PH12022551229A1 - Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders - Google Patents
Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disordersInfo
- Publication number
- PH12022551229A1 PH12022551229A1 PH1/2022/551229A PH12022551229A PH12022551229A1 PH 12022551229 A1 PH12022551229 A1 PH 12022551229A1 PH 12022551229 A PH12022551229 A PH 12022551229A PH 12022551229 A1 PH12022551229 A1 PH 12022551229A1
- Authority
- PH
- Philippines
- Prior art keywords
- liver
- gaa
- polypeptide
- disease
- igf2
- Prior art date
Links
- 206010053185 Glycogen storage disease type II Diseases 0.000 title abstract 4
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 title abstract 4
- 210000004185 liver Anatomy 0.000 title abstract 4
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 title abstract 3
- 201000004502 glycogen storage disease II Diseases 0.000 title abstract 3
- 208000015439 Lysosomal storage disease Diseases 0.000 title abstract 2
- 241000702421 Dependoparvovirus Species 0.000 title 1
- 230000001225 therapeutic effect Effects 0.000 title 1
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 5
- 229920001184 polypeptide Polymers 0.000 abstract 4
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 4
- 230000008685 targeting Effects 0.000 abstract 3
- 101001076292 Homo sapiens Insulin-like growth factor II Proteins 0.000 abstract 2
- 102100025947 Insulin-like growth factor II Human genes 0.000 abstract 2
- 108010076504 Protein Sorting Signals Proteins 0.000 abstract 2
- 108010028144 alpha-Glucosidases Proteins 0.000 abstract 2
- 210000003712 lysosome Anatomy 0.000 abstract 2
- 230000001868 lysosomic effect Effects 0.000 abstract 2
- 239000013598 vector Substances 0.000 abstract 2
- 101710145225 Cation-independent mannose-6-phosphate receptor Proteins 0.000 abstract 1
- 102100037182 Cation-independent mannose-6-phosphate receptor Human genes 0.000 abstract 1
- 101001028831 Homo sapiens Cation-independent mannose-6-phosphate receptor Proteins 0.000 abstract 1
- 108010031792 IGF Type 2 Receptor Proteins 0.000 abstract 1
- 102000038460 IGF Type 2 Receptor Human genes 0.000 abstract 1
- 208000033868 Lysosomal disease Diseases 0.000 abstract 1
- 239000002253 acid Substances 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 108010045758 lysosomal proteins Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 239000013608 rAAV vector Substances 0.000 abstract 1
- 230000003248 secreting effect Effects 0.000 abstract 1
- 230000004960 subcellular localization Effects 0.000 abstract 1
Classifications
-
- 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
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- C07K14/475—Growth factors; Growth regulators
-
- 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
- C07K14/62—Insulins
-
- 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
- C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
-
- 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)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- 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
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
- C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
- C12N2810/851—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from growth factors; from growth regulators
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962937556P | 2019-11-19 | 2019-11-19 | |
| US201962937583P | 2019-11-19 | 2019-11-19 | |
| US202063023570P | 2020-05-12 | 2020-05-12 | |
| PCT/US2020/061223 WO2021102107A1 (en) | 2019-11-19 | 2020-11-19 | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PH12022551229A1 true PH12022551229A1 (en) | 2023-07-31 |
Family
ID=75981066
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PH1/2022/551229A PH12022551229A1 (en) | 2019-11-19 | 2020-11-19 | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230038520A1 (https=) |
| EP (1) | EP4061946A4 (https=) |
| JP (1) | JP2023503046A (https=) |
| KR (1) | KR20220098384A (https=) |
| CN (1) | CN116096895A (https=) |
| AU (1) | AU2020388634A1 (https=) |
| CA (1) | CA3159018A1 (https=) |
| IL (1) | IL293068A (https=) |
| MX (1) | MX2022005916A (https=) |
| PH (1) | PH12022551229A1 (https=) |
| WO (1) | WO2021102107A1 (https=) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| US20240287148A1 (en) * | 2021-07-15 | 2024-08-29 | Cornell University | Engineered biomolecules for nutrient reprogramming |
