公开(公告)号：US11802278B2

公开(公告)日：2023-10-31

申请号：US18045650

申请日：2022-10-11

Applicant: 4D MOLECULAR THERAPEUTICS INC.

Inventor： David H. Kirn ,  Melissa Kotterman ,  Peter Francis ,  David Schaffer ,  Paul Szymanski ,  Kevin Whittlesey

IPC: C12N9/40 ,  A61K9/00 ,  A61P3/00 ,  C12N15/86 ,  A61K48/00

CPC classification number: C12N9/2465 ,  A61K9/0019 ,  A61K48/0066 ,  A61K48/0075 ,  A61K48/0083 ,  A61P3/00 ,  C12N15/86 ,  C12Y302/01022 ,  A61K48/00 ,  C12N2840/00

Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.

公开(公告)号：US20180055950A1

公开(公告)日：2018-03-01

申请号：US14608153

申请日：2015-01-28

Applicant: The Regents of the University of California

Inventor： Brian P. Head ,  Piyush M. Patel ,  Hemal Patel ,  David M. Roth

IPC: A61K48/00 ,  C12N15/86 ,  A61K38/17 ,  A61K31/7088

CPC classification number: A61K48/00 ,  A61K31/7088 ,  A61K38/17 ,  A61K38/177 ,  A61K48/0058 ,  C12N15/86 ,  C12N2740/16043 ,  C12N2830/002 ,  C12N2830/48 ,  C12N2840/00

Abstract: The invention provides an expression system for producing Caveolin-1 in neuronal cells or neural stem cells comprising a neuron-specific regulatory element and a nucleic acid sequence encoding Caveolin-1.

公开(公告)号：US20150064150A1

公开(公告)日：2015-03-05

申请号：US14488058

申请日：2014-09-16

Applicant: BLUEBIRD BIO, INC.

Inventor： Maria Joann DENARO ,  Mitchell Howard FINER ,  Gabor VERES ,  Julian DOWN

IPC: A61K35/48 ,  A61K35/28

CPC classification number: A61K35/51 ,  A61K35/28 ,  A61K38/177 ,  A61K48/005 ,  A61K48/0058 ,  C07K14/705 ,  C12N5/0686 ,  C12N7/00 ,  C12N15/86 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2800/24 ,  C12N2830/00 ,  C12N2830/008 ,  C12N2830/50 ,  C12N2830/60 ,  C12N2840/00

Abstract: The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.

公开(公告)号：US20140135305A1

公开(公告)日：2014-05-15

申请号：US14104699

申请日：2013-12-12

Applicant: PTC THERAPEUTICS, INC.

Inventor： Sergey V. Paushkin ,  Charles Romfo ,  Ellen Welch ,  Nikolai Naryshkin

IPC: C07D498/04 ,  C07D413/06 ,  C07D413/12 ,  C07D491/113 ,  C07D413/14 ,  C07D263/57 ,  C07D413/04

CPC classification number: C07D498/04 ,  C07D263/57 ,  C07D413/04 ,  C07D413/06 ,  C07D413/12 ,  C07D413/14 ,  C07D491/113 ,  C07K14/475 ,  C07K2319/61 ,  C12N2840/00 ,  C12Q1/6883 ,  C12Q2600/136 ,  C12Q2600/158

Abstract: The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.

Abstract translation: 本发明提供核酸构建体，用于鉴定和验证增加SMN2外显子7包含在从SMN2基因转录的mRNA中的化合物的方法，提高从SMN2基因产生的SMN蛋白质水平的化合物和药物组合物，以及用于 其用于治疗SMA。

公开(公告)号：US20110003884A1

公开(公告)日：2011-01-06

申请号：US12526140

申请日：2008-02-11

Applicant: Konstantin V. Pugachev ,  Qing-sheng Gao ,  Alexander A. Rumyantsev

Inventor： Konstantin V. Pugachev ,  Qing-sheng Gao ,  Alexander A. Rumyantsev

IPC: A61K31/7088 ,  C12N15/63

CPC classification number: C12N15/86 ,  A61K39/12 ,  A61K39/145 ,  A61K2039/5256 ,  A61K2039/53 ,  C07K2319/03 ,  C07K2319/50 ,  C12N2760/16134 ,  C12N2760/16171 ,  C12N2770/24143 ,  C12N2840/00 ,  Y02A50/386 ,  Y02A50/388 ,  Y02A50/39 ,  Y02A50/396

Abstract: This invention relates to viral vectors and methods employing these vectors. The vectors of the invention can be base on flaviviruses, such as chimeric flavi viruses, which may be used to deliver heterologous antigens, such as influenza virus antigens.

