CA3093708A1 - Compositions and methods for treating fanconi anemia - Google Patents

Compositions and methods for treating fanconi anemia Download PDF

Info

Publication number
CA3093708A1
CA3093708A1 CA3093708A CA3093708A CA3093708A1 CA 3093708 A1 CA3093708 A1 CA 3093708A1 CA 3093708 A CA3093708 A CA 3093708A CA 3093708 A CA3093708 A CA 3093708A CA 3093708 A1 CA3093708 A1 CA 3093708A1
Authority
CA
Canada
Prior art keywords
stringency
cells
cell population
enriched cell
gene
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3093708A
Other languages
English (en)
French (fr)
Inventor
Brian Beard
Julian SEVILLA NAVARRO
Gaurav Shah
Kinnari PATEL
Raj PRABHAKAR
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Spacecraft Seven LLC
Original Assignee
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Rocket Pharmaceuticals 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 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus, Rocket Pharmaceuticals Inc filed Critical Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Publication of CA3093708A1 publication Critical patent/CA3093708A1/en
Pending legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors 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)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Public Health (AREA)
  • Immunology (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)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
CA3093708A 2018-04-11 2019-04-11 Compositions and methods for treating fanconi anemia Pending CA3093708A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862656292P 2018-04-11 2018-04-11
US62/656,292 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
CA3093708A1 true CA3093708A1 (en) 2019-10-17

Family

ID=68162990

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3093708A Pending CA3093708A1 (en) 2018-04-11 2019-04-11 Compositions and methods for treating fanconi anemia

Country Status (11)

Country Link
US (2) US12539316B2 (cg-RX-API-DMAC10.html)
JP (2) JP7576754B2 (cg-RX-API-DMAC10.html)
KR (1) KR20210003738A (cg-RX-API-DMAC10.html)
CN (1) CN112153973A (cg-RX-API-DMAC10.html)
AU (2) AU2019252662A1 (cg-RX-API-DMAC10.html)
BR (1) BR112020020841A2 (cg-RX-API-DMAC10.html)
CA (1) CA3093708A1 (cg-RX-API-DMAC10.html)
IL (2) IL307996A (cg-RX-API-DMAC10.html)
MX (1) MX2020010718A (cg-RX-API-DMAC10.html)
SG (1) SG11202008400SA (cg-RX-API-DMAC10.html)
WO (1) WO2019200167A1 (cg-RX-API-DMAC10.html)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12539316B2 (en) 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
IL280465B2 (en) 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
MY204234A (en) 2018-08-27 2024-08-16 Regeneron Pharma Use of raman spectroscopy in downstream purification
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
AU2021413252A1 (en) * 2020-12-31 2023-06-08 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
WO2023183909A2 (en) * 2022-03-25 2023-09-28 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
EP0377676B1 (en) 1987-09-17 1995-05-24 Massachusetts Institute Of Technology Human erythroid-specific transcriptional enhancer
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
JP3866760B2 (ja) 1993-02-22 2007-01-10 ザ ロックフェラー ユニバーシティ 一過性トランスフェクションによる高力価のヘルパーフリーのレトロウイルスの生産
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
DE69435223D1 (de) 1993-10-25 2009-09-03 Canji Inc Rekombinanter Adenoviren-Vektor und Verfahren zur Verwendung
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
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
AU2001263006A1 (en) 2000-05-18 2001-11-26 Hyseq, Inc. Novel nucleic acids and polypeptides
US6627442B1 (en) 2000-08-31 2003-09-30 Virxsys Corporation Methods for stable transduction of cells with hiv-derived viral vectors
GB0024550D0 (cg-RX-API-DMAC10.html) 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
CA2462628C (en) 2001-10-02 2019-08-20 Institut Clayton De La Recherche Restricted expression lentiviral vectors
US20060051755A1 (en) 2002-04-15 2006-03-09 Rigel Pharmaceuticals Methods of assaying for cell cycle modulators
ATE446077T1 (de) 2002-05-02 2009-11-15 Univ Rochester Vektoren mit beiden isoformen von beta- hexosaminidase
WO2004094642A2 (en) 2003-04-24 2004-11-04 Fondazione Centro San Raffaele Del Monte Tabor Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof
JP2007054069A (ja) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
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
AU2008262478B9 (en) 2007-05-29 2014-06-19 Aldevron, L.L.C. Vectors and methods for genetic immunization
EP2520643B8 (en) 2007-12-07 2019-11-27 Miltenyi Biotec B.V. & Co. KG Sample processing systems and methods
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
EP2486137B1 (en) 2009-10-05 2018-05-30 Ya-Fang Mei Replication-competent ad11p based viral vectors
RU2638802C2 (ru) 2011-05-16 2017-12-15 Джензим Корпорейшн Применение антагонистов cxcr4
EP3868888A1 (en) 2012-02-29 2021-08-25 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Retroviral transduction using poloxamers
CN116622704A (zh) 2012-07-25 2023-08-22 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
US10280391B2 (en) 2012-12-11 2019-05-07 Pall Technology Uk Limited Recipient for cell cultivation
KR102955556B1 (ko) 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
US20160194660A1 (en) 2012-12-21 2016-07-07 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
CA2947466A1 (en) 2014-05-01 2015-11-05 University Of Washington In vivo gene engineering with adenoviral vectors
WO2015179540A1 (en) 2014-05-20 2015-11-26 Regents Of The University Of Minnesota Method for editing a genetic sequence
US11030531B2 (en) * 2014-06-06 2021-06-08 Trustees Of Boston University 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
US10350245B2 (en) 2015-01-21 2019-07-16 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
MX2017011615A (es) 2015-03-10 2018-04-11 Univ Columbia Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1.
WO2016183593A2 (en) 2015-05-14 2016-11-17 The Regents Of The University Of California Prenatal therapy
WO2017083750A1 (en) 2015-11-11 2017-05-18 Intrexon Corporation Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies
EP3405215B1 (en) 2016-01-19 2022-06-15 The Regents of the University of California Methods for the treatment of danon disease and other disorders of autophagy
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
KR20250134722A (ko) 2016-02-12 2025-09-11 블루버드 바이오, 인코포레이티드. Vcn 인핸서 조성물 및 이의 사용 방법
RS67367B1 (sr) 2016-04-14 2025-11-28 Ferring Ventures Ltd Bioreaktor sa fiksiranim slojem sa pumpom/sistemom creva sa konstantnim protokom
KR102673203B1 (ko) * 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
US20220323612A1 (en) 2016-06-13 2022-10-13 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
US20190345450A1 (en) 2016-06-17 2019-11-14 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential
IL320371A (en) 2016-09-08 2025-06-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Gene therapy for patients with fanconi anemia
CN110199028A (zh) 2016-12-06 2019-09-03 蓝鸟生物公司 用于ii型黏多糖贮积症的基因治疗
MX2019006552A (es) 2016-12-06 2019-10-15 Bluebird Bio Inc Terapia génica para mucopolisacaridosis de 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
EP3615044A4 (en) 2017-04-27 2021-01-20 Fred Hutchinson Cancer Research Center THERAPEUTIC FORMULAS CONTAINING CD34 + STEM CELLS DERIVED FROM NEGATIVE SELECTION
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12539316B2 (en) 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
EP3820537A4 (en) 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
IL280465B2 (en) 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
JP2021533831A (ja) 2018-08-16 2021-12-09 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 ウイルスベクターのための生成方法
US20220143215A1 (en) 2019-02-12 2022-05-12 Spacecraft Seven, Llc Gene therapy vectors for treatment of danon disease
KR20220012266A (ko) 2019-05-23 2022-02-03 스페이스크래프트 세븐, 엘엘씨 영아 악성 골화석증을 위한 유전자 요법 벡터
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
AU2021321412A1 (en) 2020-08-05 2023-04-06 Spacecraft Seven, Llc Adeno-associated viral vector for GLUT1 expression and uses thereof
IL300187A (en) 2020-08-05 2023-03-01 Spacecraft Seven Llc Gene therapy CSRP3 (protein 3 rich in cysteine and glycine)
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
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
CA3240080A1 (en) 2021-12-08 2023-06-15 Christopher Dean HERZOG Junctophilin-2 (jph2) gene therapy using aav vector
CA3240616A1 (en) 2021-12-10 2023-06-15 Christopher Dean HERZOG Troponin c (tnnc1) gene therapy using aav vector
MX2024009685A (es) 2022-02-08 2024-08-19 Spacecraft Seven Llc Vector viral adenoasociado para la expresion de glut1 y usos del mismo.
JP2025512574A (ja) 2022-04-22 2025-04-17 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ベクターを使用するＢ細胞リンパ腫２関連Ａｎｔｈａｎｏｇｅｎｅ ３ （ＢＡＧ３）遺伝子治療

