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Применить Всего найдено 15. Отображено 15.
09-01-2020 дата публикации

IN VIVO GENE THERAPY FOR IMMUNE DEFICIENCIES

Номер: US20200009266A1
Автор: Adair Jennifer E., Chan Frieda, Humbert Olivier, Kiem Hans-Peter, Rawlings David, Scharenberg Andrew, Torgerson Troy
Принадлежит:

In vivo gene therapies for immune deficiencies are described. The in vivo gene therapies utilize a foamy viral vector including a PGK promoter with a therapeutic gene. The foamy viral vector can be beneficially administered with cell mobilization into the peripheral blood. 1. A method of treating X-linked severe combined immunodeficiency (SCID-X1) in a subject in need thereof comprising administering a therapeutically effective amount of (i) a formulation comprising a foamy viral vector comprising a PGK promoter associated with a sequence encoding a γC protein; (ii) G-CSF; and (iii) AMD3100 , thereby treating SCID-X1 in the subject in need thereof.2. The method of claim 1 , wherein the foamy viral vector comprises a sequence selected from SEQ ID NOs: 1-3 claim 1 , 26 claim 1 , and 28-30.3. The method of claim 1 , wherein the foamy viral vector comprises a sequence encoding a sequence selected from SEQ ID NOs: 4 claim 1 , 5 claim 1 , and 27.4. The method of claim 1 , wherein the γC protein restores functionality to a γC-dependent signaling pathway.5. The method of claim 4 , wherein the functionality of a γC-dependent signaling pathway is determined by measuring tyrosine phosphorylation of STAT3 and/or STAT5 in cells from the subject following in vitro stimulation with IL-21 and IL-2 claim 4 , respectively.6. The method of claim 5 , wherein the tyrosine phosphorylation of STAT3 and/or STAT5 is measured by intracellular antibody staining.7. The method of claim 5 , wherein the cells are peripheral blood mononuclear cells (PBMCs).8. The method of claim 1 , wherein the therapeutically effective amount of the foamy viral vector is 1×10to 10×10infection units (IU).9. The method of claim 1 , wherein the therapeutically effective amount of G-CSF is 1 μg/kg to 10 μg/kg.10. The method of claim 1 , wherein the therapeutically effective amount of AMD3100 is 1 mg/kg to 10 mg/kg.11. The method of claim 1 , wherein the therapeutically effective amount of G-CSF is administered prior ...

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Номер записи: 1
25-02-2021 дата публикации

POINT-OF-CARE AND/OR PORTABLE PLATFORM FOR GENE THERAPY

Номер: US20210052658A1
Автор: Adair Jennifer E., Kiem Hans-Peter
Принадлежит: Fred Hutchinson Cancer Research Center

A platform for ex vivo isolation, production, and formulation of genetically-modified cells is described. The platform utilizes a software-enabled point-of-care and/or portable device making gene therapy more widely available. 180.-. (canceled)81. A method performed by a device , the method comprising:receiving, by a chamber, cells and a genetic modifier wherein the receiving results in genetic modification of the cells to produce genetically-modified cells within the chamber,centrifuging the genetically-modified cells within the chamber to create a pelleted cell suspension;removing, from the chamber, a supernatant volume from the pelleted cell suspension;diluting the pelleted cell suspension with a media comprising a pharmaceutically acceptable carrier within the chamber to create a diluted pelleted cell suspension;centrifuging the diluted pelleted cell suspension within the chamber and removing an additional volume of supernatant to form a cell product formulation; andtransferring the cell product formulation into a sterile receptacle through a conduit connected to the chamber.82. The method of claim 81 , wherein the sterile receptacle is sterilely welded to the conduit.83. The method of claim 81 , further comprising administering the transferred cell product formulation to a subject from the sterile receptacle.84. The method of claim 81 , wherein the cells comprise hematopoietic stem cells (HSC) claim 81 , hematopoietic progenitor cells (HPC) claim 81 , hematopoietic stem and progenitor cells (HSPC) claim 81 , T cells claim 81 , natural killer cells claim 81 , B cells claim 81 , macrophages claim 81 , monocytes claim 81 , mesenchymal stem cells (MSC) claim 81 , white blood cells (WBC) claim 81 , mononuclear cells (MNC) claim 81 , endothelial cells (EC) claim 81 , stromal cells claim 81 , or bone marrow fibroblasts.85. The method of claim 81 , wherein the cells comprise CD34+ HSPC.86. The method of claim 81 , wherein the genetic modifier comprises a non- ...

