Webinar: Establishing a Sustainable Platform for CRISPR-Based Genome Editing Therapies

An Academic-Industry Partnership Model for Addressing Inborn Errors of Immunity

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.

Key Takeaways

• 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.

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

Listen to hear about an academic-industry partnership model for addressing inborn errors of immunity

Meet the Speakers

Fyodor Urnov Ph.D.
Professor of Molecular Therapeutics at UC Berkeley and a Scientific Director at its Innovative Genomics Institute (IGI)
Fyodor Urnov co-developed the toolbox of human genome and epigenome editing, co-named genome editing, and was on the team that advanced all of its first-in-human applications to the clinic. He also led the effort that identified the genome editing target for an approved medicine to treat sickle cell disease and beta- thalassemia.

A major goal for the field of genome editing and a key focus of Fyodor's work is expanding access to CRISPR therapies for genetic disease. As part of that effort, Fyodor directs the Danaher-IGI Beacon for CRISPR Cures – a first-in-class academia – industry partnership developing and advancing to the clinic scalable CRISPR-based approaches to treat diseases of the immune system.

Sadik H. Kassim
Chief Technology Officer
Danaher
Sadik is a highly regarded scientist and executive with extensive experience in biotechnology with a specific focus on cell and gene therapy bioprocessing and translational research. Currently, he is Chief Technology Officer at Danaher with a focus on genomic medicines. He previously was Chief Technology Officer at Vor Bio where he built the technical operations team responsible for process development, analytical development, supply chain, and manufacturing support of a CRISPR gene-edited HSPC product and oversaw the company’s CAR-T efforts. Prior to Vor, Sadik was Executive Director at Kite Pharma and led development of manufacturing processes for autologous CAR-T and TCR-based cell therapies. As the Chief Scientific Officer at Mustang Bio, Sadik managed the foundational build-out of the company’s preclinical and manufacturing activities. Earlier in his career, Sadik was Head of Early Analytical Development for Novartis’ Cell and Gene Therapies Unit and worked on research teams at the National Cancer Institute with Dr. Steven Rosenberg, the University of Pennsylvania Gene Therapy Program with Dr. Jim Wilson, and Johnson and Johnson’s Immunology Discovery group. Sadik and his teams have contributed to the successful BLA and MAA applications for three of the commercially available CAR-T therapies: Kymriah®, Yescarta®, and Tecartus®. Sadik holds a B.S. degree in cell and molecular biology from Tulane University and earned his Ph.D. in Microbiology and Immunology from Louisiana State University.