Typesetting丨Shuicheng WenRecently, a subsidiary of Eli Lilly and CompanyPrevail Therapeutics Ends Partnership withPrecision BioSciences' partnership, abandoning the jointly developed projects including Duchenne Muscular Dystrophy（DMD）Including 3 gene editing therapies. Affected by this news,Precision BioSciences' stock price plummeted by more than 10%, and its current total market value has fallen below 80 million US dollars.The cooperation between the two parties dates back to four years ago, in November 2020, when Eli Lilly and Company...Precision BioSciences, Inc.Advance payment of 100 million US dollars, and to itsInvestment of $35 million, to co-develop three in vivo gene editing therapies. According to the agreement,Precision BioSciences is responsible for preclinical and IND studies, while Eli Lilly and Company takes charge of subsequent clinical development and commercialization.The total amount of cooperation is as high as$2.6 Billion。Less than a month later, Eli Lilly and Company acquired Prevail Therapeutics for approximately 1 billion US dollars. Thereafter,Prevail is responsible forCollaboration with Precision BioSciences. July 2023,Prevail Therapeutics Revises Original Collaboration Terms, Reducing Milestone Payments for Three Gene-Editing Therapies。According to the press release published on April 16, 2024,Prevail Therapeutics Continues to Collaborate withPrecision BioSciences Loses Interest in Collaboration, Decides to End Partnership.CEO of Precision BioSciences, Inc.Michael Amoroso,We are pleased to have had a productive gene-editing collaboration with Prevail and appreciate their contributions to the success of these programs. Together, we advanced three programs from concept to clinical candidate, and Precision has completed the work plans for these programs, bringing us to a significant development decision point.The cooperation between the two parties is with Precision BioSciences, Inc.ARCUS NucleaseGeneCentered on the editing platform,ARCUSIt is a proprietary genomic editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific nucleases to cut DNA, enabling knockouts and knock-ins of DNA within cells and organisms. ARCUS is based on a naturally occurring homing nuclease I-CreIRedesigned and transformed, I-CreIIt evolved in Chlamydomonas reinhardtii, enabling highly specific cleavage in cellular DNA and achieving gene knock-in at the cleavage site through homologous recombination. Compared to CRISPR-Cas9,ARCUS nuclease is smaller and may be more suitable for in vivo gene editing through AAV delivery.Previously,Precision BioSciences has integrated variousARCUS nuclease has been applied to knock out cholesterol metabolism genes,Transthyretin Amyloid Fibril Formation Gene, Inactivation of Hepatitis B Virus, as well as the construction of allogeneic CAR-T cells and other fields. They have developed a mitochondrial-targeting ARCUS nuclease—mitoARCUS.Co-founder and Chief Scientist of Precision BioSciencesJeff SmithDr. stated,InDuchenne Muscular Dystrophy（DMD）In the project, compelling in vivo proof-of-concept data was generated by ARCUS gene editing targeting the "hotspot" mutation regions of the dystrophin gene.Precision PublicCompany in the Newsstated that, due to the strong concept validation data already generated by these projects, opportunities for independent development or collaboration with other companies will be explored.It is worth mentioning that, in May 2023,Eli Lilly SubsidiaryPrevail Therapeutics Announces Collaboration withGene editing company Scribe Therapeutics collaborates to develop in vivo CRISPR gene editing therapies for specific neurological and neuromuscular diseases.。In this collaboration, Prevail will pay Scribe a $75 million upfront payment, and Scribe will grant Prevail an exclusive license to its patented miniaturized CRISPR gene editing system, CasX. Through this partnership, Scribe also has the opportunity to receive up to $1.5 billion in milestone payments.。The Miniaturized Novel CRISPR Gene Editing System CasX by Nobel LaureateJennifer DoudnaDeveloped by the professor in 2016,In 2018, Professor Jennifer Doudna and others foundedFounded Scribe, which is committed to developing genome editing and delivery tools based on CasX — CasX-Editors, achieving better attributes of gene editing tools from three aspects — higher activity, stronger specificity, and easier deliverability.
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