SG11201803701YA - Methods and compositions for gene editing in hematopoietic stem cells - Google Patents
Methods and compositions for gene editing in hematopoietic stem cellsInfo
- Publication number
- SG11201803701YA SG11201803701YA SG11201803701YA SG11201803701YA SG11201803701YA SG 11201803701Y A SG11201803701Y A SG 11201803701YA SG 11201803701Y A SG11201803701Y A SG 11201803701YA SG 11201803701Y A SG11201803701Y A SG 11201803701YA SG 11201803701Y A SG11201803701Y A SG 11201803701YA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- pennsylvania
- hematopoietic stem
- philadelphia
- methods
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 5
- 210000003958 hematopoietic stem cell Anatomy 0.000 title abstract 4
- 239000000203 mixture Substances 0.000 title abstract 3
- 238000010362 genome editing Methods 0.000 title abstract 2
- 235000014036 Castanea Nutrition 0.000 abstract 2
- 241001070941 Castanea Species 0.000 abstract 2
- 108010019670 Chimeric Antigen Receptors Proteins 0.000 abstract 2
- 210000004027 cell Anatomy 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 102220240796 rs553605556 Human genes 0.000 abstract 2
- 210000000130 stem cell Anatomy 0.000 abstract 2
- BHELIUBJHYAEDK-OAIUPTLZSA-N Aspoxicillin Chemical compound C1([C@H](C(=O)N[C@@H]2C(N3[C@H](C(C)(C)S[C@@H]32)C(O)=O)=O)NC(=O)[C@H](N)CC(=O)NC)=CC=C(O)C=C1 BHELIUBJHYAEDK-OAIUPTLZSA-N 0.000 abstract 1
- 206010028980 Neoplasm Diseases 0.000 abstract 1
- 239000000427 antigen Substances 0.000 abstract 1
- 108091007433 antigens Proteins 0.000 abstract 1
- 102000036639 antigens Human genes 0.000 abstract 1
- 230000001363 autoimmune Effects 0.000 abstract 1
- 201000011510 cancer Diseases 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000003394 haemopoietic effect Effects 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 229920001184 polypeptide Polymers 0.000 abstract 1
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 1
- 238000002560 therapeutic procedure Methods 0.000 abstract 1
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
- A61K35/14—Blood; Artificial blood
- A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
-
- 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
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/461—Cellular immunotherapy characterised by the cell type used
- A61K39/4611—T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/463—Cellular immunotherapy characterised by recombinant expression
- A61K39/4631—Chimeric Antigen Receptors [CAR]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
- A61K39/4643—Vertebrate antigens
- A61K39/4644—Cancer antigens
- A61K39/464402—Receptors, cell surface antigens or cell surface determinants
- A61K39/464411—Immunoglobulin superfamily
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/05—Immunological preparations stimulating the reticulo-endothelial system, e.g. against cancer
-
- 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
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
- A61P35/02—Antineoplastic agents specific for leukemia
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/515—Animal cells
- A61K2039/5156—Animal cells expressing foreign proteins
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/515—Animal cells
- A61K2039/5158—Antigen-pulsed cells, e.g. T-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
- 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 [CRISPRs]
