The research collaboration will leverage ARCUS genome editing technology, and initially include three gene targets


Eli Lilly and Company's Corporate Center in Indianapolis, Indiana in 2019. (Credit: Momoneymoproblemz/Wikipedia.)

Lilly and Precision BioSciences have entered into a research collaboration and exclusive license agreement to use the latter’s ARCUS genome editing platform for the research and development of potential treatments for genetic disorders.

The collaboration will initially focus on developing Duchenne muscular dystrophy (DMD) and two other undisclosed gene targets.

Under the terms of the agreement, Lilly is expected to make an upfront payment of $100m in cash, along with an equity investment of $35m in Precision’s common stock.

Precision is entitled to receive up to $420m in potential development and commercialisation milestones, in addition to tiered royalties on product sales.

Also, the company is expected to conduct the pre-clinical research and IND-enabling activities, followed by Lilly undertaking responsibility for clinical development and commercialisation.

Lilly holds the right to select up to three additional gene targets for the collaboration, while Precision can co-fund clinical development of one product in exchange for an increased royalty rate on sales of the co-funded product.

The transaction is subject to approval under the Hart-Scott-Rodino (HSR) Antitrust Improvements Act and certain other customary closing conditions.

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

“We look forward to working closely with Precision’s scientific team and leveraging their platform to develop and deliver breakthrough medicines for untreated genetic disorders.”

Lilly new therapeutic modalities vice president Andrew Adams said: “This collaboration with Precision BioSciences represents another milestone in the realization 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.”

According to the company, genome editing technologies enable precise editing of the DNA of a living organism, opening up the possibility of correcting genetic problems at their source.

ARCUS is a unique genome editing platform discovered and developed by scientists at Precision BioSciences.

The platform leverages sequence-specific DNA-cutting enzymes that to either insert (knock-in), remove (knock-out), or repair DNA of living cells and organisms.

ARCUS platform builds on a naturally occurring genome editing enzyme I-CreI, a homing endonuclease found in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA.

Precision BioSciences chief scientific officer and co-founder Derek Jantz said: “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, including a potentially transformative treatment for Duchenne muscular dystrophy.

“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.”