CRISPR pioneer Jennifer Doudna, Ph.D., looks set to continue to push the boundaries of gene editing, as she announces plans to team up with life sciences giant Danaher to create a center focused on generating new therapies for rare and other diseases.
The center, which will be based at the headquarters of Doudna’s own Innovative Genomics Institute (IGI) and referred to as the Danaher-IGI Beacon for CRISPR Cures, “aims to use CRISPR-based gene editing to permanently address hundreds of diseases with a unified research, development and regulatory approach,” according to a Jan. 9 release from Danaher.
Doudna will oversee the center’s work alongside Fyodor Urnov, Ph.D., Doudna’s fellow Berkeley professor who is also the IGI's Director of Technology and Translation.
From its side, Danaher has pledged to “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 counts as one of Danaher’s ‘beacons’ programs, which fund academic research aimed at innovative technology ranging from precision diagnostics to biomanufacturing and data sciences. The collaboration with the IGI will be the largest beacon program to date, with Urnov appointed the beacon’s director.
The collaboration’s initial focus is on developing gene-editing therapies for two rare genetic disorders: a life-threatening, hyper-inflammatory disorder called hemophagocytic lymphohistiocytosis; and Artemis-SCID, an immune deficiency that is typically treated with a bone marrow transplant.
Both disorders are known as "inborn errors of immunity" (IEIs), which are thought to be particularly amendable to the Danaher/IGI approach, the company explained in the release. This is because the Danaher-IGI Beacon will have access to an extensive patient registry and offer a transplant-based route of administration that “bypasses some key challenges in delivering CRISPR molecules to appropriate tissues.”
With around 500 diseases classified as IEIs, the center’s ultimate goal is for the resulting scalable platform to be used to rapidly create new therapies to treat many other disorders in this group as well as more common conditions that could be treated by editing a single gene.
“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,” Doudna said in the release.
“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,” she added.
“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."