吉利德停止与Precision合作开发的基因编辑项目

sir

DURHAM, N.C., July 06, 2020 (GLOBE NEWSWIRE) -- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company dedicated to improving life with its novel and proprietary ARCUS® genome editing platform, today announced that the Company will regain full rights and all data it generated for the in vivo chronic hepatitis B virus (HBV) program developed under its 2018 collaboration agreement with Gilead Sciences.This was a highly productive and well-aligned collaboration, and we deeply value the opportunity to advance our ARCUS genome editing technology and a potential cure for HBV alongside a global leader in infectious disease,” commented Derek Jantz, Ph.D., Chief Scientific Officer, Precision BioSciences. “Key learnings from this program and how to develop liver-directed gene editing therapeutic candidates are directly applicable to our in vivo pipeline. While we consider new partnership opportunities for HBV, we are focused on progressing our internal lead proprietary gene correction program for primary hyperoxaluria type 1 for which we expect to nominate a clinical candidate later this year.Under the terms of the collaboration agreement, Precision BioSciences was primarily responsible for the development, formulation, and preclinical evaluation of the investigational nucleases for HBV; Gilead funded the research and development and was responsible for the clinical development and commercialization of potential therapies. Upon the collaboration’s conclusion, effective September 4, 2020, Precision BioSciences will regain full clinical development and commercialization rights to the program.
[p=21, null, left]The Company does not anticipate any changes to its cash runway. As of March 31, 2020, Precision had cash and cash equivalents of $154.2 million, which is expected to sufficiently fund operations into the second half of 2021.

About Precision’s In Vivo Program for Chronic Hepatitis B Virus (HBV)
By directly targeting cccDNA and integrated HBV genomes, preclinical data have shown that ARCUS gene editing may be a promising approach for developing a potential HBV cure. Preclinical data presented earlier this year at the 2020 American Society of Genetic & Cell Therapy (ASGCT) Annual Meeting demonstrated that an optimized ARCUS nuclease effectively targeted and degraded cccDNA up to 75% with subsequent knockdown of surface antigen in HBV-infected primary human hepatocytes. Data from murine studies also showed that an ARCUS nuclease delivered by lipid nanoparticle (LNP) edited an HBV DNA target up to 70% after a single administration in vivo.
北卡罗来纳州达勒姆，2020年7月6日（环球新闻社）--Precision BioSciences，Inc.（纳斯达克：DTIL），一家临床阶段生物技术公司，致力于通过其新颖和专有的ARCUS®基因组编辑平台改善生活，今天宣布，公司将重新获得根据2018年与吉列科学公司的合作协议开发的体内慢性乙型肝炎病毒（HBV）项目的全部权利和所
Precision BioSciences首席科学官Derek Jantz博士评论道：“这是一次高效且协调一致的合作，我们非常珍惜这一机会，以推动我们的ARCUS基因组编辑技术，并与全球传染病领导者一道治愈HBV。”。“从这项计划中获得的主要经验以及如何开发肝导向基因编辑治疗候选方案直接适用于我们的体内管道。在考虑与HBV建立新的合作伙伴关系的同时，我们正致力于推进针对原发性高草酸尿1型的内部领先专有基因矫正计划，我们预计今年晚些时候将提名一名临床候选人。”

根据合作协议的条款，Precision BioSciences主要负责HBV研究核酸酶的开发、制备和临床前评估；Gilead资助研发，并负责潜在疗法的临床开发和商业化。合作结束后，自2020年9月4日起，Precision BioSciences将重新获得该项目的全部临床开发和商业化权利。

该公司预计其现金流不会发生任何变化。截至2020年3月31日，Precision的现金和现金等价物为1.542亿美元，预计这将为2021年下半年的运营提供充足的资金。

关于Precision治疗慢性乙型肝炎病毒（HBV）的体内程序
通过直接靶向cccDNA和整合HBV基因组，临床前数据表明ARCUS基因编辑可能是开发一种有潜力的HBV治疗方法。今年早些时候在2020年美国遗传与细胞治疗学会（ASGCT）年会上提出的临床前数据表明，优化的弓形核酸酶有效地靶向并降解cccDNA达75%，随后在HBV感染的原代人肝细胞中敲除表面抗原。来自小鼠研究的数据还显示，脂质纳米粒（LNP）传递的弓形核酸酶在体内一次给药后可编辑HBV DNA靶点达70%。


在动物实验当中基因编辑的效率只有75%，太低了没有清除cccDNA的可能，而且一旦脱靶副作用是难以预测的

newchinabok

sir 发表于 2020-7-7 09:29
DURHAM, N.C., July 06, 2020 (GLOBE NEWSWIRE) -- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinic ...

基因编辑还有很长路要走，根本不指望了