CN120302995A - 携带显性负突变dnRhoA基因的自互补scAAV载体及其用于治疗眼部疾病的方法 - Google Patents
携带显性负突变dnRhoA基因的自互补scAAV载体及其用于治疗眼部疾病的方法 Download PDFInfo
- Publication number
- CN120302995A CN120302995A CN202380063545.4A CN202380063545A CN120302995A CN 120302995 A CN120302995 A CN 120302995A CN 202380063545 A CN202380063545 A CN 202380063545A CN 120302995 A CN120302995 A CN 120302995A
- Authority
- CN
- China
- Prior art keywords
- particle
- scaav
- recombinant
- injection
- dominant negative
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- 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)
-
- 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
-
- 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
-
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
- A61P27/06—Antiglaucoma agents or miotics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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)
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- 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
- C12N2750/14145—Special targeting system for 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Ophthalmology & Optometry (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263407150P | 2022-09-15 | 2022-09-15 | |
| US63/407,150 | 2022-09-15 | ||
| US202263414996P | 2022-10-11 | 2022-10-11 | |
| US63/414,996 | 2022-10-11 | ||
| US202263426725P | 2022-11-19 | 2022-11-19 | |
| US63/426,725 | 2022-11-19 | ||
| PCT/US2023/074351 WO2024059823A1 (en) | 2022-09-15 | 2023-09-15 | Self-complementary AAV vectors carrying dominant negative RhoA and methods of use to treat ocular diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN120302995A true CN120302995A (zh) | 2025-07-11 |
Family
ID=90275835
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202380063545.4A Pending CN120302995A (zh) | 2022-09-15 | 2023-09-15 | 携带显性负突变dnRhoA基因的自互补scAAV载体及其用于治疗眼部疾病的方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240100193A1 (enExample) |
| EP (1) | EP4587055A1 (enExample) |
| JP (1) | JP2025536506A (enExample) |
| KR (1) | KR20250067171A (enExample) |
| CN (1) | CN120302995A (enExample) |
| CA (1) | CA3265312A1 (enExample) |
| WO (1) | WO2024059823A1 (enExample) |
Family Cites Families (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019094326A1 (en) * | 2017-11-08 | 2019-05-16 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for treating cancer using rhoa dominant negative forms |
-
2023
- 2023-09-15 CN CN202380063545.4A patent/CN120302995A/zh active Pending
- 2023-09-15 EP EP23866545.9A patent/EP4587055A1/en active Pending
- 2023-09-15 JP JP2025514713A patent/JP2025536506A/ja active Pending
- 2023-09-15 CA CA3265312A patent/CA3265312A1/en active Pending
- 2023-09-15 US US18/468,299 patent/US20240100193A1/en active Pending
- 2023-09-15 KR KR1020257011853A patent/KR20250067171A/ko active Pending
- 2023-09-15 WO PCT/US2023/074351 patent/WO2024059823A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| US20240100193A1 (en) | 2024-03-28 |
| CA3265312A1 (en) | 2024-03-21 |
| EP4587055A1 (en) | 2025-07-23 |
| KR20250067171A (ko) | 2025-05-14 |
| JP2025536506A (ja) | 2025-11-07 |
| WO2024059823A1 (en) | 2024-03-21 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US11819478B2 (en) | Selective gene therapy expression system | |
| JP7097398B2 (ja) | ミオシリン(myoc)緑内障を処置するためのアデノ随伴ウイルスベクター | |
| RU2759335C2 (ru) | Генное редактирование глубоких интронных мутаций | |
| AU2015231294B2 (en) | rAAV-based compositions and methods for treating amyotrophic lateral sclerosis | |
| AU2017227776B2 (en) | AAV vectors for treatment of dominant retinitis pigmentosa | |
| US20210371480A1 (en) | Compositions and methods for treating age-related macular degeneration and other diseases | |
| KR20210009317A (ko) | 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 초래된 질환에 대한 유전자 요법 | |
| WO2023240236A1 (en) | Compositions and methods for the treatment of spinal muscular atrophy related disorders | |
| EA034575B1 (ru) | Эффективная доставка больших генов посредством двойных aav векторов | |
| RS61079B1 (sr) | Optimizovani rpe65 promotor i kodirajuće sekvence | |
| CN111867635A (zh) | 包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法 | |
| WO2018134168A1 (en) | Methods of expressing a polynucleotide of interest in the cone photoreceptors | |
| ES2946512T3 (es) | Terapia génica para el tratamiento de una enfermedad degenerativa de la retina | |
| CN111936172A (zh) | 用于治疗视网膜病症的组合物和方法 | |
| CA3096088A1 (en) | Compositions and methods for treating macular dystrophy | |
| CN120322449A (zh) | Atp7b基因疗法 | |
| JP2024535939A (ja) | 肝臓指向性カプシドタンパク質及びアルファ-ガラクトシダーゼを含むaav粒子並びにファブリー病を治療するためのそれらの使用 | |
| US20240100193A1 (en) | Self-complementary AAV vectors carrying dominant negative RhoA and methods of use to treat ocular diseases | |
| CN121311586A (zh) | 耳蜗內毛细胞特异性启动子及其应用 | |
| JP2026504367A (ja) | グルコシルセラミダーゼβ1欠損に関連する神経障害の治療のための組成物及び方法 | |
| EP4437116A1 (en) | Compositions and methods for cell-specific expression of target genes | |
| US20230414787A1 (en) | Gene knock-out for treatment of glaucoma | |
| JP2021520231A (ja) | シュタルガルト病の処置のための組成物及び方法 | |
| US20260041791A1 (en) | Compositions and methods for treatment of achromotopsia | |
| US12385064B2 (en) | Intravitreal dosing for delivery of polynucleotides to retinal cones |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PB01 | Publication | ||
| PB01 | Publication | ||
| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination |