MX2020010718A - Composiciones y metodos para el trasplante de celulas madre. - Google Patents

Composiciones y metodos para el trasplante de celulas madre.

Info

Publication number
MX2020010718A
MX2020010718A MX2020010718A MX2020010718A MX2020010718A MX 2020010718 A MX2020010718 A MX 2020010718A MX 2020010718 A MX2020010718 A MX 2020010718A MX 2020010718 A MX2020010718 A MX 2020010718A MX 2020010718 A MX2020010718 A MX 2020010718A
Authority
MX
Mexico
Prior art keywords
methods
compositions
stem cell
cell transplant
gene
Prior art date
Application number
MX2020010718A
Other languages
English (en)
Spanish (es)
Inventor
Brian Beard
Navarro Julian Sevilla
Gaurav Shah
Kinnari Patel
Raj Prabhakar
Original Assignee
Spacecraft Seven Llc
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 Spacecraft Seven Llc filed Critical Spacecraft Seven Llc
Publication of MX2020010718A publication Critical patent/MX2020010718A/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14Blood; Artificial blood
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
MX2020010718A 2018-04-11 2019-04-11 Composiciones y metodos para el trasplante de celulas madre. MX2020010718A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862656292P 2018-04-11 2018-04-11
PCT/US2019/027083 WO2019200167A1 (en) 2018-04-11 2019-04-11 Compositions and methods for stem cell transplant

Publications (1)

Publication Number Publication Date
MX2020010718A true MX2020010718A (es) 2021-01-20

Family

ID=68162990

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020010718A MX2020010718A (es) 2018-04-11 2019-04-11 Composiciones y metodos para el trasplante de celulas madre.

Country Status (11)

Country Link
US (1) US12539316B2 (https=)
JP (2) JP7576754B2 (https=)
KR (1) KR20210003738A (https=)
CN (1) CN112153973A (https=)
AU (2) AU2019252662A1 (https=)
BR (1) BR112020020841A2 (https=)
CA (1) CA3093708A1 (https=)
IL (2) IL307996A (https=)
MX (1) MX2020010718A (https=)
SG (1) SG11202008400SA (https=)
WO (1) WO2019200167A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
CN119264211A (zh) 2018-08-27 2025-01-07 瑞泽恩制药公司 拉曼光谱在下游纯化中的应用
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
JP2024502820A (ja) * 2020-12-31 2024-01-23 ブイオーアール バイオファーマ インコーポレーテッド Cd34遺伝子改変のための組成物及び方法
US20250221931A1 (en) * 2022-03-25 2025-07-10 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia

Family Cites Families (87)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2779192B2 (ja) 1987-09-17 1998-07-23 マサチユセツツ・インスチチユート・オブ・テクノロジー ヒト赤血球特異的転写エンハンサー
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
DE69434860T2 (de) 1993-02-22 2007-03-15 The Rockefeller University Herstellung von helfer-freien retroviren mit hohem titer mittels transienter transfektion
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
AU687117B2 (en) 1993-10-25 1998-02-19 Canji, Inc. Recombinant adenoviral vector and methods of use
US5484720A (en) 1994-09-08 1996-01-16 Genentech, Inc. Methods for calcium phosphate transfection
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6027721A (en) 1996-05-20 2000-02-22 Cytotherapeutics, Inc. Device and method for encapsulated gene therapy
US5952190A (en) 1996-10-04 1999-09-14 Fanconi Anemia Research Fund, Inc. cDNA for fanconi anemia complementation group A
US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
WO2001088088A2 (en) 2000-05-18 2001-11-22 Hyseq, Inc. Novel nucleic acids and polypeptides
US7994278B1 (en) 1999-08-06 2011-08-09 Nobel Biosciences Llc Biologically active polypeptides derived from a novel early stage pregnancy factor designated maternin (MA)
HK1048302A1 (zh) 1999-08-13 2003-03-28 大正制药株式会社 前列腺素衍生物
HK1050210B (en) 1999-10-12 2007-08-24 Institut Pasteur Lentiviral triplex dna, and vectors and recombinant cells containing lentiviral triplex dna
US20020013294A1 (en) 2000-03-31 2002-01-31 Delong Mitchell Anthony Cosmetic and pharmaceutical compositions and methods using 2-decarboxy-2-phosphinico derivatives
US6627442B1 (en) 2000-08-31 2003-09-30 Virxsys Corporation Methods for stable transduction of cells with hiv-derived viral vectors
GB0024550D0 (https=) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
ATE527347T1 (de) 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
RU2305708C2 (ru) 2001-10-02 2007-09-10 Энститю Клейтон Де Ля Решерш Рекомбинантный лентивирусный вектор, клетка-хозяин, трансдуцированная лентивирусным вектором, способ ее трансдукции и применение
AU2003223661A1 (en) 2002-04-15 2003-11-03 Rigel Pharmaceuticals, Inc. Methods of assaying for cell cycle modulators
CA2483915A1 (en) 2002-05-02 2003-11-13 University Of Rochester Vectors having both isoforms of b-hexosaminidase
ES2298745T3 (es) 2003-04-24 2008-05-16 Fondazione Centro San Raffaele Del Monte Tabor Promotores bidireccionales de sintesis y utilizacion de los mismos.
US20070048285A1 (en) 2005-08-24 2007-03-01 Christopher Baum Self-inactivating retroviral vector
BE1016793A4 (fr) 2005-10-04 2007-06-05 Artelis Procede de culture de cellules et dispositif permettant sa mise en oeuvre.
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
WO2008136670A2 (en) 2007-05-02 2008-11-13 Erasmus University Medical Center Rotterdam Improved methods and means for lentiviral gene delivery
EP2333091B1 (en) 2007-05-29 2017-06-21 Nature Technology Corporation Vectors and methods for genetic immunization
ES2660180T3 (es) 2007-12-07 2018-03-21 Miltenyi Biotec Gmbh Sistemas y métodos para procesamiento de células
DE102008063606A1 (de) 2008-12-18 2010-06-24 Eufets Ag Ein-Vektor Tet-System und dessen Verwendung
WO2010126766A1 (en) 2009-04-30 2010-11-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
DE102009021592A1 (de) 2009-05-15 2010-11-18 Medizinische Hochschule Hannover ASLV-Vektorsystem
WO2010138750A2 (en) 2009-05-27 2010-12-02 The Salk Institute For Biological Studies Generation of genetically corrected disease-free induced pluripotent stem cells
JP5996429B2 (ja) 2009-09-03 2016-09-21 ジェネンテック, インコーポレイテッド 関節リウマチの治療、診断及びモニターするための方法
WO2011043719A1 (en) 2009-10-05 2011-04-14 Ya-Fang Mei Replicating viral vectors for gene therapy
KR20140045411A (ko) 2011-05-16 2014-04-16 젠자임 코포레이션 Cxcr4 길항제의 용도
EP2820138B1 (en) 2012-02-29 2020-10-28 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Retroviral transduction using poloxamers
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
EP2931871B1 (en) 2012-12-11 2026-03-04 Pall Technology UK limited Recipient for cell cultivation
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
WO2014100073A2 (en) 2012-12-21 2014-06-26 Merck Sharp & Dohme Corp. Expression vectors for recombinant protein production in mammalian cells
