TW202128740A - 變體ｉｇｆ２構築體 - Google Patents
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- C—CHEMISTRY; METALLURGY
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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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
- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
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- 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
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- C—CHEMISTRY; METALLURGY
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
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- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/2405—Glucanases
- C12N9/2451—Glucanases acting on alpha-1,6-glucosidic bonds
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/485—Exopeptidases (3.4.11-3.4.19)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
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| BR112020021962A2 (pt) * | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| CN115379850A (zh) | 2019-12-24 | 2022-11-22 | 裘美娜治疗公司 | 再生性多肽及其用途 |
| IL303239A (en) | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | Compositions and their uses for the treatment of Engelmann syndrome |
| WO2022271466A1 (en) | 2021-06-21 | 2022-12-29 | Juvena Therapeutics, Inc. | Regenerative polypeptides and uses thereof |
| WO2022271981A2 (en) | 2021-06-23 | 2022-12-29 | Lycia Therapeutics, Inc. | Bifunctional compounds containing igf-2 polypeptides |
| JP2025524569A (ja) * | 2022-07-06 | 2025-07-30 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | Ｃｌｎ１ポリヌクレオチドのアデノ随伴ウイルス送達 |
| WO2024151982A1 (en) * | 2023-01-13 | 2024-07-18 | Amicus Therapeutics, Inc. | Gene therapy constructs for the treatment of pompe disease |
| KR20250161626A (ko) * | 2023-03-20 | 2025-11-17 | 스파크 테라퓨틱스, 인코포레이티드 | Ppt1 유전자 치료 |
| US20240360431A1 (en) * | 2023-04-30 | 2024-10-31 | Sichuan Real&Best Biotech Co., Ltd. | Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease |
| PL445164A1 (pl) * | 2023-06-07 | 2024-12-09 | Recepton Spółka Z Ograniczoną Odpowiedzialnością | Cząsteczki indukujące internalizację receptorów i białek zewnątrzkomórkowych |
| WO2025108375A1 (zh) * | 2023-11-21 | 2025-05-30 | 深圳湾实验室 | 用于蛋白降解的构建体及其应用 |
| WO2025259950A1 (en) * | 2024-06-14 | 2025-12-18 | Cz Biohub Sf, Llc | Genetically encoded lysosome targeting chimeras for cell-mediated delivery |
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| WO2002087510A2 (en) * | 2001-04-30 | 2002-11-07 | Symbiontics, Inc. | Subcellular targeting of therapeutic proteins |
| US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| CA2553955C (en) * | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| JP4988599B2 (ja) * | 2005-01-07 | 2012-08-01 | 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 | Ｉｇｆ−１融合ポリペプチドおよびその治療的使用 |
| HRP20180291T1 (hr) * | 2011-05-27 | 2018-03-23 | Amicus Therapeutics, Inc. | Postupci za sparivanje ciljajućeg peptida na rekombinantne lizosomalne enzime za poboljšano liječenje lizosomskih bolesti nakupljanja |
| WO2014082080A2 (en) * | 2012-11-26 | 2014-05-30 | Callidus Biopharma, Inc. | Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases |
| ES2729997T3 (es) * | 2012-11-27 | 2019-11-07 | Biomarin Pharm Inc | Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas |
| AU2014228231B2 (en) * | 2013-03-15 | 2019-02-21 | Amicus Therapeutics, Inc. | Chemical crosslinkers |
| MA40460A (fr) * | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisation correspondante |
| EP3374504B1 (en) * | 2015-11-09 | 2025-03-19 | CureVac SE | Optimized nucleic acid molecules |
| AU2017315328A1 (en) * | 2016-08-24 | 2019-03-21 | Immunexpress Pty Ltd | Systemic inflammatory and pathogen biomarkers and uses therefor |
| BR112020021962A2 (pt) * | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| JP7585233B2 (ja) * | 2019-04-30 | 2024-11-18 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ポンペ病の治療のために有用な組成物 |
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| MX2022004345A (es) | 2022-07-27 |
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