AR129215A1 - Anticuerpos anti-complemento y agentes del complemento vectorizados y administración de estos - Google Patents
Anticuerpos anti-complemento y agentes del complemento vectorizados y administración de estosInfo
- Publication number
- AR129215A1 AR129215A1 ARP230101077A ARP230101077A AR129215A1 AR 129215 A1 AR129215 A1 AR 129215A1 AR P230101077 A ARP230101077 A AR P230101077A AR P230101077 A ARP230101077 A AR P230101077A AR 129215 A1 AR129215 A1 AR 129215A1
- Authority
- AR
- Argentina
- Prior art keywords
- transgene
- tissue cells
- expression cassette
- human
- aav
- Prior art date
Links
- 230000002391 anti-complement effect Effects 0.000 title 1
- 108010008730 anticomplement Proteins 0.000 title 1
- 230000000295 complement effect Effects 0.000 title 1
- 108700019146 Transgenes Proteins 0.000 abstract 5
- 206010064930 age-related macular degeneration Diseases 0.000 abstract 4
- 208000002780 macular degeneration Diseases 0.000 abstract 4
- 239000000427 antigen Substances 0.000 abstract 3
- 108091007433 antigens Proteins 0.000 abstract 3
- 102000036639 antigens Human genes 0.000 abstract 3
- 239000012634 fragment Substances 0.000 abstract 3
- 238000000034 method Methods 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 230000001105 regulatory effect Effects 0.000 abstract 2
- 239000013607 AAV vector Substances 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 239000013612 plasmid Substances 0.000 abstract 1
- 239000013608 rAAV vector Substances 0.000 abstract 1
- 238000007910 systemic administration Methods 0.000 abstract 1
- 229940126622 therapeutic monoclonal antibody Drugs 0.000 abstract 1
- 230000010415 tropism Effects 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/545—Medicinal preparations containing antigens or antibodies characterised by the dose, timing or administration schedule
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
- C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/52—Constant or Fc region; Isotype
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/55—Fab or Fab'
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
- C07K2317/569—Single domain, e.g. dAb, sdAb, VHH, VNAR or nanobody®
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
- C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Ophthalmology & Optometry (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
Se describen composiciones y métodos para la administración de un anticuerpo monoclonal terapéutico completamente humano modificado postraduccionalmente, o un fragmento de unión al antígeno de este, que se une a C3 o C5 a un sujeto humano para el tratamiento de una indicación ocular, particularmente AMD. También se proporcionan composiciones y métodos para la administración de hCHL1 a un sujeto humano para el tratamiento de una indicación ocular, en particular la AMD. La secuencia de nucleótidos que codifica el anticuerpo se administra en un vector de rAAV que se dirige a las células del tejido ocular para la expresión del transgén. Reivindicación 1: Una composición farmacéutica para tratar la degeneración macular asociada con la edad (AMD) en un sujeto humano que lo necesite, que comprende un vector de virus adenoasociado (AAV) que tiene: (a) una cápside viral que tiene un tropismo por las células del tejido ocular; y (b) un genoma artificial que comprende un casete de expresión flanqueado por repeticiones terminales invertidas (ITR) de AAV, donde el casete de expresión comprende un transgén que codifica una cadena pesada y una cadena ligera de un anticuerpo anti-C3 o anti-C5 sustancialmente de longitud completa o de longitud completa, o un fragmento de unión al antígeno de este, unido operativamente a una o más secuencias reguladoras