KR20240164824A - 조작된 소형 핵 rna(snrna)를 포함하는 조성물 및 방법 - Google Patents

조작된 소형 핵 rna(snrna)를 포함하는 조성물 및 방법 Download PDF

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KR20240164824A
KR20240164824A KR1020247032973A KR20247032973A KR20240164824A KR 20240164824 A KR20240164824 A KR 20240164824A KR 1020247032973 A KR1020247032973 A KR 1020247032973A KR 20247032973 A KR20247032973 A KR 20247032973A KR 20240164824 A KR20240164824 A KR 20240164824A
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sequence
rna
seq
nucleic acid
esnrna
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마크밀러 리아 라델리
다니엘라 로스
다니엘 에이 노울랜드
란잔 바트라
레이철 에이 아담스
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로카나바이오 인크.
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • A61P25/00—Drugs for disorders of the nervous system
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020247032973A 2022-03-04 2023-03-06 조작된 소형 핵 rna(snrna)를 포함하는 조성물 및 방법 Pending KR20240164824A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263316659P 2022-03-04 2022-03-04
US63/316,659 2022-03-04
US202263379983P 2022-10-18 2022-10-18
US63/379,983 2022-10-18
PCT/US2023/063798 WO2023168458A1 (en) 2022-03-04 2023-03-06 Compositions and methods comprising engineered small nuclear rna (snrna)

Publications (1)

Publication Number Publication Date
KR20240164824A true KR20240164824A (ko) 2024-11-20

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KR1020247032973A Pending KR20240164824A (ko) 2022-03-04 2023-03-06 조작된 소형 핵 rna(snrna)를 포함하는 조성물 및 방법

Country Status (10)

Country Link
US (3) US20230279397A1 (https=)
EP (1) EP4486888A1 (https=)
JP (1) JP2025507958A (https=)
KR (1) KR20240164824A (https=)
CN (1) CN119137273A (https=)
AU (1) AU2023228712A1 (https=)
CA (1) CA3245423A1 (https=)
IL (1) IL315378A (https=)
MX (1) MX2024010833A (https=)
WO (1) WO2023168458A1 (https=)

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AU2023228712A1 (en) 2022-03-04 2024-10-17 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising engineered small nuclear rna (snrna)
AU2023364059A1 (en) 2022-10-18 2025-05-08 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising programmable snrnas for rna editing
KR20250115422A (ko) 2022-12-01 2025-07-30 리제너론 파마슈티칼스 인코포레이티드 반복적 요소의 적절한 패키징을 위한 아데노 연관 바이러스 벡터
WO2025072530A2 (en) 2023-09-26 2025-04-03 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating dmd
WO2025090633A1 (en) 2023-10-23 2025-05-01 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies
WO2025126158A2 (en) 2023-12-15 2025-06-19 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) targeting sod1
WO2025160364A2 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for the treatment of pompe disease

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US6692910B2 (en) 1997-07-28 2004-02-17 The University Of Connecticut Inhibition of a target messenger RNA with a modified U1 small nuclear RNA
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
WO2002083908A1 (es) 2001-03-30 2002-10-24 Fundacion Para La Investigacion Medica Aplicada Procedimiento de inhibicion reversible de la expresion genica mediante ribonucleoproteinas modificadas
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
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Publication number Publication date
EP4486888A1 (en) 2025-01-08
IL315378A (en) 2024-11-01
US12037588B2 (en) 2024-07-16
US20230279397A1 (en) 2023-09-07
US20250136981A1 (en) 2025-05-01
WO2023168458A1 (en) 2023-09-07
JP2025507958A (ja) 2025-03-21
US20240018521A1 (en) 2024-01-18
CN119137273A (zh) 2024-12-13
CA3245423A1 (en) 2023-09-07
AU2023228712A1 (en) 2024-10-17
MX2024010833A (es) 2024-12-06

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