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
CN104419683A (zh) * 2013-09-10 2015-03-18 中国科学院生物物理研究所 制备范可尼贫血症患者自体诱导多能干细胞的方法及其应用
GB201318347D0 (en) 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
WO2015073922A2 (en) 2013-11-15 2015-05-21 Northwestern University Inhibition of oxidative stress in atrial fibrillation
US20170037431A1 (en) 2014-05-01 2017-02-09 University Of Washington In vivo Gene Engineering with Adenoviral Vectors
US20170175143A1 (en) * 2014-05-20 2017-06-22 Regents Of The University Of Minnesota Method for editing a genetic sequence
WO2015188191A1 (en) 2014-06-06 2015-12-10 Wong Wilson W Dna recombinase circuits for logical control of gene expression
GB201417042D0 (en) 2014-09-29 2014-11-12 Fkd Therapies Oy Method
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
WO2016118780A1 (en) 2015-01-21 2016-07-28 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
MA41506A (fr) 2015-02-13 2017-12-19 Takeda Vaccines Inc Procédés de production de virus pour produire des vaccins
KR20240119152A (ko) 2015-03-10 2024-08-06 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕 재조합 glut1 아데노-관련 바이러스 벡터 작제물 및 glut1 발현을 회복하기 위한 관련된 방법
WO2016183593A2 (en) 2015-05-14 2016-11-17 The Regents Of The University Of California Prenatal therapy
SG11201803934YA (en) 2015-11-11 2018-06-28 Intrexon Corp Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies
PT3405215T (pt) 2016-01-19 2022-09-20 Univ California Métodos para o tratamento da doença de danon e outros transtornos da autofagia
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
IL261002B2 (en) 2016-02-12 2024-07-01 Bluebird Bio Inc Vcn enhancer compositions and methods of using the same
CN116144500A (zh) 2016-04-14 2023-05-23 崔泽尔有限公司 具有恒流泵/管道系统的固定床生物反应器
HUE055862T2 (hu) * 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
CA3058735A1 (en) 2016-06-13 2017-12-21 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
WO2017218948A2 (en) 2016-06-17 2017-12-21 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential
KR20240151726A (ko) 2016-09-08 2024-10-18 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 판코니 빈혈 환자를 위한 유전자 치료
JP2020500562A (ja) 2016-12-06 2020-01-16 ブルーバード バイオ, インコーポレイテッド ムコ多糖症ii型のための遺伝子治療
BR112019011635A2 (pt) 2016-12-06 2019-11-12 Bluebird Bio Inc terapia genética para mucopolissacaridose, tipo i
US20180326022A1 (en) 2017-04-21 2018-11-15 The Trustees Of The University Of Pennsylvania Compositions and methods for improving heart function and treating heart failure
WO2018201065A1 (en) 2017-04-27 2018-11-01 Fred Hutchinson Cancer Research Center Therapeutic formulations containing cd34+ stem cells derived from negative selection
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
KR102918544B1 (ko) 2018-07-12 2026-01-27 로켓 파마슈티컬스, 리미티드 다논병을 치료하기 위한 유전자 요법 벡터
KR20210046004A (ko) 2018-07-30 2021-04-27 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 조혈 세포의 유전자 변형 방법
BR112021002765A2 (pt) 2018-08-16 2021-07-20 Spacecraft Seven, Llc métodos de produção para vetores virais
BR112021015751A2 (pt) 2019-02-12 2022-02-08 Spacecraft Seven Llc Vetores de terapia genética para tratamento da doença de danon
SG11202112347RA (en) 2019-05-23 2021-12-30 Spacecraft Seven Llc Gene therapy vectors for infantile malignant osteopetrosis
CA3177341A1 (en) 2020-05-20 2021-11-25 Chester Bittencort SACRAMENTO Engineered parkin and uses thereof
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
CN116113700A (zh) 2020-08-05 2023-05-12 太空飞船七有限责任公司 用于glut1表达的腺相关病毒载体及其用途
KR20230042468A (ko) 2020-08-05 2023-03-28 스페이스크래프트 세븐, 엘엘씨 Csrp3 (시스테인 및 글리신 풍부 단백질 3) 유전자 요법
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
CN116887868A (zh) 2020-12-07 2023-10-13 太空飞船七有限责任公司 达农病的治疗
JP2024546103A (ja) 2021-12-08 2024-12-17 スペースクラフト セブン リミテッド ライアビリティ カンパニー Aavベクターを用いたジャンクトフィリン-2(jph2)遺伝子治療
EP4444893A4 (en) 2021-12-10 2026-02-25 Spacecraft Seven Llc GENE THERAPY FOR TROPONIN C (TNNC1) USING AN AAV VECTOR
IL314602A (en) 2022-02-08 2024-09-01 Spacecraft Seven Llc Adeno-associated viral vector for GLUT1 expression and uses thereof
KR20250012749A (ko) 2022-04-22 2025-01-24 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 b-세포 림프종 2-관련 안타노겐 3(bag3) 유전자 요법