que promueven la expresión del transgén en células de tejido ocular humano; donde dicho vector de AAV se formula para administración subretiniana, intravítrea, intranasal, intracameral, supracoroidea o sistémica a dicho sujeto humano. Reivindicación 82: Una célula hospedadora que comprende: un plásmido que comprende un casete de expresión cis flanqueado por ITR de AAV, donde el casete de expresión cis comprende un transgén que codifica un mAb anti-C3 o mAb anti-C5 sustancialmente de longitud completa o de longitud completa o un fragmento de unión al antígeno de estos, unido operativamente a una o más secuencias reguladoras que promueven la expresión del transgén en células de tejido ocular humano.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263338009P | 2022-05-03 | 2022-05-03 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR129215A1 true AR129215A1 (es) | 2024-07-31 |
Family
ID=86688572
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP230101077A AR129215A1 (es) | 2022-05-03 | 2023-05-03 | Anticuerpos anti-complemento y agentes del complemento vectorizados y administración de estos |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP4518972A2 (es) |
| AR (1) | AR129215A1 (es) |
| TW (1) | TW202400803A (es) |
| WO (1) | WO2023215806A2 (es) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2024271004A1 (en) * | 2023-05-16 | 2026-01-15 | Regenxbio Inc. | Vectorized anti-complement antibodies and administration thereof |
| WO2025217230A1 (en) * | 2024-04-08 | 2025-10-16 | Regenxbio Inc. | Vectorized anti-complement antibodies and complement agents and administration thereof |
Family Cites Families (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1994018317A1 (en) | 1993-02-12 | 1994-08-18 | The Board Of Trustees Of The Leland Stanford Junior University | Regulated transcription of targeted genes and other biological events |
| ATE544776T1 (de) | 1994-12-29 | 2012-02-15 | Massachusetts Inst Technology | Chimäre dna-bindeproteine |
| WO1996041865A1 (en) | 1995-06-07 | 1996-12-27 | Ariad Gene Therapeutics, Inc. | Rapamcycin-based regulation of biological events |
| JP2003524368A (ja) | 1997-08-26 | 2003-08-19 | アリアド ジーン セラピューティクス インコーポレイテッド | ニ量化ドメイン、三量化ドメインまたは四量化ドメインおよび補足的非相同転写活性化ドメイン、転写抑制ドメイン、ｄｎａ結合ドメインまたはリガンド結合ドメインを含む融合蛋白 |
| IL134643A0 (en) | 1997-08-27 | 2001-04-30 | Ariad Gene Therapeutics Inc | Chimeric transcriptional activators and compositions and uses related thereto |
| WO1999036553A2 (en) | 1998-01-15 | 1999-07-22 | Ariad Gene Therapeutics, Inc. | Regulation of biological events using multimeric chimeric proteins |
| AU766513B2 (en) | 1998-02-13 | 2003-10-16 | Board Of Trustees Of The Leland Stanford Junior University | Novel dimerizing agents, their production and use |
| AU781958C (en) | 1999-08-09 | 2006-03-30 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| US7067526B1 (en) | 1999-08-24 | 2006-06-27 | Ariad Gene Therapeutics, Inc. | 28-epirapalogs |
| HU230406B1 (hu) | 2001-11-13 | 2016-04-28 | The Trustees Of The University Of Pennsylvania | Eljárás adeno-asszociált vírus(AAV)szekvenciák detektálására és/vagy azonosítására és az azzal azonosított új szekvenciák izolálására |
| FI3517134T3 (fi) | 2001-12-17 | 2024-04-03 | Univ Pennsylvania | Adeno-assosioituneen viruksen (aav) serotyypin 8 sekvenssejä, niitä sisältäviä vektoreita ja niiden käyttöjä |
| US20090010920A1 (en) | 2003-03-03 | 2009-01-08 | Xencor, Inc. | Fc Variants Having Decreased Affinity for FcyRIIb |
| US8399618B2 (en) | 2004-10-21 | 2013-03-19 | Xencor, Inc. | Immunoglobulin insertions, deletions, and substitutions |
| DK2292779T3 (en) | 2003-09-30 | 2017-02-27 | Univ Pennsylvania | ADENOASS-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME AND APPLICATIONS THEREOF |
