AU2020375040A1 - 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
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- AU2020375040A1 AU2020375040A1 AU2020375040A AU2020375040A AU2020375040A1 AU 2020375040 A1 AU2020375040 A1 AU 2020375040A1 AU 2020375040 A AU2020375040 A AU 2020375040A AU 2020375040 A AU2020375040 A AU 2020375040A AU 2020375040 A1 AU2020375040 A1 AU 2020375040A1
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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| 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 |
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| AU2020375040A1 true AU2020375040A1 (en) | 2022-05-19 |
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| AU2020375040A Pending AU2020375040A1 (en) | 2019-11-01 | 2020-10-30 | Methods and compositions for treating a premature termination codon-mediated disorder |
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| US (1) | US20240148772A1 (https=) |
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| WO (1) | WO2021087401A1 (https=) |
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| 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 |
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