The future of CRISPR-based therapies received a windfall this week with the launch of the Danaher-IGI Beacon for CRISPR Cures. The goal of the center is to use CRISPR-based gene editing to permanently address hundreds of diseases with a unified research, development, and regulatory approach. The Beacon for CRISPR Cures plans to do this by developing platform approaches that can be easily modified to develop gene-editing medicines for hundreds of devastating illnesses. The model aims to dramatically reduce preclinical and clinical development time and expense for investigational rare disease therapies that currently struggle to attract funding.

The center brings together a triple-threat of resources, from academia and industry, with the hope of making a tectonic shift in the field of CRISPR-based cures. The first of the three comes from industry. Danaher consists of many (more than 15) diverse businesses: Integrated DNA Technologies (IDT), Cytiva, Molecular Devices, Leica, Beckman Coulter, and Aldevron, to name a few. Danaher 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 second is academic, bringing the interdisciplinary expertise of the scientists at the Innovative Genomics Institute (IGI) to the effort.

Jennifer Doudna
Jennifer Doudna, PhD [Brian Ach / Stringer/Getty Images]
The last threat is the talent at the helm of the initiative. Who better to oversee such a mission than Jennifer Doudna, PhD, Nobel laureate and CRISPR pioneer, and Fyodor Urnov, PhD, IGI’s director of technology and translation and director of the new Beacon—both of whom are professors at the University of California, Berkeley.

“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,” noted Doudna. “We know how to get CRISPR molecules into the tissues where they need to be. 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.”

Fyodor Urnov
Fyodor Urnov, PhD

“It is imperative that the public health impact of CRISPR expand rapidly beyond the initial, modest-in-size cohort of diseases currently pursued by the biotechnology sector,” asserted Urnov. “The unique nature of CRISPR makes it ideal for developing and deploying a platform capability for CRISPR cures on demand. Danaher and the IGI are in a unique position to join our respective strengths, build such a platform, and create a first-of-its-kind CRISPR cures ‘cookbook’ that can be used by any team wishing to take on other diseases.”

As a first step, the Danaher-IGI Beacon aims to develop gene-editing therapies for two rare genetic disorders that are “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 collaboration, which enables a new research program at IGI, is the largest Danaher Beacon to date. The Beacon’s 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.

Rainer Blair, president and CEO of Danaher, said: “CRISPR has enormous therapeutic promise, but there is no standard scientific or regulatory framework for how to get it to patients. To take on a challenge this big, we all need to work together with a sense of urgency across academia, industry, and government. We are thrilled to join forces with some of the finest scientific minds in gene editing at the IGI, bringing R&D and manufacturing talent, technology, and expertise from across several of our operating companies in an effort to create transformative solutions for incredibly important but too often underserved patient communities.”

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