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Номер записи: 2
10-06-2021 дата публикации

GENOMIC SAFE HARBORS FOR GENETIC THERAPIES IN HUMAN STEM CELLS AND ENGINEERED NANOPARTICLES TO PROVIDE TARGETED GENETIC THERAPIES

Номер: US20210171983A1
Автор: Adair Jennifer E., SHAHBAZI Reza
Принадлежит: Fred Hutchinson Cancer Research Center

Genomic safe harbors (GSH) for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies are described. The GSH and/or associated nanoparticles can be used to safely and efficiently treat a variety of genetic, infectious, and malignant diseases. 1. A method of genetically modifying a blood cell through homology-directed repair (HDR) within a universal hematopoietic stem cell (HSC) safe harbor loci of SEQ ID NO: 132 wherein the method comprises: the first layer comprises a crRNA with a 3′ end and a 5′ end, wherein the 3′ end is conjugated to a spacer with a thiol modification, and the 5′ end is conjugated to Cpf1, and wherein the thiol modification is covalently linked to the surface of the AuNP and wherein the crRNA comprises SEQ ID NO: 195; and', 'the second layer comprises a donor template comprising a therapeutic gene and homology-directed repair templates; and wherein the second layer is farther from the surface of the AuNP than the first layer, 'contacting the blood cell with a gold nanoparticle (AuNP) associated with at least two active layers wherein'}thereby genetically modifying the blood cell through HDR within a universal HSC safe harbor loci of SEQ ID NO: 132.2. A gold nanoparticle (AuNP) associated with two active layers whereinthe first layer comprises a crRNA with a 3′ end and a 5′ end, wherein the 3′ end is conjugated to a spacer with a thiol modification and the 5′ end is conjugated to Cpf1, and wherein the thiol modification is covalently linked to the surface of the AuNP and wherein the crRNA comprises SEQ ID NO: 195; andthe second layer comprises a donor template comprising a therapeutic gene and homology-directed repair templates; and wherein the second layer is farther from the surface of the AuNP than the first layer.3. A method of genetically modifying a blood cell at a target sequence within chromosome 11 wherein the target sequence comprises SEQ ID NOs. 1-194 , 197-208 , 210 , 213 , 242 , 245 , ...

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Номер записи: 3
21-06-2018 дата публикации

POINT-OF-CARE AND/OR PORTABLE PLATFORM FOR GENE THERAPY

Номер: US20180169148A1
Автор: Adair Jennifer E., Kiem Hans-Peter
Принадлежит:

A platform for ex vivo isolation, production, and formulation of genetically-modified cells is described. The platform utilizes a software-enabled point-of-care and/or portable device making gene therapy more widely available. 1. A method of isolating , genetically-modifying , and formulating target cells obtained from a subject sample , the method comprising the steps of: a circuit for processing the subject sample, the circuit comprising a sample input, a buffer input, and a treatment chamber;', 'a plurality of valves for selectively closing one or more flow paths of the circuit;', 'one or more target cell selector(s) for separating the target cells of the subject sample from non-target cells of the subject sample;', 'at least one pump to perfuse the subject sample through at least a portion of the circuit;', 'instructions executable by one or more processors to control operation of the treatment chamber, the plurality of valves, the target cell selector(s), and the pump; and, '(i) loading the subject sample into a point-of-care and/or portable device comprising transferring the subject sample from the treatment chamber to the target cell selector(s);', 'separating the target cells from the non-target cells via operation of the target cell selector(s) simultaneously or in tandem if more than one selector is used;', 'transferring the target cells back into to the treatment chamber;', 'introducing a genetic modifier to the target cells within the treatment chamber and/or the target cell selector(s) to generate genetically-modified target cells; and', 'formulating the genetically-modified target cells into a formulation for administration to a subject., '(ii) initiating execution of the instructions by the one or more processors to cause at least one of the plurality of valves, the one or more target cell selectors, or the at least one pump to perform acts comprising2. (canceled)3. (canceled)4. (canceled)5. A method of further comprising determining an initial volume ...

