CA2945335A1 - Crispr-cas-related methods, compositions and components for cancer immunotherapy - Google Patents
Crispr-cas-related methods, compositions and components for cancer immunotherapy Download PDFInfo
- Publication number
- CA2945335A1 CA2945335A1 CA2945335A CA2945335A CA2945335A1 CA 2945335 A1 CA2945335 A1 CA 2945335A1 CA 2945335 A CA2945335 A CA 2945335A CA 2945335 A CA2945335 A CA 2945335A CA 2945335 A1 CA2945335 A1 CA 2945335A1
- Authority
- CA
- Canada
- Prior art keywords
- tables
- nucleotides
- domain
- nucleic acid
- grna
- 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
Links
- 238000000034 method Methods 0.000 title claims abstract description 164
- 239000000203 mixture Substances 0.000 title claims abstract description 54
- 238000002619 cancer immunotherapy Methods 0.000 title description 5
- 108020005004 Guide RNA Proteins 0.000 claims abstract description 555
- 230000008685 targeting Effects 0.000 claims abstract description 434
- 108090000623 proteins and genes Proteins 0.000 claims abstract description 390
- 210000001744 T-lymphocyte Anatomy 0.000 claims abstract description 304
- 101000617285 Homo sapiens Tyrosine-protein phosphatase non-receptor type 6 Proteins 0.000 claims abstract description 238
- 108091033409 CRISPR Proteins 0.000 claims abstract description 233
- 102100039498 Cytotoxic T-lymphocyte protein 4 Human genes 0.000 claims abstract description 232
- 102100035273 E3 ubiquitin-protein ligase CBL-B Human genes 0.000 claims abstract description 231
- 101000889276 Homo sapiens Cytotoxic T-lymphocyte protein 4 Proteins 0.000 claims abstract description 231
- 101000737265 Homo sapiens E3 ubiquitin-protein ligase CBL-B Proteins 0.000 claims abstract description 231
- 101710153660 Nuclear receptor corepressor 2 Proteins 0.000 claims abstract description 215
- 102100021657 Tyrosine-protein phosphatase non-receptor type 6 Human genes 0.000 claims abstract description 199
- 102100040678 Programmed cell death protein 1 Human genes 0.000 claims abstract description 194
- 101000611936 Homo sapiens Programmed cell death protein 1 Proteins 0.000 claims abstract description 193
- 230000000295 complement effect Effects 0.000 claims abstract description 82
- 206010028980 Neoplasm Diseases 0.000 claims abstract description 51
- 238000003776 cleavage reaction Methods 0.000 claims abstract description 48
- 230000007017 scission Effects 0.000 claims abstract description 48
- 201000011510 cancer Diseases 0.000 claims abstract description 31
- 108010019670 Chimeric Antigen Receptors Proteins 0.000 claims abstract description 28
- 102100029452 T cell receptor alpha chain constant Human genes 0.000 claims abstract 30
- 125000003729 nucleotide group Chemical group 0.000 claims description 460
- 239000002773 nucleotide Substances 0.000 claims description 458
- 150000007523 nucleic acids Chemical class 0.000 claims description 335
- 102000039446 nucleic acids Human genes 0.000 claims description 311
- 108020004707 nucleic acids Proteins 0.000 claims description 311
- 210000004027 cell Anatomy 0.000 claims description 124
- 230000004048 modification Effects 0.000 claims description 102
- 238000012986 modification Methods 0.000 claims description 102
- 230000005783 single-strand break Effects 0.000 claims description 65
- 230000005782 double-strand break Effects 0.000 claims description 52
- 239000013607 AAV vector Substances 0.000 claims description 46
- 230000035772 mutation Effects 0.000 claims description 43
- 102000004169 proteins and genes Human genes 0.000 claims description 33
- 238000013518 transcription Methods 0.000 claims description 32
- 108020001507 fusion proteins Proteins 0.000 claims description 29
- 102000037865 fusion proteins Human genes 0.000 claims description 29
- 108091008874 T cell receptors Proteins 0.000 claims description 27
- 102000016266 T-Cell Antigen Receptors Human genes 0.000 claims description 26
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 24
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 24
- 230000000694 effects Effects 0.000 claims description 24
- 101710163270 Nuclease Proteins 0.000 claims description 22
- 108700039691 Genetic Promoter Regions Proteins 0.000 claims description 20
- 230000001939 inductive effect Effects 0.000 claims description 18
- 230000008901 benefit Effects 0.000 claims description 16
- 230000002829 reductive effect Effects 0.000 claims description 15
- 230000006698 induction Effects 0.000 claims description 8
- 208000006265 Renal cell carcinoma Diseases 0.000 claims description 6
- 208000025324 B-cell acute lymphoblastic leukemia Diseases 0.000 claims description 5
- 208000024893 Acute lymphoblastic leukemia Diseases 0.000 claims description 4
- 208000014697 Acute lymphocytic leukaemia Diseases 0.000 claims description 4
- 206010009944 Colon cancer Diseases 0.000 claims description 4
- 208000001333 Colorectal Neoplasms Diseases 0.000 claims description 4
- 208000031671 Large B-Cell Diffuse Lymphoma Diseases 0.000 claims description 4
- 206010025323 Lymphomas Diseases 0.000 claims description 4
- 208000015914 Non-Hodgkin lymphomas Diseases 0.000 claims description 4
- 208000006664 Precursor Cell Lymphoblastic Leukemia-Lymphoma Diseases 0.000 claims description 4
- 238000012163 sequencing technique Methods 0.000 claims description 4
- 208000010839 B-cell chronic lymphocytic leukemia Diseases 0.000 claims description 3
- 206010006187 Breast cancer Diseases 0.000 claims description 3
- 208000026310 Breast neoplasm Diseases 0.000 claims description 3
- 102000004127 Cytokines Human genes 0.000 claims description 3
- 108090000695 Cytokines Proteins 0.000 claims description 3
- 206010029260 Neuroblastoma Diseases 0.000 claims description 3
- 206010033128 Ovarian cancer Diseases 0.000 claims description 3
- 206010061535 Ovarian neoplasm Diseases 0.000 claims description 3
- 206010060862 Prostate cancer Diseases 0.000 claims description 3
- 208000000236 Prostatic Neoplasms Diseases 0.000 claims description 3
- 201000001441 melanoma Diseases 0.000 claims description 3
- 239000011541 reaction mixture Substances 0.000 claims description 3
- 208000031261 Acute myeloid leukaemia Diseases 0.000 claims description 2
- 206010003571 Astrocytoma Diseases 0.000 claims description 2
- 208000000461 Esophageal Neoplasms Diseases 0.000 claims description 2
- 206010058467 Lung neoplasm malignant Diseases 0.000 claims description 2
- 208000000172 Medulloblastoma Diseases 0.000 claims description 2
- 206010027406 Mesothelioma Diseases 0.000 claims description 2
- 208000034578 Multiple myelomas Diseases 0.000 claims description 2
- 208000033776 Myeloid Acute Leukemia Diseases 0.000 claims description 2
- 206010030155 Oesophageal carcinoma Diseases 0.000 claims description 2
- 206010061902 Pancreatic neoplasm Diseases 0.000 claims description 2
- 206010035226 Plasma cell myeloma Diseases 0.000 claims description 2
- 206010039491 Sarcoma Diseases 0.000 claims description 2
- 201000004101 esophageal cancer Diseases 0.000 claims description 2
- 201000010536 head and neck cancer Diseases 0.000 claims description 2
- 208000014829 head and neck neoplasm Diseases 0.000 claims description 2
- 206010073071 hepatocellular carcinoma Diseases 0.000 claims description 2
- 231100000844 hepatocellular carcinoma Toxicity 0.000 claims description 2
- 201000005202 lung cancer Diseases 0.000 claims description 2
- 208000020816 lung neoplasm Diseases 0.000 claims description 2
- 208000015486 malignant pancreatic neoplasm Diseases 0.000 claims description 2
