Establishing a Sustainable Platform for CRISPR-Based Genome Editing Therapies: An Academic-Industry Partnership Model for Addressing Inborn Errors of Immunity

This talk was given at ASGCT 2025 in New Orleans, LA on Thursday, May 15, 2025.

CRISPR-based genome editing therapies hold great promise, yet widespread application remains limited by high cost and complex manufacturing, especially for ultra-rare diseases. In 2024, Danaher Corporation and the Innovative Genomics Institute (IGI) collaborated to address these challenges by leveraging automation, quality control and academic innovation.

Dr. Fyodor Urnov and Dr. Sadik Kassim presented a novel, scalable platform for CRISPR therapies at ASGCT that targeted inborn errors of immunity (IEIs).

Key highlights from their presentation:

• Streamlining of critical reagent manufacturing processes
• Creation of platform technologies for CRISPR-based cures
• Novel characterization assays to assess safety and efficacy

This platform technology is designed to expand to other genes and diseases, aiming to make gene editing therapies more accessible. Their standardized cookbook supports academic-industry collaborations as a promising strategy to overcome the cost and timeline barriers limiting CRISPR’s full potential.