公开(公告)号：US20190002869A1

公开(公告)日：2019-01-03

申请号：US16029273

申请日：2018-07-06

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： Hao Yin ,  Wen Xue ,  Daniel G. Anderson ,  Joseph R. Dorkin ,  Tyler E. Jacks

IPC: C12N15/10 ,  C12N15/88 ,  C12N15/86 ,  C12N15/85 ,  A61K48/00 ,  C12N15/11 ,  C12N7/00

Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.

公开(公告)号：US10047355B2

公开(公告)日：2018-08-14

申请号：US15374227

申请日：2016-12-09

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： Hao Yin ,  Wen Xue ,  Daniel G. Anderson ,  Joseph R. Dorkin ,  Tyler E. Jacks

IPC: C12N15/10 ,  C12N15/85 ,  C12N15/86 ,  C12N7/00 ,  C12N15/88 ,  A61K48/00

Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.

公开(公告)号：US11279928B2

公开(公告)日：2022-03-22

申请号：US15741211

申请日：2016-06-29

Applicant: Massachusetts Institute of Technology

Inventor： Hao Yin ,  Daniel G. Anderson ,  Robert Samuel Langer

IPC: C12N15/11 ,  C12N9/22 ,  A61K48/00 ,  C12N9/96 ,  C12N15/90 ,  C07H21/02 ,  C07H21/04

Abstract: The disclosure relates to compositions comprising and methods for chemical modification of single guide RNA (sgRNA), tracrRNA and/or crRNA used individually or in combination with one another or Cas system components. Compositions comprising modified ribonucleic acids have been designed with chemical modification for even higher efficiency as unmodified native strand of sgRNA. Administration of modified ribonucleic acids will allow decreased immune response when administered to a subject, increased stability, increased editing efficiency and facilitated in vivo delivery of sgRNA via various delivery platforms. The disclosure also relates to methods of decreasing off-target effect of CRISPR and a CRISPR complex.

公开(公告)号：US20190233816A1

公开(公告)日：2019-08-01

申请号：US16256003

申请日：2019-01-24

Applicant: Massachusetts Institute of Technology

Inventor： Robert Samuel LANGER ,  Hao Yin ,  Daniel G. Anderson ,  Wen Xue ,  Chun-Qing Song

IPC: C12N15/113 ,  A61K31/7105 ,  C12N15/85 ,  C12N15/90 ,  C12N9/22

CPC classification number: C12N15/113 ,  A61K31/7105 ,  C12N9/22 ,  C12N15/111 ,  C12N15/85 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/343 ,  C12N2320/53 ,  C12Y301/11002 ,  C12N2310/322 ,  C12N2310/3531

Abstract: The disclosure relates to compositions comprising and methods for chemical modification of single guide RNA (sgRNA), tracrRNA and/or crRNA used individually or in combination with one another or Cas system components. Compositions comprising modified ribonucleic acids have been designed with chemical modification for even higher efficiency as unmodified native strand of sgRNA. Administration of modified ribonucleic acids will allow decreased immune response when administered to a subject, increased stability, increased editing efficiency and facilitated in vivo delivery of sgRNA via various delivery platforms. The disclosure also relates to methods of decreasing off-target effect of CRISPR and a CRISPR complex.

公开(公告)号：US11845933B2

公开(公告)日：2023-12-19

申请号：US16075568

申请日：2017-02-03

Applicant: Massachusetts Institute of Technology

Inventor： Hao Yin ,  Daniel G. Anderson ,  Robert S. Langer

IPC: C12N15/113 ,  C12N15/11 ,  C12N9/22 ,  C12N9/16 ,  C12N15/90 ,  A61K48/00 ,  C12N15/85

CPC classification number: C12N15/11 ,  A61K48/005 ,  C12N9/16 ,  C12N9/22 ,  C12N15/113 ,  C12N15/85 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2320/32 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/321 ,  C12N2310/3521

Abstract: The disclosure relates to compositions comprising and methods for chemical modification of single guide RNA (sgRNA), tracrRNA and/or crRNA used individually or in combination with one another or Cas system components. Compositions comprising modified ribonucleic acids have been designed with chemical modification for even higher efficiency as unmodified native strand of sgRNA. Administration of modified ribonucleic acids will allow decreased immune response when administered to a subject, increased stability, increased editing efficiency and facilitated in vivo delivery of sgRNA via various delivery platforms. The disclosure also relates to methods of decreasing off-target effect of CRISPR and a CRISPR complex.