- 201000002528 pancreatic cancer Diseases 0.000 claims description 2
- 208000008443 pancreatic carcinoma Diseases 0.000 claims description 2
- 230000004927 fusion Effects 0.000 claims 2
- 230000015788 innate immune response Effects 0.000 claims 2
- 241000237519 Bivalvia Species 0.000 claims 1
- 235000020639 clam Nutrition 0.000 claims 1
- 238000010453 CRISPR/Cas method Methods 0.000 abstract description 8
- 230000001225 therapeutic effect Effects 0.000 abstract description 3
- 108091007741 Chimeric antigen receptor T cells Proteins 0.000 abstract 1
- 102100030569 Nuclear receptor corepressor 2 Human genes 0.000 description 176
- 239000013598 vector Substances 0.000 description 76
- 108700026244 Open Reading Frames Proteins 0.000 description 49
- -1 PD CD] Proteins 0.000 description 47
- 238000003197 gene knockdown Methods 0.000 description 44
- 230000006780 non-homologous end joining Effects 0.000 description 41
- 230000004075 alteration Effects 0.000 description 40
- 108091026890 Coding region Proteins 0.000 description 34
- 230000001404 mediated effect Effects 0.000 description 33
- 241000193996 Streptococcus pyogenes Species 0.000 description 32
- 238000013459 approach Methods 0.000 description 29
- 241000894007 species Species 0.000 description 27
- 230000035897 transcription Effects 0.000 description 25
- 108091028043 Nucleic acid sequence Proteins 0.000 description 24
- 238000012217 deletion Methods 0.000 description 16
- 230000037430 deletion Effects 0.000 description 16
- 238000011144 upstream manufacturing Methods 0.000 description 16
- 230000009467 reduction Effects 0.000 description 15
- 108091081024 Start codon Proteins 0.000 description 14
- 230000006052 T cell proliferation Effects 0.000 description 14
- 150000001413 amino acids Chemical class 0.000 description 14
- 230000001580 bacterial effect Effects 0.000 description 14
- 230000008030 elimination Effects 0.000 description 14
- 238000003379 elimination reaction Methods 0.000 description 14
- 239000000427 antigen Substances 0.000 description 12
- 108091007433 antigens Proteins 0.000 description 12
- 102000036639 antigens Human genes 0.000 description 12
- 239000013603 viral vector Substances 0.000 description 12
- 108020004414 DNA Proteins 0.000 description 11
- 101150064015 FAS gene Proteins 0.000 description 11
- 201000010099 disease Diseases 0.000 description 11
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 11
- 230000004083 survival effect Effects 0.000 description 11
- 238000012546 transfer Methods 0.000 description 11
- 108091023043 Alu Element Proteins 0.000 description 10
- 101100519206 Homo sapiens PDCD1 gene Proteins 0.000 description 10
- 101150087384 PDCD1 gene Proteins 0.000 description 10
- 238000003556 assay Methods 0.000 description 10
- 210000000349 chromosome Anatomy 0.000 description 10
- 101150056981 BID gene Proteins 0.000 description 9
- 241000194020 Streptococcus thermophilus Species 0.000 description 9
- 108020004999 messenger RNA Proteins 0.000 description 9
- 238000012360 testing method Methods 0.000 description 9
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 8
- 102000017420 CD3 protein, epsilon/gamma/delta subunit Human genes 0.000 description 8
- 101150116845 Cblb gene Proteins 0.000 description 8
- 101150091887 Ctla4 gene Proteins 0.000 description 8
- 230000035755 proliferation Effects 0.000 description 8
- 108010077544 Chromatin Proteins 0.000 description 7
- 230000000259 anti-tumor effect Effects 0.000 description 7
- 230000015556 catabolic process Effects 0.000 description 7
- 230000003915 cell function Effects 0.000 description 7
- 210000003483 chromatin Anatomy 0.000 description 7
- 238000006731 degradation reaction Methods 0.000 description 7
- 230000006870 function Effects 0.000 description 7
- 230000002163 immunogen Effects 0.000 description 7
- 108700028369 Alleles Proteins 0.000 description 6
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 6
- 238000006640 acetylation reaction Methods 0.000 description 6
- 238000004520 electroporation Methods 0.000 description 6
- 238000002474 experimental method Methods 0.000 description 6
- 238000003780 insertion Methods 0.000 description 6
- 230000037431 insertion Effects 0.000 description 6
- 230000011987 methylation Effects 0.000 description 6
- 238000007069 methylation reaction Methods 0.000 description 6
- 239000013612 plasmid Substances 0.000 description 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 description 6
- 210000004881 tumor cell Anatomy 0.000 description 6
- 230000033540 T cell apoptotic process Effects 0.000 description 5
- 210000004369 blood Anatomy 0.000 description 5
- 239000008280 blood Substances 0.000 description 5
- 238000011002 quantification Methods 0.000 description 5
- 230000004044 response Effects 0.000 description 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 4
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 4
- 241000588650 Neisseria meningitidis Species 0.000 description 4
- 125000000539 amino acid group Chemical group 0.000 description 4
- 239000012830 cancer therapeutic Substances 0.000 description 4
- 230000000415 inactivating effect Effects 0.000 description 4
- 230000002401 inhibitory effect Effects 0.000 description 4
- 230000000977 initiatory effect Effects 0.000 description 4
- 230000000670 limiting effect Effects 0.000 description 4
- 230000008520 organization Effects 0.000 description 4
- 230000008439 repair process Effects 0.000 description 4
- 102100035102 E3 ubiquitin-protein ligase MYCBP2 Human genes 0.000 description 3
- 101000662902 Homo sapiens T cell receptor beta constant 2 Proteins 0.000 description 3
- 108700018351 Major Histocompatibility Complex Proteins 0.000 description 3
- 230000006044 T cell activation Effects 0.000 description 3
- 102100037298 T cell receptor beta constant 2 Human genes 0.000 description 3
- 125000003275 alpha amino acid group Chemical group 0.000 description 3
- 238000000137 annealing Methods 0.000 description 3
- 231100000433 cytotoxic Toxicity 0.000 description 3
- 230000001472 cytotoxic effect Effects 0.000 description 3
- 239000003623 enhancer Substances 0.000 description 3
- 238000001943 fluorescence-activated cell sorting Methods 0.000 description 3
- 238000005304 joining Methods 0.000 description 3
- 238000004519 manufacturing process Methods 0.000 description 3
- 239000000463 material Substances 0.000 description 3
- 230000004962 physiological condition Effects 0.000 description 3
- 229920002401 polyacrylamide Polymers 0.000 description 3
- 102000004196 processed proteins & peptides Human genes 0.000 description 3
- 108090000765 processed proteins & peptides Proteins 0.000 description 3
- 230000020382 suppression by virus of host antigen processing and presentation of peptide antigen via MHC class I Effects 0.000 description 3
- 229940035893 uracil Drugs 0.000 description 3
- 108020005345 3' Untranslated Regions Proteins 0.000 description 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 2
- 108090000672 Annexin A5 Proteins 0.000 description 2
- 102000004121 Annexin A5 Human genes 0.000 description 2
- 238000010356 CRISPR-Cas9 genome editing Methods 0.000 description 2
- 206010011968 Decreased immune responsiveness Diseases 0.000 description 2
- 241000702421 Dependoparvovirus Species 0.000 description 2
- 208000002250 Hematologic Neoplasms Diseases 0.000 description 2
- 101001000998 Homo sapiens Protein phosphatase 1 regulatory subunit 12C Proteins 0.000 description 2
- 241000124008 Mammalia Species 0.000 description 2
