SAN CARLOS, Calif.--(BUSINESS WIRE)--Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, announced the first pediatric patient has been dosed in a multi-center open-label Phase 2 study of GLM101, a mannose-1-phosphate replacement therapy, in patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG)..

加利福尼亚州圣卡洛斯（商业新闻短讯）--Glycomine，Inc.，一家专注于开发孤儿疾病新疗法的临床阶段生物技术公司，宣布第一名儿科患者已在一项多中心开放标签的GLM101（一种甘露糖-1-磷酸替代疗法）2期研究中服用，用于治疗磷酸甘露糖突变酶2-先天性糖基化障碍（PMM2-CDG）患者。。

“The initiation of dosing in pediatrics is an important milestone for the company,” said Steven Axon, CEO of Glycomine. “We are encouraged by the biomarker and clinical improvements we have observed in the dose-range finding portion of the trial in adult patients, and we are excited to move forward into pediatrics with GLM101, the first potential disease-modifying therapy to be evaluated in PMM2-CDG.”.

Glycomine首席执行官史蒂文·阿克森（StevenAxon）表示：“儿科给药的开始对公司来说是一个重要的里程碑。“我们对在成年患者试验的剂量范围发现部分观察到的生物标志物和临床改进感到鼓舞，我们很高兴能够使用GLM101进入儿科，这是第一种在PMM2-CDG中评估的潜在疾病缓解疗法。”。

“We are delighted to continue our collaboration with Glycomine and move forward with GLM101 to initiate dosing in pediatrics,” added Mercedes Serrano, M.D., Ph.D., Sant Joan de Déu Hospital in Barcelona. “We are happy to be a part of an effort that has the potential to make a positive impact on PMM2-CDG patients and the community.”.

巴塞罗那圣琼德杜医院梅赛德斯·塞拉诺医学博士补充道：“我们很高兴继续与Glycomine合作，并与GLM101合作，开始儿科给药。”。“我们很高兴能够成为有可能对PMM2-CDG患者和社区产生积极影响的努力的一部分。”。

Following a dose range finding study that enrolled 10 adult PMM2-CDG patients, this Phase 2 study in pediatric patients with a confirmed diagnosis of PMM2-CDG will be an open-label, 24-week treatment with GLM101 (ClinicalTrials.gov Identifier: NCT05549219). The purpose of the study is to evaluate potential pharmacodynamic activity and clinical efficacy, as well as safety, tolerability, and pharmacokinetics of GLM101..

在招募了10名成年PMM2-CDG患者的剂量范围研究之后，这项针对确诊为PMM2-CDG的儿科患者的2期研究将是GLM101（ClinicalTrials.gov标识符：NCT05549219）的开放标签24周治疗。该研究的目的是评估GLM101的潜在药效学活性和临床疗效，以及安全性，耐受性和药代动力学。。

About GLM101

关于GLM101

GLM101 is a mannose-1-phosphate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG Type Ia. GLM101 has received Orphan Drug Designation in the U.S. and Europe and Rare Pediatric Disease Designation in the U.S. PMM2-CDG is caused by genetic mutations that lead to a deficiency of the enzyme phosphomannomutase 2 (encoded by the PMM2 gene).

GLM101是一种正在开发的甘露糖-1-磷酸替代疗法，用于治疗磷酸甘露糖突变酶2-先天性糖基化疾病（PMM2-CDG），以前称为CDG Ia型。GLM101在美国和欧洲被指定为孤儿药，在美国被指定为罕见儿科疾病。PMM2-CDG是由导致磷酸甘露糖突变酶2（由PMM2基因编码）缺乏的基因突变引起的。

GLM101 is designed to deliver mannose-1-phosphate directly into cells and thereby bypass the PMM2 enzyme deficiency and address disease-causing PMM2 mutations to restore pathway function..

GLM101旨在将甘露糖-1-磷酸直接递送到细胞中，从而绕过PMM2酶缺乏症，并解决引起疾病的PMM2突变以恢复途径功能。。

About Glycomine, Inc.

关于Glycomine，Inc。

Glycomine is a clinical-stage biotechnology company developing novel drugs for serious rare disorders of metabolism and protein misfolding for which no other therapeutic options exist. The company's approach is to use replacement therapies – substrates, enzymes, or proteins – and to target those molecules to clinically relevant cellular compartments.

Glycomine是一家临床阶段的生物技术公司，正在开发治疗严重罕见的代谢异常和蛋白质错误折叠的新药，目前尚无其他治疗选择。该公司的方法是使用替代疗法-底物，酶或蛋白质-并将这些分子靶向临床相关的细胞区室。

The company is based in San Carlos, California and supported by leading international life sciences investors. For more info, visit www.glycomine.com..

该公司总部位于加利福尼亚州圣卡洛斯，由领先的国际生命科学投资者支持。有关更多信息，请访问www.glycomine.com。。