MX2021011889A - Tratamiento genico para patologias oculares. - Google Patents

Tratamiento genico para patologias oculares.

Info

Publication number
MX2021011889A
MX2021011889A MX2021011889A MX2021011889A MX2021011889A MX 2021011889 A MX2021011889 A MX 2021011889A MX 2021011889 A MX2021011889 A MX 2021011889A MX 2021011889 A MX2021011889 A MX 2021011889A MX 2021011889 A MX2021011889 A MX 2021011889A
Authority
MX
Mexico
Prior art keywords
therapeutic
gene therapy
eye
eye pathologies
vectors
Prior art date
Application number
MX2021011889A
Other languages
English (en)
Inventor
Everen Sherri Van
Olivier Danos
Jesse I Yoo
Samir Maganbhai Patel
Avanti Arvind Ghanekar
Anthony Ray O''berry
Kim Rees Irwin-Pack
Darin Thomas Curtiss
Original Assignee
Regenxbio Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Regenxbio Inc filed Critical Regenxbio Inc
Publication of MX2021011889A publication Critical patent/MX2021011889A/es

Links

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
    • A61K48/0075—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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C07K16/22—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against 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
    • 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
    • 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
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • 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
    • 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
    • C07K2317/40—Immunoglobulins specific features characterized by post-translational modification
    • C07K2317/41—Glycosylation, sialylation, or fucosylation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/515—Complete light chain, i.e. VL + CL
    • 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
    • C07K2317/522—CH1 domain
    • 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
    • C07K2317/567—Framework region [FR]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/04—Uses of viruses as vector in vivo

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Mycology (AREA)

Abstract

Composiciones y métodos para la administración de productos terapéuticos (tales como proteínas terapéuticas (anticuerpos), ARN terapéuticos (shARN, siARN y miARN) y aptámeros terapéuticos) a la retina/humor vítreo en los ojos de sujetos humanos para tratar patologías oculares, que implican vectores virales recombinantes tales como vectores de virus adenoasociado recombinante (rAAV).
MX2021011889A 2019-04-03 2020-04-02 Tratamiento genico para patologias oculares. MX2021011889A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962828949P 2019-04-03 2019-04-03
US201962856533P 2019-06-03 2019-06-03
US201962946158P 2019-12-10 2019-12-10
PCT/US2020/026356 WO2020206098A1 (en) 2019-04-03 2020-04-02 Gene therapy for eye pathologies

Publications (1)

Publication Number Publication Date
MX2021011889A true MX2021011889A (es) 2021-10-26

Family

ID=70480820

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021011889A MX2021011889A (es) 2019-04-03 2020-04-02 Tratamiento genico para patologias oculares.

Country Status (13)

Country Link
US (1) US20220143221A1 (es)
EP (1) EP3946467A1 (es)
JP (1) JP2022521851A (es)
KR (1) KR20220004987A (es)
CN (1) CN113966236A (es)
AU (1) AU2020253462A1 (es)
BR (1) BR112021019825A2 (es)
CA (1) CA3135843A1 (es)
IL (1) IL286825A (es)
MX (1) MX2021011889A (es)
SG (1) SG11202110789VA (es)
TW (1) TW202102679A (es)
WO (1) WO2020206098A1 (es)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3452103A1 (en) 2016-04-15 2019-03-13 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
EA202091509A1 (ru) 2017-12-19 2020-09-22 Акуос, Инк. Aav-опосредованная доставка терапевтических антител во внутреннее ухо
JP2022530845A (ja) * 2019-04-27 2022-07-04 オクジェン アイエヌシー． 眼疾患に対するａｄｅｎｏ随伴ウイルス連ウイルスベクター媒介遺伝子治療
EP4133093A4 (en) * 2020-04-08 2024-05-22 President And Fellows Of Harvard College TGFBETA THERAPY FOR EYE AND NEURODEGENERATIVE DISEASES
CN111826386B (zh) * 2020-07-30 2022-02-01 西南大学 一种调控棉花纤维呈色的融合基因及其表达载体和应用
KR20230083287A (ko) * 2020-10-07 2023-06-09 리젠엑스바이오 인크. Cln2 질환의 안구 징후에 대한 유전자 요법
AU2022270607A1 (en) * 2021-05-03 2023-11-23 North Carolina State University Compositions and methods related to the treatment of ocular diseases in equines
CA3222463A1 (en) * 2021-06-18 2022-12-22 Peter Widdowson Retinal disorders
MX2024008509A (es) * 2022-01-09 2024-07-19 Kriya Therapeutics Inc Construcciones de vectores para la distribucion de acidos nucleicos que codifican anticuerpos terapeuticos anti-igf-1r y metodos para usarlos.
WO2024048688A1 (ja) * 2022-08-31 2024-03-07 国立大学法人京都大学 光応答改変型オプシン類
WO2024073669A1 (en) 2022-09-30 2024-04-04 Regenxbio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
CN116965768B (zh) * 2023-07-07 2024-01-19 中山大学中山眼科中心 一种自动定量分析眼内前房炎症程度的系统