- 108091092724 Noncoding DNA Proteins 0.000 description 2
- 102100035620 Protein phosphatase 1 regulatory subunit 12C Human genes 0.000 description 2
- 210000000173 T-lymphoid precursor cell Anatomy 0.000 description 2
- 230000015572 biosynthetic process Effects 0.000 description 2
- 230000001419 dependent effect Effects 0.000 description 2
- 239000003814 drug Substances 0.000 description 2
- 229940079593 drug Drugs 0.000 description 2
- 238000010362 genome editing Methods 0.000 description 2
- 210000002865 immune cell Anatomy 0.000 description 2
- 230000006028 immune-suppresssive effect Effects 0.000 description 2
- 238000009169 immunotherapy Methods 0.000 description 2
- 238000000338 in vitro Methods 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 2
- 230000003993 interaction Effects 0.000 description 2
- 150000002605 large molecules Chemical class 0.000 description 2
- 229920002521 macromolecule Polymers 0.000 description 2
- 230000036210 malignancy Effects 0.000 description 2
- 230000007246 mechanism Effects 0.000 description 2
- 230000036961 partial effect Effects 0.000 description 2
- 230000008488 polyadenylation Effects 0.000 description 2
- 229920001184 polypeptide Polymers 0.000 description 2
- 230000008569 process Effects 0.000 description 2
- 230000019491 signal transduction Effects 0.000 description 2
- 230000011664 signaling Effects 0.000 description 2
- 238000003786 synthesis reaction Methods 0.000 description 2
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 description 2
- 210000001519 tissue Anatomy 0.000 description 2
- 238000010361 transduction Methods 0.000 description 2
- 230000026683 transduction Effects 0.000 description 2
- 208000030507 AIDS Diseases 0.000 description 1
- 229930024421 Adenine Natural products 0.000 description 1
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 description 1
- 241000180579 Arca Species 0.000 description 1
- 102100024222 B-lymphocyte antigen CD19 Human genes 0.000 description 1
- 102000055105 BH3 Interacting Domain Death Agonist Human genes 0.000 description 1
- 108700000712 BH3 Interacting Domain Death Agonist Proteins 0.000 description 1
- 241000283690 Bos taurus Species 0.000 description 1
- 238000010354 CRISPR gene editing Methods 0.000 description 1
- 108010021064 CTLA-4 Antigen Proteins 0.000 description 1
- 241000282472 Canis lupus familiaris Species 0.000 description 1
- 241000283707 Capra Species 0.000 description 1
- 241000700198 Cavia Species 0.000 description 1
- 102000000844 Cell Surface Receptors Human genes 0.000 description 1
- 108010001857 Cell Surface Receptors Proteins 0.000 description 1
- 241000699800 Cricetinae Species 0.000 description 1
- HMFHBZSHGGEWLO-SOOFDHNKSA-N D-ribofuranose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H]1O HMFHBZSHGGEWLO-SOOFDHNKSA-N 0.000 description 1
- 108090000626 DNA-directed RNA polymerases Proteins 0.000 description 1
- 102000004163 DNA-directed RNA polymerases Human genes 0.000 description 1
- 241000283086 Equidae Species 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- 102100021186 Granulysin Human genes 0.000 description 1
- 101710168479 Granulysin Proteins 0.000 description 1
- 102000001398 Granzyme Human genes 0.000 description 1
- 108060005986 Granzyme Proteins 0.000 description 1
- HVLSXIKZNLPZJJ-TXZCQADKSA-N HA peptide Chemical compound C([C@@H](C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](C(C)C)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CC=1C=CC(O)=CC=1)C(=O)N[C@@H](C)C(O)=O)NC(=O)[C@H]1N(CCC1)C(=O)[C@@H](N)CC=1C=CC(O)=CC=1)C1=CC=C(O)C=C1 HVLSXIKZNLPZJJ-TXZCQADKSA-N 0.000 description 1
- 208000031886 HIV Infections Diseases 0.000 description 1
- 208000037357 HIV infectious disease Diseases 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 101000980825 Homo sapiens B-lymphocyte antigen CD19 Proteins 0.000 description 1
- 101000662909 Homo sapiens T cell receptor beta constant 1 Proteins 0.000 description 1
- 101000914514 Homo sapiens T-cell-specific surface glycoprotein CD28 Proteins 0.000 description 1
- 101000914484 Homo sapiens T-lymphocyte activation antigen CD80 Proteins 0.000 description 1
- 102000015696 Interleukins Human genes 0.000 description 1
- 108010063738 Interleukins Proteins 0.000 description 1
- 241000186805 Listeria innocua Species 0.000 description 1
- 208000031422 Lymphocytic Chronic B-Cell Leukemia Diseases 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- 108700019961 Neoplasm Genes Proteins 0.000 description 1
- 102000048850 Neoplasm Genes Human genes 0.000 description 1
- 108010055279 Oncogene Protein v-cbl Proteins 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 102000004503 Perforin Human genes 0.000 description 1
- 108010056995 Perforin Proteins 0.000 description 1
- KHGNFPUMBJSZSM-UHFFFAOYSA-N Perforine Natural products COC1=C2CCC(O)C(CCC(C)(C)O)(OC)C2=NC2=C1C=CO2 KHGNFPUMBJSZSM-UHFFFAOYSA-N 0.000 description 1
- 241000288906 Primates Species 0.000 description 1
- 101710089372 Programmed cell death protein 1 Proteins 0.000 description 1
- 241000700159 Rattus Species 0.000 description 1
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 description 1
- 241000283984 Rodentia Species 0.000 description 1
- 241000194019 Streptococcus mutans Species 0.000 description 1
- 241000282887 Suidae Species 0.000 description 1
- 102100037272 T cell receptor beta constant 1 Human genes 0.000 description 1
- 102100027213 T-cell-specific surface glycoprotein CD28 Human genes 0.000 description 1
- 102100027222 T-lymphocyte activation antigen CD80 Human genes 0.000 description 1
- 108091028113 Trans-activating crRNA Proteins 0.000 description 1
- 108091023040 Transcription factor Proteins 0.000 description 1
- 102000040945 Transcription factor Human genes 0.000 description 1
- 102100031988 Tumor necrosis factor ligand superfamily member 6 Human genes 0.000 description 1
- 108050002568 Tumor necrosis factor ligand superfamily member 6 Proteins 0.000 description 1
- 238000009825 accumulation Methods 0.000 description 1
- 230000003213 activating effect Effects 0.000 description 1
- 230000004913 activation Effects 0.000 description 1
- 239000012190 activator Substances 0.000 description 1
- 229960000643 adenine Drugs 0.000 description 1
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 230000006023 anti-tumor response Effects 0.000 description 1
- 210000000612 antigen-presenting cell Anatomy 0.000 description 1
- 238000002617 apheresis Methods 0.000 description 1
- 230000006907 apoptotic process Effects 0.000 description 1
- 210000003719 b-lymphocyte Anatomy 0.000 description 1
- 229960000074 biopharmaceutical Drugs 0.000 description 1
- 238000004364 calculation method Methods 0.000 description 1
- 150000001720 carbohydrates Chemical class 0.000 description 1
- 235000014633 carbohydrates Nutrition 0.000 description 1
- 230000036755 cellular response Effects 0.000 description 1
- 230000008859 change Effects 0.000 description 1
- 208000032852 chronic lymphocytic leukemia Diseases 0.000 description 1
- 150000001875 compounds Chemical class 0.000 description 1
- 230000003750 conditioning effect Effects 0.000 description 1
- 239000012641 cytoplasmic effector Substances 0.000 description 1
- 210000001151 cytotoxic T lymphocyte Anatomy 0.000 description 1
- 230000006378 damage Effects 0.000 description 1
- 230000003247 decreasing effect Effects 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 238000009826 distribution Methods 0.000 description 1
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 description 1
- 239000012636 effector Substances 0.000 description 1
- 230000002255 enzymatic effect Effects 0.000 description 1
- 230000001747 exhibiting effect Effects 0.000 description 1
- 238000000684 flow cytometry Methods 0.000 description 1
- MHMNJMPURVTYEJ-UHFFFAOYSA-N fluorescein-5-isothiocyanate Chemical compound O1C(=O)C2=CC(N=C=S)=CC=C2C21C1=CC=C(O)C=C1OC1=CC(O)=CC=C21 MHMNJMPURVTYEJ-UHFFFAOYSA-N 0.000 description 1
- 239000012634 fragment Substances 0.000 description 1
- 230000037433 frameshift Effects 0.000 description 1
- 238000010363 gene targeting Methods 0.000 description 1
- 208000024908 graft versus host disease Diseases 0.000 description 1
- 230000002489 hematologic effect Effects 0.000 description 1
- 208000024200 hematopoietic and lymphoid system neoplasm Diseases 0.000 description 1
- 208000033519 human immunodeficiency virus infectious disease Diseases 0.000 description 1
- 238000011577 humanized mouse model Methods 0.000 description 1
- 238000009396 hybridization Methods 0.000 description 1
- 230000028993 immune response Effects 0.000 description 1
- 230000036039 immunity Effects 0.000 description 1
- 230000001506 immunosuppresive effect Effects 0.000 description 1
- 230000001976 improved effect Effects 0.000 description 1
- 230000002779 inactivation Effects 0.000 description 1
- 238000001802 infusion Methods 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 238000007689 inspection Methods 0.000 description 1
- 230000009545 invasion Effects 0.000 description 1
- 210000000265 leukocyte Anatomy 0.000 description 1
- 239000003446 ligand Substances 0.000 description 1
- 239000011159 matrix material Substances 0.000 description 1
- 239000012528 membrane Substances 0.000 description 1
- 239000013642 negative control Substances 0.000 description 1
- 230000009438 off-target cleavage Effects 0.000 description 1
- 230000009437 off-target effect Effects 0.000 description 1
- 201000003733 ovarian melanoma Diseases 0.000 description 1
- 208000011932 ovarian sarcoma Diseases 0.000 description 1
- 230000037361 pathway Effects 0.000 description 1
- 229930192851 perforin Natural products 0.000 description 1
- 229920000642 polymer Polymers 0.000 description 1
- 244000144977 poultry Species 0.000 description 1
- XJMOSONTPMZWPB-UHFFFAOYSA-M propidium iodide Chemical compound [I-].[I-].C12=CC(N)=CC=C2C2=CC=C(N)C=C2[N+](CCC[N+](C)(CC)CC)=C1C1=CC=CC=C1 XJMOSONTPMZWPB-UHFFFAOYSA-M 0.000 description 1
- 230000029610 recognition of host Effects 0.000 description 1
- 230000017702 response to host Effects 0.000 description 1
- 230000001177 retroviral effect Effects 0.000 description 1
- 150000003384 small molecules Chemical class 0.000 description 1
- 239000007787 solid Substances 0.000 description 1
- 238000010186 staining Methods 0.000 description 1
- 230000002123 temporal effect Effects 0.000 description 1
- 229940113082 thymine Drugs 0.000 description 1
- 230000002103 transcriptional effect Effects 0.000 description 1
Classifications
-
- 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
- C12N15/1138—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 against receptors or cell surface proteins
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
- A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/70503—Immunoglobulin superfamily
- C07K14/7051—T-cell receptor (TcR)-CD3 complex
-
- 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
- C12N15/11—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
- C12N15/1135—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 against oncogenes or tumor suppressor genes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N5/0636—T lymphocytes
-
- 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 [RNase]; Deoxyribonucleases [DNase]
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/55—Vector systems having a special element relevant for transcription from bacteria
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Immunology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Cell Biology (AREA)
- Epidemiology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Oncology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Virology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Hematology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461981636P | 2014-04-18 | 2014-04-18 | |
| US61/981,636 | 2014-04-18 | ||
| US201562138246P | 2015-03-25 | 2015-03-25 | |
| US62/138,246 | 2015-03-25 | ||
| PCT/US2015/026504 WO2015161276A2 (en) | 2014-04-18 | 2015-04-17 | Crispr-cas-related methods, compositions and components for cancer immunotherapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2945335A1 true CA2945335A1 (en) | 2015-10-22 |
Family
ID=53059426
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2945335A Pending CA2945335A1 (en) | 2014-04-18 | 2015-04-17 | Crispr-cas-related methods, compositions and components for cancer immunotherapy |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20170175128A1 (enExample) |
| EP (2) | EP3132030B1 (enExample) |
| JP (5) | JP2017513485A (enExample) |
| KR (3) | KR102595473B1 (enExample) |
| CN (1) | CN108138183A (enExample) |
| AU (3) | AU2015247323B2 (enExample) |
| CA (1) | CA2945335A1 (enExample) |
| IL (4) | IL292512B2 (enExample) |
| SG (3) | SG10201809157VA (enExample) |
| WO (1) | WO2015161276A2 (enExample) |
Families Citing this family (240)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6261500B2 (ja) | 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 | ヌクレアーゼ切断特異性の評価および改善 |
| BR112014009346B1 (pt) | 2011-10-17 | 2020-09-15 | Massachusetts Institute Of Technology | Entrega intracelular |
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| KR102243597B1 (ko) | 2013-08-16 | 2021-04-26 | 메사추세츠 인스티튜트 오브 테크놀로지 | 세포로의 선택적 물질 전달 |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| PT3066201T (pt) | 2013-11-07 | 2018-06-04 | Massachusetts Inst Technology | Métodos e composições relacionadas com crispr com arng reguladores |
| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
| KR20170032406A (ko) | 2014-07-15 | 2017-03-22 | 주노 쎄러퓨티크스 인코퍼레이티드 | 입양 세포 치료를 위한 조작된 세포 |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| KR20170075013A (ko) | 2014-10-31 | 2017-06-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | Cart 세포에서 유전자 발현의 변경 및 그의 용도 |
| CN107109362A (zh) | 2014-10-31 | 2017-08-29 | 麻省理工学院 | 递送生物分子至免疫细胞 |
| CN107002089B (zh) | 2014-11-14 | 2021-09-07 | 麻省理工学院 | 化合物和组合物向细胞中的破坏和场实现的递送 |
| CN107532162A (zh) | 2014-12-12 | 2018-01-02 | 托德·M·伍尔夫 | 用于利用寡核苷酸编辑细胞中核酸的组合物和方法 |
| WO2016094880A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs) |
| WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
| WO2016094867A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Protected guide rnas (pgrnas) |
| WO2016106244A1 (en) | 2014-12-24 | 2016-06-30 | The Broad Institute Inc. | Crispr having or associated with destabilization domains |
| WO2016115179A1 (en) | 2015-01-12 | 2016-07-21 | Massachusetts Institute Of Technology | Gene editing through microfluidic delivery |
| JP6929791B2 (ja) | 2015-02-09 | 2021-09-01 | デューク ユニバーシティ | エピゲノム編集のための組成物および方法 |
| US20180112213A1 (en) * | 2015-03-25 | 2018-04-26 | Editas Medicine, Inc. | Crispr/cas-related methods, compositions and components |
| BR112017020750A2 (pt) * | 2015-03-27 | 2018-06-26 | Harvard College | células t modificadas e métodos de produção e utilização das mesmas |
| JP2018515139A (ja) | 2015-05-08 | 2018-06-14 | 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 | 万能ドナー幹細胞および関連する方法 |
