METHODS OF TREATING POMPE DISEASE
    33.
    发明申请

    公开(公告)号:US20220273615A1

    公开(公告)日:2022-09-01

    申请号:US17630929

    申请日:2020-09-30

    Abstract: Disclosed herein are novel uses of ADMDP stereoisomers or their derivatives for the manufacture of a medicament for treating Pompe disease. Accordingly, the present disclosure provides a method of treating Pompe disease in a subject. The method includes the step of, administering to the subject a therapeutically effective amount of a compound of formula (I), or a salt, an ester or a solvate thereof, wherein R1 and R2 are independently H or alkyl optionally substituted by —NH2 or —OH, so as to ameliorate, alleviate mitigate and/or prevent symptoms associated with the Pompe disease. According to certain embodiments of the present disclosure, the compound of formula (I) may serve a stabilizer of α-glucosidase via preventing its denaturalization of deactivation.

    Methods for modifying human antibodies by glycan engineering

    公开(公告)号:US11377485B2

    公开(公告)日:2022-07-05

    申请号:US16150164

    申请日:2018-10-02

    Abstract: Methods for making modified Fc regions of antibodies and antibody fragments, both human and humanized, and having enhanced stability and efficacy, are provided. Antibodies comprising Fc regions with core fucose residues removed, and attached to oligosaccharides comprising terminal sialyl residues, are provided. Antibodies comprising homogeneous glycosylation of Fc regions with specific oligosaccharides are provided. Fc regions conjugated with homogeneous glycoforms of monosaccharides and trisaccharides, are provided. Methods of preparing human antibodies with modified Fc using glycan engineering, are provided.

    Encapsulated functionalized diamond crystal

    公开(公告)号:US11366119B2

    公开(公告)日:2022-06-21

    申请号:US15990189

    申请日:2018-05-25

    Abstract: This invention is a functionalized diamond crystal with high dispersibility in high ionic strength solution and/or with specific targeting ability, which comprise a diamond crystal and a fatty acid layer. The fatty acid layer works a surfactant and provides a specific targeting feature for the diamond crystal. The feature of the surfactant makes the diamond crystal being easily dispersed in biological surrounding (e.g., phosphate saline buffer) and the feature of specific targeting ability provides the diamond crystal with specific recognizing specific targets. This invention allows researchers to use diamond crystal as a marker for specific labelling.

    Nuclear paraspeckle assembly transcript 1 as therapeutic targeting in neurodegeneration

    公开(公告)号:US11357788B2

    公开(公告)日:2022-06-14

    申请号:US16355545

    申请日:2019-03-15

    Abstract: The present disclosure provides a method of reducing neurodegeneration and/or TDP43 associated aggregation, comprising knocking down the expression of Nuclear Paraspeckle Assembly Transcript 1 (NEAT1) or LncRNA NEAT1. Also provided are methods for screening a candidate agent that reduces neurodegeneration and/or TDP43 associated aggregation in a cell, treating or preventing a neurodegenerative disorder, delaying or preventing the onset of a neurodegenerative disorder or reducing a risk for developing a neurodegenerative disorder in a subject and determining whether a subject is suffering from, or at a risk of developing a neurodegenerative disorder, comprising measuring the presence of cytoplasmic NEAT1 in a biological sample, wherein the presence is indicative of the risk of developing a neurodegenerative disorder.

Patent Agency Ranking