公开(公告)号：US12110499B2

公开(公告)日：2024-10-08

申请号：US16608182

申请日：2018-04-24

申请人： Seattle Children's Hospital ,  Fred Hutchinson Cancer Center

发明人： Andrew Scharenberg ,  Kyle Jacoby ,  Hans-Peter Kiem ,  David J. Rawlings ,  Christopher Lux ,  Sowmya Pattabhi ,  Olivier M. Humbert

IPC分类号： C12N15/90 ,  A61K35/28 ,  A61P9/00 ,  C07K14/805 ,  C12N15/11 ,  C12N15/86

CPC分类号： C12N15/907 ,  A61K35/28 ,  A61P9/00 ,  C07K14/805 ,  C12N15/11 ,  C12N15/86 ,  C12N2310/20 ,  C12N2750/14143 ,  C12N2800/80

摘要： The present disclosure provides improved compositions for the homology directed repair of the human globin locus for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy.

公开(公告)号：US20240218025A1

公开(公告)日：2024-07-04

申请号：US18554417

申请日：2022-04-15

申请人： Jennifer M. CHERONE ,  Alister PW FUNNELL ,  John A. ANNOPOULOS ,  Eric David HAUGEN ,  Altius Institute for Biomedical Sciences

发明人： Jennifer M. Cherone ,  Alister PW Funnell ,  John A. Stamatoyannopoulos ,  Eric David Haugen

IPC分类号： C07K14/195 ,  A61K38/00 ,  C07K14/805 ,  C12N15/86

CPC分类号： C07K14/195 ,  C07K14/805 ,  C12N15/86 ,  A61K38/00 ,  C12N2740/15043

摘要： The present disclosure provides methods and poly peptides for opening closed chromatin. The present disclosure provides methods and polypeptides for increasing expression of fetal hemoglobin G (HBG) in a cell by, for example, reducing binding of an endogenous transcription repressor to a regulatory sequence and/or potentiating binding of a transcriptional activator to a sequence in the fetal γ-globin gene promoter. The present disclosure also provides methods and DNA binding polypeptides (DBPs) for modulating expression of a gene in a cell which gene includes a regulatory region bound by a transcription factor (TF), wherein the DBP competes with the TF for binding to a regulatory sequence comprising CACC box or GATA site.

公开(公告)号：US20240131083A1

公开(公告)日：2024-04-25

申请号：US18269199

申请日：2021-12-21

申请人： Evelo Biosciences, Inc.

发明人： Alexander Bank

IPC分类号： A61K35/74 ,  A61K9/48 ,  A61K38/42 ,  C07K14/805 ,  C12N1/20 ,  G01N33/72

CPC分类号： A61K35/74 ,  A61K9/4858 ,  A61K38/42 ,  C07K14/805 ,  C12N1/205 ,  G01N33/721 ,  C12R2001/01

摘要： Methods and compositions related to pharmaceutical agents, pharmaceutical compositions, and solid dosage forms comprising animal hemoglobin and bacteria or agents of bacterial origin are provided herein.

公开(公告)号：US20230416775A1

公开(公告)日：2023-12-28

申请号：US17982209

申请日：2022-11-07

申请人： Sangamo Therapeutics, Inc.

发明人： Gregory J. Cost ,  Dmitry Guschin ,  Philip D. Gregory ,  Michael C. Holmes ,  Jeffrey C. Miller ,  David Paschon ,  Edward J. Rebar ,  Andreas Reik ,  Fyodor Urnov ,  Lei Zhang

IPC分类号： C12N15/85 ,  C12N15/52 ,  C12N9/22 ,  C07K14/805 ,  C07K14/47

CPC分类号： C12N15/85 ,  C12N15/52 ,  C07K2319/81 ,  C07K14/805 ,  C07K14/4702 ,  C12N9/22

摘要： Methods and compositions for a genetic disease are provided.

公开(公告)号：US20230340077A1

公开(公告)日：2023-10-26

申请号：US18071742

申请日：2022-11-30

申请人： Luyef Biotechnologies INC.

发明人： Stephanie Brain ,  Andrea Villanueva ,  María Soledad Gutierrez ,  Kris Blanchard ,  Pamela Barroso

IPC分类号： C07K14/805 ,  C12N15/80 ,  C12N9/02 ,  C12N9/88

CPC分类号： C07K14/805 ,  C12N15/80 ,  C12N9/0008 ,  C12Y102/01012 ,  C12N9/88 ,  C12Y401/01001

摘要： A method to express myoglobin in a fungus is described. The method includes optimizing a codon for a fungal host (e.g., Trichoderma reesei), inserting the optimized codon into a plasmid (e.g., a pTrEno plasmid or one of its derivatives), and ultimately collecting the secreted myoglobin from a feeding media in response to the myoglobin being expressed extracellularly. Optionally, the myoglobin is purified.