| EP3294888A1 (en) * | 2015-05-11 | 2018-03-21 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating hiv infection and aids |
| CA2986310A1 (en) * | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| US20180161368A1 (en) | 2015-05-29 | 2018-06-14 | Juno Therapeutics, Inc. | Composition and methods for regulating inhibitory interactions in genetically engineered cells |
| EP3307887A1 (en) | 2015-06-09 | 2018-04-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for improving transplantation |
| AU2016279062A1 (en) | 2015-06-18 | 2019-03-28 | Omar O. Abudayyeh | Novel CRISPR enzymes and systems |
| WO2017008063A1 (en) | 2015-07-09 | 2017-01-12 | Massachusetts Institute Of Technology | Delivery of materials to anucleate cells |
| MA42895A (fr) | 2015-07-15 | 2018-05-23 | Juno Therapeutics Inc | Cellules modifiées pour thérapie cellulaire adoptive |
| ES2939084T3 (es) | 2015-07-29 | 2023-04-18 | Onk Therapeutics Ltd | Células asesinas naturales y líneas de células asesinas naturales modificadas que tienen citotoxicidad aumentada |
| US10906951B2 (en) | 2015-07-29 | 2021-02-02 | Onk Therapeutics Limited | Modified natural killer cells and natural killer cell lines having increased cytotoxicity |
| WO2017023803A1 (en) | 2015-07-31 | 2017-02-09 | Regents Of The University Of Minnesota | Modified cells and methods of therapy |
| US11613759B2 (en) | 2015-09-04 | 2023-03-28 | Sqz Biotechnologies Company | Intracellular delivery of biomolecules to cells comprising a cell wall |
| WO2017066497A2 (en) | 2015-10-13 | 2017-04-20 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| EP3365356B1 (en) | 2015-10-23 | 2023-06-28 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
| EP3368670A1 (en) | 2015-10-30 | 2018-09-05 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus |
| WO2017095967A2 (en) | 2015-11-30 | 2017-06-08 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| SG10202109655VA (en) * | 2015-12-04 | 2021-10-28 | Novartis Ag | Compositions and methods for immunooncology |
| JP7033535B2 (ja) * | 2016-01-12 | 2022-03-10 | スクイーズ バイオテクノロジーズ カンパニー | 複合体の細胞内送達 |
| WO2017147278A1 (en) | 2016-02-25 | 2017-08-31 | The Children's Medical Center Corporation | Customized class switch of immunoglobulin genes in lymphoma and hybridoma by crispr/cas9 technology |
| KR102587132B1 (ko) * | 2016-03-04 | 2023-10-11 | 에디타스 메디신, 인코포레이티드 | 암 면역요법을 위한 crispr-cpf1-관련 방법, 조성물 및 구성성분 |
| EP3219799A1 (en) | 2016-03-17 | 2017-09-20 | IMBA-Institut für Molekulare Biotechnologie GmbH | Conditional crispr sgrna expression |
| JP2019512271A (ja) * | 2016-03-21 | 2019-05-16 | デイナ ファーバー キャンサー インスティチュート，インコーポレイテッド | Ｔ細胞疲弊状態特異的遺伝子発現調節因子およびその使用 |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| WO2017185054A1 (en) * | 2016-04-22 | 2017-10-26 | Intellia Therapeutics, Inc. | Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four |
| CN107304434A (zh) * | 2016-04-25 | 2017-10-31 | 上海宇玫博生物科技有限公司 | 一种免疫细胞治疗的双功能新型载体 |
| CN109414449A (zh) * | 2016-05-06 | 2019-03-01 | 托德·M·伍尔夫 | 利用和不利用可设计核酸酶编辑基因组的改进方法 |
| US20190136230A1 (en) * | 2016-05-06 | 2019-05-09 | Juno Therapeutics, Inc. | Genetically engineered cells and methods of making the same |
| DK3476865T5 (da) | 2016-06-28 | 2024-09-09 | Biocytogen Pharmaceuticals Beijing Co Ltd | Fremgangsmåde til konstruktion af pd-1-gen-modificeret humaniseret dyremodel og anvendelse deraf |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | 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 | アデノシン核酸塩基編集因子およびそれらの使用 |
| US11078481B1 (en) | 2016-08-03 | 2021-08-03 | KSQ Therapeutics, Inc. | Methods for screening for cancer targets |
| CN109996811A (zh) * | 2016-08-03 | 2019-07-09 | 约翰·F·迪帕西奥 | 用于用嵌合抗原受体治疗t细胞恶性肿瘤的car-t细胞的基因编辑 |
| EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| EP4485466A3 (en) | 2016-08-17 | 2025-04-02 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| US10576167B2 (en) | 2016-08-17 | 2020-03-03 | Factor Bioscience Inc. | Nucleic acid products and methods of administration thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| US11078483B1 (en) | 2016-09-02 | 2021-08-03 | KSQ Therapeutics, Inc. | Methods for measuring and improving CRISPR reagent function |
| WO2018067618A1 (en) | 2016-10-03 | 2018-04-12 | Juno Therapeutics, Inc. | Hpv-specific binding molecules |
| CN110381963A (zh) | 2016-10-13 | 2019-10-25 | 朱诺治疗学股份有限公司 | 涉及色氨酸代谢途径调节剂的免疫治疗方法和组合物 |
| EP3526320A1 (en) | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| CA3041068A1 (en) | 2016-10-18 | 2018-04-26 | Regents Of The University Of Minnesota | Tumor infiltrating lymphocytes and methods of therapy |
| GB2573664B (en) * | 2016-10-27 | 2022-09-28 | Intima Bioscience Inc | Viral methods of T cell therapy |
| US11332713B2 (en) | 2016-11-16 | 2022-05-17 | KSQ Therapeutics, Inc. | Gene-regulating compositions and methods for improved immunotherapy |
| CN110121558B (zh) * | 2016-11-28 | 2023-06-13 | 日商那帕洁制药股份有限公司 | 化学修饰siRNA |
| KR20190098747A (ko) | 2016-12-05 | 2019-08-22 | 주노 쎄러퓨티크스 인코퍼레이티드 | 입양 세포 치료법을 위한 조작된 세포의 제조방법 |
| JP7206214B2 (ja) | 2016-12-13 | 2023-01-17 | シアトル チルドレンズ ホスピタル （ディービーエイ シアトル チルドレンズ リサーチ インスティテュート） | インビトロ及びインビボで操作された細胞において発現された化学誘導シグナル伝達複合体の外因性薬物活性化の方法 |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| MX2019007840A (es) * | 2016-12-30 | 2020-08-03 | Celularity Inc | Celulas asesinas naturales modificadas geneticamente. |
| CN110520156A (zh) * | 2017-01-29 | 2019-11-29 | 唐泽群 | 针对外源抗原和/或自身抗原的免疫调控方法 |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| US10828330B2 (en) * | 2017-02-22 | 2020-11-10 | IO Bioscience, Inc. | Nucleic acid constructs comprising gene editing multi-sites and uses thereof |
| CA3054307A1 (en) | 2017-02-22 | 2018-08-30 | Io Biosciences, Inc. | Nucleic acid constructs comprising gene editing multi-sites and uses thereof |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Cancer vaccine |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| JP2020513832A (ja) * | 2017-03-22 | 2020-05-21 | ノバルティス アーゲー | 免疫腫瘍学のための組成物および方法 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US10934336B2 (en) | 2017-04-13 | 2021-03-02 | The Trustees Of The University Of Pennsylvania | Use of gene editing to generate universal TCR re-directed T cells for adoptive immunotherapy |
| EP3621981A2 (en) | 2017-05-12 | 2020-03-18 | CRISPR Therapeutics AG | Materials and methods for engineering cells and uses thereof in immuno-oncology |
| US11166985B2 (en) | 2017-05-12 | 2021-11-09 | Crispr Therapeutics Ag | Materials and methods for engineering cells and uses thereof in immuno-oncology |
| 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 |
| MA48797A (fr) | 2017-05-25 | 2020-04-08 | Bluebird Bio Inc | Variants de l'endonucléase cblb , compositions et procédés d'utilisation |
| MX2019014640A (es) | 2017-06-09 | 2020-10-05 | Editas Medicine Inc | Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas. |
| EP3638317A4 (en) | 2017-06-15 | 2021-03-17 | The Regents of The University of California | TARGETED NONVIRAL DNA INSERTIONS |
| CN109963944A (zh) * | 2017-06-20 | 2019-07-02 | 江苏恒瑞医药股份有限公司 | 体外敲除T细胞中靶基因的方法以及该方法中使用的crRNA |
| SG10201705285SA (en) * | 2017-06-27 | 2019-01-30 | Agency Science Tech & Res | Antisense oligonucleotides |
| JP2020530307A (ja) | 2017-06-30 | 2020-10-22 | インティマ・バイオサイエンス，インコーポレーテッド | 遺伝子治療のためのアデノ随伴ウイルスベクター |
| WO2019003193A1 (en) * | 2017-06-30 | 2019-01-03 | Novartis Ag | METHODS FOR TREATING DISEASES USING GENE EDITING SYSTEMS |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| JP7654240B2 (ja) | 2017-09-19 | 2025-04-01 | マサチューセッツ インスティテュート オブ テクノロジー | キメラ抗原受容体ｔ細胞治療のための組成物およびその使用 |
