Glycomine, a biotechnology startup working on a treatment for a rare genetic disorder, has raised a $115 million Series C round to push its lead program deeper into clinical testing.

Glycomine是一家致力于治疗罕见遗传病的生物技术初创公司，已经筹集了1.15亿美元的C轮融资，以推动其主要项目深入临床试验。

The funding announced Wednesday for the San Carlos, California-based company will support development of its therapy GLM101, which is designed to treat phosphomannomutase 2-congenital disorder of glycosylation, or PMM2-CDG.

周三宣布的为加利福尼亚州圣卡洛斯市的公司提供的资金将支持其治疗药物GLM101的开发，该药物旨在治疗磷酸甘露糖变位酶2-先天性糖基化障碍（PMM2-CDG）。

PMM2-CDG is the most common among a

PMM2-CDG 是最常见的

family of inherited disorders

遗传性疾病的家族

that cause errors in glycosylation, the biological process by which sugar chains called glycans are added to proteins. This type of malfunction can affect the structure and function of cellular proteins throughout the body, leading to an array of symptoms, developmental delays and impaired motor function.

这些错误会导致糖基化过程出现问题，糖基化是将称为聚糖的糖链添加到蛋白质中的生物过程。这种类型的故障会影响全身细胞蛋白质的结构和功能，导致一系列症状、发育迟缓和运动功能受损。

There are no approved treatments for the roughly 15,000 people who are estimated to have the condition in the U.S. and Europe, though a .

据估计，美国和欧洲大约有15,000人患有此病，但目前尚无获批的治疗方法，尽管有一个。

few

很少

programs

程序

are in clinical testing.

正在进行临床测试。

“That need is extremely high,” said Steven Axon, Glycomine’s CEO. “There's no other therapies for these patients. There's only supportive care.”

“这种需求非常高，”Glycomine首席执行官史蒂文·阿克森说。“这些患者没有其他疗法，只有支持性护理。”

Glycomine aims to change that by delivering into the body a component used in glycosylation — mannose-1-phosphate — that’s deficient in people with PMM2-CDG.

Glycomine 旨在通过向体内输送一种糖基化过程中所需的成分——甘露糖-1-磷酸——来改变这一状况，这种成分在PMM2-CDG患者中是缺乏的。

The company’s approach is somewhat akin to the enzyme replacement therapies used to treat

该公司的方法与用于治疗的酶替代疗法有些相似

lysosomal storage disorders

溶酶体贮积症

like Fabry or Pompe disease. Glycomine’s therapy is replacing a missing sugar molecule rather than an enzyme, however, and corrects a defective chemical process instead of clearing an accumulating toxin.

像法布里病或庞贝病。然而，Glycomine的疗法是替换缺失的糖分子而不是酶，并且修正了有缺陷的化学过程，而不是清除积累的毒素。

Last year, Glycomine released

去年，Glycomine发布了

Phase 2 data

第二阶段数据

showing the drug appeared to impact ataxia, a common symptom of PMM2-CDG, in a small group of patients. The Series C round will carry the company through a more rigorous, randomized and placebo-controlled Phase 2b study that should start in the middle of this year and enroll between 40 to 50 participants, Axon said..

显示该药物似乎对共济失调（PMM2-CDG 的常见症状）在一小群患者中有影响。Axon 公司表示，C 轮融资将推动公司进行一项更为严格、随机和安慰剂对照的 2b 期研究，该研究应该在今年年中开始，计划招募 40 至 50 名参与者。

The company hopes to report data in 2026 that “will really tell us whether the effect we were seeing in the earlier clinical program has been confirmed,” he said.

他表示，公司希望在2026年报告数据，“这些数据将真正告诉我们早期临床项目中看到的效果是否得到了证实。”

If those results are positive, Axon said the company may explore a partnership with a pharmaceutical company.

如果结果是积极的，Axon 公司表示可能会探索与制药公司的合作。

“Having this randomized controlled data and really knowing that the drug works and its path to approval would be highly derisking for a pharma partner,” he said.

“拥有这些随机对照数据，并真正了解药物的有效性及其获得批准的路径，将大大降低制药合作伙伴的风险，”他说道。

Though Glycomine has not disclosed specifics about the rest of its pipeline, it’s developing drug candidates for other rare diseases outside of PMM2-CDG, according to Axon.

据 Axon 称，虽然 Glycomine 尚未披露其管线中其他药物的具体信息，但该公司正在开发针对 PMM2-CDG 之外其他罕见疾病的候选药物。

The company’s Series C funding was led by CTI Life Sciences Fund, funds managed by Abrdn, and Advent Life Sciences. Other backers include Novo Holdings, Sanofi Ventures and Abingworth. Glycomine previously raised $80 million across

公司C轮融资由CTI生命科学基金、Abrdn管理的基金和Advent生命科学领投。其他支持者包括诺和控股、赛诺菲风险投资和Abingworth。Glycomine此前筹集了8000万美元。

Series A

A轮

and

和

B rounds

B轮

.

。