Innovative Genomics Institute and Danaher Launch Collaboration to Advance Platform Approaches to Gene Editing

Rare Daily Staff

The Innovative Genomics Institute and Danaher Corporation have launched a collaborative center to develop gene-editing therapies for rare and other diseases, with a goal of creating a new model for future development of a wide range of genomic medicines.

The center, known as the Danaher-IGI Beacon for CRISPR Cures, aims to use CRISPR-based gene editing to address hundreds of diseases with a unified research, development, and regulatory approach.

“CRISPR has enormous therapeutic promise, but there is no standard scientific or regulatory framework for how to get it to patients,” said Rainer Blair, president and CEO of Danaher. “To take on a challenge this big, we all need to work together with a sense of urgency across academia, industry, and government.”

Jennifer Doudna, who won the Nobel prize for her pioneering work on CRISPR and founder of the IGI, will oversee work at the center at the IGI headquarters alongside Fyodor Urnov, IGI’s director of technology and translation and director of the new Beacon. Both are professors at the University of California, Berkeley. Brad Ringeisen, IGI’s executive director, will also play a key role in the new center.

Danaher, a global life sciences and diagnostics innovator, will make available an extensive collection of technologies and solutions for the manufacturing of CRISPR-based therapies, and will also work to develop new technologies and approaches intended to simplify and standardize preclinical and clinical development.

The collaboration, which enables a substantial new research program at IGI, is the largest Danaher Beacon to date. The Beacons program funds pioneering academic research with the goal of developing innovative technologies and applications for human health. Focus areas for Beacons include genomic medicines, precision diagnostics, next-generation biomanufacturing, human systems, and data sciences.

As a first step, the Danaher-IGI Beacon aims to develop gene-editing therapies for two rare genetic disorders called “inborn errors of immunity” (IEIs), hemophagocytic lymphohistiocytosis (HLH) and Artemis-SCID. IEIs have several advantages that the parties believe make them amenable to the combined Danaher/IGI approach, including an extensive patient registry and a transplant-based route of administration that bypasses some key challenges in delivering CRISPR molecules to appropriate tissues.

Collectively, IEIs comprise some 500 distinct diseases that together affect many hundreds of thousands of people worldwide. Yet they are not currently the focus of any major gene-editing trials, largely because designing and testing therapies for each very small IEI patient population would be challenging, slow, and cost-prohibitive. Because of CRISPR’s unique ability to be easily reprogrammed to address any gene mutation, the goal of the Beacon for CRISPR Cures is to develop a scalable platform approach that would enable a new medicine to be rapidly built, even for diseases beyond IEIs.

The Beacon’s goal is for the platform developed at the new center to be expandable across many IEIs, other rare diseases, or more common conditions that could be treated by editing a single specific gene.

The IGI will teaming up with leading clinicians focusing on IEIs at the University of California, San Francisco and the University of California, Los Angeles. The IGI will lend its unique expertise in CRISPR engineering, nonclinical models of inborn errors of immunity, manufacture of gene-edited cell products, regulatory interactions with the FDA in this disease space, and the design and execution of clinical trials for patients affected with these severe diseases.

Danaher operating companies will provide tools, reagents, resources, and expertise to simplify preclinical and clinical development and develop new standards for safety and efficacy. Among them is Integrated DNA Technologies, which will contribute industry-leading capabilities in the synthesis, modification, purification, and quality control of CRISPR nucleases and guide RNA, drawing on its newly opened therapeutic oligonucleotide manufacturing facility.

Aldevron, which has previously worked with IGI to advance the use of CRISPR-based gene editing in the brain, will also play a key role in the collaboration, alongside other Danaher operating companies including Cytiva, Beckman Coulter Life Sciences, Leica Biosystems, and Leica Microsystems.

“Combining the strengths of the IGI and Danaher companies in this new center is a uniquely powerful way to deliver on the promise of CRISPR cures. We know how to get CRISPR molecules into the tissues where they need to be,” said IGI’s Doudna. “We know the patient communities. And we have the world experts on these diseases on our team. What we need is a blueprint describing all the science and technology required to treat a person using CRISPR. Once that is achieved, I am convinced that CRISPR can become the standard of clinical care for many diseases.”