In a continuing string of deals in the gene therapy segment, Eli Lilly announced research collaboration and exclusive license agreement with Precision BioSciences. As part of the agreement, Eli Lilly will utilize Precision Biosciences genome editing platform ARCUS® for the research and development of in vivo therapies for genetic disorders, primarily focussed on Duchenne Muscular Dystrophy and two other undisclosed gene targets.
Under the terms of the agreement, Eli Lilly will pay USD 100 million in upfront payment and a USD 35 million equity investment in Precision Biosciences and Precision will also be eligible to receive upto USD 420 million in consecutive development and commercialization milestone payments per product, as well as tiered royalties going up to low-teens on product sales after commercialization. Precision will be responsible for preclinical development and IND-enabling activities, while Eli Lilly will be responsible for the clinical development and commercialization. Precision Biosciences also has the option to co-fund clinical development of one product out of the collaboration in return for a higher royalty rate on the co-funded product sales.
The ARCUS® platform is based on a naturally existing gene editing enzyme homing endonuclease I-CreI from algae Chlamydomonas reinhardtii to make highly specific edits (repair, knock-in and knock-out) in the DNA and has a built-in safety switch to prevent any unwanted off-target DNA edits – a major limitation and clinical risk inherent to many gene editing platforms. The built-in safety switch is the unique differentiator of the platform, along with its small size of about 364 amino acids, making its delivery to specific cells and tissues easier.
Eli Lilly has been diving into the gene therapy segment with high corporate confidence and a focus on evolving scientific intricacy. A monogenic disorder such as Duchenne Muscular Dystrophy is a viable option for applying gene editing technology because of the known, well-studied target and with currently a lean competition. Sarepta’s drugs Exondys 51 and Vyondys 53 have been found to work only for about one-fifth of the patients. Other therapies in the pipeline include Pfizer’s phase III gene therapy, Sarepta’s phase I/III micro-dystrophin construct gene therapy and Ultragenyx’s recent partnership with Solid Biosciences for its phase I/II gene therapy. There have been notable investments in upstream gene therapy research in the last one month which alludes to the continuing corporate momentum in the space and a welcoming sentiment amongst the big pharma to dive deep into next-gen gene therapy.