Danaher and Nobel Laureate Jennifer Doudna Launch Danaher-IGI Beacon for CRISPR Cures to Develop Gene Editing Therapies for Hundreds of Diseases

Shanghai, January 12, 2024 /PRNewswire/ -- Danaher has partnered with Jennifer Doudna and the Innovative Genomics Institute (IGI) she founded to launch the Danaher-IGI Beacon for CRISPR Cures project. This initiative is dedicated to developing CRISPR gene-editing solutions aimed at using gene-editing therapies to treat hundreds of diseases.

Washington, D.C. – January 9, 2024 – Danaher Corporation and the Innovative Genomics Institute (IGI) announced the establishment of a joint research and development center to collaboratively develop gene-editing therapies for treating rare diseases and other conditions, creating a new model for the future development of various gene-based medicines.The R&D center is namedDanaher-IGI Beacon for CRISPR Cures, aiming to leverage through integrated research, development, and regulation based onCRISPRGene editing technology permanently treats hundreds of diseases.

Nobel laureate, CRISPR pioneer, and IGI founder Jennifer Doudna, together with Fyodor Urnov, IGI’s Director of Technology and Translation and the new Beacon Project Director, will lead the development of research initiatives at IGI headquarters. Both are professors at the University of California, Berkeley. IGI Executive Director Brad Ringeisen will also play a key role in the new R&D center. As an innovator in global life sciences and medical diagnostics, Danaher will provide extensive technical support and solutions for the production of CRISPR therapies while developing new technologies and methods to streamline and standardize preclinical and clinical development.

This collaboration is the largest-scale one for Danaher to date.BeaconProject, will enableIGILaunch substantial new research projects.Danaher Beacon Project aims to fund scientific research institutes in conducting cutting-edge scientific research and developing innovative technologies and applications that improve human health. The project focuses on areas including genomic medicine, precision diagnostics, next-generation biologics manufacturing, human systems, and data science.

Danaher Corporation President and CEO Rainer M. Blair stated, "CRISPR holds tremendous promise for disease treatment, but there is currently no standardized framework in academia or regulatory fields to guide how we apply it to patients. To address such a significant challenge, we need to bring together academia, industry, and government to collaborate. We are thrilled to partner with some of the most outstanding researchers in the field of gene editing from IGI, combining the talent, technology, and expertise from Danaher's various operating companies in research, development, and production. Together, we will create revolutionary solutions for patients who are critically underserved."

Doudna stated, "The new joint R&D center combines the respective strengths of IGI and Danaher, offering a unique and powerful way to fulfill the promise of CRISPR therapies. We know how to deliver CRISPR molecules to target tissues. We understand the patient population. Our team includes world-class experts researching these diseases. Together, we need to map out a blueprint that outlines all the science and technology required for using CRISPR therapeutics. Once that is achieved, I firmly believe CRISPR will become the standard for clinical treatment of many diseases."

As a first step, the Danaher-IGI Beacon project will develop gene-editing therapies for two types of inborn errors of immunity (IEIs) — hemophagocytic lymphohistiocytosis (HLH) and Artemis-deficient severe combined immunodeficiency (Artemis-SCID). The two parties believe that several key characteristics of IEIs make them suitable for the Danaher/IGI joint approach, including extensive patient records and a transplant-based delivery route, thereby overcoming some of the key challenges in delivering CRISPR molecules to target tissues.

In general, IEI encompasses approximately 500 distinct diseases, affecting hundreds of thousands of people worldwide. However, they are not currently the primary focus of any major gene-editing trials, largely because designing and testing therapies for each very small population of IEI patients is challenging, slow-moving, and costly. Given CRISPR's unique ability to be easily reprogrammed to address any genetic mutation, the goal of the Danaher-IGI Beacon for CRISPR Cures is to develop a scalable platform-based approach that enables the rapid creation of new treatments, even for diseases beyond IEIs. The aim of the Beacon project is to expand the platform-based methods developed by the joint research center to cover more IEIs and other rare diseases, as well as more common conditions treatable by editing a single specific gene.

Urnov stated, "The impact of CRISPR on public health must expand rapidly beyond the initially limited range of diseases currently being advanced by the biotech industry. The uniqueness of CRISPR makes it ideal for developing and deploying a platform approach to on-demand CRISPR treatments. By combining the strengths of Danaher and IGI, we can establish such a platform and pioneer CRISPR-based therapies for other teams aiming to treat more diseases."

In collaboration with top IEI clinicians from UCSF and UCLA, IGI will provide unique expertise to support CRISPR engineering, non-clinical models of congenital immune deficiencies, the manufacturing of gene-edited cell products, regulatory efforts in this disease area, as well as the design and execution of clinical trials for patients suffering from these severe conditions.

Danaher’s operating companies will provide instruments, reagents, resources, and expertise to streamline preclinical and clinical development and establish new standards for safety and efficacy. Among them, IDT will leverage its newly commissioned therapeutic oligonucleotide production facility to deliver industry-leading capabilities in the synthesis, modification, purification, and quality control of CRISPR nucleases and guide RNAs. Aldevron has previously collaborated with IGI to advance the application of CRISPR-based gene-editing technology in the brain. Other Danaher operating companies, including Cytiva, Beckman Coulter Life Sciences, Leica Biosystems, and Leica Microsystems, will also play key roles in this collaboration.