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -' Organization International Bureau ires.. 00) (43) International Publication Date ..... ..r ,„,„01 11 May 2017(11.05.2017) WIPO I PCT (10) WO International Publication Number 111111111111311111111111111111111111111111111111111111111111111111111311111111111111111 2017/079400 Al (51) International Patent Classification: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, A61K 35/15 (2015.01) A61K 48/00 (2006.01) BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, A61K 35/17 (2015.01) A61P 35/00 (2006.01) DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, A61K 45/06 (2006.01) A61P 35/02 (2006.01) HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, (21) International Application Number: MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, PCT/US2016/060273 OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, (22) International Filing Date: SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, 3 November 2016 (03.11.2016) TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (25) Filing Language: English (84) Designated States (unless otherwise indicated, for every (26) Publication Language: English kind of regional protection available): ARIPO (BW, GH, (30) Priority Data: GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 62/250,561 4 November 2015 (04.11.2015) US TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, (71) Applicant . THE TRUSTEES OF THE UNIVERSITY DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, OF PENNSYLVANIA [US/US]; 3160 Chestnut Street, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, Suite 200, Philadelphia, Pennsylvania 19104 (US). SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, (72) Inventors: GILL, Saar; 30 North Third Street, Apt. 4A, GW, KM, ML, MR, NE, SN, TD, TG). Philadelphia, Pennsylvania 19106 (US). KIM, Miriam; Declarations under Rule 4.17: 2400 Chestnut Street, Apt. 2604, Philadelphia, of inventorship (Rule 4.17(iv)) Pennsylvania 19103 (US). Published: (74) Agents: DOYLE, Kathryn et al.; Saul Ewing LLP, Centre Square West, 1500 Market Street, 38th Floor, Philadelphia, — with international search report (Art 21(3)) Pennsylvania 19102 (US). — with sequence listing part of description (Rule 5.2(a)) Designated (81) States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (54) Title: METHODS AND COMPOSITIONS FOR GENE EDITING IN HEMATOPOIETIC STEM CELLS A. trunc-gRNAvI trunc-gRNAv2 hum- RNAv3 trunc-gR NAv5 1-1 . .4t i I i CD33n ;40.3 S r x :310 1 0 / 0 it Cr\ IN O ir.... (57) : The present invention relates to compositions and 1-1 c:::, cells. One aspect of the invention includes a modified hematopoietic stem or progenitor cell comprising a nucleic acid capable of de N creasing expression of an endogenous gene or a portion thereof, wherein 0 antigen domain targeted by a chimeric antigen receptor (CAR). Another ,1 . modified hematopoietic stem or progenitor cell. Also included are fled cell for adoptive therapy and treating a condition, such as an autoimmune 1)33neg 364 ^890 , 1 t Figures 1 methods of generating modified hematopoietic stem or progenitor - the endogenous gene encodes a polypeptide comprising an aspect of the invention includes a method for generating a methods and pharmaceutical compositions comprising the modi - disease or cancer.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562250561P | 2015-11-04 | 2015-11-04 | |
PCT/US2016/060273 WO2017079400A1 (en) | 2015-11-04 | 2016-11-03 | Methods and compositions for gene editing in hematopoietic stem cells |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201803701YA true SG11201803701YA (en) | 2018-06-28 |
Family
ID=58662736
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201803701YA SG11201803701YA (en) | 2015-11-04 | 2016-11-03 | Methods and compositions for gene editing in hematopoietic stem cells |
SG10202107602XA SG10202107602XA (en) | 2015-11-04 | 2016-11-03 | Methods and compositions for gene editing in hematopoietic stem cells |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10202107602XA SG10202107602XA (en) | 2015-11-04 | 2016-11-03 | Methods and compositions for gene editing in hematopoietic stem cells |