| US11952408B2 (en) | 2017-10-03 | 2024-04-09 | Juno Therapeutics, Inc. | HPV-specific binding molecules |
| WO2019072241A1 (en) | 2017-10-13 | 2019-04-18 | Beijing Biocytogen Co., Ltd | NON-HUMAN ANIMAL GENETICALLY MODIFIED WITH PD-1 HUMAN OR CHIMERIC |
| CN111757937A (zh) | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
| KR102770787B1 (ko) | 2017-10-27 | 2025-02-19 | 더 리전트 오브 더 유니버시티 오브 캘리포니아 | 내인성 t 세포 수용체의 표적화된 대체 |
| WO2019089982A1 (en) | 2017-11-01 | 2019-05-09 | Juno Therapeutics, Inc. | Method of assessing activity of recombinant antigen receptors |
| CN111556893A (zh) * | 2017-11-06 | 2020-08-18 | 爱迪塔斯医药股份有限公司 | 免疫疗法t细胞中cblb的crispr-cas9编辑的方法、组合物和组分 |
| US20210187018A1 (en) * | 2017-12-07 | 2021-06-24 | Flagship Pioneering Innovations V, Inc. | Cytobiologics and therapeutic uses thereof |
| WO2019118508A1 (en) | 2017-12-12 | 2019-06-20 | The Trustees Of The University Of Pennsylvania | Genetically modified immune cells targeting ny-eso-1 and methods of use thereof |
| US11766455B2 (en) * | 2017-12-14 | 2023-09-26 | Ezy Biotech Llc | Subject-specific tumor inhibiting cells and the use thereof |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| WO2019178427A1 (en) | 2018-03-14 | 2019-09-19 | Arbor Biotechnologies, Inc. | Novel crispr dna targeting enzymes and systems |
| KR20200133219A (ko) | 2018-03-15 | 2020-11-26 | 케이에스큐 세러퓨틱스 인코포레이티드 | 개선된 면역요법을 위한 유전자-조절 조성물 및 방법 |
| BR112020018658A2 (pt) | 2018-03-15 | 2020-12-29 | KSQ Therapeutics, Inc. | Composições de regulação gênica e métodos para imu-noterapia aprimorada |
| US20210017509A1 (en) * | 2018-03-23 | 2021-01-21 | The Trustees Of Columbia University In The City Of New York | Gene Editing for Autosomal Dominant Diseases |
| BR112020019313A2 (pt) | 2018-04-05 | 2021-01-05 | Juno Therapeutics Inc | Receptores de células t e células modificadas que expressam os mesmos |
| CA3095084A1 (en) | 2018-04-05 | 2019-10-10 | Juno Therapeutics, Inc. | T cells expressing a recombinant receptor, related polynucleotides and methods |
| KR20210029707A (ko) | 2018-04-05 | 2021-03-16 | 주노 쎄러퓨티크스 인코퍼레이티드 | 재조합 수용체를 발현하는 세포의 생산 방법 및 관련 조성물 |
| US20190316102A1 (en) * | 2018-04-13 | 2019-10-17 | Sigma-Aldrich Co. Llc | Modification of immune-related genomic loci using paired crispr nickase ribonucleoproteins |
| RU2020134965A (ru) | 2018-04-27 | 2022-04-27 | Спейскрафт Севен, Ллк | Генная терапия при дегенерации цнс |
| SG11202007874XA (en) | 2018-04-27 | 2020-09-29 | Seattle Childrens Hospital Dba Seattle Childrens Res Inst | Rapamycin resistant cells |
| CN112105420A (zh) * | 2018-05-11 | 2020-12-18 | 克里斯珀医疗股份公司 | 用于治疗癌症的方法和组合物 |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| EP3806903B1 (en) | 2018-06-14 | 2024-02-14 | 2seventy bio, Inc. | Cd79a chimeric antigen receptors |
| CN108866004A (zh) * | 2018-06-26 | 2018-11-23 | 奥妙生物技术（广州）有限公司 | Shp-1敲除的t细胞及其构建方法 |
| KR20210043574A (ko) | 2018-07-09 | 2021-04-21 | 플래그쉽 파이어니어링 이노베이션스 브이, 인크. | 푸소좀 조성물 및 이의 용도 |
| US12473533B2 (en) | 2018-07-09 | 2025-11-18 | The Regents Of The University Of California | Gene targets for T-cell-based immunotherapy |
| WO2020022802A1 (ko) * | 2018-07-25 | 2020-01-30 | 주식회사 툴젠 | 인위적인 유전자 조작을 통한 자가면역질환 치료 |
| US20210317448A1 (en) * | 2018-07-26 | 2021-10-14 | Toolgen Incorporated | Gene editing of anticoagulants |
| WO2020041380A1 (en) | 2018-08-20 | 2020-02-27 | The Broad Institute, Inc. | Methods and compositions for optochemical control of crispr-cas9 |
| US20210348177A1 (en) | 2018-09-05 | 2021-11-11 | The Regents Of The University Of California | Generation of heritably gene-edited plants without tissue culture |
| US20200080056A1 (en) * | 2018-09-06 | 2020-03-12 | The Trustees Of The University Of Pennsylvania | CRISPR-Cas9 Knock-out of SHP-1/2 to Reduce T cell Exhaustion in Adoptive Cell Therapy |
| WO2020055962A1 (en) * | 2018-09-11 | 2020-03-19 | Board Of Regents, The University Of Texas System | A genetic mouse model of autoimmune adverse events and immune checkpoint blockade therapy |
| SG11202100935TA (en) | 2018-09-28 | 2021-02-25 | Massachusetts Inst Technology | Collagen-localized immunomodulatory molecules and methods thereof |
| WO2020072059A1 (en) * | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
| AU2019362879A1 (en) * | 2018-10-16 | 2021-05-27 | Intellia Therapeutics, Inc. | Compositions and methods for immunotherapy |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| CN111757889B (zh) * | 2018-10-29 | 2021-05-25 | 中国农业大学 | 新型CRISPR/Cas12f酶和系统 |
| CN113631718A (zh) | 2018-11-14 | 2021-11-09 | 旗舰先锋创新V股份有限公司 | 用于特定隔室货物递送的组合物和方法 |
| US12264323B2 (en) | 2018-12-17 | 2025-04-01 | The Broad Institute, Inc. | CRISPR CPF1 direct repeat variants |
| WO2020131862A1 (en) | 2018-12-17 | 2020-06-25 | The Broad Institute, Inc. | Crispr-associated transposase systems and methods of use thereof |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| EP3917541A4 (en) * | 2019-01-28 | 2022-12-07 | Bar Ilan University | Combinations, nanoparticles and methods for controlling natural killer cell activation and function |
| BR112021016046A2 (pt) | 2019-02-15 | 2021-11-09 | Editas Medicine Inc | Células assassinas naturais modificadas (nk) para imunoterapia |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| CA3136119A1 (en) | 2019-04-10 | 2020-10-15 | University Of Utah Research Foundation | Htra1 modulation for treatment of amd |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| WO2020222176A1 (en) | 2019-04-30 | 2020-11-05 | Crispr Therapeutics Ag | Allogeneic cell therapy of b cell malignancies using genetically engineered t cells targeting cd19 |
| MA55811A (fr) | 2019-05-01 | 2022-03-09 | Editas Medicine Inc | Cellules exprimant un récepteur recombinant à base d'un locus modifié du tgfbr2, et polynucléotides et méthodes associés |
| JP7754722B2 (ja) | 2019-05-01 | 2025-10-15 | ジュノー セラピューティクス インコーポレイテッド | 改変されたｃｄ２４７遺伝子座からキメラ受容体を発現する細胞、関連ポリヌクレオチド、および方法 |
| GB2600568B (en) | 2019-05-03 | 2024-07-31 | Specific Biologics Inc | Lipid-encapsulated dual-cleaving endonuclease for DNA and gene editing |
| US20220235340A1 (en) | 2019-05-20 | 2022-07-28 | The Broad Institute, Inc. | Novel crispr-cas systems and uses thereof |
| US12188045B2 (en) | 2019-06-07 | 2025-01-07 | KSQ Therapeutics, Inc. | Guide RNA combinations and methods of use |
| MA56212A (fr) * | 2019-06-14 | 2022-04-20 | 2Seventy Bio Inc | Compositions et méthodes de traitement du cancer |
| US11642409B2 (en) | 2019-06-26 | 2023-05-09 | Massachusetts Insttute of Technology | Immunomodulatory fusion protein-metal hydroxide complexes and methods thereof |
| WO2021022043A2 (en) | 2019-07-30 | 2021-02-04 | Pairwise Plants Services, Inc. | Morphogenic regulators and methods of using the same |
| WO2021041922A1 (en) | 2019-08-30 | 2021-03-04 | The Broad Institute, Inc. | Crispr-associated mu transposase systems |
| WO2021061648A1 (en) | 2019-09-23 | 2021-04-01 | Massachusetts Institute Of Technology | Methods and compositions for stimulation of endogenous t cell responses |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| US20220348929A1 (en) * | 2019-12-09 | 2022-11-03 | Caribou Biosciences, Inc. | Crispr abasic restricted nucleotides and crispr accuracy via analogs |
| EP4183868A4 (en) * | 2020-01-23 | 2024-08-21 | Kangstem Biotech Co., Ltd. | READY-TO-USE IMMUNE CELLS AND STEM CELLS, AND PHARMACEUTICAL COMPOSITION COMPRISING THEM |
