Implementing Novel Development Strategies for Clinical Trials

18 Jan 2023
Patient and Clinical Considerations Track
Syneos Health

1:30pm Chair Introduction

Samik Basu, Chief Scientific Officer, Cabaletta Bio


1:35pm Presentation: Begin with the End in Mind: Clinical Trial Readiness in Rare Disease

  • Discuss the epidemiology of rare disease, clinical and economic burden, and challenges rare disease face with a traditional drug development model
  • Discuss precision therapies and implications different approaches have on the traditional clinical trial model
  • Discuss the role of clinical trial readiness studies in clinical trial development and patient community engagement on the pathway to approval

Kimberly Goodspeed, Associate Professor, University of Texas SouthWestern


1:50pm Presentation: Product Development with the End-In-Mind: How Commercial Realities Should Inform Tour Clinical & Evidence Generation Programs

  • Over the last few years, more than two dozen cell and gene therapies have launched in the US. Many of these CGTs have faced unique challenges post-approval
  • With an increased interest in development of such therapies, manufacturers must understand the specific hurdles associated with different treatments. These hurdles can diminish the potential of CGTs and limit the value they can bring not only to treatment developers, but also to patients
  • In this presentation, Syneos Health will dissect six key commercial challenges faced by CGTs which manufacturers must address in order to ensure success and outline what steps should be taken during the clinical development process to better prepare for commercialization

Pete Robinson, Vice President, Medical and Scientific Management Head, Cell and Gene Therapy Consortium, Syneos Health


2:05pm Presentation: Real World Evidence Impact on Clinical Development Strategy Design

  • Types and sources of real world data/real world evidence relevant for advanced therapies
  • RWD collection strategies beyond registries
  • Elucidating and integrating patient preferences into development strategies

Michael von Forstner, Head of Patient Safety, Biogen


2:20pm Presentation: Going Bigger, Stronger, and Faster With TcBuster Transposon System for Cell Therapy Development

  • TcBuster cargo capacity meets the needs of next generation therapies expressing multiple proteins
  • The manufacture of TcBuster mRNA and DNA transposons is more consistent and accessible than viral manufacturing
  • Screen more and get through candidate selection faster to help choose the best and safest products for clinical trials

David Hermanson, Senior Manager, CGT Applications, Bio-Techne


2:35pm Closing Panel with Q&A

With all session participants, Joined by: 

Tunç Toker, Chief Operating Officer, Galen Therapeutics


David Hermanson
Senior Manager, CGT Applications
Kimberly Goodspeed
Assistant Professor
University of Texas Southwestern Medical Center
Michael Von Forstner
Global Head of Clinical Safety and Pharmacovigilance
Pete Robinson
Vice President, Medical and Scientific Management, Head, Cell and Gene Therapy Consortium
Syneos Health
Samik Basu
Chief Scientific Officer
Cabaletta Bio
Tunç Toker
Chief Operating Officer
Galen Therapeutics