December 6, 2023

Welcome to hubXchange’s Rare Diseases Clinical Strategies East Coast 2023, bringing together executives from pharma and biotech to address and find solutions to the key issues faced in the cardiovascular drug discovery space, through a series of roundtable discussions.

Discussion topics will cover Clinical Biomarkers, Preclinical Studies, Clinical Development, Clinical Operations

Take advantage of this unique highly interactive meeting format designed for maximum engagement and collaboration with your peers.

Please note this is an In-Person meeting.

VENUE DETAILS: Hilton Boston Woburn Hotel, 2 Forbes Road, Woburn MA 01801


  • Identifying and validating novel biomarkers and end points for rare diseases
  • Developing biomarkers that accurately reflect disease status or treatment response while minimizing false positives and false negatives
  • Innovation and development of new gene transfer technologies:
    Managing potential immunogenicity risks and leveraging both preclinical and clinical data for best solutions
  • Overcoming the absence of animal models
  • Challenges in designing and conducting clinical trials in rare diseases
  • What is the best practice to ensure your rare disease trial is a success?
  • Understand the patient barriers within rare disease
  • Innovative data collection methods in rare disease trials: remote monitoring, wearables, and digital health

Full Xchange Agenda

Click on each track for detailed agenda

Clinical Biomarkers

Titles and Bullets
08:00 – 08:30
08:30 – 09:00

Opening Address & Keynote Presentation

09:05 – 10:05

Identifying and validating novel biomarkers and end points for rare diseases 

10:10 – 10:40
1-2-1 Meetings/Networking Break
10:40 – 11:10
1-2-1 Meetings / Networking Break
11:10 – 11:20
Morning Refreshments
11:20 – 12:20

Sponsor-led Roundtable

12:20 – 13:20

Networking Lunch

13:20 – 13:50
Spotlight Presentation
13:55 – 14:25
1-2-1 Meetings / Networking Break
14:25 – 14:55
1-2-1 Meetings / Networking Break
15:00 – 15:30

Poster Session

15:35 – 16:35
Defining biomarkers early in development to accelerate the path to clinical treatments for rare genetic disorders
  • Importance of clinical biomarkers in rare disease drug development
  • Innovative methods and techniques to define biomarkers
  • Power of patient organizations and advocacy groups in biomarker development

Vice President, Head of Rare Diseases and New Modalities, BIAL

Smitha is the Vice-President, Head of New Modalities and Rare Diseases at Bial. Innovative treatments are essential to address the high unmet medical needs in Rare Diseases. Prior to joining Bial, Smitha created the strategy and led the new modalities group for developing novel medicines for pain at Grunenthal. Prior to that, she was in the Genomic Medicine Unit at Sanofi Genzyme. Smitha received her Ph.D. in Neuroscience from Columbia University, NY, did her postdoctoral fellowship in University of California in San Francisco, CA, and was a Research Scientist at the Whitehead and Picower Institutes, MIT.

Smitha Jagadish
16:35 – 17:35
Evening Drinks Reception


East Coast Rare Diseases Clinical STRATEGIES Xchange | East Coast 2023