| US11896619B2 (en) | 2020-03-10 | 2024-02-13 | Massachusetts Institute Of Technology | Compositions and methods for immunotherapy of NPM1c-positive cancer |
| WO2021216623A1 (en) | 2020-04-21 | 2021-10-28 | Aspen Neuroscience, Inc. | Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom |
| WO2021216622A1 (en) | 2020-04-21 | 2021-10-28 | Aspen Neuroscience, Inc. | Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom |
| US20210340524A1 (en) | 2020-05-01 | 2021-11-04 | Massachusetts Institute Of Technology | Methods for identifying chimeric antigen receptor-targeting ligands and uses thereof |
| US12433954B2 (en) | 2020-05-01 | 2025-10-07 | Massachusetts Institute Of Technology | Methods of activating anti-CD19 chimeric antigen receptor (CAR) T cells using amphiphilic ligand conjugates comprising CAR-targeting protein sequence motifs |
| MX2022014008A (es) | 2020-05-08 | 2023-02-09 | Broad Inst Inc | Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo. |
| AU2021267379A1 (en) * | 2020-05-08 | 2022-12-08 | Metagenomi, Inc. | Enzymes with RuvC domains |
| US20230181641A1 (en) | 2020-05-13 | 2023-06-15 | Juno Therapeutics, Inc. | Process for producing donor-batched cells expressing a recombinant receptor |
| AU2021282578A1 (en) * | 2020-06-03 | 2023-02-02 | Mammoth Biosciences, Inc. | Programmable nucleases and methods of use |
| EP4171616A1 (en) | 2020-06-26 | 2023-05-03 | Juno Therapeutics GmbH | Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods |
| US11332744B1 (en) | 2020-10-26 | 2022-05-17 | Arsenal Biosciences, Inc. | Safe harbor loci |
| JP2023549780A (ja) | 2020-11-04 | 2023-11-29 | ジュノー セラピューティクス インコーポレイテッド | 改変されたインバリアントｃｄ３免疫グロブリンスーパーファミリー鎖遺伝子座からキメラ受容体を発現する細胞ならびに関連するポリヌクレオチドおよび方法 |
| US11459372B2 (en) | 2020-11-30 | 2022-10-04 | Crispr Therapeutics Ag | Gene-edited natural killer cells |
| WO2022132756A1 (en) * | 2020-12-14 | 2022-06-23 | Emendobio Inc. | Biallelic knockout of trac |
| WO2022144632A1 (en) | 2020-12-30 | 2022-07-07 | Crispr Therapeutics Ag | Compositions and methods for differentiating stem cells into nk cells |
| EP4284932A1 (en) * | 2021-02-01 | 2023-12-06 | Epsilen Bio S.r.l. | Gene silencing |
| EP4288541A1 (en) | 2021-02-05 | 2023-12-13 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| EP4301755A1 (en) | 2021-03-03 | 2024-01-10 | Juno Therapeutics, Inc. | Combination of a t cell therapy and a dgk inhibitor |
| JP2024511420A (ja) | 2021-03-22 | 2024-03-13 | ジュノー セラピューティクス インコーポレイテッド | ウイルスベクター粒子の効力を評価する方法 |
| WO2022215982A1 (ko) * | 2021-04-05 | 2022-10-13 | 주식회사 셀렌진 | Trac 유전자에 상보적인 가이드 rna 및 이의 용도 |
| WO2022232839A1 (en) | 2021-04-30 | 2022-11-03 | The Board Of Trustees Of The Leland Stanford Junior University | Methods for improved production of primary cd34+ cells |
| EP4355879A4 (en) | 2021-06-14 | 2025-07-23 | Kamau Therapeutics Inc | METHODS FOR GENETIC MODIFICATION OF HEMATOPOIETIC STEM AND PROGENITOR CELLS FOR ERYTHROCYTE-SPECIFIC EXPRESSION OF THERAPEUTIC PROTEINS |
| WO2022269393A1 (en) * | 2021-06-23 | 2022-12-29 | Crispr Therapeutics Ag | Engineered cells with improved protection from natural killer cell killing |
| CA3225138A1 (en) | 2021-06-23 | 2022-12-29 | Editas Medicine, Inc. | Engineered cells for therapy |
| WO2023278811A1 (en) | 2021-07-01 | 2023-01-05 | Indapta Therapeutics, Inc. | Engineered natural killer (nk) cells and related methods |
| CA3227103A1 (en) | 2021-07-30 | 2023-02-02 | Matthew P. GEMBERLING | Compositions and methods for modulating expression of frataxin (fxn) |
| US20240252684A1 (en) | 2021-07-30 | 2024-08-01 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| MX2024002539A (es) | 2021-08-27 | 2024-03-19 | Metagenomi Inc | Enzimas con dominios ruvc. |
| PE20241173A1 (es) | 2021-10-14 | 2024-05-28 | Arsenal Biosciences Inc | Celulas inmunitarias que tienen arnhc coexpresados y sistemas de compuerta logica |
| WO2023077149A1 (en) | 2021-11-01 | 2023-05-04 | Sigma-Aldrich Co. Llc | Electroporation enhancers for crispr-cas systems |
| WO2023081715A1 (en) | 2021-11-03 | 2023-05-11 | Viracta Therapeutics, Inc. | Combination of car t-cell therapy with btk inhibitors and methods of use thereof |
| WO2023081900A1 (en) | 2021-11-08 | 2023-05-11 | Juno Therapeutics, Inc. | Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods |
| EP4448775A1 (en) | 2021-12-17 | 2024-10-23 | Sana Biotechnology, Inc. | Modified paramyxoviridae attachment glycoproteins |
| TW202342498A (zh) | 2021-12-17 | 2023-11-01 | 美商薩那生物科技公司 | 經修飾副黏液病毒科融合醣蛋白 |
| IL313660A (en) | 2021-12-22 | 2024-08-01 | Camp4 Therapeutics Corp | Modulation of gene transcription using antisense oligonucleotides targeting regulatory RNAs |
| EP4463135A2 (en) | 2022-01-10 | 2024-11-20 | Sana Biotechnology, Inc. | Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses |
| EP4463549A2 (en) | 2022-01-14 | 2024-11-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression |
| WO2023137471A1 (en) | 2022-01-14 | 2023-07-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation |
| EP4472646A1 (en) | 2022-02-01 | 2024-12-11 | Sana Biotechnology, Inc. | Cd3-targeted lentiviral vectors and uses thereof |
| EP4473097A1 (en) | 2022-02-02 | 2024-12-11 | Sana Biotechnology, Inc. | Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses |
| EP4508073A1 (en) | 2022-04-14 | 2025-02-19 | Board of Regents of the University of Nebraska | Cell therapy for alzheimer's disease |
| US20230372395A1 (en) | 2022-05-19 | 2023-11-23 | Massachusetts Institute Of Technology | Car cells targeting an inserted ligand |
| JP2025524469A (ja) | 2022-06-24 | 2025-07-30 | チューン セラピューティクス インコーポレイテッド | 標的を定めた遺伝子抑制を介して低密度リポタンパク質を低減するための組成物、系、及び方法 |
| KR20250060188A (ko) | 2022-06-30 | 2025-05-07 | 인답타 세라뷰틱스 인코포레이티드 | 조작된 자연 살해(nk) 세포 및 항체 요법의 조합 및 관련 방법 |
| CA3261865A1 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics, Inc. | TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS |
| AU2023325407A1 (en) | 2022-08-19 | 2025-02-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression |
| WO2024044655A1 (en) | 2022-08-24 | 2024-02-29 | Sana Biotechnology, Inc. | Delivery of heterologous proteins |
| CN120152717A (zh) | 2022-09-08 | 2025-06-13 | 朱诺治疗学股份有限公司 | T细胞疗法和连续或间歇dgk抑制剂给药的组合 |
| EP4590821A2 (en) | 2022-09-19 | 2025-07-30 | Tune Therapeutics, Inc. | Compositions, systems, and methods for modulating t cell function |
| KR20250072709A (ko) * | 2022-09-19 | 2025-05-26 | 에멘도바이오 인코포레이티드 | Ctla4의 이중 대립유전자 녹아웃 |
| EP4590688A1 (en) | 2022-09-21 | 2025-07-30 | Sana Biotechnology, Inc. | Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof |
| EP4602174A1 (en) | 2022-10-13 | 2025-08-20 | Sana Biotechnology, Inc. | Viral particles targeting hematopoietic stem cells |
| WO2024100604A1 (en) | 2022-11-09 | 2024-05-16 | Juno Therapeutics Gmbh | Methods for manufacturing engineered immune cells |
| WO2024119157A1 (en) | 2022-12-02 | 2024-06-06 | Sana Biotechnology, Inc. | Lipid particles with cofusogens and methods of producing and using the same |
| WO2024163678A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| WO2024163683A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist) |
| CN121002052A (zh) | 2023-02-03 | 2025-11-21 | C3S2 有限公司 | 用于非病毒生产工程化免疫细胞的方法 |
| WO2024186656A1 (en) | 2023-03-03 | 2024-09-12 | Arsenal Biosciences, Inc. | Systems targeting psma and ca9 |
| WO2024220574A1 (en) | 2023-04-18 | 2024-10-24 | Sana Biotechnology, Inc. | Universal protein g fusogens and adapter systems thereof and related lipid particles and uses |
| WO2024220598A2 (en) | 2023-04-18 | 2024-10-24 | Sana Biotechnology, Inc. | Lentiviral vectors with two or more genomes |
