Sangamo Therapeutics was hit hard by the loss of Pfizer as a partner at the end of last year, but a new $1.4 billion alliance with Eli Lilly has gone some way to soften the blow and provide a much-needed cash injection.

去年年底辉瑞退出合作，令桑加莫制药公司受到重创，但与礼来公司新达成的 14 亿美元联盟协议在一定程度上缓解了这一打击，并提供了急需的现金注入。

The agreement – which includes an $18 million upfront fee – gives Lilly rights to deploy Sangamo's neurotropic adeno-associated virus (AAV) capsid STAC-BBB across one potential disease target, along with an option to expand the programme to include another four.

该协议包括 1800 万美元的预付款，使礼来公司有权在 Sangamo 的神经向性腺相关病毒 (AAV) 衣壳 STAC-BBB 的一个潜在疾病靶点上进行部署，并可选择将该计划扩展至另外四个靶点。

STAC-BBB was selected out of 100 million capsids that were screened for their ability to cross into the central nervous system after intravenous administration and deliver genetic sequences to nerve cells (neuronal transduction). In non-human primate studies, it has been shown to achieve 700-fold higher expression of messenger RNA than a regular AAV vector..

STAC-BBB是从一亿个经过筛选的衣壳中选出的，这些衣壳因其在静脉注射后能够穿越进入中枢神经系统并将基因序列递送到神经细胞（神经元转导）而被筛选。在非人灵长类动物研究中，它已被证明比普通AAV载体实现高出700倍的信使RNA表达。

The capsid has also been designed to 'de-target' peripheral tissues, according to Sangamo, so should allow gene therapies to be activated selectively in the CNS and reduce the risk of off-target side effects.

根据桑加莫公司的说法，这种衣壳还被设计为“脱离”周围组织，因此应该能够允许基因疗法在中枢神经系统中选择性激活，并降低脱靶副作用的风险。

The deal with Lilly comes after Pfizer delivered a

与礼来的交易是在辉瑞公司交付了之后进行的

bombshell

重磅炸弹

to Sangamo in December when it handed back rights to experimental gene therapy for haemophilia A, giroctocogene fitelparvovec, despite positive phase 3 results. That decision is thought to be related to the slow uptake in the market of BioMarin's rival gene therapy Roctavian (valoctocogene roxaparvovec)..

去年12月，当Sangamo收回其用于治疗A型血友病的实验性基因疗法giroctocogene fitelparvovec的权利时，尽管3期试验结果积极，但该决定被认为与BioMarin的竞争对手基因疗法Roctavian（valoctocogene roxaparvovec）在市场上的缓慢接受度有关。

Lilly is the third company to partner with Sangamo on STAC-BBB after

礼来是继 Sangamo 之后第三家与 STAC-BBB 合作的公司

Astellas Gene Therapies

安斯泰来基因疗法

, which paid $20 million upfront to kick off a $1.3 billion partnership in December, and

，该公司于去年12月支付了2000万美元预付款，以启动一项价值13亿美元的合作关系，并且

Roche

罗氏

which made an initial payment of $50 million in a deal worth upwards of $1.9 billion last August.

去年八月，该交易价值超过19亿美元，其中5000万美元为预付款。

The deals have provided invaluable top-up cash to Sangamo, which has been forced to

这些交易为Sangamo提供了宝贵的额外现金，该公司已被迫

shed

棚屋

staff and research programmes in the last couple of years after losing other partnerships with Novartis and Biogen on its zinc finger gene-editing technology and abandoning a gene therapy for sickle cell disease.

在失去与诺华和百健在锌指基因编辑技术上的其他合作关系，并放弃了一项镰状细胞病的基因疗法之后，该公司在过去的几年里进行了员工和研究项目的调整。

The Richmond, California-based company – which has changed its strategy to focus on epigenetic regulation therapies treating neurological diseases and novel AAV capsids – ended 2024 with just under $42 million in cash reserves, down from $81 million a year earlier, and said last month that was only sufficient to fund operations 'into the middle of the second quarter of 2025,'.

这家总部位于加利福尼亚州里士满的公司——已将其战略转变为专注于治疗神经系统疾病的表观遗传调控疗法和新型AAV衣壳——在2024年底拥有近4200万美元的现金储备，低于一年前的8100万美元，并于上月表示，这笔资金仅足够维持运营至“2025年第二季度中期”。

Lilly has a prior AAV capsid collaboration with Sangamo on the go via its subsidiary Prevail Therapeutics, worth up to $1 billion and signed in 2023, that is focusing on multiple undisclosed neurological targets.

礼来公司通过其子公司普雷维尔治疗公司与桑加莫在2023年签署了一项价值高达10亿美元的AAV衣壳合作项目，该项目专注于多个未公开的神经学靶点。