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1.WO/2022/002040LIPID COMPOUNDS AND LIPID NANOPARTICLE COMPOSITIONS
WO 06.01.2022
Int.Class A61P 35/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
Appl.No PCT/CN2021/103076 Applicant SUZHOU ABOGEN BIOSCIENCES CO., LTD. Inventor YING, Bo
Provided herein are lipid compounds that can 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) for therapeutic or prophylactic purposes, including vaccination. Also provided herein are lipid nanoparticle compositions comprising said lipids.
2.20210371494CIRCULAR RNA COMPOSITIONS AND METHODS
US 02.12.2021
Int.Class C07K 14/725
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
725T-cell receptors
Appl.No 17384460 Applicant Massachusetts Institute of Technology Inventor Alexander Wesselhoeft

Circular RNA and transfer vehicles, along with related compositions and methods are described herein. In some embodiments, the inventive circular RNA comprises group I intron fragments, spacers, an IRES, duplex forming regions, and an expression sequence. In some embodiments, the expression sequence encodes a chimeric antigen receptor (CAR). In some embodiments, circular RNA of the invention has improved expression, functional stability, immunogenicity, ease of manufacturing, and/or half-life when compared to linear RNA. In some embodiments, inventive methods and constructs result in improved circularization efficiency, splicing efficiency, and/or purity when compared to existing RNA circularization approaches.

3.20210363547DIFFERENTIAL KNOCKOUT OF AN ALLELE OF A HETEROZYGOUS ELANE GENE
US 25.11.2021
Int.Class C12N 15/90
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
Appl.No 17053629 Applicant David Baram Inventor David Baram

Methods for inactivating in a cell a mutant allele of the elastase, neutrophil expressed gene (ELANE gene) gene having a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN) and which cell is heterozygous at one or more polymorphic sites selected from the group consisting of: rs10414837, rs3761005, rs1683564, rs9749274, rs740021, rs201048029, rs199720952, rs28591229, rs71335276, rs58082177, rs3826946, rs10413889, rs761481944, rs3761008, rs10409474, rs3761007, rs17216649, rs10469327, rs8107095, rs10424470 and rs78302854, the method comprising

    • introducing to the cell a composition comprising:
      • a CRISPR nuclease or a sequence encoding the CRISPR nuclease; and
      • a first RNA molecule comprising a guide sequence portion having 17-20 nucleotides,
    • wherein a complex of the CRISPR nuclease and the first RNA molecule affects a double strand break in the mutant allele of the ELANE gene
      the method optionally further comprising introduction of a second RNA molecule comprising a guide sequence portion capable of complexing with a CRISPR nuclease, wherein the complex of the second RNA molecule and CRISPR nuclease affects a second double strand break in the ELANE gene.

4.113677793稳定的CRISPR复合物
CN 19.11.2021
Int.Class C12N 9/22
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
Appl.No 202080028119.3 Applicant 辛瑟高公司 Inventor 特拉维斯·毛雷斯
本文提供了多核苷酸和CRISPR效应蛋白,所述多核苷酸和CRISPR效应蛋白被配置为以CRISPR复合物的形式共价结合在一起。所述多核苷酸可以被进一步修饰以调节CRISPR复合物的活性。所述多核苷酸和CRISPR效应蛋白的修饰可以用于改进靶结合和/或裂解的效力。
5.20210355463MESSENGER RNA ENCODING CAS9 FOR USE IN GENOME-EDITING SYSTEMS
US 18.11.2021
Int.Class C12N 9/22
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
Appl.No 17319291 Applicant CRISPR THERAPEUTICS AG Inventor Christian Cobaugh

The present disclosure provides optimized mRNAs encoding a site-directed endonuclease for use in a CRISPR/Cas system. Also provided herein are delivery systems for use of the CRISPR/Cas system in methods of in vivo and ex vivo genome editing.