| US20070135620A1 (en) | 2004-11-12 | 2007-06-14 | Xencor, Inc. | Fc variants with altered binding to FcRn |
| US8367805B2 (en) | 2004-11-12 | 2013-02-05 | Xencor, Inc. | Fc variants with altered binding to FcRn |
| US7183969B2 (en) | 2004-12-22 | 2007-02-27 | Raytheon Company | System and technique for calibrating radar arrays |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| US7846724B2 (en) | 2006-04-11 | 2010-12-07 | Hoffmann-La Roche Inc. | Method for selecting CHO cell for production of glycosylated antibodies |
| EP2250256B1 (en) | 2008-02-19 | 2017-08-02 | uniQure IP B.V. | Optimisation of expression of parvoviral rep and cap proteins in insect cells |
| HUE044522T2 (hu) | 2009-04-30 | 2019-10-28 | Univ Pennsylvania | Adeno-asszociált vírus konstrukciókat tartalmazó légúti vezetõ sejtek célzására szolgáló kompozíciók |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US10053513B2 (en) | 2009-11-30 | 2018-08-21 | Janssen Biotech, Inc. | Antibody Fc mutants with ablated effector functions |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| KR102202908B1 (ko) | 2011-04-22 | 2021-01-15 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법 |
| EP2748185A1 (en) | 2011-08-24 | 2014-07-02 | The Board of Trustees of The Leland Stanford Junior University | New aav capsid proteins for nucleic acid transfer |
| US9382319B2 (en) | 2011-09-26 | 2016-07-05 | Jn Biosciences Llc | Hybrid constant regions |
| JP6385920B2 (ja) | 2012-05-09 | 2018-09-05 | オレゴン ヘルス アンド サイエンス ユニバーシティー | アデノ随伴ウイルスプラスミド及びベクター |
| TWI635098B (zh) | 2013-02-01 | 2018-09-11 | 再生元醫藥公司 | 含嵌合恆定區之抗體 |
| CA2905952A1 (en) | 2013-03-13 | 2014-10-02 | The Children's Hospital Of Philadelphia | Adeno-associated virus vectors and methods of use thereof |
| AU2014253730B2 (en) | 2013-04-20 | 2018-09-13 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus delivery of exon 2-targeted U7snRNA polynucleotide constructs |
| CN105579465B (zh) | 2013-07-22 | 2019-09-10 | 费城儿童医院 | 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途 |
| EP3561062A1 (en) | 2013-09-13 | 2019-10-30 | California Institute of Technology | Selective recovery |
| EP3060575B1 (en) | 2013-10-11 | 2018-12-05 | Massachusetts Eye & Ear Infirmary | Methods of predicting ancestral virus sequences and uses thereof |
| US10746742B2 (en) | 2014-04-25 | 2020-08-18 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG11201702073XA (en) | 2014-09-24 | 2017-04-27 | Hope City | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3277725B1 (en) | 2015-03-30 | 2020-11-25 | Regeneron Pharmaceuticals, Inc. | Heavy chain constant regions with reduced binding to fc gamma receptors |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| JP6665466B2 (ja) | 2015-09-26 | 2020-03-13 | 日亜化学工業株式会社 | 半導体発光素子及びその製造方法 |
| WO2017070491A1 (en) | 2015-10-23 | 2017-04-27 | Applied Genetic Technologies Corporation | Ophthalmic formulations |
| US20220195462A1 (en) * | 2019-04-24 | 2022-06-23 | Regenxbio Inc. | Fully-human post-translationally modified antibody therapeutics |
| AU2021356667A1 (en) * | 2020-10-07 | 2023-06-08 | Regenxbio Inc. | Adeno-associated viruses for ocular delivery of gene therapy |
-
2023
- 2023-05-03 TW TW112116523A patent/TW202400803A/zh unknown
- 2023-05-03 WO PCT/US2023/066574 patent/WO2023215806A2/en not_active Ceased
- 2023-05-03 EP EP23728549.9A patent/EP4518972A2/en active Pending
- 2023-05-03 AR ARP230101077A patent/AR129215A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4518972A2 (en) | 2025-03-12 |
| WO2023215806A3 (en) | 2023-12-21 |
| WO2023215806A2 (en) | 2023-11-09 |
| TW202400803A (zh) | 2024-01-01 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FB | Suspension of granting procedure |