The Beacon for CRISPR Cures aims to create a roadmap for rapidly developing genome-editing therapies
The story of CRISPR in medicine so far has been one of remarkable speed. It took just 11 years from the 2012 paper in which Innovative Genomics Institute (IGI) founder Jennifer Doudna and Emmanuelle Charpentier and colleagues first described CRISPR genome editing to reach the first CRISPR-based therapy approved by regulators in the UK and the US. New medical advances often take multiple decades before they start to benefit the public, and it’s common for a clinical trial using even a well-established approach to take 10 years or more.
The key question for the CRISPR field now is whether this initial speed can translate into new therapies at scale, especially for rare diseases that have been historically neglected. There is a risk that industry, academia, and regulators won’t be able to sustain the pace, delaying treatment development by years for patients who can’t afford to wait. One CRISPR therapy in 11 years is a victory, but scaling up to address thousands of rare diseases requires a new approach.
“It is imperative that the public health impact of CRISPR expands rapidly beyond the initial, modest-in-size cohort of diseases currently pursued by the biotechnology sector, and the way to get there is to put the power of CRISPR as a therapeutic platform to full use,” says Fyodor Urnov, IGI’s Director of Technology and Translation.
Beacon for CRISPR Cures
To take on the challenge of scaling up CRISPR cure development, the IGI is partnering with Danaher, a leading global life sciences and diagnostics innovator, to create a new collaborative center. The Danaher-IGI Beacon for CRISPR Cures seeks to use the unique programmable power of CRISPR genome editing to address hundreds of genetic diseases within a unified research, development, and regulatory framework. The collaboration enables a substantial new research program at the IGI and is also the largest investment yet from the Danaher Beacons program, which funds pioneering academic research with the goal of developing innovative technologies and applications for human health. The IGI team will receive support from Danaher and several of its operating companies, including IDT, Cytiva, Beckman Coulter Life Sciences, and Aldevron, which has previously collaborated with the IGI in advancing CRISPR-editing tools for neurologic diseases.
“At Danaher, we want to accelerate and scale adoption of the cutting-edge biomedical technologies that stand to benefit humanity the most,” says Rainer Blair, Danaher’s President and CEO. “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 to create transformative gene-editing solutions.”
The collaboration builds on the inherent strengths of the two organizations — the cutting-edge research in genome editing and translating it to clinical trials of the IGI and its partners at UCSF and UCLA, and the industry-leading manufacturing capabilities and technology of Danaher and its operating companies — to reach a shared goal: impact at scale.
“We can develop CRISPR cures in a laboratory, but at the end of the day we need a way to turn those into clinical products for thousands of patients,” says Doudna. “For this, we need industrial-scale solutions to make these cures and make sure they are safe and effective before delivering them to people.”
“The IGI’s central mission is to develop CRISPR solutions that benefit the public and are accessible to all,” adds Ringeisen. “Our partnership with Danaher on the Beacon for CRISPR Cures helps make that vision a reality.”
We can develop CRISPR cures in a laboratory, but at the end of the day we need a way to turn those into clinical products for thousands of patients. — Jennifer Doudna
Fixing Errors of Immunity
The Beacon for CRISPR Cures research team, led by Urnov along with Doudna and IGI Executive Director Brad Ringeisen, will initially focus on developing gene-editing therapies for two rare genetic “inborn errors of immunity” (IEIs), familial hemophagocytic lymphohistiocytosis (HLH) and Artemis-deficient severe combined immunodeficiency (ART-SCID), and taking these therapies to initial regulatory submission. These diseases are typical of many rare diseases in that they have small patient populations, making drug development challenging and cost-prohibitive, so they are often neglected by the pharmaceutical industry.
IEIs have several advantages that make them suited to Beacon for CRISPR Cures project, including an extensive patient registry and a transplant-based route of administration that bypasses some key challenges in delivering CRISPR molecules to appropriate tissues. Importantly, leading experts on these diseases at UC San Francisco and UCLA are part of the Beacon team.
The IGI team includes Matthew Kan at UCSF/UC Berkeley, Jennifer Puck and Mort Cowan at UCSF focusing on ART-SCID, and David Nguyen at UCSF/UC Berkeley, Michelle Hermiston, and Bryan Shy at UCSF focusing on HLH. Petros Giannikopoulos, Director of IGI’s Clinical Laboratory, will be the diagnostic and analytical lead. Donald Kohn at UCLA will be involved in translating the gene editing approaches developed at UCB and UCSF to clinical cell manufacturing in the UCLA Human Gene and Cell Therapy Facility.
From One to a Thousand
Because of CRISPR’s ability to be easily reprogrammed to address any gene mutation, the larger goal of the Beacon for CRISPR Cures is to think beyond the two initial diseases and use them as a model to develop a scalable platform approach in which a new medicine for any genetic disease can be rapidly developed.
“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,” says Urnov.
A platform approach could accelerate the development of cures for diseases that might otherwise struggle to get sufficient attention by industry, and allow therapies to be developed in parallel.
“UC Berkeley has the phrase ‘Fiat lux’ or ‘let there be light’ as its motto, and I find it meaningful that our partnership has the word ‘beacon’ in it. The world now has the first-ever approved CRISPR medicine — the IGI and Danaher hope to illuminate a path to a great many more,” says Doudna.
- Danaher, Innovative Genomics Institute Join Forces (Danaher press release, Jan 9, 2024)
- New CRISPR Center Brings Hope for Rare and Deadly Disease (UCSF press release, Jan 9 2024)
- Aldevron, Jennifer Doudna, and Innovative Genomics Institute Collaborate on In Vivo CRISPR Therapies for Neurologic Diseases. Resulting paper published in Molecular Therapy
- Finding & Fixing Immunodeficiencies with CRISPR
Media contact: Andy Murdock firstname.lastname@example.org