Conference on Rare Disease and Orphan Drugs

1st and 2nd October
Register Now Become a Speaker

OUR SPEAKERS

Jonathan Morton

Rare Disease Consultant

AMICULUM

Rich Horgan

Founder and President

Cure Rare Disease

Lydia L. Seiders

Patient Education Council Member

Aplastic Anemia and MDS International Foundation

Kelly Austin

Founder, Regulatory Specialist (ODD, Fast Track, Breakthrough, RMAT)

KLA Breakthrough Consulting

Harsha K Rajasimha

Founder and CEO

Jeeva Informatics Solutions

Alice Rathjen

Co-Founder and CEO

DNA Compass

Gavin Jones

Director of Rare Disease

OPEN Health

Hector De la Riva

Commercial Lead Ocular Rare Disease – Gene Therapy

Novartis

Diana Stefani-Hunyady

Co-Founder

Life Key Sciences LLC

Christine Von Raesfeld

Founder and CEO

People with Empathy

Shahar Keinan

CEO and Co-Founder

POLARISqb

Dan Donovan

Founder & CEO

rareLife solutions

CONFERENCE PROGRAM

  8:00 AM EST
Registration (Telephonic And Virtual)
  8:30 AM EST
Introduction To DBMR
  9:00 AM EST
the Rare Disease Community for the Long Haul
Dan Donovan | Founder & CEO - rareLife solutions
  10:00 AM EST
Rethinking our approach to rare disease communications during COVID-19 and beyond
Jonathan Morton | Rare Disease Consultant - AMICULUM
Agency Lead - Comradis Limited
  11:15 AM EST
Every BODY is unique: A patient perspective into precision medicine
Christine Von Raesfeld | Founder and CEO - People with Empathy
Technical Expert Panelist - Centers for Medicare & Medicaid Services
  12:30 PM EST
Advocacy and the need to hear the rare disease patients voice within strategic efforts
Lydia L. Seiders | Patient Education Council Member Aplastic Anemia and MDS International Foundation
Advisor - Liv4TheCure
Advisor - Rare Disease Ghana Initiative
  1:45 PM EST
A Case Study (Epidiolex) Demonstrating How to Use Special FDA Designations to Efficiently Reach the US Market
Kelly Austin | Founder, Regulatory Specialist (ODD, Fast Track, Breakthrough, RMAT)
KLA Breakthrough Consulting
  3:00 PM EST
The Future of Rare Diseases: Developing and Accessing the Next Generation of Therapies
Rich Horgan | Founder and President - Cure Rare Disease
  4:15 PM EST
Federated data solutions for Accessing rare genomes across borders
Alice Rathjen | Co-Founder and CEO - DNA Compass
  5:15 PM EST
How to Commercialize an Orphan Drug
  6:15 PM EST
Rare vs. Common Diseases
  7:00 PM EST
DBMR Quantum Leap Awards
  8:30 AM EST
Registration
  9:30 AM EST
Challenges being faced by Orphan drug development Companies
  10:00 AM EST
Succeeding with Star Wars Medicine - Translating the science and value of Gene Therapies
Gavin Jones | Director of Rare Disease - OPEN Health
  11:15 AM EST
Gene Therapy, a New Paradigm in Ocular Rare Diseases
Hector De la Riva | Commercial Lead Ocular Rare Disease – Gene Therapy
Novartis
  12:30 PM EST
Treatment and Advanced Therapies for Rare Diseases
Shahar Keinan | CEO and Co-Founder - POLARISqb
  1:45 PM EST
Social Entrepreneurship in Rare Diseases: It takes concerted efforts between technology and advocacy to accelerate therapies for rare diseases
Harsha K Rajasimha | Founder and CEO - Jeeva Informatics Solutions
Founder and Chairman IndoUSrare
Member of the Board of Advisors - Synergy
  3:00 PM EST
Patient involvement in drug development for rare diseases
Diana Stefani-Hunyady | Head of Global Medical Affairs - Viela Bio
Co-Founder - Life Key Sciences LLC
  4:15 PM EST
Examining industry strategies for leveraging patient data and AI to advance rare disease patient identification, R&D, and commercialization
  5:15 PM EST
Regulatory Framework for Drugs for Rare Diseases
  6:15 PM EST
Rare Mental and Behavioural Disorders
  7:00 PM EST
DBMR Closing Remarks