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Номер записи: 4
25-06-2020 дата публикации

Therapeutic formulations containing cd34+ stem cells derived from negative selection

Номер: US20200199534A1
Автор: Hans-Peter Kiem, Jennifer E. ADAIR
Принадлежит: Fred Hutchinson Cancer Research Center

Therapeutic formulations containing CD34+ stem cells derived from negative selection are described. The cells within the formulations can be genetically-modified for a number of therapeutic purposes. The disclosure is particularly useful for the treatment of patients with fragile stem cells or stem cells with low CD34+ expression levels.

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Номер записи: 5
14-11-2019 дата публикации

STRATEGIES TO ASSESS AND/OR PRODUCE CELL POPULATIONS WITH PREDICTIVE ENGRAFTMENT POTENTIAL

Номер: US20190345450A1
Автор: Adair Jennifer E., Kiem Hans-Peter, Radtke Stefan
Принадлежит: Fred Hutchinson Cancer Research Center

Strategies to assess and/or produce cell populations with predictive engraftment potential are described. The cell populations can be used for a variety of therapeutic and research purposes. 1238.-. (canceled)239. A method of sorting a stem cell population comprisingobtaining a biological sample comprising stem cells;enriching the biological sample for CD34+ cells to create a CD34+ enriched sample; andisolating CD45RA− and CD90+ cells from the CD34+ enriched sample thereby sorting the stem cell population to create a CD34+/CD45RA−/CD90+ stem cell population.240. The method of claim 239 , further comprising isolating CD133+ cells from the CD34+ enriched sample thereby sorting the stem cell population to create a CD34+/CD45RA−/CD90+/CD133+ stem cell population.241. The method of claim 239 , further comprising isolating CD117+ cells from the CD34+ enriched sample thereby sorting the stem cell population to create a CD34+/CD45RA−/CD90+/CD117+ stem cell population.242. The method of claim 239 , wherein the isolating does not utilize markers other than CD34 claim 239 , CD45RA claim 239 , and CD90 to sort the stem cell population.243. The method of claim 240 , wherein the isolating does not utilize markers other than CD34 claim 240 , CD45RA claim 240 , CD90 claim 240 , and CD133 to sort the stem cell population.244. The method of claim 241 , wherein the isolating does not utilize markers other than CD34 claim 241 , CD45RA claim 241 , CD90 claim 241 , and CD117 to sort the stem cell population.245. The method of claim 239 , wherein the sorting does not utilize CD38 or CD49f to sort the stem cell population.246. The method of claim 239 , wherein the isolating does not utilize CD3 claim 239 , CD7 claim 239 , CD10 claim 239 , CD13 claim 239 , CD14 claim 239 , CD33 claim 239 , CD38 claim 239 , CD41 claim 239 , CD56 claim 239 , CD105 claim 239 , CD127 claim 239 , CD135 claim 239 , and CD138 to sort the stem cell population.247. The method of claim 239 , wherein the biological ...

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Номер записи: 6
05-12-2019 дата публикации

POINT-OF-CARE AND/OR PORTABLE PLATFORM FOR GENE THERAPY

Номер: US20190365817A1
Автор: Adair Jennifer E., Kiem Hans-Peter
Принадлежит: Fred Hutchinson Cancer Research Center

A platform for ex vivo isolation, production, and formulation of genetically-modified cells is described. The platform utilizes a software-enabled point-of-care and/or portable device making gene therapy more widely available. 180.-. (canceled)81. A method performed by a device , the method comprising:receiving, by a treatment chamber, a subject sample comprising at least one first cell type and at least one second cell type;transferring the subject sample from the treatment chamber to at least one cell type separator;separating the at least one first cell type in the subject sample by allowing the at least one second cell type to pass through the cell type separator and retaining the at least one first cell type within the at least one cell type separator;transferring the at least one first cell type into the treatment chamber;introducing a genetic modifier to the at least one first cell type to generate genetically-modified cells;pelleting the genetically-modified cells within the treatment chamber through centrifugation to create a pelleted cell suspension;removing a specified supernatant volume from the pelleted cell suspension through at least one first conduit connected to the treatment chamber;diluting the pelleted cell suspension with media comprising a pharmaceutically acceptable carrier;centrifuging the diluted pelleted cell suspension and removing an additional volume of supernatant in the treatment chamber through the at least one first conduit to form a final cell product formulation; andtransferring the final cell product formulation into one or more sterile receptacles through a second conduit connected to the treatment chamber wherein the final cell product formulation is ready for administration to a subject upon completion of transfer into the one or more sterile receptacles.84. The method of claim 81 , wherein the introducing of the genetic modifier inserts or alters a gene selected from ABCD1 claim 81 , ABCA3 claim 81 , ABLI claim 81 , ADA claim ...