公开(公告)号：US10941395B2

公开(公告)日：2021-03-09

申请号：US16029273

申请日：2018-07-06

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： Hao Yin ,  Wen Xue ,  Daniel G. Anderson ,  Joseph R. Dorkin ,  Tyler E. Jacks

IPC: C12N15/88 ,  C12N15/10 ,  C12N15/11 ,  A61K48/00 ,  C12N7/00 ,  C12N15/85 ,  C12N15/86

Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.

公开(公告)号：US20170015994A1

公开(公告)日：2017-01-19

申请号：US15121292

申请日：2015-02-24

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： Daniel G. Anderson ,  Hao Yin ,  Roman L. Bogorad ,  Tyler E. Jacks ,  Wen Xue

IPC: C12N15/10 ,  A61K31/713 ,  A61K9/00

CPC classification number: C12N15/1024 ,  A61K9/0019 ,  A61K31/713 ,  C12N15/102 ,  C12N15/63

Abstract: Methods of transfecting cells in vivo, by administering an injectable pharmaceutical composition including a genome editing composition and a pharmaceutically acceptable carrier to a subject by hydrodynamic injection into a vessel of the subject are disclosed. Typically, the pharmaceutical composition is administered in a volume and at rate of injection suitable to transfect target eukaryotic cells in the subject with an effective amount of the genome editing composition to alter the genome of the target cells. In preferred embodiments the subject is a mammal, such as rodent, or a primate such as a human. The methods can be used to treat one or more symptoms of a genetic disease or condition.

Abstract translation: 公开了通过向受试者施用包含基因组编辑组合物和药学上可接受的载体的可注射药物组合物在体内转染细胞的方法，通过流体动力学注射到受试者的血管中。 通常，药物组合物以体积和注射速率施用，适合于用有效量的基因组编辑组合物转染受试者中的靶真核细胞以改变靶细胞的基因组。 在优选的实施方案中，受试者是哺乳动物，例如啮齿动物，或灵长类动物例如人。 该方法可用于治疗遗传性疾病或病症的一种或多种症状。

公开(公告)号：US20140329884A1

公开(公告)日：2014-11-06

申请号：US14267530

申请日：2014-05-01

Applicant: Massachusetts Institute of Technology

Inventor： Yizhou Dong ,  Akinleye C. Alabi ,  Hao Yin ,  Joseph R. Dorkin ,  Delai Chen ,  Robert S. Langer ,  Daniel Griffith Anderson ,  Ahmed A. Eltoukhy

IPC: C07D251/30 ,  A61K31/713 ,  A61K31/53

CPC classification number: C07D251/30 ,  A61K31/53 ,  A61K31/713

Abstract: The present invention provides novel 1,3,5-triazinane-2,4,6-trione derivatives, such as compounds of any one of Formulae (I) and (II), and salts thereof, and methods of preparing the compounds. Also provided are compositions including a compound of the invention and an agent (e.g., an siRNA, mRNA, or plasmid DNA). The present invention also provides methods and kits using the compositions for delivering an agent to a subject (e.g., to the liver, spleen, or lung of the subject) or cell and for treating and/or preventing a range of diseases, such as genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, and lung diseases.

Abstract translation: 本发明提供新的1,3,5-三氮杂环丁烷-2,4,6-三酮衍生物，如式（I）和（II）中任一项的化合物及其盐，以及制备该化合物的方法。 还提供了包含本发明化合物和试剂（例如siRNA，mRNA或质粒DNA）的组合物。 本发明还提供了使用组合物将试剂递送至受试者（例如，受试者的肝脏，脾脏或肺）或细胞的方法和试剂盒，并用于治疗和/或预防一系列疾病，例如遗传 疾病，增殖性疾病，血液疾病，神经系统疾病，肝脏疾病和肺部疾病。

公开(公告)号：US20190144845A9

公开(公告)日：2019-05-16

申请号：US16029273

申请日：2018-07-06

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： Hao Yin ,  Wen Xue ,  Daniel G. Anderson ,  Joseph R. Dorkin ,  Tyler E. Jacks

IPC: C12N15/10 ,  C12N15/88 ,  C12N15/86 ,  C12N15/85 ,  A61K48/00 ,  C12N15/11 ,  C12N7/00

Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.

公开(公告)号：US12163124B2

公开(公告)日：2024-12-10

申请号：US17195625

申请日：2021-03-08

Applicant: Massachusetts Institute of Technology

Inventor： Hao Yin ,  Wen Xue ,  Daniel G. Anderson ,  Joseph R. Dorkin ,  Tyler E. Jacks

IPC: C12N15/10 ,  A61K48/00 ,  C12N7/00 ,  C12N15/11 ,  C12N15/85 ,  C12N15/86 ,  C12N15/88

Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.