MX2024012616A - Uso de moduladores de irak4 para terapia genica - Google Patents

Uso de moduladores de irak4 para terapia genica

Info

Publication number
MX2024012616A
MX2024012616A MX2024012616A MX2024012616A MX2024012616A MX 2024012616 A MX2024012616 A MX 2024012616A MX 2024012616 A MX2024012616 A MX 2024012616A MX 2024012616 A MX2024012616 A MX 2024012616A MX 2024012616 A MX2024012616 A MX 2024012616A
Authority
MX
Mexico
Prior art keywords
gene therapy
vector
irak
modulator
individual
Prior art date
Application number
MX2024012616A
Other languages
English (en)
Spanish (es)
Inventor
Sourav Roy Choudhury
Mona Motwani
Christian Mueller
John Reed
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of MX2024012616A publication Critical patent/MX2024012616A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4427—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
    • A61K31/4439—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47—Quinolines; Isoquinolines
    • A61K31/475—Quinolines; Isoquinolines having an indole ring, e.g. yohimbine, reserpine, strychnine, vinblastine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5386—1,4-Oxazines, e.g. morpholine spiro-condensed or forming part of bridged ring systems
    • 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
    • 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
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
MX2024012616A 2022-04-12 2024-10-10 Uso de moduladores de irak4 para terapia genica MX2024012616A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263330239P 2022-04-12 2022-04-12
PCT/US2023/065687 WO2023201272A1 (en) 2022-04-12 2023-04-12 Use of irak4 modulators for gene therapy

Publications (1)

Publication Number Publication Date
MX2024012616A true MX2024012616A (es) 2025-02-10

Family

ID=86329443

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024012616A MX2024012616A (es) 2022-04-12 2024-10-10 Uso de moduladores de irak4 para terapia genica

Country Status (11)

Country Link
US (1) US20230405151A1 (enExample)
EP (1) EP4508225A1 (enExample)
JP (1) JP2025513850A (enExample)
KR (1) KR20250004740A (enExample)
CN (1) CN119630802A (enExample)
AU (1) AU2023252915A1 (enExample)
CA (1) CA3248481A1 (enExample)
IL (1) IL316231A (enExample)
MX (1) MX2024012616A (enExample)
TW (1) TW202400250A (enExample)
WO (1) WO2023201272A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PH12022500002A1 (en) 2019-06-28 2023-04-03 Kymera Therapeutics Inc Irak degraders and uses thereof
US12558427B2 (en) 2019-07-17 2026-02-24 Kymera Therapeutics, Inc. IRAK degraders and uses thereof
WO2021119159A1 (en) 2019-12-10 2021-06-17 Kymera Therapeutics, Inc. Irak degraders and uses thereof
CA3161878A1 (en) 2019-12-17 2021-06-24 Matthew M. Weiss Irak degraders and uses thereof

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
US6818419B2 (en) 2000-01-13 2004-11-16 Tularik Inc. IRAK-4: compositions and methods of use
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
US20100279316A1 (en) 2007-01-19 2010-11-04 Leonid Gorelik Antibodies to Phosphorylated IRAK4
JP5956331B2 (ja) 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えａａｖベクターの改良された精製方法
US20110070241A1 (en) * 2009-06-30 2011-03-24 Duke University Methods for modulating immune responses to aav gene therapy vectors
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
JP2017513954A (ja) 2014-04-22 2017-06-01 ニンバス アイリス， インコーポレイテッド Ｉｒａｋ阻害剤およびその使用
EP3200788B1 (en) 2014-09-30 2019-09-18 Merck Sharp & Dohme Corp. Inhibitors of irak4 activity
US10040798B2 (en) 2015-03-12 2018-08-07 Merck Sharp & Dohme Corp. Pyrrolopyridazine inhibitors of IRAK4 activity
WO2017004133A1 (en) 2015-06-29 2017-01-05 Nimbus Iris, Inc. Irak inhibitors and uses thereof
EP3322409A4 (en) 2015-07-15 2019-07-24 Aurigene Discovery Technologies Limited SUBSTITUTED AZA COMPOUNDS AS IRAQ-4 INHIBITORS
CN107531725B (zh) 2015-08-13 2021-03-19 北京韩美药品有限公司 Irak4抑制剂及其应用
CN110234637B (zh) 2016-10-26 2022-11-01 里格尔药品股份有限公司 用作irak抑制剂的吡唑酰胺化合物
IL315310A (en) 2017-12-26 2024-10-01 Kymera Therapeutics Inc Irak degraders and uses thereof
CA3090275A1 (en) 2018-02-14 2019-08-22 Dana-Farber Cancer Institute, Inc. Irak degraders and uses thereof
EP3774797B1 (en) 2018-04-05 2025-11-19 Merck Patent GmbH Heteroaryl compounds as type ii irak inhibitors and uses hereof
EP3814380A4 (en) 2018-06-29 2022-06-15 Dana Farber Cancer Institute, Inc. BISPECIFIC DEGRADATION PRODUCTS
AU2019389174A1 (en) 2018-11-30 2021-07-01 Kymera Therapeutics, Inc. Irak degraders and uses thereof
WO2020264490A1 (en) 2019-06-28 2020-12-30 Kymera Therapeutics, Inc. Irak degraders and uses thereof
US12558427B2 (en) 2019-07-17 2026-02-24 Kymera Therapeutics, Inc. IRAK degraders and uses thereof
EP4076524A4 (en) 2019-12-17 2023-11-29 Kymera Therapeutics, Inc. IRAQ DEGRADERS AND USES THEREOF
JP7815127B2 (ja) 2020-02-19 2026-02-17 ニューリックス セラピューティクス，インコーポレイテッド インターロイキン－１受容体関連キナーゼの二機能性分解剤およびその治療的使用
EP4149622A4 (en) * 2020-05-12 2024-12-18 Myogene Bio LLC IMMUNOSUPPRESSIVE AGENTS AND METHODS OF RE-DOSING VIRAL ADMINISTRATION FOR GENE THERAPY
EP4161521A4 (en) 2020-06-03 2024-07-10 Kymera Therapeutics, Inc. DEUTERED IRAQ DEGRADERS AND USES THEREOF
TW202210483A (zh) 2020-06-03 2022-03-16 美商凱麥拉醫療公司 Irak降解劑之結晶型

