Rare Disease Symposium 2019 Header_v1.png

Rare Disease Research Symposium 2019 - Agenda


Click for Rare Disease Landing PageClick for Calls for Abstracts

With more than 7,000 rare diseases identified, next generation treatments are required to meet the needs of diverse patients.

Across our region, researchers and providers are developing precision approaches to diagnostics and treatment for rare conditions. National and international collaborations are leveraging data science to repurpose exiting drugs, new delivery mechanisms, and emerging technologies.


  • Research aimed at uncovering new mechanisms for rare disease treatment and improving the efficacy of existing approaches.

  • Strategies for bringing a profitable repurposed rare disease drug to the market.

  • Regulatory considerations needed in preclinical studies, generating validated data for INDs promoting clinical trial acceptance.

  • Orphan Drug Sales and marketing approaches to reach doctors and patients, reach internationally, etc.

  • Intellectual Property considerations for protecting revenue on an expiring patent.

  • Building Trust between investors and researchers to accelerate innovation, increasing startup activity.


8:00-9:00 Check-in and Light Breakfast

Ross A. Daly, Associate Professor of Journalism
Interim Dean of Liberal Arts and Science, Purchase College, SUNY
Michael Welling, Co-Founder and Chief Strategy Officer, Westchester Biotech Project

Emerging Therapeutics for Brain Hemorrhage: a Rare Disease with Not So Rare Consequences
Rajiv Ratan, MD, Ph.D
, Executive Director, Burke Neurological Institute

Update on the Rare Disease Research Hub
Antonio Molina-Pachon, Ph.D.
, Founder and CEO, GenCo Pharma

Keynote: Coordination of International Research on Rare Diseases

David A Pearce, Ph.D., President, Innovation and Research, Sanford Health
Vice Chair, Consortium Assembly, International Rare Disease Research Consortium (IRDiRC)

A Platform Technology Approach to Building a Drug Portfolio
Rick Huntress, Director, Business Development, Commercial Strategy, The Jackson Laboratory

Emerging Perspectives on Clinical Research in Rare Diseases


Lessons In Patient-Centricity From Rare Disease Clinical Trials
Dalfoni Banerjee
, Principal Consultant & CEO, 3Sixty Pharma Solutions


Regulatory Issues in Rare Disease Trials
Bethany Hills
, Partner, Morrison & Foerster, LLP

Digital Strategies for Global Drug Development
Antonio Biancardi
, Senior Manager, Technical Architect, Genpact

Journalism News Conference and One-on-One Interviews -
A Model Experience for Young Science Writers

Ross A. Daly, Associate Professor of Journalism, Interim Dean of Liberal Arts and Science
Purchase College, SUNY

Anil Vaidya, Associate Director, Global Submission Medical Writing
Pfizer CV/Metabolic/Rare Diseases

Bill Levine, Commercial Director, Genetic Engineering & Biotechnology News Life Sciences
Portfolio, Mary Ann Liebert Inc. Publishers


Patient Experiences
Roundtable: Taking the Next Steps
with NY BioPharma Networking Group, Doral Arrowwood


Antonio Molina-Pachon, Ph.D., Founder and CEO, GenCo Pharma
Anil Vaidya, Associate Director, Global Submission Medical Writing, Pfizer
Joanne Gere, Co-Founder and Executive Director, Westchester Biotech Project

Subject to Change
Click for Agenda from 2018 Symposium