Also Published As

Publication number Publication date
CN112153973A (zh) 2020-12-29
SG11202008400SA (en) 2020-09-29
US20260144823A1 (en) 2026-05-28
AU2019252662A1 (en) 2020-09-24
IL277878A (en) 2020-11-30
AU2026200325A1 (en) 2026-04-09
JP2021521180A (ja) 2021-08-26
WO2019200167A1 (en) 2019-10-17
JP7576754B2 (ja) 2024-11-01
IL307996A (en) 2023-12-01
US20210154237A1 (en) 2021-05-27
RU2020129103A (ru) 2022-03-03
JP2025004171A (ja) 2025-01-14
BR112020020841A2 (pt) 2021-01-19
MX2020010718A (es) 2021-01-20
KR20210003738A (ko) 2021-01-12
EP3773636A1 (en) 2021-02-17
US12539316B2 (en) 2026-02-03

Similar Documents

Publication Publication Date Title
US12539316B2 (en) Compositions and methods for stem cell transplant
JP7664829B2 (ja) 造血細胞の遺伝子改変のための方法
US10828333B2 (en) Point-of-care and/or portable platform for gene therapy
US12496359B2 (en) Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
KR102672636B1 (ko) 판코니 빈혈 환자를 위한 유전자 치료
JP7430202B2 (ja) プラスミドの組合せおよび改変免疫細胞の調製におけるその適用
CN114746546B (zh) 工程化红细胞用于治疗痛风和高尿酸血症
US20240293466A1 (en) Differential cxcr4 expression on hematopoietic progenitor cells versus stem cells directs homing and long-term engraftment
RU2824194C2 (ru) Способы генной модификации гемопоэтических клеток

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220922

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20241223

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD

Year of fee payment: 6

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250219

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20250219

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250219

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20250415

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250415

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20250718

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20250718

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 7TH ANNIV.) - STANDARD

Year of fee payment: 7

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260217

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260217

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20260305

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20260617

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260622