| WO2024220560A1 (en) | 2023-04-18 | 2024-10-24 | Sana Biotechnology, Inc. | Engineered protein g fusogens and related lipid particles and methods thereof |
| WO2024243340A1 (en) | 2023-05-23 | 2024-11-28 | Sana Biotechnology, Inc. | Tandem fusogens and related lipid particles |
| WO2024249342A1 (en) | 2023-05-26 | 2024-12-05 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting ptpn2 expression |
| CN121195068A (zh) | 2023-05-26 | 2025-12-23 | 爱迪塔斯医药公司 | 靶向cd70表达的crispr相关方法和组合物 |
| WO2024249348A2 (en) | 2023-05-26 | 2024-12-05 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting fl1-1 expression |
| CN116539885B (zh) * | 2023-07-06 | 2023-09-29 | 上海秤信生物科技有限公司 | 用于乳腺癌早期检测的肿瘤自身抗原/抗体组合及应用 |
| WO2025029840A1 (en) | 2023-07-31 | 2025-02-06 | Tune Therapeutics, Inc. | Compositions and methods for multiplexed activation and repression of t cell gene expression |
| WO2025029835A1 (en) | 2023-07-31 | 2025-02-06 | Tune Therapeutics, Inc. | Compositions and methods for modulating il-2 gene expression |
| WO2025038494A1 (en) | 2023-08-11 | 2025-02-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation |
| WO2025054202A1 (en) | 2023-09-05 | 2025-03-13 | Sana Biotechnology, Inc. | Method of screening a sample comprising a transgene with a unique barcode |
| WO2025059073A1 (en) | 2023-09-11 | 2025-03-20 | Tune Therapeutics, Inc. | Epigenetic editing methods and systems for differentiating stem cells |
| WO2025059533A1 (en) | 2023-09-13 | 2025-03-20 | The Broad Institute, Inc. | Crispr enzymes and systems |
| WO2025096638A2 (en) | 2023-10-30 | 2025-05-08 | Turnstone Biologics Corp. | Genetically modified tumor infilitrating lymphocytes and methods of producing and using the same |
| WO2025117544A1 (en) | 2023-11-29 | 2025-06-05 | The Broad Institute, Inc. | Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof |
| WO2025137646A1 (en) | 2023-12-22 | 2025-06-26 | Recode Therapeutics, Inc. | Gene editing methods and compositions for treating cystic fibrosis |
| WO2025151838A1 (en) | 2024-01-12 | 2025-07-17 | Sana Biotechnology, Inc. | Safety switches to control in vitro and in vivo proliferation of cell therapy products |
| WO2025184529A1 (en) | 2024-03-01 | 2025-09-04 | Sana Biotechnology, Inc. | Viral particles with fusogen display and related compositions and methods |
| WO2025212519A1 (en) | 2024-04-01 | 2025-10-09 | Moonlight Bio, Inc. | Dll3 binding proteins and uses thereof |
| WO2025213127A1 (en) | 2024-04-05 | 2025-10-09 | Indapta Therapeutics, Inc. | Treatment of autoimmune diseases or conditions with natural killer cells |
| US20250345431A1 (en) | 2024-05-10 | 2025-11-13 | Juno Therapeutics, Inc. | Genetically engineered t cells expressing a cd19 chimeric antigen receptor (car) and uses thereof for allogeneic cell therapy |
| WO2025245399A1 (en) | 2024-05-23 | 2025-11-27 | Indapta Therapeutics, Inc. | Method of treating cancer with natural killer cells |
| WO2025260068A1 (en) | 2024-06-14 | 2025-12-18 | Tune Therapeutics, Inc. | Lipid nanoparticle formulation for delivery of nucleic acids to cells |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP3362867B2 (ja) * | 1991-04-26 | 2003-01-07 | 財団法人大阪バイオサイエンス研究所 | ヒト細胞表面抗原をコードするｄｎａ |
| MY189533A (en) * | 2012-05-25 | 2022-02-16 | Univ California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| CN111676196A (zh) * | 2012-05-25 | 2020-09-18 | 塞勒克提斯公司 | 工程化异体和免疫抑制耐受性t细胞的方法 |
| ES2824024T3 (es) * | 2012-10-10 | 2021-05-11 | Sangamo Therapeutics Inc | Compuestos modificadores de células T y usos de los mismos |
| US8697359B1 (en) * | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| CN105209621B (zh) * | 2012-12-12 | 2021-05-25 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| JP6346266B2 (ja) * | 2013-03-21 | 2018-06-20 | サンガモ セラピューティクス， インコーポレイテッド | 操作されたジンクフィンガータンパク質ヌクレアーゼを使用するｔ細胞受容体遺伝子の標的化された破壊 |
| AU2014266833B2 (en) * | 2013-05-13 | 2020-07-02 | Cellectis | Methods for engineering highly active T cell for immunotherapy |
| EP3309248B1 (en) * | 2013-05-29 | 2021-06-09 | Cellectis | Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system |
| EP3011031B1 (en) * | 2013-06-17 | 2020-09-30 | The Broad Institute Inc. | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
| KR102228828B1 (ko) * | 2014-03-11 | 2021-03-16 | 셀렉티스 | 동종이형 이식에 양립성인 t-세포들을 만들어내는 방법 |
| US20180112213A1 (en) * | 2015-03-25 | 2018-04-26 | Editas Medicine, Inc. | Crispr/cas-related methods, compositions and components |
-
2015
- 2015-04-17 SG SG10201809157VA patent/SG10201809157VA/en unknown
- 2015-04-17 SG SG10201912171PA patent/SG10201912171PA/en unknown
- 2015-04-17 IL IL292512A patent/IL292512B2/en unknown
- 2015-04-17 KR KR1020167032071A patent/KR102595473B1/ko active Active
- 2015-04-17 EP EP15721400.8A patent/EP3132030B1/en active Active
- 2015-04-17 US US15/303,722 patent/US20170175128A1/en not_active Abandoned
- 2015-04-17 JP JP2016562958A patent/JP2017513485A/ja active Pending
- 2015-04-17 AU AU2015247323A patent/AU2015247323B2/en active Active
- 2015-04-17 KR KR1020237036403A patent/KR102827558B1/ko active Active
- 2015-04-17 CN CN201580031806.XA patent/CN108138183A/zh active Pending
- 2015-04-17 WO PCT/US2015/026504 patent/WO2015161276A2/en not_active Ceased
- 2015-04-17 SG SG11201608701QA patent/SG11201608701QA/en unknown
- 2015-04-17 EP EP20190783.9A patent/EP3800248A3/en active Pending
- 2015-04-17 KR KR1020257021203A patent/KR20250102123A/ko active Pending
- 2015-04-17 CA CA2945335A patent/CA2945335A1/en active Pending
-
2016
- 2016-10-10 IL IL248284A patent/IL248284A0/en active IP Right Grant
-
2019
- 2019-12-16 JP JP2019226458A patent/JP2020062029A/ja active Pending
-
2020
- 2020-12-06 IL IL279230A patent/IL279230B/en unknown
-
2021
- 2021-08-26 US US17/412,608 patent/US20220106600A1/en active Pending
- 2021-09-02 IL IL286103A patent/IL286103B/en unknown
- 2021-09-20 AU AU2021236446A patent/AU2021236446B2/en active Active
-
2022
- 2022-02-24 JP JP2022026505A patent/JP7525531B2/ja active Active
-
2024
- 2024-03-18 JP JP2024042229A patent/JP7681753B2/ja active Active
- 2024-11-08 AU AU2024259841A patent/AU2024259841A1/en active Pending
-
2025
- 2025-05-09 JP JP2025078666A patent/JP2025128109A/ja active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CA2945335A1 (en) | Crispr-cas-related methods, compositions and components for cancer immunotherapy | |
| AU2021282533C1 (en) | Optimized crispr/cas9 systems and methods for gene editing in stem cells | |
| US20230002760A1 (en) | Crispr/cas-related methods, compositions and components | |
| KR102796744B1 (ko) | 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물 | |
| CA2943622A1 (en) | Crispr/cas-related methods and compositions for treating hiv infection and aids | |
| US20190136230A1 (en) | Genetically engineered cells and methods of making the same | |
| KR20180120752A (ko) | 베타 이상헤모글로빈증의 치료를 위한 crispr/cas-관련 방법 및 조성물 | |
| CA2986262C (en) | Crispr/cas-related methods and compositions for improving transplantation | |
| HK40050007A (en) | Crispr-cas-related methods, compositions and components for cancer immunotherapy | |
| HK1255296B (en) | Crispr/cas-related methods and compositions for improving transplantation |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20200415 |
|
| EEER | Examination request |
Effective date: 20200415 |
|
| EEER | Examination request |
Effective date: 20200415 |
|
| EEER | Examination request |
Effective date: 20200415 |
|
| EEER | Examination request |
Effective date: 20200415 |
|
| EEER | Examination request |
Effective date: 20200415 |