure Success in Product Launch

Liz Minko - Head, US Oncology Trade & Distribution, Takeda

16:45 - 17:3045 mins

Patient-Driven Progress

Discuss the Impact of Externally-Led Patient-Focused Drug Development Meetings

Brett Kopelan - Executive Director, Debra of America
Lucas Kempf, MD - Acting Associate Director, Rare Diseases Program, OND, CDER, FDA

16:45 - 17:3045 mins

New Launch & Commercialization

Build a Comprehensive Marketing Strategy for Effective Launch

Neil Keene - Director, Digital HCP Marketing, Ocaliva, Intercept Pharmaceuticals
Zach Crouch - Senior Director, Marketing Brand Lead, Blueprint Medicines
Shaili Shah - Associate Director, US Marketing, Intercept Pharmaceuticals

16:45 - 17:3045 mins

Partnering & Investment

Partnering and Meeting Time

16:45 - 17:3045 mins

Reimbursement & Access

Review Value-Based and Other Evolving Reimbursement Models

Leora Schiff - Principal, Altius Strategy Consulting, LLC
Kevin Mayo, PhD, EJD - Vice President & Head, Global Market Access Gene Therapies, PTC Therapeutics, Inc.
Dan Kennedy - Executive Director, Business Development, Achillion

17:30 - 18:3060 mins

Networking

Close of Day One: Networking, Wine and Cheese Reception Commences