Also Published As

Publication number Publication date
US20230405151A1 (en) 2023-12-21
JP2025513850A (ja) 2025-04-30
WO2023201272A1 (en) 2023-10-19
AU2023252915A1 (en) 2024-11-28
IL316231A (en) 2024-12-01
EP4508225A1 (en) 2025-02-19
TW202400250A (zh) 2024-01-01
KR20250004740A (ko) 2025-01-08
CN119630802A (zh) 2025-03-14
CA3248481A1 (en) 2023-10-19

Similar Documents

Publication Publication Date Title
MX2024012617A (es) Uso de un modulador irak4 para terapia genica
CO2018012082A2 (es) Administración de vectores del virus adenoasociado de β-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular
ECSP17059343A (es) ARNi VARIANTE
BR112022009895A2 (pt) Construtos de terapia de gene de microdistrofina e usos dos mesmos
CO2021012074A2 (es) Suministro de β-sarcoglicano mediante vectores de virus adenoasociado y el tratamiento de la distrofia muscular
CL2017002731A1 (es) Adenovirus oncolítico que codifica una proteína b7
SV2018005634A (es) Anticuerpos anti-bcma, moleculas de union a antigenos biespecificas que se unen a bcma y cd3, y usos de estos
CO2020001930A2 (es) Adenovirus armado con captador biespecífico de linfocitos t (bite)
DOP2016000237A (es) Terapia genica para la retinitis pigmentaria
CO2022014388A2 (es) Una terapia combinada con nirogacestat y una terapia dirigida a bcma y usos de estas
CL2008001634A1 (es) Anticuerpo de la region regulatoria negativa anti-notch1 aislado; polinucleotido que lo codifica; vector y celula huesped; metodo de produccion; composicion que lo comprende; y su uso para preparar un medicamento para tratar un trastorno asociado con un aumento en la señalizacion o expresion de notch1 en un individuo.
AR113134A1 (es) Arni variante
CO2022002864A2 (es) Administración de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular
EA202290001A1 (ru) Векторы aav с промотором миелинового белка ноль и их применение для лечения заболеваний, связанных со шванновскими клетками, таких как болезнь шарко-мари-тута
BR112018076639A2 (pt) quimioterapias de combinação
CL2021001880A1 (es) Vectores víricos que codifican variantes del fviii recombinantes con mayor expresión para la genoterapia de la hemofilia a
MX2017014257A (es) Metodos de tratamiento de trastornos linfoproliferativos asociados al virus de epstein-barr por terapia con celulas t.
EP4591924A3 (en) Therapy device
CO2022007361A2 (es) Terapia de combinación para el tratamiento de cáncer cerebral
MX2024012618A (es) Ensayo de células dendríticas para determinar la inmunogenicidad innata frente a agentes de terapia génica
MX2021011598A (es) Uso de mir101 o mir128 en el tratamiento de trastornos convulsivos.
CO2023012667A2 (es) Combinaciones farmacéuticas para el tratamiento del cáncer
CL2022001459A1 (es) Célula vector adyuvante artificial con contenido de ny-eso-1 para el tratamiento del cancer
AR121175A1 (es) ADMINISTRACIÓN DE VECTORES DE VIRUS ADENOASOCIADO DE a-SARCOGLICANO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR
AR120388A1 (es) Promotores de células de apoyo vestibulares y sus usos