Eli Lilly has shifted its focus to genome editing following a new research collaboration deal agreed with US-based specialist Precision BioSciences, fronting an initial $100m for access to its novel ARCUS platform.
Lilly and Precision will collaborate on the research and development of in vivo therapies for gene disorders, with an initial focus on Duchenne muscular dystrophy and two further undisclosed gene targets.
Precision’s ARCUS genome editing platform could deliver ‘safer, more specific gene edits’ through the use of a naturally occurring gene editing enzyme known as endonuclease I-CreI, according to the company.
By using this technology, Precision can re-engineer the enzyme’s editing abilities to ‘knock-in, knock-out and repair’ the targeted cells.
“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,” said Derek Jantz, chief scientific officer and co-founder of Precision BioSciences.
Under the terms of the deal, Lilly will pay Precision $100m upfront and also make an equity investment totalling $35m in Precision’s common stock.
In addition, Precision is eligible to receive up to $420m in potential development and commercialisation milestones per product, with tiered royalties on top of that if Lilly successfully commercialises a therapy from the collaboration.
While Precision will lead pre-clinical research and IND-enabling activities, Lilly will assume responsibility for clinical development and commercialisation.
Lilly will have the right to select up to three additional gene targets as part of the deal, and Precision can also choose to co-fund clinical development of one product in return for an increased royalty rate on product sales.
“Gene-edited therapies are emerging as a promising approach to help patients afflicted with genetic conditions,” said Ruth Gimeno, vice president of diabetes and metabolic research at Lilly.
“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,” she added.
A number of companies are seeking to develop therapies for DMD as the therapy area becomes increasingly more competitive.
In August, the US Food and Drug Administration (FDA) approved Japanese pharmaceutical company Nippon Shinyaku’s (NS Pharma) DMD drug Viltepso (viltolarsen).
Sarepta is also prominent in the DMD therapy area and the company’s second approved DMD therapy Vyondys 53 (golodirsen) received approval last December.
Vyondys 53 is Sarepta’s second approved DMD therapy after Exondys 51 (eteplirsen). It was the first disease-modifying treatment to be introduced in the US four years ago for the inherited muscle-wasting disease.