Country Status (17)
Country | Link |
---|---|
US (3) | US10548922B2 (en) |
EP (1) | EP3370741A4 (en) |
JP (3) | JP6866385B2 (en) |
KR (1) | KR20180073679A (en) |
CN (1) | CN108463227A (en) |
AU (2) | AU2016349280B2 (en) |
BR (1) | BR112018009098A8 (en) |
CA (1) | CA3004053A1 (en) |
CL (1) | CL2018001198A1 (en) |
EA (1) | EA201891092A1 (en) |
HK (1) | HK1259151A1 (en) |
IL (1) | IL259084A (en) |
MX (1) | MX2018005612A (en) |
MY (1) | MY185961A (en) |
PH (1) | PH12018500965A1 (en) |
SG (2) | SG11201803701YA (en) |
WO (1) | WO2017079400A1 (en) |
Families Citing this family (44)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP6261500B2 (en) | 2011-07-22 | 2018-01-17 | プレジデント アンド フェローズ オブ ハーバード カレッジ | Evaluation and improvement of nuclease cleavage specificity |
US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
ES2692206T3 (en) | 2014-11-26 | 2018-11-30 | Miltenyi Biotec Gmbh | Combined immunotherapy of antigen and hematopoietic recognition receptors for the treatment of diseases |
CA3001859A1 (en) | 2015-10-16 | 2017-04-20 | The Trustees Of Columbia University In The City Of New York | Compositions and methods for inhibition of lineage specific antigens |
US20190225955A1 (en) | 2015-10-23 | 2019-07-25 | President And Fellows Of Harvard College | Evolved cas9 proteins for gene editing |
SG11201803701YA (en) * | 2015-11-04 | 2018-06-28 | Univ Pennsylvania | Methods and compositions for gene editing in hematopoietic stem cells |
KR102547316B1 (en) | 2016-08-03 | 2023-06-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | Adenosine nucleobase editing agents and uses thereof |
AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
KR20240007715A (en) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | Aav delivery of nucleobase editors |
IL266362B1 (en) * | 2016-11-02 | 2024-03-01 | Univ Basel | Immunologically discernible cell surface variants for use in cell therapy |
US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
MX2019010196A (en) * | 2017-02-28 | 2019-12-19 | Vor Biopharma Inc | Compositions and methods for inhibition of lineage specific proteins. |
US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
EP3592777A1 (en) | 2017-03-10 | 2020-01-15 | President and Fellows of Harvard College | Cytosine to guanine base editor |
US11268082B2 (en) | 2017-03-23 | 2022-03-08 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
US11795443B2 (en) | 2017-10-16 | 2023-10-24 | The Broad Institute, Inc. | Uses of adenosine base editors |
JP2021523745A (en) | 2018-05-16 | 2021-09-09 | シンテゴ コーポレイション | Methods and systems for guide RNA design and use |
IL260445B (en) * | 2018-07-05 | 2019-08-29 | Lempo Therapeutics Ltd | Methods and compositions for modulating myeloperoxidase (mpo) expression |
KR20220036908A (en) | 2018-08-28 | 2022-03-23 | 보르 바이오파마 인크. | Genetically engineered hematopoietic stem cells and uses thereof |
KR20210061397A (en) * | 2018-09-19 | 2021-05-27 | 후지필름 셀룰러 다이내믹스, 인코포레이티드 | Protein L for activation and expansion of chimeric antigen receptor-modified immune cells |
KR20210105344A (en) * | 2018-10-31 | 2021-08-26 | 휴머니건, 아이엔씨. | Substances and methods for the treatment of cancer |
CN109852627B (en) * | 2018-12-26 | 2022-07-05 | 中国人民解放军军事科学院军事医学研究院 | Plasmid for CRISPR system, construction method thereof and application of plasmid in targeted silencing of target gene of plague bacteria |
AU2020207962A1 (en) * | 2019-01-18 | 2021-07-22 | University Of Southern California | Methods and compositions to improve the safety and efficacy of cellular therapies |
US20220202900A1 (en) * | 2019-02-22 | 2022-06-30 | The Trustees Of The University Of Pennsylvania | Compositions and methods for crispr/cas9 knock-out of cd33 in human hematopoietic stem / progenitor cells for allogenic transplantation in patients with relapsed - refractory acute myeloid leukemia |
