GNE myopathy is a distal myopathy that is thought to be caused by a mutation in the GNE gene that encodes an enzyme in the biosynthetic process of aceneuramic acid (typical sialic acid). The investigators will examine the efficacy and safety of aceneuramic acid (SA-ER tablets) 6g daily for 48 weeks in patients with GNE myopathy in a placebo-controlled, double-blind, controlled trial.

Start Date08 Feb 2021

Sponsor / Collaborator

Nobelpharma Co., Ltd.

CTR20200160 / TerminatedPhase 3

一项旨在检查DAS181治疗免疫功能低下受试者下呼吸道副流感病毒感染的有效性和安全性的III期、随机化、安慰剂对照研究

[Translation] A Phase III, randomized, placebo-controlled study to examine the efficacy and safety of DAS181 for the treatment of lower respiratory tract parainfluenza virus infections in immunocompromised subjects

主要目的是检查DAS181治疗免疫功能低下受试者下呼吸道副流感病毒感染的有效性和安全性：证明DAS181相比安慰剂在临床结局方面的疗效；评估DAS181组相比安慰剂组PoI中DAS181治疗的安全性和耐受性。

[Translation]

The primary objectives are to examine the efficacy and safety of DAS181 in the treatment of parainfluenza virus infections in the lower respiratory tract of immunocompromised subjects: to demonstrate the efficacy of DAS181 compared with placebo in clinical outcomes; and to evaluate the safety and tolerability of DAS181 treatment in the PoI in the DAS181 group compared with the placebo group.

Start Date20 Nov 2020

Sponsor / Collaborator

Quintiles Enterprise Management (Shanghai) Co., Ltd.

NCT04354389 / WithdrawnPhase 2/3

DAS181 for COVID-19: A Phase II Multicenter, Randomized, Placebo-Controlled, Double-Blind Study

It is a multicenter, randomized, placebo-controlled, double-blind study. The study population is defined as subjects diagnosed with lower respiratory tract COVID-19 who require supplemental oxygen ≥2 LPM at the time of randomization.

Start Date25 Jul 2020

Sponsor / Collaborator

Ansun Biopharma, Inc.

100 Clinical Results associated with sialic acid

100 Translational Medicine associated with sialic acid

0 Patents (Medical) associated with sialic acid

1,087

Literatures (Medical) associated with sialic acid

01 Dec 2024·European Journal of Medicinal Chemistry

Trivalent oleanolic acid-glucose conjugates: Synthesis and efficacy against Influenza A virus

Article

Author: Yang, Fan ; Zhang, Jihong ; Cai, Ming ; Zhang, Yuan ; Yu, Fei ; Zhen, Jie ; Ou, Xia

Influenza A virus (IAV) leads to significant morbidity and mortality due to the seasonal epidemics and spread. We have demonstrated that oleanolic acid (OA) C28 glucose conjugates and OA trimers are capable of effectively blocking the recognition and interaction between the influenza virus and host cells. In this study, a series of OA-glucose trimers were designed and synthesized through the CuAAC reaction. All trimers underwent screening for anti-IAV activities in vitro. Among these, compounds 13a and 13b showed inhibitory activity against the influenza virus, with IC₅₀ values of 0.68 μM and 0.47 μM, respectively, demonstrating greater potency than oseltamivir (IC₅₀ = 1.36 μM). Results from the time-of-addition experiment and hemagglutination inhibition assay suggest that these OA-glucose trimers may disrupt the recognition between the HA protein of IAV and sialic acid receptors on host cells, thus blocking viral entry. Furthermore, it was found that compound 13b effectively inhibits IAV infection in BALB/c mice. This study has elucidated the structure-activity relationships of OA trimers against the influenza virus and highlighted the utility of multivalent OA conjugates for enhancing ligand-target interactions in anti-influenza virus drug design, laying a groundwork for future research into the antiviral applications of these natural products.

01 Nov 2024·Advances in Colloid and Interface Science

Phenylboronic acid-functionalized biomaterials for improved cancer immunotherapy via sialic acid targeting

Review

Author: Jangid, Ashok Kumar ; Kim, Kyobum

Phenylboronic acid (PBA) is recognized as one of the most promising cancer cell binding modules attributed to its potential to form reversible and dynamic boronic ester covalent bonds. Exploring the advanced chemical versatility of PBA is crucial for developing new anticancer therapeutics. The presence of a specific Lewis acidic boron atom-based functional group and a Π-ring-connected ring has garnered increasing interest in the field of cancer immunotherapy. PBA-derivatized functional biomaterials can form reversible bonds with diols containing cell surface markers and proteins. This review primarily focuses on the following topics: (1) the importance and versatility of PBA, (2) different PBA derivatives with pKa values, (3) specific key features of PBA-mediated biomaterials, and (4) cell surface activity for cancer immunotherapy applications. Specific key features of PBA-mediated materials, including sensing, bioadhesion, and gelation, along with important synthesis strategies, are highlighted. The utilization of PBA-mediated biomaterials for cancer immunotherapy, especially the role of PBA-based nanoparticles and PBA-mediated cell-based therapeutics, is also discussed. Finally, a perspective on future research based on PBA-biomaterials for immunotherapy applications is presented.

01 Nov 2024·Free Radical Biology and Medicine

Microscopic mechanistic study of the penetration distributions for plasma reactive oxygen and nitrogen species based on sialic acid targeting on the cell membrane surface

Article

Author: Wang, Daohan ; Zhao, Tong ; Niu, Yanxiong ; Cui, Yanxiu ; Wang, Xiaolong ; Zhang, Yuantao

The ability of cold atmospheric plasmas (CAPs) to produce a wide range of active constituents while maintaining a low or even room temperature of the gas has made it a novel research area of great interest. During plasma action, cancer cell membrane surface components are susceptible to oxidative modification by reactive oxygen and nitrogen species (RONS). In this study, the process of oxidative modification of membrane surface components sialic acid by RONS was investigated based on molecular dynamics simulations, and the penetration mechanism of long-lived particles ONOOH and its homolytic products at the membrane-water interface and the effect of appropriate electric field action were studied. The results showed that cancer cells with high sialic acid expression were less stable than healthy cells. Plasma treatment may promote the ONOOH homolysis process, and its homolysis product OH free radical is more likely to adsorb near sialic acid molecules by hydrogen bonding, resulting in oxidative modification. The interaction force between OH free radical and sialic acid molecules is stronger than ONOOH, which helps to further understand the oxidative modification reaction in membrane environment. At the same time, appropriate electric field stimulation can enhance the depth of penetration of RONS to more effectively treat the pathological state of biological tissues. The study proposes the use of membrane surface sialic acid as a cancer therapeutic target and provides guidance for improving the depth of RONS penetration and maximizing the survival of healthy cells, which contributes to the further clinical translation of plasma biomedicine.

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