公开(公告)号：US11779861B2

公开(公告)日：2023-10-10

申请号：US16345676

申请日：2017-10-27

申请人： Massachusetts Institute of Technology

发明人： Xiao Su ,  Trevor Alan Hatton

IPC分类号： B01D15/38 ,  C07K1/24 ,  C12N15/10 ,  H01M4/90 ,  G01N27/327 ,  H01M8/18 ,  H01M10/36 ,  C07K14/765 ,  C07K14/805 ,  C12N9/08 ,  C12N9/22 ,  C12N9/36 ,  C12N9/76 ,  C12N13/00

CPC分类号： B01D15/3885 ,  B01D15/3804 ,  C07K1/24 ,  C07K14/765 ,  C07K14/805 ,  C12N9/0065 ,  C12N9/22 ,  C12N9/2462 ,  C12N9/6427 ,  C12N13/00 ,  C12N15/10 ,  C12N15/1003 ,  C12Y111/01007 ,  C12Y301/27005 ,  C12Y302/01017 ,  C12Y304/21001 ,  G01N27/3276 ,  G01N27/3277 ,  H01M4/90 ,  H01M8/188 ,  H01M10/36 ,  B01D15/388 ,  H01M4/9008 ,  H01M4/9083 ,  Y02E60/50 ,  C12Q2523/303 ,  C12Q2523/307

摘要： Various aspects described herein relate to electrochemical devices, e.g., for separation of one or more biomolecules from a solution, and methods of using the same. Methods for using the electrochemical devices for biocatalysis are also described herein.

公开(公告)号：US20190183982A1

公开(公告)日：2019-06-20

申请号：US16224197

申请日：2018-12-18

申请人： Milwaukee School of Engineering

发明人： Wujie Zhang ,  Rebecca Ann Schroeder ,  Sydney Jeanene Stephens ,  Haley Eleanor Stephens ,  Kellen Daniel O'Connell ,  Nataline Marie Duerig ,  Devon McCune ,  Jung Chull Lee ,  Gene A. Wright

IPC分类号： A61K38/42 ,  A61K9/00 ,  C07K14/805 ,  A61K31/732 ,  A61K31/722

CPC分类号： A61K38/42 ,  A61K9/0026 ,  A61K31/722 ,  A61K31/732 ,  C07K14/805

摘要： Disclosed herein are synthetic particles that are shaped like red blood cells. The particles include pectin and oligochitosan and optionally a bioactive agent. In addition, methods of making the synthetic particles via electrospray techniques are provided.

公开(公告)号：US20180346552A1

公开(公告)日：2018-12-06

申请号：US15995675

申请日：2018-06-01

申请人： Children's Hospital Medical Center

发明人： Punam Malik

IPC分类号： C07K14/805 ,  C12N15/86 ,  A61K48/00

CPC分类号： C07K14/805 ,  A61K48/005 ,  C12N15/86 ,  C12N2740/10043 ,  C12N2740/16043 ,  C12N2830/008 ,  C12N2830/40 ,  C12N2830/50

摘要： Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or β-thalassemia.

公开(公告)号：US10072067B2

公开(公告)日：2018-09-11

申请号：US15115530

申请日：2015-01-30

申请人： Children's Hospital Medical Center

发明人： Punam Malik

IPC分类号： C07K14/805 ,  C12N15/86

CPC分类号： C07K14/805 ,  A61K48/005 ,  C12N15/86 ,  C12N2740/10043 ,  C12N2740/16043 ,  C12N2830/008 ,  C12N2830/40 ,  C12N2830/50

摘要： Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or β-thalassemia.

公开(公告)号：US20180223311A1

公开(公告)日：2018-08-09

申请号：US15943082

申请日：2018-04-02

申请人： Sangamo Therapeutics, Inc.

发明人： Gregory J. Cost ,  Philip D. Gregory ,  Dmitry Guschin ,  Michael C. Holmes ,  Jeffrey C. Miller ,  David Paschon ,  Edward J. Rebar ,  Andreas Reik ,  Fyodor Urnov ,  Lei Zhang

IPC分类号： C12N15/85 ,  C12N9/22 ,  C07K14/47 ,  C07K14/805 ,  C12N15/52

CPC分类号： C12N15/85 ,  C07K14/4702 ,  C07K14/805 ,  C07K2319/81 ,  C12N9/22 ,  C12N15/52

摘要： Methods and compositions for a genetic disease are provided.