6.20210340542RNA MOLECULES, METHODS OF PRODUCING CIRCULAR RNA, AND TREATMENT METHODS
US 04.11.2021
Int.Class C12N 15/115
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith
Appl.No 16625562 Applicant CORNELL UNIVERSITY Inventor Samie R. JAFFREY

The present invention relates to a RNA molecule comprising a first ribozyme, a first ligation sequence, an effector molecule, a second ligation sequence, and a second ribozyme. Methods of producing circular RNA molecules and treatment methods are also disclosed.

7.1020210127718섬모병증의 치료
KR 22.10.2021
Int.Class A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Appl.No 1020217028846 Applicant 에트리스 게엠베하 Inventor 루돌프, 칼스텐
본 개시내용은 섬모병증을 앓고 있는 대상체에서 섬모병증을 치료하는데 사용하기 위한 폴리리보뉴클레오티드를 포함하는 제약 조성물로서, 여기서 폴리리보뉴클레오티드는 상기 섬모병증과 연관된 결함을 갖는 단백질의 기능적 버전을 코딩하고, 여기서 상기 대상체의 호흡기계에 대한 상기 제약 조성물의 투여는 대상체가 호흡기계의 염증을 나타낼 때 실시되는 것인 제약 조성물에 관한 것이다. 추가로, 본 개시내용은 섬모발생에 대한 폴리리보뉴클레오티드의 효과를 분석하는 방법으로서, 여기서 상기 폴리리보뉴클레오티드는 섬모발생에 수반되고/거나 그에 요구되는 단백질을 코딩하는 것인 방법에 관한 것이다.
8.1020210125536접선 유동 여과에 의한 mRNA 정제
KR 18.10.2021
Int.Class C12N 15/10
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
10Processes for the isolation, preparation or purification of DNA or RNA
Appl.No 1020217028845 Applicant 에트리스 게엠베하 Inventor 가이거, 요하네스
본 개시내용은 mRNA 분자를 정제하는 방법으로서, (Ia) 침전된 mRNA 분자를 포함하는 현탁액으로부터 침전된 mRNA 분자를 정제하는 단계, (Ib) 정제된 침전된 mRNA 분자를 세척하고 용해시키는 단계, (IIa) 킬레이트제를 포함하는 용액을 사용하여 mRNA 분자를 정제하는 단계, 이어서 (IIb) 정제된 mRNA 분자를 세척하는 단계를 포함하며, 여기서 단계 (Ia) 내지 (IIb)는 접선 유동 여과를 사용하여 수행되는 것인 방법을 제공한다.
9.WO/2021/207085SYNTHETIC ELABORATION OF NATIVE DNA BY RASS (SENDR)
WO 14.10.2021
Int.Class C07H 21/00
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
Appl.No PCT/US2021/025799 Applicant THE SCRIPPS RESEARCH INSTITUTE Inventor DAWSON, Philip
Provided herein are methods for site selective chemical modification of an oligonucleotide comprising immobilizing the oligonucleotide on an inert resin to form oligonucleotide-resin complex; and contacting the oligonucleotide-resin complex with a phosphorus (V) based phosphorus (V) reagent in an organic solvent to produce a modified oligonucleotide. Further provide herein are methods of targeting delivery of an oligonucleotide therapeutic to a specific location in the patient body, and/or improving stability and half life of an oligonucleotide therapeutic by using modified oligonucleotides.
10.WO/2021/204175LIPID NANOPARTICLE COMPOSITION
WO 14.10.2021
Int.Class C07C 229/16
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
CACYCLIC OR CARBOCYCLIC COMPOUNDS
229Compounds containing amino and carboxyl groups bound to the same carbon skeleton
02having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
04the carbon skeleton being acyclic and saturated
06having only one amino and one carboxyl group bound to the carbon skeleton
10the 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
16to carbon atoms of hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
Appl.No PCT/CN2021/085879 Applicant SUZHOU ABOGEN BIOSCIENCES CO., LTD. Inventor YING, Bo
Provided herein are lipids that can 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) for therapeutic or prophylactic purposes, including vaccination.