Regeneron Pharmaceuticals is putting more money into gene editing, announcing Monday

再生元制药公司正在投入更多资金用于基因编辑，并于周一宣布。

a partnership with Tessera Therapeutics

与Tessera Therapeutics的合作关系

to develop an experimental program for a rare liver and lung disease.

开发一种针对罕见肝肺疾病的实验性项目。

At the center of the deal is a treatment Tessera, a well-funded startup backed by Flagship Pioneering, is developing for alpha-1 antitrypsin deficiency. Regeneron is paying Tessera $150 million upfront, in the form of cash and an equity investment, to collaborate on the program and split future development costs and profits.

这笔交易的核心是Tessera公司正在开发的一种针对α-1抗胰蛋白酶缺乏症的治疗方法。Tessera是一家由Flagship Pioneering支持、资金充裕的初创公司。再生元将向Tessera支付1.5亿美元的预付款，以现金和股权投资的形式，合作开展该项目并分担未来的开发成本和利润。

Tessera could receive another $125 million in unspecified near and mid-term development milestone payments.   .

Tessera 还可能获得一笔金额达 1.25 亿美元的款项，这笔款项与尚未确定的近期和中期开发里程碑付款有关。

Tessera will lead the initial first-in-human trial, with Regeneron taking the reins for future development and eventually commercialization.

特赛拉将领导最初的首次人体试验，而再生元将接手未来的开发并最终实现商业化。

Alpha-1 antitrypsin deficiency, or AATD, is a rare genetic condition that can cause lung and liver disease, such as

Alpha-1抗胰蛋白酶缺乏症（AATD）是一种罕见的遗传性疾病，可能引发肺部和肝脏疾病，例如

chronic obstructive pulmonary disease and cirrhosis

慢性阻塞性肺疾病和肝硬化

. The disorder is typically caused by a mutation in a gene called SERPINA1 and that provides instructions for a protective protein known as alpha-1 antitrypsin, or AAT.

这种疾病通常是由一个名为SERPINA1的基因突变引起的，该基因提供了产生一种称为α-1抗胰蛋白酶（AAT）的保护性蛋白质的指令。

Treatment options for AATD are limited, don’t reverse existing damage and largely only affect the disease’s impact on the lungs. As a result, the disease has become a top target among

AATD的治疗选择有限，不能逆转现有的损伤，而且在很大程度上只影响肺部的疾病。因此，这种疾病已成为众多研究中的首要目标之一。

drugmakers

制药商

in recent years. Several companies are pursuing a variety of technological approaches, from

近年来，多家公司正在追求各种技术方法，从

RNA editing

RNA编辑

to

至

base editing

碱基编辑

and

和

RNA interference

RNA 干扰

.

。

Some

一些

have suffered

已经遭受

clinical

临床的

setbacks

挫折

.

。

Tessera’s program, TSRA-196, is designed to precisely correct the underlying genetic mutation in AATD and durably restore production of the AAT protein through a one-time treatment. It’s also an “in vivo” gene editing treatment that does its work inside the body, rather than via a laborious process that involves manipulating cells in a lab. .

Tessera的项目TSRA-196旨在精确修正AATD中的根本基因突变，并通过一次性治疗持久恢复AAT蛋白的生产。这也是一种“体内”基因编辑疗法，它在体内发挥作用，而不是通过在实验室中操作细胞的繁琐过程。

“Alpha-1 antitrypsin deficiency is a serious disease with limited treatment options today and is particularly well suited for Tessera’s gene editing approach. Together with Tessera, we have an opportunity to pioneer new frontiers in genetic medicine and redefine what is possible for AATD patients,” said George Yancopoulos, Regeneron’s chief scientific officer, in a statement.  .

“α-1抗胰蛋白酶缺乏症是一种严重疾病，目前治疗选择有限，特别适合Tessera的基因编辑方法。与Tessera一起，我们有机会开拓基因医学的新领域，重新定义AATD患者的可能性，”Regeneron首席科学官George Yancopoulos在一份声明中表示。

TSRA-196 is in early research, however. Tessera presented

TSRA-196 处于早期研究阶段。Tessera 公司展示了

preclinical data

临床前数据

earlier this year and expects to ask U.S. regulators by the end of 2025 to begin human testing.

今年早些时候，并预计将在 2025 年底前请求美国监管机构开始人体试验。

Though Regeneron is best known for the eye drug Eylea, it’s made genetic medicine a focus in recent years. Deals involving companies like

虽然再生元公司最出名的是眼科药物Eylea，但近年来它已将基因药物作为重点。涉及像

Intellia Therapeutics

Intellia治疗公司

,

，

ViGeneron

维真生物

,

，

Decibel Therapeutics

分贝疗法公司

and

和

Mammoth Biosciences

猛犸生物科学

have given the company a pipeline of gene therapy and gene editing medicines. One of those treatments, for a

已经为公司提供了基因治疗和基因编辑药物的渠道。其中一种治疗方法，针对

rare type of inherited hearing loss, could soon get to market.

罕见的遗传性听力损失，很快就能上市。

Tessera, meanwhile, is led by former AbbVie executive

与此同时，Tessera由前艾伯维高管领导。

Michael Severino

迈克尔·塞韦里诺

and has raised

并且已经筹集了

more than half a billion dollars

超过5亿美元

in private funding since its inception.

自成立以来，私人资金。