AR127217A1 - Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticas - Google Patents
Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticasInfo
- Publication number
- AR127217A1 AR127217A1 ARP220102662A ARP220102662A AR127217A1 AR 127217 A1 AR127217 A1 AR 127217A1 AR P220102662 A ARP220102662 A AR P220102662A AR P220102662 A ARP220102662 A AR P220102662A AR 127217 A1 AR127217 A1 AR 127217A1
- Authority
- AR
- Argentina
- Prior art keywords
- hereditary angioedema
- tris
- agent
- years
- genotherapy
- Prior art date
Links
- 206010019860 Hereditary angioedema Diseases 0.000 title abstract 4
- 239000013598 vector Substances 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title 1
- 230000001225 therapeutic effect Effects 0.000 title 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 2
- 229940009550 c1 esterase inhibitor Drugs 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 239000002245 particle Substances 0.000 abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 abstract 2
- 102000055157 Complement C1 Inhibitor Human genes 0.000 abstract 1
- 108050007539 Plasma protease C1 inhibitor Proteins 0.000 abstract 1
- 239000007983 Tris buffer Substances 0.000 abstract 1
- 239000006172 buffering agent Substances 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 230000002338 cryopreservative effect Effects 0.000 abstract 1
- 210000004185 liver Anatomy 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
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
- 239000004094 surface-active agent Substances 0.000 abstract 1
- 238000013518 transcription Methods 0.000 abstract 1
- 230000035897 transcription Effects 0.000 abstract 1
Classifications
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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/10—Antioedematous agents; Diuretics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/81—Protease inhibitors
- C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
- C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
- C07K14/8121—Serpins
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
-
- 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
- C12N2830/42—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)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biochemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
En la presente descripción se proporcionan composiciones farmacéuticas y métodos para tratar el angioedema hereditario en un sujeto humano. Reivindicación 1: Un método para tratar a un sujeto humano con angioedema hereditario (AEH), que comprende administrar al sujeto una dosis única en el intervalo de aproximadamente 2E13 vg/kg a aproximadamente 6E14 vg/kg de partículas de virus adenoasociado recombinante (rAAV) que comprenden (a) una cápside de AAV con tropismo hepático, y (b) una construcción de vector recombinante que comprende un ácido nucleico que codifica una proteína inhibidora de la esterasa C1 funcional (C1-INH) enlazada operativamente a una región reguladora de transcripción específica del hígado heteróloga. Reivindicación 68: Una composición farmacéutica que comprende partículas de rAAV a una concentración de al menos aproximadamente 1E13 vg/ml a aproximadamente 1E14 vg/ml, un agente amortiguador tris(hidroximetil)aminometano (Tris), un agente de isotonicidad, un agente crioconservador y un tensioactivo que es estable durante el almacenamiento a aproximadamente -60ºC (menos sesenta grados centígrados) o menos durante al menos aproximadamente 1 año, 1,5 años, o 2 años.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163251558P | 2021-10-01 | 2021-10-01 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR127217A1 true AR127217A1 (es) | 2023-12-27 |
Family
ID=84358324
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220102662A AR127217A1 (es) | 2021-10-01 | 2022-09-30 | Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticas |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP4408481A2 (es) |
| AR (1) | AR127217A1 (es) |
| TW (1) | TW202332472A (es) |
| WO (1) | WO2023056436A2 (es) |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| ATE78293T1 (de) | 1983-05-27 | 1992-08-15 | Texas A & M Univ Sys | Verfahren zur herstellung eines rekombinanten baculovirus-expressionsvektors. |
| ZA848495B (en) | 1984-01-31 | 1985-09-25 | Idaho Res Found | Production of polypeptides in insect cells |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| DE60239103D1 (de) | 2001-10-16 | 2011-03-17 | Nat Inst Of Advanced Ind Scien | Neue n-acetylglucosamin transferase, codierende nukleinsäuren, spezifische antikörper, sowie verwendung zur krebsdiagnose |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| IL161827A0 (en) | 2001-11-13 | 2005-11-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
| KR102057540B1 (ko) | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| US10214731B2 (en) * | 2015-05-28 | 2019-02-26 | Cornell University | Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| WO2020023612A1 (en) * | 2018-07-24 | 2020-01-30 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
| BR112022005392A2 (pt) | 2019-09-27 | 2022-09-06 | Biomarin Pharm Inc | Caracterização de partículas virais de terapia genética que usa cromatografia por exclusão de tamanho e tecnologias de dispersão de luz multiangular |
| CN115176022A (zh) * | 2019-10-23 | 2022-10-11 | 夏尔人类遗传性治疗公司 | 用于遗传性血管性水肿的基于腺相关病毒载体的基因疗法 |
| CN114829391A (zh) | 2019-11-14 | 2022-07-29 | 生物马林药物股份有限公司 | 用肝特异性基因疗法载体治疗遗传性血管性水肿 |
| AU2022214192A1 (en) * | 2021-01-27 | 2023-07-27 | Spark Therapeutics, Inc. | Compositions and methods for treating hereditary angioedema |
-
2022
- 2022-09-30 AR ARP220102662A patent/AR127217A1/es unknown
- 2022-09-30 TW TW111137262A patent/TW202332472A/zh unknown
- 2022-09-30 EP EP22803136.5A patent/EP4408481A2/en active Pending
- 2022-09-30 WO PCT/US2022/077372 patent/WO2023056436A2/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4408481A2 (en) | 2024-08-07 |
| WO2023056436A2 (en) | 2023-04-06 |
| WO2023056436A3 (en) | 2023-05-11 |
| TW202332472A (zh) | 2023-08-16 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FB | Suspension of granting procedure |