Session Chairs: Michael Molony, Insmed Incorporated, Isabella Palazzolo, Intellia Therapeutics, Inc., and Marcos Timon, Spanish Agency of Medicines and Medical Products

This session will provide a comprehensive exploration of the current landscape of genome and epigenome editing technologies, including CRISPR/Cas9, Zinc Finger Proteins, Large Serine Integrases, and other emerging editing mechanisms. We will delve into the significant potential these technologies hold for treating genetic diseases, while also addressing the critical ethical and regulatory considerations surrounding their application in human clinical trials.

Key Discussion Points:

Recent Breakthroughs: Highlighting the latest advancements in gene editing technologies and their transformative potential for therapeutic applications.

Epigenetic Therapies: Examining how these technologies can be harnessed for epigenetic therapies, including an in-depth discussion of any concerns related to patient safety.

CMC Challenges: Addressing the chemistry, manufacturing, and controls (CMC) challenges encountered throughout the clinical development phases, such as strategies for targeting delivery of in vivo therapies to new organs/tissues, and manufacturing and control challenges for multiple gene editions.

Ex Vivo Gene Therapy: Showcasing success stories of genome editing applications in ex vivo gene therapy, with a focus on their journey from research to clinical trials.

Impurity Characterization: Providing insights into the progress made in characterizing impurities related to genome editing for complex products, and its implications for product safety and efficacy.

This presentation aims to stimulate a comprehensive discussion among researchers, clinicians, and regulators on the responsible advancement of genome editing technologies in the clinical setting. By addressing both the opportunities and challenges, this session will contribute to the ongoing dialogue on how to safely and effectively integrate these cutting-edge technologies into therapeutic practices.

Session Speakers:

Challenges in Getting Genome Editing Medicines into the Clinic

Phillip Ramsey, Sangamo Therapuetics, Inc.

Advancing Analytical Development for Prime Editing Therapeutics: A Platform-Based Approach

Yune Kunes, Prime Medicine, Inc.

Splicing the Dots: Potency Assurance Strategy for RNA-Editing Gene Therapies

John Kerwin, Ascidian Therapeutics, Inc.

Session Panelists:

Kiran Musunuru, University of Pennsylvania

Marcos Timón, AEMPS

Yune Kunes

Appearances