CA3130789A1 (en) | 2019-03-07 | 2020-09-10 | The Regents Of The University Of California | Crispr-cas effector polypeptides and methods of use thereof |
CA3130488A1 (en) | 2019-03-19 | 2020-09-24 | David R. Liu | Methods and compositions for editing nucleotide sequences |
CN114787352A (en) * | 2019-08-28 | 2022-07-22 | Vor生物制药股份有限公司 | Compositions and methods for CD123 modification |
EP4022063A1 (en) * | 2019-08-28 | 2022-07-06 | Vor Biopharma Inc. | Compositions and methods for cll1 modification |
KR20210109971A (en) | 2020-02-28 | 2021-09-07 | 현대자동차주식회사 | Manufacturing method of negative electrode for lithium ion battery and lithium ion battery including negative electrode manufactured by the method |
GB2614813A (en) | 2020-05-08 | 2023-07-19 | Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
WO2022228471A1 (en) * | 2021-04-27 | 2022-11-03 | 上海驯鹿生物技术有限公司 | Gene-edited hematopoietic stem cell and combined use thereof with car-t cell |
WO2022232411A2 (en) * | 2021-04-28 | 2022-11-03 | Eisai R&D Mangement Co., Ltd. | Antisense oligonucleotides and their use for treatment of neurodegenerative disorders |
WO2023086422A1 (en) * | 2021-11-09 | 2023-05-19 | Vor Biopharma Inc. | Compositions and methods for erm2 modification |
WO2023137335A2 (en) * | 2022-01-11 | 2023-07-20 | Actinium Pharmaceuticals, Inc. | Methods for treating cd33-positive hematological malignancies |
Family Cites Families (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20100172882A1 (en) * | 2007-01-11 | 2010-07-08 | Glazer Peter M | Compositions and methods for targeted inactivation of hiv cell surface receptors |
AU2013329186B2 (en) * | 2012-10-10 | 2019-02-14 | Sangamo Therapeutics, Inc. | T cell modifying compounds and uses thereof |
US9573988B2 (en) * | 2013-02-20 | 2017-02-21 | Novartis Ag | Effective targeting of primary human leukemia using anti-CD123 chimeric antigen receptor engineered T cells |
AU2014273490B2 (en) * | 2013-05-29 | 2019-05-09 | Cellectis | Methods for engineering T cells for immunotherapy by using RNA-guided Cas nuclease system |
IL280215B (en) * | 2014-04-07 | 2022-07-01 | Novartis Ag | Treatment of cancer using anti-cd19 chimeric antigen receptor |
CA3001859A1 (en) * | 2015-10-16 | 2017-04-20 | The Trustees Of Columbia University In The City Of New York | Compositions and methods for inhibition of lineage specific antigens |
SG11201803701YA (en) * | 2015-11-04 | 2018-06-28 | Univ Pennsylvania | Methods and compositions for gene editing in hematopoietic stem cells |
-
2016
- 2016-11-03 SG SG11201803701YA patent/SG11201803701YA/en unknown
- 2016-11-03 BR BR112018009098A patent/BR112018009098A8/en not_active Application Discontinuation
- 2016-11-03 CN CN201680076538.8A patent/CN108463227A/en active Pending
- 2016-11-03 SG SG10202107602XA patent/SG10202107602XA/en unknown
- 2016-11-03 MX MX2018005612A patent/MX2018005612A/en unknown
- 2016-11-03 CA CA3004053A patent/CA3004053A1/en active Pending
- 2016-11-03 WO PCT/US2016/060273 patent/WO2017079400A1/en active Application Filing
- 2016-11-03 EA EA201891092A patent/EA201891092A1/en unknown
- 2016-11-03 KR KR1020187015530A patent/KR20180073679A/en unknown
- 2016-11-03 AU AU2016349280A patent/AU2016349280B2/en not_active Expired - Fee Related
- 2016-11-03 EP EP16862941.8A patent/EP3370741A4/en active Pending
- 2016-11-03 MY MYPI2018701723A patent/MY185961A/en unknown
- 2016-11-03 JP JP2018543036A patent/JP6866385B2/en active Active
-
2018
- 2018-05-01 IL IL259084A patent/IL259084A/en unknown
- 2018-05-03 CL CL2018001198A patent/CL2018001198A1/en unknown
- 2018-05-03 US US15/970,605 patent/US10548922B2/en active Active
- 2018-05-04 PH PH12018500965A patent/PH12018500965A1/en unknown
-
2019
