Lilly partners with Precision BioSciences on genome-editing therapies

Eli Lilly entered into an exclusive licensing agreement to use Precision BioSciences' ARCUS genome-editing platform for the development of in vivo therapies for genetic disorders, with an initial focus on Duchenne muscular dystrophy and two other undisclosed targets, the companies said Friday. Eli Lilly will pay $100 million in cash to Precision BioSciences upfront, whilst also making an equity investment of $35 million.

Further, the licensing deal calls for Precision BioSciences to receive potential milestones of up to $420 million per product, as well as tiered royalties ranging from the mid-single digits to low-teens on product sales. Shares in the company gained as much as nearly 27% on the news.

Tackling undruggable targets

Andrew Adams, vice president of new therapeutic modalities at Eli Lilly, said the collaboration "represents another milestone in the realisation of our vision to create medicines with transformational potential, using new therapeutic modalities such as gene editing to tackle targets and indications which were previously undruggable."

The ARCUS technology uses sequence-specific nucleases that are designed to insert, remove or repair DNA of living cells and organisms. The platform is derived from the natural genome-editing I-CreI enzyme, a homing endonuclease that Precision BioSciences says can be optimised to control for potency and specificity. It noted that ARCUS is capable of a "range of therapeutic editing" given its specificity, ability to make efficient edits and small size.

The company will lead preclinical research and investigational new drug-enabling activities, after which Eli Lilly will handle clinical development and eventual marketing of any approved products. Aside from the initial three gene targets, the agreement gives Eli Lilly the right to select three additional targets. In addition, Precision BioSciences can co-fund clinical development of one product in exchange for a higher royalty rate on co-funded product sales.

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