Abstract

The present invention discloses lipid compounds for delivery of therapeutic agents and preparation method and its use thereof, specifically, the present invention discloses a compound of Formula I or a pharmaceutically acceptable salt, stereoisomer, tautomer, solvate, chelate, non-covalent complex, or precursor thereof, the ionizable lipid compounds of the present invention can effectively deliver nucleic acid molecules, small molecule compounds and other drugs, and by comparison, the lipid nanoparticles of the present invention have better particle size distribution, high encapsulation efficiency, and the delivery effect can be significantly better than the comparison lipid nanoparticles, which can meet the demand for in vivo delivery.

IPC Classes  ?

• C07C 219/06 - Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
• C07C 9/22 - Acyclic saturated hydrocarbons with more than fifteen carbon atoms
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
• A61P 3/00 - Drugs for disorders of the metabolism

Abstract

Provided are lipid compounds for delivery of therapeutic agents and preparation method and its use thereof, specifically, provided a compound of Formula I or a pharmaceutically acceptable salt, stereoisomer, tautomer, solvate, chelate, non-covalent complex, or precursor thereof, the ionizable lipid compounds can effectively deliver nucleic acid molecules, small molecule compounds and other drugs, and by comparison, the lipid nanoparticles have better particle size distribution, high encapsulation efficiency, and the delivery effect can be significantly better than the comparison lipid nanoparticles, which can meet the demand for in vivo delivery (I).

IPC Classes  ?

• C07C 9/22 - Acyclic saturated hydrocarbons with more than fifteen carbon atoms
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
• A61P 3/00 - Drugs for disorders of the metabolism

Abstract

Provided are a gene editing protein, a corresponding gene editing system thereof, and a use thereof. Specifically, the gene editing protein shows gene editing activity in vitro, can effectively edit or cut a target gene, and can effectively treat disorders or diseases of a subject in need.

IPC Classes  ?

• C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 9/22 - Ribonucleases
• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
• C12N 15/62 - DNA sequences coding for fusion proteins
• C07K 14/195 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from bacteria
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy

Abstract

Provided are a Cas protein, and a composition comprising same and a CRISPR-Cas system. Furthermore, provided are a method for using the composition and the CRISPR-Cas system and the use thereof. Also provided is a cell comprising the Cas protein, the composition and the CRISPR-Cas system.

IPC Classes  ?

• C12N 9/22 - Ribonucleases
• C12N 15/55 - Hydrolases (3)
• C12N 15/62 - DNA sequences coding for fusion proteins
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
• C07K 19/00 - Hybrid peptides
• C12Q 1/6816 - Hybridisation assays characterised by the detection means
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy

Abstract

Provided are a Cas protein and a variant thereof, and a corresponding gene editing system and a use thereof. Specifically, the Cas protein has good gene editing activity, can effectively edit or cut a target gene, and can effectively treat disorders or diseases of subjects in need. Also provided are an editing system and a use of a fusion protein comprising a Cas protein or a variant thereof.

IPC Classes  ?

• C12N 9/22 - Ribonucleases
• C12N 9/78 - Hydrolases (3.) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
• C07K 19/00 - Hybrid peptides
• C12N 15/55 - Hydrolases (3)
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 15/70 - Vectors or expression systems specially adapted for E. coli
• C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
• C12N 5/10 - Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
• C12Q 1/6816 - Hybridisation assays characterised by the detection means
• A61K 38/46 - Hydrolases (3)
• A61K 38/50 - Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
• A61P 35/00 - Antineoplastic agents
• A61P 31/00 - Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
• A61P 25/00 - Drugs for disorders of the nervous system
• A61P 37/02 - Immunomodulators
• A61P 27/16 - Otologicals

Abstract

A Cas13 protein, and a CRISPR-Cas system and the use thereof. The amino acid sequences of the Cas13 proteins are as shown in SEQ ID NO.1-SEQ ID NO.9, which are named as Cas13a_10, Cas13a_11, Cas13b_3, Cas13b_4, Cas13b_5, Cas13T_9, Cas13T_15, Cas13T_16, and Cas13Z_2, respectively. The nine Cas13 proteins have low homology to reported Cas13 proteins, show RNA nuclease activity, and have great application prospects in the field of gene editing.

IPC Classes  ?

• C12N 9/22 - Ribonucleases
• C07K 19/00 - Hybrid peptides
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
• C12N 5/10 - Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
• C12Q 1/68 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving nucleic acids

Abstract

A lipid compound for delivering a therapeutic agent, a preparation method therefor, and a use thereof. A lipid compound is a compound having a structural formula I or a pharmaceutically acceptable form thereof. The lipid compound may be used in combination with other lipid components such as a neutral lipid, cholesterol, and a polymer-bound lipid to form lipid nanoparticles for delivery of therapeutic agents (e.g., nucleic acid molecules) to achieve therapeutic or prophylactic purposes (e.g., vaccination), thereby enriching the types of ionizable lipid compounds.

IPC Classes  ?