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US878575A (en) 1906-12-31 1908-02-11 Isaac Stevenson Ore dredging and separating apparatus.
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
KR19990022651A (ko) 1995-06-07 1999-03-25 데이비드 엘. 버스테인 생물학적 사건에 대한 라파마이신 기재 조절방법
AU9036198A (en) 1997-08-26 1999-03-16 Ariad Gene Therapeutics, Inc. Fusion proteins comprising a dimerization, trimerization or tetramerization domain and an additional heterologous transcription activation, transcription repression, dna binding or ligand binding domain
JP2001514007A (ja) 1997-08-27 2001-09-11 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 キメラ転写アクチベーター、ならびにそれに関連する組成物および使用
AU755784B2 (en) 1998-01-15 2002-12-19 Ariad Pharmaceuticals, 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
DK1916258T3 (da) 1999-08-09 2014-07-28 Genzyme Corp Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
PL222683B1 (pl) 2001-11-13 2016-08-31 Univ Pennsylvania Rekombinowane wirusy stowarzyszone z adenowirusem (AAV), sposoby ich wytwarzania, pakująca komórka gospodarza, kompozycja zawierająca wirus, zastosowanie wirusa, wyizolowane wirusy AAV, białka,sztuczne białka kapsydu, cząsteczki, komórki gospodarza, sposoby dostarczania transgenu, sposób identyfikacji serotypu, zestaw diagnostyczny, sposób izolacji nowych wirusów, rekombinowana komórka
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
US20040208847A1 (en) * 2003-03-28 2004-10-21 Fabienne Rolling Method and vectors for selectively transducing retinal pigment epithelium cells
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
DK2359867T3 (en) 2005-04-07 2015-01-05 Univ Pennsylvania A method for increasing an AAV vector function
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
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
JP5704361B2 (ja) 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
DK2673289T3 (da) 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf
SG194583A1 (en) 2011-04-22 2013-12-30 Univ California Adeno-associated virus virions with variant capsid and methods of use thereof
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
US9677088B2 (en) 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
ES2729166T3 (es) 2012-05-24 2019-10-30 Altaviz Llc Inyector de fluido viscoso
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
EP3060575B1 (en) 2013-10-11 2018-12-05 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
US10064752B2 (en) * 2014-09-11 2018-09-04 Orbit Biomedical Limited Motorized suprachoroidal injection of therapeutic agent
EP3193985B1 (en) 2014-09-19 2020-08-12 Oxular Limited Ophthalmic delivery device
WO2016083669A1 (en) 2014-11-28 2016-06-02 Visionisti Oy Ocular therapeutics tool
EP3261440B1 (en) * 2015-02-20 2022-04-06 University of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
GB2543645B (en) 2015-09-17 2019-01-02 Oxular Ltd Ophthalmic Injection device
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
EP3400014A1 (en) * 2016-01-08 2018-11-14 Clearside Biomedical, Inc. Methods and devices for treating posterior ocular disorderswith aflibercept and other biologics
AU2017235845B2 (en) 2016-03-16 2022-06-09 Oxular Limited Ophthalmic delivery device and ophthalmic drug compositions
JP2019515027A (ja) 2016-04-15 2019-06-06 レジェンクスバイオ インコーポレーテッド 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療
EP3452103A1 (en) * 2016-04-15 2019-03-13 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
SG11202002396TA (en) * 2017-09-27 2020-04-29 Regenxbio Inc Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab
KR102675205B1 (ko) 2017-11-04 2024-06-13 알타비즈 엘엘씨 가스 구동식 유체 주입 시스템
WO2019090193A1 (en) 2017-11-04 2019-05-09 Altaviz, Llc Injection devices and methods for making and using them