- 2019-01-23 HK HK19101177.2A patent/HK1259151A1/en unknown
- 2019-12-19 US US16/721,156 patent/US11771719B2/en active Active
-
2021
- 2021-04-07 JP JP2021065361A patent/JP7378439B2/en active Active
-
2023
- 2023-06-14 JP JP2023097365A patent/JP2023115069A/en active Pending
- 2023-09-14 US US18/467,223 patent/US20240066064A1/en active Pending
-
2024
- 2024-01-01 AU AU2024200000A patent/AU2024200000A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
US10548922B2 (en) | 2020-02-04 |
JP2023115069A (en) | 2023-08-18 |
MX2018005612A (en) | 2018-11-09 |
CA3004053A1 (en) | 2017-05-11 |
EP3370741A1 (en) | 2018-09-12 |
CN108463227A (en) | 2018-08-28 |
JP7378439B2 (en) | 2023-11-13 |
BR112018009098A8 (en) | 2019-02-26 |
IL259084A (en) | 2018-06-28 |
EP3370741A4 (en) | 2019-04-24 |
HK1259151A1 (en) | 2019-11-29 |
AU2024200000A1 (en) | 2024-03-21 |
EA201891092A1 (en) | 2018-10-31 |
US20240066064A1 (en) | 2024-02-29 |
US11771719B2 (en) | 2023-10-03 |
AU2016349280A1 (en) | 2018-05-24 |
MY185961A (en) | 2021-06-14 |
AU2016349280B2 (en) | 2023-10-05 |
PH12018500965A1 (en) | 2018-11-12 |
US20180250339A1 (en) | 2018-09-06 |
WO2017079400A1 (en) | 2017-05-11 |
JP2018533625A (en) | 2018-11-15 |
JP2021106595A (en) | 2021-07-29 |
CL2018001198A1 (en) | 2018-09-21 |
BR112018009098A2 (en) | 2018-11-06 |
US20200281975A1 (en) | 2020-09-10 |
SG10202107602XA (en) | 2021-08-30 |
KR20180073679A (en) | 2018-07-02 |
JP6866385B2 (en) | 2021-04-28 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
SG11201803701YA (en) | Methods and compositions for gene editing in hematopoietic stem cells | |
SG11201909949XA (en) | Targeted immunotolerance | |
SG11201908719QA (en) | Biomarkers and car t cell therapies with enhanced efficacy | |
SG11201807489PA (en) | Cells expressing multiple chimeric antigen receptor (car) molecules and uses therefore | |
SG11201908391XA (en) | Methods for modulating an immune response | |
SG11201808622SA (en) | Chimeric receptors to flt3 and methods of use thereof | |
SG11201805268XA (en) | T cell receptors specific for the ny-eso-1 tumor antigen-hla-a*02 complex | |
SG11201908527SA (en) | High affinity mage-a1-specific tcrs and uses thereof | |
SG11201811432WA (en) | Rna for cancer therapy | |
SG11201804375WA (en) | Compositions and methods for internalizing enzymes | |
SG11201805449PA (en) | Methods of making chimeric antigen receptor -expressing cells | |
SG11201900885VA (en) | Treatment of cancer using a chimeric antigen receptor in combination with an inhibitor of a pro-m2 macrophage molecule | |
SG11201900344YA (en) | Treatment and prevention of cytokine release syndrome using a chimeric antigen receptor in combination with a kinase inhibitor | |
SG11201909955XA (en) | Formulations of anti-lag3 antibodies and co-formulations of anti-lag3 antibodies and anti-pd-1 antibodies | |
SG11201805709RA (en) | Anti-pro/latent myostatin antibodies and methods of use thereof | |
SG11201809040YA (en) | Hybrid aerosol-generating element and method for manufacturing a hybrid aerosol-generating element | |
SG11201805792PA (en) | Chimeric proteins and methods of regulating gene expression | |
SG11201908624SA (en) | Aryl hydrocarbon receptor antagonists and uses thereof | |
SG11201805451TA (en) | Immune effector cell therapies with enhanced efficacy | |
SG11201806988UA (en) | Improved differentiation method | |
SG11201804161VA (en) | Compositions comprising bacterial strains | |
SG11201407580YA (en) | Composition comprising two antibodies engineered to have reduced and increased effector function | |
SG11201900772YA (en) | Gene editing of car-t cells for the treatment of t cell malignancies with chimeric antigen receptors | |
SG11201805645QA (en) | Lsd1 inhibitors | |
SG11201407561UA (en) | ANTI-Ly6E ANTIBODIES AND IMMUNOCONJUGATES AND METHODS OF USE |