Also Published As

Publication number Publication date
IL307996A (en) 2023-12-01
WO2019200167A1 (en) 2019-10-17
JP2021521180A (ja) 2021-08-26
US12539316B2 (en) 2026-02-03
EP3773636A1 (en) 2021-02-17
BR112020020841A2 (pt) 2021-01-19
KR20210003738A (ko) 2021-01-12
JP2025004171A (ja) 2025-01-14
SG11202008400SA (en) 2020-09-29
AU2026200325A1 (en) 2026-04-09
JP7576754B2 (ja) 2024-11-01
AU2019252662A1 (en) 2020-09-24
IL277878A (en) 2020-11-30
US20210154237A1 (en) 2021-05-27
RU2020129103A (ru) 2022-03-03
CA3093708A1 (en) 2019-10-17
CN112153973A (zh) 2020-12-29

Similar Documents

Publication Publication Date Title
MX2020010718A (es) Composiciones y metodos para el trasplante de celulas madre.
SA517390590B1 (ar) Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة
DOP2020000071A (es) Proteínas trispecificas y métodos de uso
MX392977B (es) Composiciones y metodos para programar celulas terapeuticas utilizando nanoportadores de acidos nucleicos dirigidos.
BR112022003021A2 (pt) Modificação de pacotes mensageiros de plantas com lipídeos carregados
CO2020001872A2 (es) Preparación de exosomas terapéuticos mediante el uso de proteínas de membrana
MX2024012897A (es) Proteinas de anticuerpo injertadas con citocina y metodos de uso en el tratamiento del cancer
BR112022005602A2 (pt) Células efetoras de múltiplos direcionamentos e uso das mesmas
PH12018500965A1 (en) Methods and compositions for gene editing in hematopoietic stem cells
CL2017001296A1 (es) Anticuerpos contra cd73 y sus usos
EP4036079A3 (en) Compounds and compositions for intracellular delivery of agents
PE20240950A1 (es) PROTEINAS DE FUSION FC HETERODIMERICAS IL 15/IL 15R(alfa)
MX2019011040A (es) Composiciones y metodos para potenciar la expresion genica.
PE20190563A1 (es) Genes de mini-distrofina optimizados y casetes de expresion y su uso
MX2016001044A (es) Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos.
MX2017008817A (es) Composiciones y metodos para la glicosilacion de proteinas.
FI3186395T4 (fi) Kantasolujen siirto kantasoluihin kohdistuvan aineen ja immunoregulatorisen signaloinnin modulaation yhdistelmällä
PE20160244A1 (es) Anticuerpos contra el inhibidor del activador de plasminogeno tipo 1 (pai-1) y usos de los mismos
EP3939606A3 (en) Improved methods and compounds for eliminating immune responses to therapeutic agents
MX359201B (es) Polipeptidos que se unen al complemento humano 5 (c5).
CL2021001254A1 (es) Vectores vegetales, composiciones y usos relacionados con los mismos
AR081354A1 (es) Metodos y reactivos par una transferencia genica eficaz y dirigida a monocitos y macrofagos
MX2021000322A (es) Enzimas de variante fosfopentomutasa modificada geneticamente.
CL2023003260A1 (es) Composiciones y métodos para el tratamiento de la hipoacusia neurosensorial mediante sistemas de vector dual de estereocilina.
BR112018009732A8 (pt) ?polinucleotídeo, vetor de vírus adeno-associado, partícula de vírus adeno-associado, célula hospedeira, métodos para produzir uma partícula de vírus adeno-associado, para tratar hemofilia a, para transduzir uma célula hospedeira, e, uso de uma partícula de vírus adeno-associado?