• C07C 333/04 - Monothiocarbamic acidsDerivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
• C07C 229/16 - Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
• C07C 329/06 - Esters of monothiocarbonic acids having sulfur atoms of thiocarbonic groups bound to acyclic carbon atoms
• A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• A61P 3/00 - Drugs for disorders of the metabolism
• A61P 9/00 - Drugs for disorders of the cardiovascular system
• A61P 35/00 - Antineoplastic agents
• A61P 31/00 - Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics

Abstract

A lipid compound for delivering a therapeutic agent, and a preparation method therefor and the use thereof. A lipid compound, which is a compound having structural formula I or a pharmaceutically acceptable form thereof. The lipid compound may be used in combination with other lipid components such as a neutral lipid, cholesterol and a polymer-bound lipid so as to form a lipid nanoparticle used for delivering a therapeutic agent (e.g., nucleic acid molecules) to achieve therapeutic or prophylactic purposes (e.g., vaccination), thereby increasing the types of ionizable lipid compounds.

IPC Classes  ?

• C07C 333/04 - Monothiocarbamic acidsDerivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
• C07C 237/12 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by carboxyl groups
• C07C 231/02 - Preparation of carboxylic acid amides from carboxylic acids or from esters, anhydrides, or halides thereof by reaction with ammonia or amines
• A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
• A61K 9/51 - Nanocapsules
• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61P 31/12 - Antivirals
• A61P 31/04 - Antibacterial agents
• A61P 35/00 - Antineoplastic agents
• A61P 3/00 - Drugs for disorders of the metabolism

Abstract

Provided are an enzyme capable of mediating integration of a large DNA fragment into a mammalian genome and the use thereof. Specifically, provided is a novel enzyme capable of integrating a large DNA fragment into a genome, comprising an integrase as shown in SEQ ID NO: 1-7. Further provided are a polynucleotide comprising a nucleotide sequence encoding the integrase and a host cell comprising the polynucleotide. Also provided is a method of recombining a target nucleic acid to a human genome by means of using the integrase and in the integrase.

IPC Classes  ?

• C12N 9/12 - Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
• C12N 9/22 - Ribonucleases
• C12N 15/90 - Stable introduction of foreign DNA into chromosome
• C12N 15/54 - Transferases (2)
• C12N 15/33 - Genes encoding viral proteins
• C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides

Goods & Services

Biological preparations for medical purposes; biological preparations for veterinary purposes; enzyme preparations for medical purposes; stem cells for medical purposes; stem cells for veterinary purposes; biological tissue cultures for medical purposes; biological tissue cultures for veterinary purposes; surgical implants comprising living tissue; biochemical drugs; blood products.

Abstract

A lipid compound for delivery of a therapeutic agent, a preparation method therefor and a use thereof. The lipid compound is a compound having structural formula (I) or a pharmaceutically acceptable form thereof. The lipid compound may be used in combination with other lipid components, such as neutral lipids, cholesterol, and polymer conjugated lipids, to form lipid nanoparticles for delivery of therapeutic agents (e.g., nucleic acid molecules) to achieve therapeutic or prophylactic purposes (e.g., vaccination), enriching the types of ionizable lipid compounds.

IPC Classes  ?

• C07C 211/21 - Monoamines
• C07D 295/13 - Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
• A61P 35/00 - Antineoplastic agents

Abstract

A Cas13 protein, a CRISPR-Cas system, and a use thereof, relating to the field of biotechnology. Provided is a Cas13 protein, comprising the Cas13 proteins having the amino acid sequences represented by SEQ ID NO.1 to SEQ ID NO.10, which are named as Cas13a_2, Cas13b_1, Cas13b_2, Cas13d_2, Cas13T_1, Cas13T_4, Cas13T_5, Cas13T_6, Cas13T_7, and Cas13T_8, respectively. The 10 Cas13 proteins have low homology to reported Cas13 proteins, and show RNA nuclease activity. Therefore, the 10 Cas13 proteins all have great application prospects in gene editing.

IPC Classes  ?

• C12N 9/22 - Ribonucleases
• C12N 15/70 - Vectors or expression systems specially adapted for E. coli
• C12R 1/19 - Escherichia coli

Goods & Services

Biological preparations for medical purposes; biological preparations for veterinary purposes; enzyme preparations for medical purposes; stem cells for medical purposes; stem cells for veterinary purposes; biological tissue cultures for medical purposes; biological tissue cultures for veterinary purposes; surgical implants comprising living tissue; biochemical drugs for the treatment and prevention of cancer, immune diseases, genetic disorders and infectious diseases; blood products, namely, blood, blood plasma, and components thereof, for medical use

Abstract

The present invention belongs to the technical field of biology, and provides a deaminase and an adenine base editor. Also provided are a deaminase mutant and a corresponding adenine base editor. The deaminase mutant comprises multiple amino acid mutations compared with the parental deaminase, thereby possessing improved base editing efficiency and good application prospects.

IPC Classes  ?

• C12N 9/78 - Hydrolases (3.) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
• C07K 19/00 - Hybrid peptides
• C12N 15/62 - DNA sequences coding for fusion proteins
• C12N 15/55 - Hydrolases (3)
• A61K 38/50 - Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
• A61P 3/06 - Antihyperlipidemics