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Informa
Key Sessions

Nicole Boice

Patient and Caregiver Perspectives Keynote Panel

Global Genes

Janet Lambert

Science Luminaries Keynote

Alliance for Regenerative Medicine

John Hallinan

Regulatory and Policy Update Address Keynote

Massachusetts Biotechnology Council

Jeremy Springhorn

Focus on Funding Keynote Panel

Syros Pharmaceuticals

Jen Beachell

Technology Spotlight Keynote

Beachell Consulting LLC

Partnering and Meeting Time

Jun 13
Show Filter
07:00 - 08:00 60 mins
Networking
Continental Breakfast
07:00 - 08:00 60 mins
Main Agenda
Conference Registration Open
08:00 - 08:15 15 mins
Main Agenda
Chair’s Opening Remarks
  • Alison Silva - CEO, Cotinga Pharmaceuticals
08:15 - 15:15 420 mins
Partnering Meetings
One-to-one meetings
08:15 - 09:00 45 mins
Info
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

Explore New Developments in Cures, Community, Collaboration and the Care Continuum for Patients and Caregivers

09:00 - 09:45 45 mins
Info
Keynotes
Science Luminaries Keynote
  • Moderator Janet Lambert - CEO, Alliance for Regenerative Medicine
  • Presenter David Lennon, PhD - President, AveXis
  • Presenter Alan Beggs, PhD - Director, The Manton Center for Orphan Disease Research, Division of Genetics & Genomics, Boston Children’s Hospital
  • Presenter Sam Wadsworth - Chief Scientific Officer, Ultragenyx Gene Therapy

Advancements in Gene Therapy that Are Tearing Down Barriers to Discovery in Rare Disease

09:45 - 10:30 45 mins
Info
Keynotes
Regulatory and Policy Update Address Keynote
  • Moderator John Hallinan - Chief Business Officer, Massachusetts Biotechnology Council
  • Panelist Janet Maynard, MD, MHS - Director, Office of Orphan Products Development, Food and Drug Administration
  • Panelist Samir Shaikh - Deputy Director of Patient Affairs Staff, Office of Medical Products and Tobacco, U.S. Food and Drug Administration
  • Panelist Martine Zimmermann - Global Head, Regulatory Affairs, Alexion

Legislative Updates and New Initiatives within the FDA’s Orphan Drug Program

10:30 - 11:00 30 mins
Networking
Networking and Refreshment Break
11:00 - 11:45 45 mins
Info
Keynotes
Focus on Funding Keynote Panel
  • Moderator Jeremy Springhorn - Chief Business Officer, Syros Pharmaceuticals
  • Panelist Phil Ross - Vice Chairman, Investment Banking, JP Morgan
  • Panelist Chris Garabedian - Chairman and CEO, Xontogeny
  • Panelist Neil Littman - Vice President, Business Development, Notable Labs
  • Panelist Yury Kukushkin - Principal, 4BIO Capital

The Impact of Disruptive Investors, Drivers of Value and Funding Models for Success

11:45 - 12:30 45 mins
Info
Keynotes
Technology Spotlight Keynote
  • Moderator Jen Beachell - President & Rare Disease Executive, Beachell Consulting LLC
  • Panelist John Reynders - Vice President, R&D Strategy, Program Management & Data Sciences, Alexion Pharmaceuticals, Inc.
  • Panelist Tim Considine - SVP, Strategic Development, Recursion Pharmaceutical
  • Panelist Niven Narain, PhD - Co-Founder, President & CEO, Berg Health
  • Panelist Tiffany Freitas - Chief Business Officer, PathAI

Artificial Intelligence (AI) and Advanced Data Analytics — Reshaping Complex Diagnostics and Decision-Making in Rare Disease Drug Development

