Epicrispr Biotechnologies has raised another $90 million to accelerate development of a first-of-its-kind treatment for a rare genetic muscle disorder.

Epicrispr Biotechnologies 公司再次筹集 9,000 万美元，以加速开发一种针对罕见遗传性肌肉疾病的首创疗法。

That treatment, EPI-321, harnesses what’s known as “epigenetic editing,” an emerging form of genetic medicine that

该疗法EPI-321利用了被称为“表观遗传编辑”的技术，这是一种新兴的基因医学形式

uses CRISPR tools to switch genes on or off

利用CRISPR工具开启或关闭基因

rather than alter DNA. With EPI-321, Epicrispr is using this strategy to silence the genetic driver of

而非改变DNA。Epicrispr公司正利用这一策略，通过EPI-321来沉默基因驱动因子

f

f

acioscapulohumeral muscular dystrophy, or

肩肱型肌营养不良，或

FSHD, a progressive neuromuscular condition. The company has completed enrollment in an early-stage study.

FSHD是一种进行性神经肌肉疾病。该公司已完成一项早期阶段研究的受试者入组。

The Series C round announced Tuesday “

周二宣布的C轮融资

marks a pivotal milestone for Epicrispr as we advance EPI-321 and the next generation of programmable epigenetic medicines,” said CEO Amber Salzman, in a statement.

“这标志着Epicrispr的一个关键里程碑，因为我们正在推进EPI-321以及下一代可编程表观遗传药物。”首席执行官Amber Salzman在一份声明中表示。

Epigenetic editing has captured the attention of researchers, companies and

表观遗传编辑已引起研究人员和公司的关注，以及

investors alike

投资者也同样

because of its potential to subtly dial up or down gene expression without breaking or rewriting DNA. Proponents believe that technique might be less risky than DNA editing approaches and help genetic medicine reach a wider range of diseases.

因为它具有在不破坏或重写DNA的情况下微妙地上调或下调基因表达的潜力。支持者认为，该技术可能比DNA编辑方法风险更低，并有助于让基因医学惠及更广泛的疾病。

In FSHD, for instance, the overexpression of a gene called DUX4 causes muscle atrophy and degeneration. Rather than cut into that gene, EPI-321 binds to a specific area of DUX4 and makes a chemical modification that suppresses production of its encoded protein. The hope is that effect will stop the death of muscle cells and improve function.

例如，在面肩肱型肌营养不良症（FSHD）中，名为 DUX4 的基因过表达会导致肌肉萎缩和退化。EPI-321 并非对该基因进行切割，而是结合到 DUX4 的特定区域，并通过化学修饰抑制其编码蛋白的产生。希望这一作用能够阻止肌肉细胞死亡并改善功能。

Early clinical data have shown the potential to boost muscle volume and impact biological markers associated with DUX4 suppression. .

早期临床数据显示，其具有增加肌肉体积并影响与DUX4抑制相关的生物标志物的潜力。

“While these are still early data from a small number of patients, they provide important initial evidence supporting EPI-321’s potential to address the underlying biology of

“尽管这些数据仍来自少量患者的早期研究，但它们提供了重要的初步证据，支持EPI-321在解决潜在生物学机制方面的潜力。”

FSHD

面肩肱型肌营养不良症

,” Salzman wrote in an email to

”，萨尔兹曼在一封电子邮件中写道

BioPharma

生物制药

Dive.

潜入。

Results with six months of study follow-up should come in early October, she said.

她表示，为期六个月的随访研究结果将于10月初公布。

Multiple other drugmakers have zeroed in on FSHD, many of which aim to

其他多家制药公司也已将焦点集中在面肩肱型肌营养不良症（FSHD）上，其中许多公司旨在

block DUX4

DUX4 区块

in one way or another. Prospective treatments from

以某种方式。来自的前瞻性治疗

Novartis

诺华

,

，

Arrowhead Pharmaceuticals

箭头制药公司

,

，

Dyne Therapeutics

戴恩治疗公司

and

和

Sarepta Therapeutics

萨雷普塔治疗公司

, among others, are in human testing.

，除其他外，正在进行人体试验。

Epicrispr has raised

Epicrispr 已筹集

$213 million

2.13亿美元

since its inception

自其成立以来

. Octagon Capital and Janus Henderson Investors and included co-led the latest financing, which included more than half a dozen other backers, among them Fidelity Management & Research, Sanofi Ventures and Cormorant Asset Management.

Octagon Capital 与 Janus Henderson Investors 共同领投了最新一轮融资，其他投资方还包括 Fidelity Management & Research、Sanofi Ventures 和 Cormorant Asset Management 等六家以上机构。

Anran Li, an investment analyst at Octagon Capital, will join the startup’s board of directors.

八角资本的投资分析师李安然将加入该初创公司的董事会。

The

这

new financing

新融资

gives Epicrispr “the resources and flexibility” to advance EPI-321, invest in its pipeline and “build the company for the long term,”

为Epicrispr提供了“资源和灵活性”，以推进EPI-321的研发，投资其产品管线，并“为公司长远发展奠定基础”。

Salzman said.

萨尔兹曼说。