Lilly, Precision to Develop Gene Editing Therapies for DMD, Other Disorders

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by Forest Ray PhD |

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A new collaboration between Eli Lilly and Precision BioSciences aims to develop gene editing therapies for genetic disorders, initially focusing on Duchenne muscular dystrophy (DMD) and two other undisclosed targets, the two companies announced.

Precision licensed its ARCUS genome editing platform to Lilly and will lead preclinical research to support regulatory clearance for starting clinical trials. Lilly will be in charge of clinical development and commercialization.

“Collaborating with Lilly, a global healthcare leader with strong clinical and commercial experience in difficult-to-treat diseases, will help us accelerate our work aimed to solve genetic diseases with unique editing challenges,” Derek Jantz, PhD, chief scientific officer and co-founder of Precision, said in a press release.

Genome editing is a way to treat genetic disorders by directly changing a person’s DNA. Duchenne, for instance, is caused by a mutation in the DMD gene that results in muscle cells making little to no functional dystrophin, a protein needed to strengthen muscle fibers and protect them from damage.

Because mutations in a single gene cause the disorder, targeting that gene via gene editing has become an attractive therapeutic approach in DMD.

Precision’s ARCUS platform is derived from an enzyme called I-CreI, found in algae. ARCUS is designed to “home in” on specific sites where it makes targeted edits in a patient’s genome. After making its edits, the enzyme deactivates itself through a built-in safety mechanism. This self-inactivation should limit the chance of undesired or “off-target” changes, according to Precision.

ARCUS is designed to be highly versatile, enabling researchers to add, remove, and repair genes of interest. One other advantage of ARCUS, said Precision, is that the enzyme is smaller than other gene editing technologies, making it easier to deliver to specific cells.

“Gene-edited therapies are emerging as a promising approach to help patients afflicted with genetic conditions,” said Ruth Gimeno, PhD, vice president of diabetes and metabolic research at Lilly.

Under the terms of the agreement, Lilly will pay Precision $100 million in cash upfront and will make a $35 million equity investment. Precision may receive up to $420 million more through meeting development and commercialization milestones, as well as royalties based on the sale of any successful products. Precision may increase these royalties by co-funding clinical development of one product.

Finally, Lilly has the option of choosing up to three additional gene targets for this partnership.

“We look forward to working with Lilly to leverage our deep understanding of in vivo gene editing and experience with ARCUS to develop new therapies,” Jantz added, “including a potentially transformative treatment for Duchenne muscular dystrophy.”

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