CL2025003844A1 - Molécula de arn diseñada genéticamente con expresión ajustable - Google Patents

Molécula de arn diseñada genéticamente con expresión ajustable

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Publication number
CL2025003844A1
CL2025003844A1 CL2025003844A CL2025003844A CL2025003844A1 CL 2025003844 A1 CL2025003844 A1 CL 2025003844A1 CL 2025003844 A CL2025003844 A CL 2025003844A CL 2025003844 A CL2025003844 A CL 2025003844A CL 2025003844 A1 CL2025003844 A1 CL 2025003844A1
Authority
CL
Chile
Application number
CL2025003844A
Inventor
Zhang Weiguo
Dong Yijie
Chen Rui
Qi Rui
Original Assignee
Rinuagene Biotechnology Co Ltd
Rinuagene International Hk Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Rinuagene Biotechnology Co Ltd, Rinuagene International Hk Ltd filed Critical Rinuagene Biotechnology Co Ltd
Publication of CL2025003844A1 publication Critical patent/CL2025003844A1/es

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
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    • A61K9/51—Nanocapsules; Nanoparticles
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    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12R2001/00—Microorganisms ; Processes using microorganisms
    • C12R2001/01—Bacteria or Actinomycetales ; using bacteria or Actinomycetales
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

Esta solicitud se relaciona con una molécula de ARN diseñada genéticamente, una molécula de ADN que codifica la molécula de ARN diseñada genéticamente, y el uso de la molécula de ARN diseñada genéticamente. La molécula de ARN diseñada genéticamente comprende una secuencia de cola de poli(A) que contiene un sitio de unión de miARN. La cola de poli(A) permite la expresión precisa de un gen diana en un órgano, un tejido y/o una célula.
CL2025003844A 2023-06-09 2025-12-05 Molécula de arn diseñada genéticamente con expresión ajustable CL2025003844A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2023099512 2023-06-09
CN2023121779 2023-09-26
CN202410711025 2024-06-03

Publications (1)

Publication Number Publication Date
CL2025003844A1 true CL2025003844A1 (es) 2026-03-13

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ID=93712947

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CL2025003844A CL2025003844A1 (es) 2023-06-09 2025-12-05 Molécula de arn diseñada genéticamente con expresión ajustable

Country Status (9)

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US (1) US20260092293A1 (es)
EP (1) EP4726038A1 (es)
KR (1) KR20260022409A (es)
CN (1) CN119101681A (es)
AU (1) AU2024285035A1 (es)
CL (1) CL2025003844A1 (es)
MX (1) MX2025014784A (es)
TW (1) TW202513796A (es)
WO (1) WO2024251232A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025167868A1 (zh) * 2024-02-05 2025-08-14 深圳深信生物科技有限公司 修饰的核酸及其应用

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016005004A1 (en) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
EP3458474B1 (en) * 2016-05-18 2022-07-06 ModernaTX, Inc. Combinations of mrnas encoding immune modulating polypeptides and uses thereof
US11680275B2 (en) * 2017-06-06 2023-06-20 University Of Massachusetts Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome
WO2020056239A1 (en) * 2018-09-14 2020-03-19 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
EP4306643A3 (en) 2018-10-11 2024-03-20 ethris GmbH Plasmid containing a sequence encoding an mrna with a segmented poly(a) tail
US20230279408A1 (en) 2020-08-07 2023-09-07 The Hong Kong University Of Science And Technology Compositions and methods for increasing protein expression
US20240041785A1 (en) * 2020-11-16 2024-02-08 BioNTech SE Compositions and methods for stabilization of lipid nanoparticle mrna vaccines
IL303315A (en) * 2020-12-02 2023-07-01 Seqirus Inc Multicistronic RNA components and their uses
CA3171589A1 (en) * 2021-05-03 2022-11-03 Moritz THRAN Improved nucleic acid sequence for cell type specific expression
EP4355882A2 (en) * 2021-06-15 2024-04-24 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
WO2023031392A2 (en) * 2021-09-03 2023-03-09 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids comprising phosphatidylserine
CN115820740A (zh) * 2021-09-16 2023-03-21 北京锦篮基因科技有限公司 用于治疗ⅱ型粘多糖贮积症的重组腺相关病毒载体及其应用

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WO2024251232A1 (zh) 2024-12-12
KR20260022409A (ko) 2026-02-19
US20260092293A1 (en) 2026-04-02
TW202513796A (zh) 2025-04-01
MX2025014784A (es) 2026-01-07
EP4726038A1 (en) 2026-04-15
AU2024285035A1 (en) 2026-01-15
CN119101681A (zh) 2024-12-10

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