公开(公告)号：US20240335517A1

公开(公告)日：2024-10-10

申请号：US18493262

申请日：2023-10-24

申请人： VITAMERICA UG (HAFTUNGSBESCHRANKT)

发明人： Daniel Henry WYROBNIK ,  Isaac Harry WYROBNIK

IPC分类号： A61K38/52 ,  A23L29/00 ,  A23L33/10 ,  A23L33/18 ,  A23L33/20 ,  A61K9/48 ,  A61K38/44 ,  A61K38/54 ,  A61P1/00

CPC分类号： A61K38/52 ,  A23L29/06 ,  A23L33/10 ,  A23L33/18 ,  A23L33/20 ,  A61K9/485 ,  A61K9/4858 ,  A61K38/44 ,  A61K38/54 ,  A61P1/00 ,  C12Y101/99011 ,  C12Y302/0102 ,  C12Y302/01026 ,  C12Y503/01005 ,  A23V2002/00 ,  A61K2300/00

摘要： There is provided a method for treating or reducing the effects of fructose intolerance and health problems associated with excessive fructose intake by administration of glucose isomerase. Other embodiments are also disclosed.

公开(公告)号：US11744861B2

公开(公告)日：2023-09-05

申请号：US16493853

申请日：2018-03-13

申请人： Poseida Therapeutics, Inc.

发明人： Eric M. Ostertag ,  Devon Shedlock ,  Julian David Down

IPC分类号： A61K35/17 ,  A61K9/00 ,  A61K31/664 ,  A61K31/7076 ,  A61K35/28 ,  A61K38/17 ,  A61K38/19 ,  A61K38/48 ,  A61K38/52 ,  A61K39/39 ,  A61K39/395 ,  A61K45/06 ,  C12N5/0783 ,  C12N9/12 ,  C12N9/22 ,  C12N15/11

CPC分类号： A61K35/17 ,  A61K9/0019 ,  A61K9/0085 ,  A61K31/664 ,  A61K31/7076 ,  A61K35/28 ,  A61K38/177 ,  A61K38/1774 ,  A61K38/193 ,  A61K38/4873 ,  A61K38/52 ,  A61K39/39 ,  A61K39/3955 ,  A61K45/06 ,  C12N5/0636 ,  C12N9/1241 ,  C12N9/22 ,  C12N15/11 ,  C12Y207/07 ,  C12Y304/22062 ,  C12Y502/01008 ,  C12N2310/20 ,  C12N2510/00 ,  C12N2800/80

摘要： Disclosed are methods of eliminating at least on target cell in a subject, comprising administering to the subject an effective amount of a composition comprising a plurality of immune cells, wherein each immune cell of the plurality expresses one or more chimeric ligand receptor(s) (CLR(s)) that each specifically bind to a target ligand on the at least one target cell, wherein specifically binding of the one or more CLR(s) to the target activates the immune cell, and wherein the activated immune cell induces death of the target cell. Exemplary target cells include, but are not limited to, hematopoietic stem cells (HSCs).

公开(公告)号：US20230270685A1

公开(公告)日：2023-08-31

申请号：US17989941

申请日：2022-11-18

申请人： ModernaTX, Inc.

发明人： Paolo Martini ,  Vladimir Presnyak ,  Kerry Benenato ,  Stephen Hoge ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe

IPC分类号： A61K9/51 ,  C12N9/90 ,  A61K9/127 ,  A61P3/00 ,  A61P43/00 ,  A61K38/52 ,  A61K48/00 ,  C12N15/52

CPC分类号： A61K9/5123 ,  A61K9/1272 ,  A61K38/52 ,  A61K48/005 ,  A61P3/00 ,  A61P43/00 ,  C12N9/90 ,  C12N15/52 ,  C12Y504/99002 ,  A61K38/00

摘要： The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof. In some embodiments, the disclosure includes methods of treating methylmalonic acidemia in a subject in need thereof comprising administering an mRNA encoding an MCM polypeptide.

公开(公告)号：US20210220273A1

公开(公告)日：2021-07-22

申请号：US17058334

申请日：2019-05-30

申请人： Translate Bio, Inc.

发明人： Yi Zhang ,  Shrirang Karve ,  Frank DeRosa ,  Michael Heartlein

IPC分类号： A61K9/127 ,  A61K9/00 ,  A61K39/385 ,  A61K38/45 ,  A61K38/52 ,  C07J41/00

摘要： Disclosed are cationic lipids which are compounds of Formula I. Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

公开(公告)号：US10918738B2

公开(公告)日：2021-02-16

申请号：US16233970

申请日：2018-12-27

申请人： The Regents of the University of California

发明人： H. Kirk Hammond ,  Mei Hua Gao

IPC分类号： A61K48/00 ,  A01N63/00 ,  C07K14/575 ,  C07K14/65 ,  C12N15/86 ,  A61K47/69 ,  C12N15/864 ,  A61K38/22 ,  A61K31/436 ,  A61K31/65 ,  C07K14/075 ,  C12N15/861 ,  A61K38/25 ,  A61K38/30 ,  A61K38/52

摘要： In alternative embodiments, the invention provides methods for treating, ameliorating or protecting (preventing) an individual or a patient against a disease, an infection or a condition responsive to an increased paracrine polypeptide level in vivo comprising: providing a paracrine polypeptide-encoding nucleic acid or gene operatively linked to a transcriptional regulatory sequence; or an expression vehicle, a vector, a recombinant virus, or equivalent, having contained therein a paracrine-encoding nucleic acid or gene, and the expression vehicle, vector, recombinant virus, or equivalent can express the paracrine-encoding nucleic acid or gene in a cell or in vivo; and administering or delivering the paracrine polypeptide-encoding nucleic acid or gene operatively linked to a transcriptional regulatory sequence, or the expression vehicle, vector, recombinant virus, or equivalent, to an individual or a patient in need thereof, thereby treating, ameliorating or protecting (preventing) the individual or patient against the disease, infection or condition responsive to an increased paracrine polypeptide level.

