RICHMOND, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced it has entered into a license agreement with Genentech, a member of the Roche Group, to develop intravenously administered genomic medicines to treat certain neurodegenerative diseases.

基因组药物公司 Sangamo Therapeutics, Inc.（纳斯达克股票代码：SGMO）今天宣布与罗氏集团旗下的基因泰克公司（Genentech）达成了一项许可协议，共同开发用于治疗某些神经退行性疾病的静脉注射基因组药物。

Sangamo has granted Genentech an exclusive license to Sangamo’s proprietary zinc finger repressors that are directed to the tau gene, which is critically involved in Alzheimer’s disease and other tauopathies, as well as an undisclosed second neurology target. Sangamo has also agreed to exclusively license to Genentech, for tau and the second neurology target, Sangamo’s proprietary, neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has demonstrated potent blood-brain barrier penetration and brain transduction in nonhuman primates..

Sangamo已向Genentech授予Sangamo专有锌指阻遏物的独家许可证，该锌指阻遏物针对tau基因，该基因与阿尔茨海默病和其他tau蛋白病以及未公开的第二神经学靶标密切相关。Sangamo还同意向Genentech独家许可tau和第二个神经学靶标Sangamo专有的嗜神经腺相关病毒（AAV）衣壳STAC-BBB，该衣壳已在非人灵长类动物中证明了有效的血脑屏障穿透和脑转导。。

“Sangamo has been pioneering the field of genomic medicine for years to address devastating neurodegenerative diseases with limited current treatment options,” said Sandy Macrae, Chief Executive Officer of Sangamo. “We strongly believe in the power of our zinc finger technology to regulate the expression of key genes involved in disease.

Sangamo首席执行官桑迪·麦克雷（SandyMacrae）表示：“多年来，Sangamo一直在基因组医学领域开拓先河，以解决目前治疗选择有限的毁灭性神经退行性疾病。”。“我们坚信锌指技术能够调节疾病关键基因的表达。

The recent discovery of our industry-leading intravenously delivered AAV capsid, STAC-BBB, has the potential to address longstanding challenges in delivering therapeutics to the central nervous system. We are excited to share this powerful combination with Genentech to advance potential treatment options for devastating neurodegenerative disorders, and we are hopeful this could be the first of multiple capsid collaborations to come with other partners.”.

最近发现的业界领先的静脉注射AAV衣壳STAC-BBB有可能解决向中枢神经系统提供治疗的长期挑战。我们很高兴与基因泰克分享这一强大的组合，以推进破坏性神经退行性疾病的潜在治疗选择，我们希望这可能是与其他合作伙伴合作的多个衣壳合作中的第一个。”。

“We are uniquely positioned with our collective experience, expertise and resources in neurological research to explore transformative approaches, including gene therapy, that treat neurodegenerative diseases,” said Boris L. Zaïtra, Head of Roche Corporate Business Development. “Through groundbreaking research and partnerships with companies such as Sangamo, we are committed to pursuing important breakthroughs in both early diagnosis and treatment.

罗氏公司业务发展负责人鲍里斯·扎伊特拉（BorisL.Zaïtra）表示：“我们凭借在神经学研究方面的集体经验、专业知识和资源，在探索治疗神经退行性疾病的变革方法（包括基因治疗）方面拥有独特的地位。”。“通过开创性的研究以及与Sangamo等公司的合作，我们致力于在早期诊断和治疗方面取得重大突破。

Our relentless pursuit of scientific innovation is taking us into areas of enormous unmet medical need and progress in treating diseases of the brain and nervous system.”.

我们对科学创新的不懈追求正在将我们带入巨大的未满足的医疗需求领域，并在治疗大脑和神经系统疾病方面取得进展。”

Under the terms of the agreement, Sangamo is responsible for completing a technology transfer and certain preclinical activities, and Genentech is responsible for all clinical development, regulatory interactions, manufacturing and global commercialization. Genentech is expected to pay Sangamo $50 million in near-term upfront license fees and milestone payments.

根据协议条款，Sangamo负责完成技术转让和某些临床前活动，Genentech负责所有临床开发、监管互动、制造和全球商业化。Genentech预计将向Sangamo支付5000万美元的近期预付许可费和里程碑付款。

Sangamo is eligible to earn up to $1.9 billion in development and commercial milestones spread across multiple potential products under the agreement and tiered royalties on net sales of such products, subject to certain specified reductions..

Sangamo有资格在协议下跨越多个潜在产品的开发和商业里程碑中获得高达19亿美元的收入，并根据这些产品的净销售额收取分层版税，但须进行某些特定的削减。。

Sangamo continues to engage in business development discussions with additional potential collaboration partners about the Sangamo STAC-BBB capsid delivery platform, its epigenetic regulation capabilities, and other assets, including Fabry disease.

Sangamo继续与其他潜在合作伙伴就Sangamo STAC-BBB衣壳递送平台，其表观遗传调控能力以及包括法布里病在内的其他资产进行业务发展讨论。

About Sangamo Therapeutics

关于Sangamo Therapeutics

Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and that its capsid discovery platform can expand delivery beyond currently available intrathecal delivery capsids, including in the central nervous system.

Sangamo Therapeutics是一家基因组医学公司，致力于将开创性的科学转化为药物，以改变患有严重神经系统疾病但没有足够或任何治疗选择的患者和家庭的生活。Sangamo认为，其锌指表观遗传调节剂非常适合解决潜在的破坏性神经系统疾病，其衣壳发现平台可以将递送范围扩大到目前可用的鞘内递送衣壳，包括中枢神经系统。