Sessions

How Do We Best Serve Rare and Small Patient Population
Disruptive and Novel Therapeutics for Oncology Indications
Reaching More Patients with CGT: Screening, Genomics, Databases and Patient Diversity
Decentralizing Advanced Therapies: Are We Moving Closer to Bedside Therapeutics?
Working With Patient Advocacy Organizations to Inform Development
Working With Qualified Treatment Centers for Best Patient Outcomes
Implementing Novel Development Strategies for Clinical Trials
What Is The Public and Patient Perception of Cell and Gene Therapies

How Do We Best Serve Rare and Small Patient Population

20 Jan 2023
11:00
Patient and Clinical Considerations Track
SPONSORED BY
PharmaLex GmbH (Biopharma excellence)

11:00am Chair Introduction

Chairperson TBC

 

11:05am Presentation: TBA

Wilson Bryan, Director, Office of Tissues and Advanced Therapies, FDA

 

11:20am Presentation: TBA

 Brad Carlin, Senior Advisor, Statistics and Data Science, PharmaLex – US

 

11:35am Presentation: A Parents’ Journey Through Drug Development”: How Angelman Syndrome is Making the Impossible Possible

  • This talk will discuss the journey of a motivated and sophisticated community who moved from a vision for transformative treatment to starting a company which launched the first in human ASO for Angelman syndrome.
  • This will incorporate a day in the life of parents as This type of roadmap is navigated

Allyson Berent, Chief Science Officer, Foundation for Angelman Syndrome Therapeutics (FAST)

 

11:50am Presentation: TBA

Sharon Hesterlee, Chief Research Officer, Muscular Dystrophy Association USA

 

12:05pm Closing Panel with Q&A

With all session participants

Speakers

Wilson Bryan
Director, Office of Tissues and Advanced Therapies
FDA
Brad Carlin
Senior Advisor, Statistics and Data Science
PharmaLex GmbH (Biopharma excellence)
Allyson Berent
Chief Science Officer
Foundation for Angelman Syndrome Therapeutics
Sharon Hesterlee
Executive Vice President, Chief Research Officer
Muscular Dystrophy Association USA