Gene Therapies Entering the Mainstream – What Are the Next Gene Therapy Treatments?

19 Jan 2023
Modalities and Indications Track

1:30pm Chair Introduction

Nicole Paulk, Associate Professor, University of California, San Francisco


1:35pm Presentation: Collaboration: The Critical Ingredient to Advancing Gene Therapy Cures

  • Role of collaboration
    • Collaboration can help expedite advances in the field of cell and gene therapies and ensure the efficient development of the highest quality of advanced, potentially curative therapies
    • True collaboration, guided by patients as our North Star, will be the key to not only finding cures for rare diseases, but also to moving the entire field of regenerative medicines forward
  • Multi-platform approach to rare disease gene therapies
    • Using our multi-platform approach, we pursue rare genetic diseases that have previously been untapped and work with patients, HCPs and other key industry stakeholders
    • Highlight pipeline/data
  • Path to Commercial stage
    • Discuss upcoming milestones and learnings on path to commercialization (e.g., communicate early and often; maintain transparency)

Kinnari Patel, President and Chief Operating Officer, Rocket Pharmaceuticals


1:50pm Presentation: Realizing the Potential in Gene Therapy Manufacturing

  • Industry trends
  • Challenges in AAV production
  • Solutions designed to support next-generation therapies
  • Case Study: Fast Track to GMP

Nina Bauer, Global Head of Commercial, Biologics and Viral Vector CTDMO, MilliporeSigma


2:05pmam Presentation: Tackling The Leading Cause of Death in the World

  • As the life sciences industry has gained a greater understanding of the genetic causes of heart diseases, the potential to treat the underlying causes of these conditions has come closer to reality
  • Tenaya will discuss the tools required to bring gene therapy to the heart and address the opportunities to tackle both rare and prevalent cardiovascular conditions leveraging genetic targets and innovations (like the design of capsids with more desirable properties to improve delivery) to address the leading cause of death worldwide

Faraz Ali, Chief Executive Officer, Tenaya Therapeutics


2:20pmam Presentation: Next Generation Optogenetics

  • Intro to RayTx
  • Intro to Optogenetics
  • Development of RTx-015 (lead asset First to Market, First in Class Precision Optogenetic Medicine) 

Jenny Holt, Senior Vice President, Program and Portfolio Management, Ray Therapeutics


2:35pm Closing Panel with Q&A

With all session participants


Nicole Paulk
University of California San Francisco
Faraz Ali
Chief Executive Officer
Tenaya Therapeutics
Jenny Holt
Senior Vice President, Programme and Portfolio Management
Ray Therapeutics
Nina Bauer
Global Head of Commercial, Biologics and Viral Vector CTDMO