Patent classifications
C07H15/08
GALNAC PHOSPHORAMIDITES, NUCLEIC ACID CONJUGATES THEREOF AND THEIR USE
This invention generally relates to the field of phosphoramidite derivatives. In particular, the invention relates to N-Acetylgalactosamine phosphoramidite molecules and to conjugates of nucleic acid molecules with N-Acetylgalactosamine containing molecules. Also provided are methods for preparation of these molecules and possible uses thereof, in particular in medicine.
Modified oligonucleotides and compound that can be used for synthesizing same
The present disclosure falls within the field of biomedical technology, and in particular relates to modified oligonucleatides and a compound that can be used for synthesizing same and a method for modifying oligonucleotides. The present disclosure also relates to the use of the modified oligonucleotides for preventing and/or treating diseases associated with the liver in a subject.
PROCESS FOR THE PREPARATION OF GALNAC OLIGONUCLEOTIDE CONJUGATES
The invention comprises a process for the preparation of GalNAc oligonucleotide conjugates which comprises the coupling of a GalNAc cluster compound of the formula
##STR00001##
or corresponding salts, enantiomers and/or a stereoisomer thereof, with an oligonucleotide in the presence of an O-dicarboximidouronium salt as coupling agent.
Substituted-6,8-dioxabicyclo[3.2.1]octane-2,3-diol compounds as targeting agents of ASGPR
Compounds of Formula (A) are described herein and the uses thereof for the treatment of diseases, conditions and/or disorders mediated by pharmaceutical compositions and the uses thereof as asialoglycoprotein receptor (ASGPR) targeting agents. ##STR00001##
Substituted-6,8-dioxabicyclo[3.2.1]octane-2,3-diol compounds as targeting agents of ASGPR
Compounds of Formula (A) are described herein and the uses thereof for the treatment of diseases, conditions and/or disorders mediated by pharmaceutical compositions and the uses thereof as asialoglycoprotein receptor (ASGPR) targeting agents. ##STR00001##
UTILITY OF NEMATODE SMALL MOLECULES
The present invention relates to methods of treating immune disorders and/or inflammation using certain modulator compounds. In one embodiment, the present invention provides a method of treating an immune and inflammatory disorders disorder by administering a composition comprising a therapeutically effective dosage of an ascaroside compound, or a mixture of ascaroside compounds, or a mixture containing at least one ascaroside.
UTILITY OF NEMATODE SMALL MOLECULES
The present invention relates to methods of treating immune disorders and/or inflammation using certain modulator compounds. In one embodiment, the present invention provides a method of treating an immune and inflammatory disorders disorder by administering a composition comprising a therapeutically effective dosage of an ascaroside compound, or a mixture of ascaroside compounds, or a mixture containing at least one ascaroside.
NOVEL AMPHIPHILIC COMPOUND HAVING DENDRONIC HYDROPHOBIC GROUP AND APPLICATION THEREOF
The present invention relates to an amphiphilic compound having a dendronic hydrophobic group, a method for preparing the same, and a method for extraction, solubilization, stabilization, or crystallization of a membrane protein by using the same. The use of the compound according to the present invention leads to an excellent membrane protein solubilization effect and a stable storage of a membrane protein in an aqueous solution for a long time, and thus can be utilized for functional analysis and structural analysis of the membrane protein. Especially, the amphiphilic compound having a dendronic hydrophobic group showed very remarkable characteristics in the visualization of protein composites through an electronic microscope. The membrane protein structural and functional analysis is one of the fields of greatest interest in current biology and chemistry, and more than half of the new drugs that are currently being developed are targeted at membrane proteins, and thus the amphiphilic compound of the present invention can be applied to membrane protein structure studies closely related to the development of new drugs.
NOVEL AMPHIPHILIC COMPOUND HAVING DENDRONIC HYDROPHOBIC GROUP AND APPLICATION THEREOF
The present invention relates to an amphiphilic compound having a dendronic hydrophobic group, a method for preparing the same, and a method for extraction, solubilization, stabilization, or crystallization of a membrane protein by using the same. The use of the compound according to the present invention leads to an excellent membrane protein solubilization effect and a stable storage of a membrane protein in an aqueous solution for a long time, and thus can be utilized for functional analysis and structural analysis of the membrane protein. Especially, the amphiphilic compound having a dendronic hydrophobic group showed very remarkable characteristics in the visualization of protein composites through an electronic microscope. The membrane protein structural and functional analysis is one of the fields of greatest interest in current biology and chemistry, and more than half of the new drugs that are currently being developed are targeted at membrane proteins, and thus the amphiphilic compound of the present invention can be applied to membrane protein structure studies closely related to the development of new drugs.
INOSITOL DERIVATIVES FOR USE IN PATHOLOGICAL CRYSTALLIZATION
The present invention relates to inositol derivatives covalently modified with one, two or three solubility functions, particularly polyethylene glycol moieties, for use in therapy or prevention of conditions related to pathological calcium crystallization, such as cardiovascular calcifications, nephrocalcinosis, calcinosis cutis, chondrocalcinosis and kidney stones.