Also Published As

Publication number Publication date
BR112021019825A2 (pt) 2021-12-21
SG11202110789VA (en) 2021-10-28
CN113966236A (zh) 2022-01-21
WO2020206098A9 (en) 2021-02-04
EP3946467A1 (en) 2022-02-09
TW202102679A (zh) 2021-01-16
US20220143221A1 (en) 2022-05-12
IL286825A (en) 2021-10-31
AU2020253462A1 (en) 2021-10-28
JP2022521851A (ja) 2022-04-12
WO2020206098A1 (en) 2020-10-08
CA3135843A1 (en) 2020-10-08
KR20220004987A (ko) 2022-01-12

Similar Documents

Publication Publication Date Title
MX2021011889A (es) Tratamiento genico para patologias oculares.
EP4317185A3 (en) Fully-human post-translationally modified antibody therapeutics
MX2021013486A (es) Viriones de virus adenoasociados con capside variante y metodos para su uso.
Pareek et al. MiR-155 induction in microglial cells suppresses Japanese encephalitis virus replication and negatively modulates innate immune responses
MX2021012867A (es) Terapéutica de anticuerpos postraduccionalmente modificados completamente humanos.
ZA201705628B (en) Methods and compositions for treating genetic eye diseases
MX2021006646A (es) Vector viral adenoasociado recombinante para el suministro de genes.
MD3596222T2 (ro) Livrarea cu vector de virus adeno-asociat a micro-distrofinei specifică mușchiului pentru tratarea distrofiei musculare
MX2024005644A (es) Administracion de vectores del virus adenoasociado de microdistrofina para tratar la distrofia muscular.
EA201990644A1 (ru) Антисмысловые олигонуклеотиды для лечения глазного заболевания
MX2020010465A (es) Vectores de virus para direccionamiento a tejidos oftalmicos.
EA201692206A1 (ru) Векторы на основе aav для генной терапии в сетчатке и cns
TR201900649T4 (tr) Oküler hastalıkların tedavisi için mrna tedavisi.
MY195439A (en) Adeno-Associated Virus Vector Delivery of B-Sarcoglycan and Microrna-29 and the Treatment of Muscular Dystrophy
CO2021000227A2 (es) Suministro mediante vectores de virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular
SG10201808218YA (en) Gene therapy for retinitis pigmentosa
MX2023014921A (es) Anticuerpo anti-cd154 con caracteristicas de union, funcionalidad y seguridad mejoradas y uso en inmunoterapia humana.
WO2020097511A3 (en) Messenger rna therapy for treatment of ocular diseases
MX2021003303A (es) Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano.
MX2022005063A (es) Tratamiento de enfermedades oculares con antagonistas de receptor de endotelina.
TN2019000112A1 (en) Ophthalmic formulation comprising citicoline carried by liposome for the treatment of glaucoma
WO2009058970A3 (en) A novel gene therapy approach for treating the metabolic disorder obesity
AR118573A1 (es) Tratamiento génico para patologías oculares
Araújo et al. Development of strategies to modulate gene expression of angiogenesis-related molecules in the retina
MX2016006256A (es) Tratamiento de glaucoma usando laquinimod.