Clinical Development Of CRISPR-Based Therapies In Oncology

By Matthew Confeld, PHARMD, PhD, Clinical Trials Methodology Fellow, Jana Knezevic, PHD, Associate Director of Project Management, Oncology and Rare Disease, and Michael F. Murphy, MD, PHD, Chief Medical And Scientific Officer

CRISPR/Cas9 gene editing offers immense potential for revolutionizing oncology drug development. However, the novelty of CRISPR-based therapies introduces complex operational challenges in clinical trial design and execution.

Key challenges include:

• Immunological considerations: The bacterial origin of Cas proteins and delivery vectors can trigger immune responses, necessitating careful evaluation of regional differences and alternative delivery methods.
• IP management complexities: Cell-based therapies modified with CRISPR technology require intricate IP management, including donor sample collection, transportation, and return.
• Operational hurdles: Maintaining cell or vector viability during transportation, performing invasive reintroduction procedures, and adhering to stringent regulatory requirements pose significant operational challenges.
• Ethical implications: The potential for genetic modifications to induce cell proliferation, differentiation, or migration raises ethical concerns and demands robust oversight.
• To successfully develop CRISPR-based cancer therapies, sponsors and partners must navigate these challenges while adapting to evolving clinical research standards for advanced therapy medicinal products.