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Номер записи: 7
11-06-2020 дата публикации

Reduced and minimal manipulation manufacturing of genetically-modified cells

Номер: WO2020118110A1
Автор: Jennifer E. ADAIR, Reza SHAHBAZI
Принадлежит: Fred Hutchinson Cancer Research Center

Nanoparticles to genetically modify selected cell types within a biological sample that has been subjected to reduced or minimal manipulation are described. The nanoparticles deliver all components required for precise genome engineering and overcome numerous drawbacks associated with current clinical practices to genetically engineer cells for therapeutic purposes.

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Номер записи: 8
28-07-2016 дата публикации

Point-of-care and/or portable platform for gene therapy

Номер: WO2016118780A1
Автор: Hans-Peter Kiem, Jennifer E. ADAIR
Принадлежит: Fred Hutchinson Cancer Research Center

A platform for ex vivo isolation, production, and formulation of genetically-modified cells is described. The platform utilizes a software-enabled point-of-care and/or portable device making gene therapy more widely available.

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Номер записи: 9
13-12-2018 дата публикации

Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies

Номер: CA3102054A1
Автор: Jennifer E. ADAIR, Reza SHAHBAZI
Принадлежит: Fred Hutchinson Cancer Research Center

Genomic safe harbors (GSH) for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies are described. The GSH and/or associated nanoparticles can be used to safely and efficiently treat a variety of genetic, infectious, and malignant diseases.

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Номер записи: 10
08-02-2018 дата публикации

Strategies to assess and/or produce cell populations with predictive engraftment potential

Номер: WO2017218948A3
Автор: Hans-Peter Kiem, Jennifer E. ADAIR, Stefan RADTKE
Принадлежит: Fred Hutchinson Cancer Research Center

Strategies to assess and/or produce cell populations with predictive engraftment potential are described. The cell populations can be used for a variety of therapeutic and research purposes.

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Номер записи: 11
04-03-2020 дата публикации

Therapeutic formulations containing cd34+ stem cells derived from negative selection

Номер: EP3615044A1
Автор: Hans-Peter Kiem, Jennifer E. ADAIR
Принадлежит: Fred Hutchinson Cancer Research Center

Therapeutic formulations containing CD34+ stem cells derived from negative selection are described. The cells within the formulations can be genetically-modified for a number of therapeutic purposes. The disclosure is particularly useful for the treatment of patients with fragile stem cells or stem cells with low CD34+ expression levels.

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Номер записи: 12
07-09-2023 дата публикации

Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies

Номер: US20230279441A1
Автор: Jennifer E. ADAIR, Reza SHAHBAZI
Принадлежит: Fred Hutchinson Cancer Research Center

Genomic safe harbors (GSH) for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies are described. The GSH and/or associated nanoparticles can be used to safely and efficiently treat a variety of genetic, infectious, and malignant diseases.

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Номер записи: 13
25-04-2024 дата публикации

Systems and methods for gene therapy

Номер: WO2024086595A2
Автор: Jennifer E. ADAIR, Lois BAYIGGA
Принадлежит: Fred Hutchinson Cancer Center

Systems and methods to simplify ex vivo gene therapy are described. The systems and methods provide ex vivo manufacturing of cells using target-cell enriching magnetic beads, vector-magnetic bead complexes, and a magnetic field. The present disclosure reduces the amount of required vector to 10 infectious particles per cell and does not require the use of transduction culture or cytokines. Cell manufacturing can be completed within one day, such that treated subjects do not require chemotherapy between cell collection and re-administration.

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Номер записи: 14
06-06-2024 дата публикации

Systems and methods for gene therapy

Номер: WO2024086595A3
Автор: Jennifer E. ADAIR, Lois BAYIGGA
Принадлежит: Fred Hutchinson Cancer Center

Systems and methods to simplify ex vivo gene therapy are described. The systems and methods provide ex vivo manufacturing of cells using target-cell enriching magnetic beads, vector-magnetic bead complexes, and a magnetic field. The present disclosure reduces the amount of required vector to 10 infectious particles per cell and does not require the use of transduction culture or cytokines. Cell manufacturing can be completed within one day, such that treated subjects do not require chemotherapy between cell collection and re-administration.

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Номер записи: 15