12:30 - 13:30 60 mins
Networking
Networking Luncheon
13:30 - 13:45 15 mins
Reimbursement & Access
Track Chair's Opening Remarks
13:30 - 13:45 15 mins
Partnering & Investment
Track Chair's Opening Remarks
  • Chris Garabedian - Chairman and CEO, Xontogeny
13:30 - 13:45 15 mins
New Launch & Commercialization
Track Chair's Opening Remarks
  • Robert Metz - Senior VP, Business Operations & External Affairs, Horizon Pharma
13:30 - 13:45 15 mins
Patient-Driven Progress
Track Chairs' Opening Remarks
  • Kristin Anthony - President, PTEN Hamartoma Tumor Syndrome Foundation
  • Jocelyn Duff - Founder & CEO, Cure CMT4J
13:45 - 14:30 45 mins
Reimbursement & Access
Overcome Critical Challenges in Expanded Access Programs — New Models and Emerging Regulations
  • Stuart Bell, PhD - Vice President Consulting, Medicines Access, Inceptua
13:45 - 14:30 45 mins
Partnering & Investment
Investing Today in Tomorrow’s Life-Transforming Treatments — Opportunities for Venture Capitalists to Move Science to Medicine
  • Moderator Michael Pistone - Director, Innovation Acceleration, Innovation Ventures, Cincinnati Children's Hospital
  • Panelist Imran Babar, PhD - Chief Business Officer, Cydan II Inc.
13:45 - 14:30 45 mins
New Launch & Commercialization
Build a Cross Functional Foundation for Success in Orphan Product Launch
  • Moderator Suzette DiMascio, CHE, CMCE, CPC - President and CEO, CSI Specialty Group
  • Panelist Wendy Erler - Vice President, Head Patient Advocacy, Alexion Pharmaceuticals
  • Panelist Rob Roth - Vice President, Marketing & Communication, Albireo
  • Panelist Philina Lee - Vice President, Commercial Strategy & Operations, Blueprint Medicines
  • Panelist Lora Marden - Director, U.S. Gamifant Marketing, Sobi North America
13:45 - 14:30 45 mins
Patient-Driven Progress
The Role of Disease Awareness Campaigns for Rare Diseases
  • Jodie Gillon - Global Medical Lead, Patient Engagement Rare Diseases, Pfizer
  • Isabelle Lousada - Founder & CEO, Amyloidosis Research Consortium
  • Mary McGowan, MHRM - Executive Director, The Myositis Association
14:30 - 15:15 45 mins
Reimbursement & Access
Leverage RealWorld Evidence for Improved Outcomes and Access
  • 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
14:30 - 15:15 45 mins
Partnering & Investment
Partnering and Meeting Time
14:30 - 15:15 45 mins
New Launch & Commercialization
Leverage Novel Collaborations in Orphan Drug Commercialization
  • Bruce Bloom - CEO, Cures Within Reach
  • Neil Littman - Vice President, Business Development, Notable Labs
  • Robert Metz - Senior VP, Business Operations & External Affairs, Horizon Pharma
14:30 - 15:15 45 mins
Patient-Driven Progress
Discuss Opportunities for Patients and Advocates to Engage in Early Research and Scientific Development
  • Moderator Florian Eichler, MD - Director, Center for Rare Neurological Diseases, Massachusetts General Hospital
  • Panelist Patricia Musolino - Assistant Professor of Neurology, Genetic, Vascular & Critical Care, Center for Genomic Medicine, MGH-Harvard Medical School
  • Panelist Kathleen O’Sullivan-Fortin, Esq – ALD/AMN - Advocate, Member Board of Directors, ALD Connect
  • Panelist Robert Burgess, PhD - Professor, JAX Center for Precision Genomics, Jackson Lab
  • Panelist Rebecca Oberman - Executive Director, ML4 Foundation
15:45 - 16:30 45 mins
Reimbursement & Access
Discuss Innovative Methods for Demonstrating Value to Support Market Access
  • Clark Paramore - Head of Value Demonstration, bluebird bio
15:45 - 16:30 45 mins
Partnering & Investment
Early-Stage Investing Considerations — Leverage Bio/Pharma’s Expertise and Resources to Find and Build a Patient Network
  • Panelist Andy Holt - VP, Business Development & Manufacturing Support, Asklepios BioPharmaceuticals
  • Panelist Eric Waehner - VP, Business Development, Recordati Rare Diseases, Inc.
  • Panelist Debra Miller - CEO and Founder, CureDuchenne
  • Panelist Yael Weiss - Vice President, Business Development, Ultrageynx
15:45 - 16:30 45 mins
New Launch & Commercialization
Employ Optimal Distribution Strategies to Ensure Success in Product Launch
  • Alex Ross - Director, Trade Relations, Horizon Pharma
15:45 - 16:30 45 mins
Patient-Driven Progress
Develop New Models for Patient-Centric Registries
  • Pat Furlong - Founding President & CEO, Parent Project Muscular Dystrophy
16:30 - 17:15 45 mins
Reimbursement & Access
Review Value-Based and Other Evolving Reimbursement Models
  • Leora Schiff - Principal, Altius Strategy Consulting, LLC
16:30 - 17:15 45 mins
Partnering & Investment
Partnering and Meeting Time
16:30 - 17:15 45 mins
New Launch & Commercialization
Build a Comprehensive Marketing Strategy for Effective Launch
  • Moderator Neil Keene - Director, Digital HCP Marketing, Ocaliva, Intercept Pharmaceuticals
  • Panelist Zach Crouch - Brand Lead, Blueprint Medicines
  • Panelist Shaili Shah - Associate Director, US Marketing, Intercept Pharmaceuticals
16:30 - 17:15 45 mins
Patient-Driven Progress
Discuss the Impact of Externally-Led Patient-Focused Drug Development Meetings
  • Brett Kopelan - Executive Director, Debra of America
17:15 - 18:15 60 mins
Networking
Close of Day One: Networking, Wine and Cheese Reception Commences