BR112022006842A2 - Construtos de igf2 variante - Google Patents
Construtos de igf2 varianteInfo
- Publication number
- BR112022006842A2 BR112022006842A2 BR112022006842A BR112022006842A BR112022006842A2 BR 112022006842 A2 BR112022006842 A2 BR 112022006842A2 BR 112022006842 A BR112022006842 A BR 112022006842A BR 112022006842 A BR112022006842 A BR 112022006842A BR 112022006842 A2 BR112022006842 A2 BR 112022006842A2
- Authority
- BR
- Brazil
- Prior art keywords
- constructs
- igf2
- treatment
- fusion proteins
- lysosomal storage
- Prior art date
Links
- 101001076292 Homo sapiens Insulin-like growth factor II Proteins 0.000 title abstract 5
- 102100025947 Insulin-like growth factor II Human genes 0.000 title abstract 5
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 2
- 102000037865 fusion proteins Human genes 0.000 abstract 2
- 108020001507 fusion proteins Proteins 0.000 abstract 2
- 230000001413 cellular effect Effects 0.000 abstract 1
- 238000002641 enzyme replacement therapy Methods 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 150000007523 nucleic acids Chemical group 0.000 abstract 1
- 230000028327 secretion Effects 0.000 abstract 1
Classifications
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2405—Glucanases
- C12N9/2451—Glucanases acting on alpha-1,6-glucosidic bonds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- C12N15/62—DNA sequences coding for fusion proteins
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/22—Hormones
- A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
-
- 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
-
- 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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/485—Exopeptidases (3.4.11-3.4.19)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962913677P | 2019-10-10 | 2019-10-10 | |
| US201962929054P | 2019-10-31 | 2019-10-31 | |
| PCT/US2020/055251 WO2021072372A1 (en) | 2019-10-10 | 2020-10-12 | Variant igf2 constructs |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022006842A2 true BR112022006842A2 (pt) | 2022-07-05 |
Family
ID=75438187
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022006842A BR112022006842A2 (pt) | 2019-10-10 | 2020-10-12 | Construtos de igf2 variante |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20240091321A1 (https=) |
| EP (1) | EP4041284A4 (https=) |
| JP (2) | JP2022552254A (https=) |
| KR (1) | KR20220077921A (https=) |
| CN (1) | CN115087458A (https=) |
| AU (2) | AU2020361703A1 (https=) |
| BR (1) | BR112022006842A2 (https=) |
| CA (1) | CA3154189A1 (https=) |
| IL (1) | IL292065A (https=) |
| MX (1) | MX2022004345A (https=) |
| TW (1) | TW202128740A (https=) |
| WO (1) | WO2021072372A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
-
2020
- 2020-10-12 KR KR1020227015116A patent/KR20220077921A/ko active Pending
- 2020-10-12 CA CA3154189A patent/CA3154189A1/en active Pending
- 2020-10-12 TW TW109135159A patent/TW202128740A/zh unknown
- 2020-10-12 US US17/767,803 patent/US20240091321A1/en active Pending
- 2020-10-12 EP EP20875073.7A patent/EP4041284A4/en active Pending
- 2020-10-12 AU AU2020361703A patent/AU2020361703A1/en not_active Abandoned
- 2020-10-12 MX MX2022004345A patent/MX2022004345A/es unknown
- 2020-10-12 CN CN202080081564.6A patent/CN115087458A/zh active Pending
- 2020-10-12 BR BR112022006842A patent/BR112022006842A2/pt not_active Application Discontinuation
- 2020-10-12 WO PCT/US2020/055251 patent/WO2021072372A1/en not_active Ceased
- 2020-10-12 JP JP2022521217A patent/JP2022552254A/ja active Pending
-
2022
- 2022-04-07 IL IL292065A patent/IL292065A/en unknown
-
2025
- 2025-02-01 AU AU2025200698A patent/AU2025200698A1/en active Pending
- 2025-10-03 JP JP2025167212A patent/JP2026021331A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2026021331A (ja) | 2026-02-10 |
| MX2022004345A (es) | 2022-07-27 |
| JP2022552254A (ja) | 2022-12-15 |
| IL292065A (en) | 2022-06-01 |
| AU2020361703A1 (en) | 2022-04-28 |
| AU2025200698A1 (en) | 2025-02-27 |
| EP4041284A1 (en) | 2022-08-17 |
| KR20220077921A (ko) | 2022-06-09 |
| US20240091321A1 (en) | 2024-03-21 |
| EP4041284A4 (en) | 2023-10-18 |
| TW202128740A (zh) | 2021-08-01 |
| CN115087458A (zh) | 2022-09-20 |
| CA3154189A1 (en) | 2021-04-15 |
| WO2021072372A1 (en) | 2021-04-15 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B11A | Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing | ||
| B11N | Dismissal: publication cancelled [chapter 11.14 patent gazette] |
Free format text: ANULADA A PUBLICACAO CODIGO 11.1 NA RPI NO 2755 DE 24/10/2023 POR TER SIDO INDEVIDA. |