| KR20240073025A (ko) * | 2021-08-25 | 2024-05-24 | 캔브리지 파마슈티컬스, 인크. | 간-향성 캡시드 단백질 및 알파-갈락토시다제를 포함하는 aav 입자 및 파브리 질환을 치료하기 위한 이의 용도 |
| TW202338095A (zh) * | 2021-08-25 | 2023-10-01 | 美商北海康成製藥有限公司 | 包含肝臟向性衣殼蛋白質及酸α－葡萄糖苷酶（ＧＡＡ）之ＡＡＶ顆粒及其用於治療龐貝氏病（ＰＯＭＰＥ ＤＩＳＥＡＳＥ）之用途 |
| MX2024009003A (es) * | 2022-01-21 | 2024-09-17 | Astrazeneca Ireland Ltd | Terapia genica para la enfermedad de gaucher. |
| KR20240145491A (ko) * | 2022-02-03 | 2024-10-07 | 아스텔라스 진 테라피스, 인크. | 폼페병의 개선된 치료를 위한 조성물 및 방법 |
| WO2023164060A2 (en) * | 2022-02-25 | 2023-08-31 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy |
| WO2023172975A1 (en) * | 2022-03-08 | 2023-09-14 | University Of Massachusetts | Methods of raav packaging |
| EP4493704A2 (en) * | 2022-03-18 | 2025-01-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications |
| WO2024055272A1 (zh) * | 2022-09-16 | 2024-03-21 | 复旦大学附属中山医院 | 能高效表达目的基因的mRNA载体系统、其构建及应用 |
| WO2024212961A1 (en) * | 2023-04-10 | 2024-10-17 | Skyline Therapeutics (Shanghai) Co., Ltd. | Recombinant aav for the gene therapy of pompe disease |
| EP4698655A1 (en) * | 2023-04-20 | 2026-02-25 | AskBio Inc. | Liver-specific regulatory nucleic acid sequences |
| KR20240159745A (ko) | 2023-04-28 | 2024-11-06 | 아주대학교산학협력단 | 유산균 유래 nadh 산화효소를 이용한 비알코올성 지방간염의 유전자 치료제 |
| WO2025018822A1 (ko) * | 2023-07-20 | 2025-01-23 | 제닉스큐어 주식회사 | 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물 |
| KR102814185B1 (ko) * | 2023-07-20 | 2025-06-12 | 제닉스큐어 주식회사 | 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물 |
| AU2024317483A1 (en) * | 2023-07-28 | 2026-01-29 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2001266740A1 (en) * | 2000-06-06 | 2001-12-17 | Genelabs Technologies, Inc. | Promoters for regulated gene expression |
| ATE521701T1 (de) * | 2003-01-22 | 2011-09-15 | Univ Duke | Verbesserte konstrukte zur expression lysosomaler polypeptide |
| US7521211B2 (en) * | 2005-04-05 | 2009-04-21 | Regeneron Pharmaceuticals, Inc | IGF-1 and IGF-2 chimeric polypeptides and therapeutic uses thereof |
| EP2162540A2 (en) * | 2007-05-22 | 2010-03-17 | Amgen Inc. | Compositions and methods for producing bioactive fusion proteins |
| HRP20170698T1 (hr) * | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| AU2010366066B2 (en) * | 2010-12-22 | 2016-01-14 | Fondazione Telethon | Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses |
| MX2013012345A (es) * | 2011-04-22 | 2015-05-07 | Genzyme Corp | Alfa glucosidasa acida modificada con procesamiento acelerado. |
| KR102423442B1 (ko) * | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| EP3293260A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293259A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| US12296026B2 (en) * | 2018-02-05 | 2025-05-13 | Astellas Gene Therapies, Inc. | Transcription regulatory elements and uses thereof |
| JP7779653B2 (ja) * | 2018-02-07 | 2025-12-03 | 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 | 治療用タンパク質送達のための方法および組成物 |
| KR20210005154A (ko) * | 2018-04-30 | 2021-01-13 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 유전자 요법 작제물 및 사용 방법 |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
-
2020
- 2020-11-19 CA CA3159018A patent/CA3159018A1/en active Pending
- 2020-11-19 EP EP20890917.6A patent/EP4061946A4/en active Pending
- 2020-11-19 PH PH1/2022/551229A patent/PH12022551229A1/en unknown
- 2020-11-19 MX MX2022005916A patent/MX2022005916A/es unknown
- 2020-11-19 IL IL293068A patent/IL293068A/en unknown
- 2020-11-19 US US17/778,175 patent/US20230038520A1/en active Pending
- 2020-11-19 AU AU2020388634A patent/AU2020388634A1/en active Pending
- 2020-11-19 KR KR1020227020169A patent/KR20220098384A/ko active Pending
- 2020-11-19 CN CN202080093548.9A patent/CN116096895A/zh active Pending
- 2020-11-19 JP JP2022529007A patent/JP2023503046A/ja active Pending
- 2020-11-19 WO PCT/US2020/061223 patent/WO2021102107A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| US20230038520A1 (en) | 2023-02-09 |
| IL293068A (en) | 2022-07-01 |
| KR20220098384A (ko) | 2022-07-12 |
| TW202132570A (zh) | 2021-09-01 |
| WO2021102107A1 (en) | 2021-05-27 |
| EP4061946A4 (en) | 2024-03-06 |
| CA3159018A1 (en) | 2021-05-27 |
| CN116096895A (zh) | 2023-05-09 |
| MX2022005916A (es) | 2022-08-04 |
| JP2023503046A (ja) | 2023-01-26 |
| AU2020388634A1 (en) | 2022-06-23 |
| EP4061946A1 (en) | 2022-09-28 |
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