What Is The Public and Patient Perception of Cell and Gene Therapies

18 Jan 2023
Patient and Clinical Considerations Track
Vertex Cell and Gene Therapies

11:00am Chair Introduction

Ryan Leahy, Vice President of Research, Phacilitate


11:05am Presentation: Advancing The Frontline

  • In March of 2020, Courtney’s 8 year old son, Colby, was diagnosed with a stage 1, low risk lymphoma and immediately started a standard frontline chemotherapy protocol lasting nearly 800 days and including nearly a dozen different non-targeted, incredibly toxic drugs, the majority of which were developed and approved for use between 1950-1970.
  • Colby’s experience, along with thousands of other children, has inspired Courtney, a board certified toxicologist and drug development professional, to educate the research and pharmaceutical community about the patient experience with frontline care in pediatric oncology and the desperate need for more target, effective, accessible and safe therapies.

Courtney Horvarth, Global Head of Strategy, Planning and Operations, Novartis


11:20am Presentation: Preparing Patients and Other Stakeholders for the Coming Wave of Cell and Gene Therapies

  • There will be several ‘firsts’ and milestones for CGT approvals in 2023, including the first approval of a CRISPR therapy and approvals for diseases with larger patient populations such as hemophilia and sickle cell disease, helping to raise public and patient awareness of CGTs
  • We will start to learn in 2023 how – and how quickly – healthcare systems will evolve to adapt to the transformative nature of CGTs
  • The sector needs to engage all stakeholders to address their hopes – and their concerns – about the coming wave of CGT therapies

Timothy D. Hunt, Chief Executive Officer, Alliance for Regenerative Medicine


11:35am Presentation:

Gene and Cell Therapy: A Patient Perspective

Teonna Woolford, Chief Executive Officer, Sickle Cell RED


11:50am Presentation: TBA

Speaker TBC


12:05pm Closing Panel with Q&A

With all session participants, joined by: 

Mona Jhaveri, Founder & Director, Music Beats Cancer

Sven Kili, Chief Executive Officer, Antion Biosciences


Courtney Horvarth
Global Head of Strategy, Planning & Operations, Translational Medicine & Childhood Cancer Advocate
Ryan Leahy
Vice President of Research
Teonna Woolford
Chief Executive Officer
Sickle Cell Reproductive Health Education Directive
Timothy D. Hunt
Chief Executive Officer
Alliance for Regenerative Medicine
Mona Jhaveri
Founder & Executive Director
Music Beats Cancer
Sven Kili
Chief Executive Officer
Antion Biosciences