CA3156791A1 - Methods and compositions for treating a premature termination codon-mediated disorder - Google Patents
Methods and compositions for treating a premature termination codon-mediated disorder Download PDFInfo
- Publication number
- CA3156791A1 CA3156791A1 CA3156791A CA3156791A CA3156791A1 CA 3156791 A1 CA3156791 A1 CA 3156791A1 CA 3156791 A CA3156791 A CA 3156791A CA 3156791 A CA3156791 A CA 3156791A CA 3156791 A1 CA3156791 A1 CA 3156791A1
- Authority
- CA
- Canada
- Prior art keywords
- trna
- seq
- gene
- expression vector
- nucleotide sequence
- Prior art date
- Legal status (The legal status 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 status listed.)
- Pending
Links
Classifications
-
- 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
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Plant Pathology (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962929428P | 2019-11-01 | 2019-11-01 | |
| US62/929,428 | 2019-11-01 | ||
| PCT/US2020/058415 WO2021087401A1 (en) | 2019-11-01 | 2020-10-30 | Methods and compositions for treating a premature termination codon-mediated disorder |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3156791A1 true CA3156791A1 (en) | 2021-05-06 |
Family
ID=75715359
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3156791A Pending CA3156791A1 (en) | 2019-11-01 | 2020-10-30 | Methods and compositions for treating a premature termination codon-mediated disorder |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20240148772A1 (https=) |
| EP (1) | EP4051291A4 (https=) |
| JP (2) | JP7712270B2 (https=) |
| KR (1) | KR20220155981A (https=) |
| CN (2) | CN115209902B (https=) |
| AU (1) | AU2020375040A1 (https=) |
| CA (1) | CA3156791A1 (https=) |
| WO (1) | WO2021087401A1 (https=) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| KR102930465B1 (ko) | 2017-11-02 | 2026-02-25 | 더 위스타 인스티튜트 오브 아나토미 앤드 바이올로지 | ACE-tRNA를 이용한 유전 재할당을 통한 정지 코돈의 구조 방법 |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| MX2022012955A (es) * | 2020-04-14 | 2023-02-23 | Flagship Pioneering Innovations Vi Llc | Composiciones de trem y usos de las mismas. |
| AU2022269633A1 (en) * | 2021-05-05 | 2023-11-02 | Tevard Biosciences, Inc. | Methods and compositions for treating a premature termination codon-mediated disorder |
| JP2024538097A (ja) * | 2021-10-13 | 2024-10-18 | フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー | Ｔｒｅｍ組成物及び使用方法 |
| AU2023255614A1 (en) | 2022-04-18 | 2024-10-24 | Vertex Pharmaceuticals Incorporated | Compositions and methods for enhancing aav therapy and decreasing tropism of aav to the liver. |
| WO2023220342A2 (en) * | 2022-05-13 | 2023-11-16 | Shape Therapeutics Inc. | Engineered tranfer rnas |
| EP4652272A1 (en) * | 2023-01-18 | 2025-11-26 | The Broad Institute Inc. | Prime editing-mediated readthrough of premature termination codons (pert) |
| WO2024155745A1 (en) * | 2023-01-18 | 2024-07-25 | The Broad Institute, Inc. | Base editing-mediated readthrough of premature termination codons (bert) |
| EP4442826A1 (en) | 2023-04-06 | 2024-10-09 | Universität Hamburg | Synthetic dna construct encoding transfer rna |
| WO2024226381A1 (en) * | 2023-04-28 | 2024-10-31 | University Of Rochester | A method of treating a premature translation termination codon n1n2n3 (ptc)-related disease by nonsense suppression in a subject |
| EP4458967A1 (en) | 2023-05-02 | 2024-11-06 | Universität Hamburg | Pharmaceutical composition comprising multiple suppressor transfer rnas |
| WO2025111506A1 (en) * | 2023-11-22 | 2025-05-30 | Tevard Biosciences, Inc. | Modified transfer rnas and methods of use |
| CN120418432A (zh) * | 2023-12-04 | 2025-08-01 | 睿愈(北京)生物医药科技有限公司 | 改善假尿苷修饰的密码子的通读的方法和组合物 |
| WO2026030209A1 (en) * | 2024-07-29 | 2026-02-05 | University Of Rochester | Use of decoding trnas to boost protein expression or function |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109112126B (zh) * | 2010-06-23 | 2024-06-14 | 库尔纳公司 | 通过抑制电压门控钠通道α亚基(SCNA)的天然反义转录物而治疗SCNA相关疾病 |
| MX365525B (es) * | 2011-09-06 | 2019-06-06 | Curna Inc | Compuestos que regulan la expresión de subunidades alfa de canales de sodio regulados por voltaje en enfermedades relacionadas con epilepsia mioclónica severa de la infancia. |
| WO2016149455A2 (en) * | 2015-03-17 | 2016-09-22 | The General Hospital Corporation | The rna interactome of polycomb repressive complex 1 (prc1) |
| CN110352244B (zh) * | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| KR102930465B1 (ko) * | 2017-11-02 | 2026-02-25 | 더 위스타 인스티튜트 오브 아나토미 앤드 바이올로지 | ACE-tRNA를 이용한 유전 재할당을 통한 정지 코돈의 구조 방법 |
-
2020
- 2020-10-30 CN CN202080090997.8A patent/CN115209902B/zh active Active
- 2020-10-30 CN CN202411915548.9A patent/CN119955780A/zh active Pending
- 2020-10-30 US US17/773,112 patent/US20240148772A1/en active Pending
- 2020-10-30 WO PCT/US2020/058415 patent/WO2021087401A1/en not_active Ceased
- 2020-10-30 CA CA3156791A patent/CA3156791A1/en active Pending
- 2020-10-30 EP EP20882711.3A patent/EP4051291A4/en active Pending
- 2020-10-30 JP JP2022526004A patent/JP7712270B2/ja active Active
- 2020-10-30 KR KR1020227018429A patent/KR20220155981A/ko not_active Ceased
- 2020-10-30 AU AU2020375040A patent/AU2020375040A1/en active Pending
-
2025
- 2025-07-10 JP JP2025116258A patent/JP2025148444A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4051291A1 (en) | 2022-09-07 |
| KR20220155981A (ko) | 2022-11-24 |
| WO2021087401A1 (en) | 2021-05-06 |
| JP2022554357A (ja) | 2022-12-28 |
| CN119955780A (zh) | 2025-05-09 |
| AU2020375040A1 (en) | 2022-05-19 |
| JP7712270B2 (ja) | 2025-07-23 |
| EP4051291A4 (en) | 2024-07-03 |
| US20240148772A1 (en) | 2024-05-09 |
| CN115209902A (zh) | 2022-10-18 |
| JP2025148444A (ja) | 2025-10-07 |
| CN115209902B (zh) | 2025-01-07 |
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