CA3114425A1 - Compositions and methods for lactate dehydrogenase (ldha) gene editing - Google Patents
Compositions and methods for lactate dehydrogenase (ldha) gene editing Download PDFInfo
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- C12Y101/01027—L-Lactate dehydrogenase (1.1.1.27)
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| PCT/US2019/053423 WO2020069296A1 (en) | 2018-09-28 | 2019-09-27 | Compositions and methods for lactate dehydrogenase (ldha) gene editing |
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| WO2020198697A1 (en) * | 2019-03-28 | 2020-10-01 | Intellia Therapeutics, Inc. | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
| WO2022081750A1 (en) | 2020-10-14 | 2022-04-21 | George Mason Research Foundation, Inc. | Ionizable lipids and methods of manufacture and use thereof |
| US20230034581A1 (en) | 2021-06-04 | 2023-02-02 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof |
| US20240415980A1 (en) | 2021-10-28 | 2024-12-19 | Regeneron Pharmaceuticals, Inc. | Crispr/cas-related methods and compositions for knocking out c5 |
| WO2023122433A1 (en) * | 2021-12-22 | 2023-06-29 | Arbor Biotechnologies, Inc. | Gene editing systems targeting hydroxyacid oxidase 1 (hao1) and lactate dehydrogenase a (ldha) |
| US20250032642A1 (en) | 2022-02-02 | 2025-01-30 | Regeneron Pharmaceuticals, Inc. | Crispr-mediated transgene insertion in neonatal cells |
| CN114657181B (zh) * | 2022-04-01 | 2023-08-25 | 安徽大学 | 一种靶向H1.4的sgRNA以及H1.4基因编辑方法 |
| JP2025514304A (ja) | 2022-04-29 | 2025-05-02 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 遺伝子治療法のための組織特異的遺伝子外セーフハーバーの同定 |
| WO2023235725A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
| CN119421951A (zh) | 2022-05-31 | 2025-02-11 | 瑞泽恩制药公司 | 用于c9orf72重复序列扩增疾病的crispr干扰疗法 |
| WO2024026474A1 (en) | 2022-07-29 | 2024-02-01 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle |
| CA3265590A1 (en) | 2022-09-28 | 2024-04-04 | Regeneron Pharmaceuticals, Inc. | MODIFIED ANTIBODY-RESISTANT RECEPTORS TO IMPROVE CELL-BASED THERAPIES |
| US20240182561A1 (en) | 2022-11-04 | 2024-06-06 | Regeneron Pharmaceuticals, Inc. | Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle |
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| WO2025029654A2 (en) | 2023-07-28 | 2025-02-06 | Regeneron Pharmaceuticals, Inc. | Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion |
| TW202519551A (zh) | 2023-07-28 | 2025-05-16 | 美商雷傑納榮製藥公司 | 用於治療酸性神經髓磷脂酶缺乏症之抗ＴｆＲ：酸性神經髓磷脂酶 |
| TW202513801A (zh) | 2023-07-28 | 2025-04-01 | 美商雷傑納榮製藥公司 | 用於治療龐貝氏症之抗ＴｆＲ：ＧＡＡ及抗ＣＤ６３：ＧＡＡ插入 |
| WO2025049524A1 (en) | 2023-08-28 | 2025-03-06 | Regeneron Pharmaceuticals, Inc. | Cxcr4 antibody-resistant modified receptors |
| WO2025045247A1 (en) * | 2023-08-31 | 2025-03-06 | Geneditbio Limited | Nucleic acids encoding crispr-associated proteins and uses thereof |
| WO2025049959A2 (en) | 2023-09-01 | 2025-03-06 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025162435A1 (en) * | 2024-02-02 | 2025-08-07 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of liver disease |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
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| WO2025235388A1 (en) | 2024-05-06 | 2025-11-13 | Regeneron Pharmaceuticals, Inc. | Transgene genomic identification by nuclease-mediated long read sequencing |
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| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
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| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
| US20150376587A1 (en) * | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| US10342761B2 (en) | 2014-07-16 | 2019-07-09 | Novartis Ag | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
| DK3204497T3 (da) * | 2014-10-10 | 2020-05-25 | Dicerna Pharmaceuticals Inc | Terapeutisk hæmning af lactatdehydrogenase og midler dertil |
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| WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
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| US11242510B2 (en) * | 2016-05-02 | 2022-02-08 | The Regents Of The University Of California | Mammalian cells devoid of lactate dehydrogenase activity |
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- 2019-09-27 AU AU2019347517A patent/AU2019347517A1/en not_active Withdrawn
- 2019-09-27 CN CN201980076179.XA patent/CN113056559A/zh active Pending
- 2019-09-27 CA CA3114425A patent/CA3114425A1/en active Pending
- 2019-09-27 KR KR1020217012025A patent/KR20210086621A/ko not_active Withdrawn
- 2019-09-27 MX MX2021003457A patent/MX2021003457A/es unknown
- 2019-09-27 SG SG11202102660RA patent/SG11202102660RA/en unknown
-
2021
- 2021-03-16 IL IL281529A patent/IL281529A/en unknown
- 2021-03-25 US US17/212,901 patent/US20210222173A1/en not_active Abandoned
- 2021-03-26 PH PH12021550686A patent/PH12021550686A1/en unknown
- 2021-04-23 CO CONC2021/0005231A patent/CO2021005231A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| MX2021003457A (es) | 2021-07-16 |
| AU2019347517A1 (en) | 2021-05-06 |
| KR20210086621A (ko) | 2021-07-08 |
| BR112021005718A2 (pt) | 2021-06-22 |
| EP3856901A1 (en) | 2021-08-04 |
| PH12021550686A1 (en) | 2022-02-28 |
| JP2022502055A (ja) | 2022-01-11 |
| SG11202102660RA (en) | 2021-04-29 |
| CO2021005231A2 (es) | 2021-06-10 |
| TW202028460A (zh) | 2020-08-01 |
| IL281529A (en) | 2021-05-31 |
| US20210222173A1 (en) | 2021-07-22 |
| CN113056559A (zh) | 2021-06-29 |
| WO2020069296A1 (en) | 2020-04-02 |
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