公开(公告)号：US10894077B2

公开(公告)日：2021-01-19

申请号：US16390917

申请日：2019-04-22

申请人： The United States of America, as represented by the Secretary, Dept. of Health and Human Services

发明人： Charles P. Venditti ,  Randy J. Chandler

IPC分类号： A61K38/52 ,  C12N9/90 ,  A61K48/00

摘要： Synthetic polynucleotides encoding human methylmalonyl-CoA mutase (synMUT) and exhibiting augmented expression in cell culture and/or in a subject are described herein. An adeno-associated viral (AAV) gene therapy vector encoding synMUT under the control of a liver-specific promoter (AAV2/8-HCR-hAAT-synMUT-RBG) successfully rescued the neonatal lethal phenotype displayed by methylmalonyl-CoA mutase-deficient mice, lowered circulating methylmalonic acid levels in the treated animals, and resulted in prolonged hepatic expression of the product of synMUT transgene in vivo, human methylmalonyl-CoA mutase (MUT).

公开(公告)号：US10745689B2

公开(公告)日：2020-08-18

申请号：US15215461

申请日：2016-07-20

申请人： Seattle Children's Hospital

发明人： Andrew M. Scharenberg ,  Michael T. Certo ,  Kamila Sabina Gwiazda

IPC分类号： C12N9/12 ,  C12Q1/68 ,  C12Q1/37 ,  A61K38/46 ,  C12N15/10 ,  A61K38/45 ,  A61K38/52 ,  C12N9/90 ,  C12N15/62 ,  C12N9/22 ,  A61K9/00 ,  A61K35/28 ,  A61K35/12

摘要： The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease cleavage.

公开(公告)号：US10568942B2

公开(公告)日：2020-02-25

申请号：US15269943

申请日：2016-09-19

申请人： PRO NATURA GESELLSCHAFT FUR GESUNDE ERNAHRUNG MBH

发明人： Daniel Henry Wyrobnik ,  Isaac Harry Wyrobnik ,  Elliad Ronen Silcoff

IPC分类号： A61K38/47 ,  A23L29/00 ,  A23L5/20 ,  A61K9/48 ,  A61K38/52 ,  C12Q1/32 ,  A61K38/44

摘要： 5-D-fructose dehydrogenase, optionally in combination with invertase and/or maltase and/or glucose isomerase, may be used to treat fructose intolerance. Other embodiments are also disclosed.

公开(公告)号：US20190390203A1

公开(公告)日：2019-12-26

申请号：US16565606

申请日：2019-09-10

申请人： Kelvin LEE ,  Abraham Lenhoff ,  Kristin Valente ,  Nick Levy ,  Yatin Gokarn

发明人： Kelvin LEE ,  Abraham Lenhoff ,  Kristin Valente ,  Nick Levy ,  Yatin Gokarn

IPC分类号： C12N15/113 ,  C12P21/00 ,  A61K47/26 ,  A61K38/52 ,  A61K38/48 ,  A61K38/45 ,  A61K38/44 ,  A61K38/42 ,  A61K38/39 ,  A61K38/17 ,  C12N9/20 ,  C12P21/02 ,  C07K16/00

摘要： The invention relates a method for producing a stable recombinant protein, comprising growing a non-naturally occurring host cell in a culture medium to produce a recombinant protein, and making a composition comprising the recombinant protein and a polysorbate. The production of endogenous lipoprotein lipase by the host cell is reduced. The endogenous lipoprotein lipase is present in the composition in a small amount, and is capable of degrading the polysorbate. The invention also relates to the relevant host cells and compositions, and preparation thereof.

公开(公告)号：US20190224257A1

公开(公告)日：2019-07-25

申请号：US16373388

申请日：2019-04-02

申请人： Tadashi Tanabe ,  Keiichi Hukuda ,  Takashi Kawakami

发明人： Tadashi Tanabe ,  Keiichi Hukuda ,  Takashi Kawakami

IPC分类号： A61K35/76 ,  A61K45/06 ,  A61K38/18 ,  C12N7/00 ,  A61K48/00 ,  A61K31/5585 ,  A61K38/52

摘要： Drug compositions of angiogenesis therapy contain gene coding for human prostacyclin synthase (hPGIS) synthesizing prostaglandin I2 with activities of vasodialation and/or anti-platelet aggregation; drug compositions contain adeno-associated virus (AAV) inserted with gene for angiogenesis factors. The administration of the drug compositions into the aimed treatment region results in transfer of AAV type 1-hPGIS to skeletal muscles and induces a notable expression of human PGIS gene in skeletal muscles. The PGI2 is produced by mediation of the gene expression in the muscle cells, secreted, induces vessel-protective, neovascularization and anti-platelet aggregation actions, which lead to an improvement in vascular ischemia.