Abstract translation: 本发明涉及使用这些载体的病毒载体和方法。 本发明的载体可以基于黄病毒，例如嵌合黄病毒，其可用于递送异源抗原，例如流感病毒抗原。

公开(公告)号：US20240018498A1

公开(公告)日：2024-01-18

申请号：US18463979

申请日：2023-09-08

Applicant: 4D MOLECULAR THERAPEUTICS INC.

Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  Peter FRANCIS ,  David SCHAFFER ,  Paul SZYMANSKI ,  Kevin WHITTLESEY

IPC: C12N9/40 ,  A61K9/00 ,  A61P3/00 ,  C12N15/86 ,  A61K48/00

CPC classification number: C12N9/2465 ,  C12Y302/01022 ,  A61K9/0019 ,  A61P3/00 ,  C12N15/86 ,  A61K48/0066 ,  A61K48/0075 ,  A61K48/0083 ,  C12N2840/00 ,  A61K48/00

Abstract: The present disclosure provides codon optimized nucleotide sequences encoding hum n alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.

公开(公告)号：US20180170990A1

公开(公告)日：2018-06-21

申请号：US15662090

申请日：2017-07-27

Applicant: ModernaTX, Inc.

Inventor： Stephane BANCEL ,  Tirtha CHAKRABORTY ,  Antonin DE FOUGEROLLES ,  Sayda M. ELBASHIR ,  Matthias JOHN ,  Atanu ROY ,  Susan WHORISKEY ,  Paul HATALA ,  Jason P. SCHRUM ,  Kenechi EJEBE ,  Jeff Lynn ELLSWORTH ,  Justin GUILD ,  Ron WEISS ,  Kristy M. WOOD

IPC: C07K14/535 ,  A61K48/00 ,  C07K14/745 ,  C07K16/28 ,  C07K16/32 ,  C12N9/02 ,  C12N9/64 ,  A61K38/19 ,  A61K38/21 ,  A61K38/36 ,  A61K38/44 ,  A61K38/48 ,  A61K39/395 ,  C07K14/475 ,  C07K14/505 ,  C07K14/525 ,  C07K14/56 ,  C07K14/565 ,  C07K14/75 ,  A61K47/10 ,  A61K38/18 ,  A61K38/17 ,  A61K9/14 ,  C07K14/47 ,  C12N15/85 ,  A61K9/50 ,  A61K9/127

CPC classification number: C07K14/535 ,  A61K9/0019 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/1277 ,  A61K9/14 ,  A61K9/5031 ,  A61K31/7088 ,  A61K38/1767 ,  A61K38/1816 ,  A61K38/1866 ,  A61K38/191 ,  A61K38/193 ,  A61K38/212 ,  A61K38/215 ,  A61K38/36 ,  A61K38/363 ,  A61K38/44 ,  A61K38/4833 ,  A61K38/4846 ,  A61K39/3955 ,  A61K47/10 ,  A61K47/54 ,  A61K47/542 ,  A61K48/00 ,  A61K48/0033 ,  A61K48/0066 ,  A61K48/0075 ,  C07K14/47 ,  C07K14/475 ,  C07K14/505 ,  C07K14/525 ,  C07K14/56 ,  C07K14/565 ,  C07K14/745 ,  C07K14/75 ,  C07K16/2887 ,  C07K16/32 ,  C07K19/00 ,  C12N9/0069 ,  C12N9/644 ,  C12N15/85 ,  C12N15/88 ,  C12N2840/00 ,  C12Y113/12007 ,  C12Y304/21005 ,  C12Y304/21022

Abstract: The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.

公开(公告)号：US09782462B2

公开(公告)日：2017-10-10

申请号：US15060707

申请日：2016-03-04

Applicant: Moderna Therapeutics, Inc.

Inventor： Stephane Bancel ,  Tirtha Chakraborty ,  Antonin de Fougerolles ,  Sayda M. Elbashir ,  Matthias John ,  Atanu Roy ,  Susan Whoriskey ,  Kristy M. Wood ,  Paul Hatala ,  Jason P. Schrum ,  Kenechi Ejebe ,  Jeff Lynn Ellsworth ,  Justin Guild

IPC: A61K48/00 ,  A61K38/48 ,  C12N15/88 ,  A61K9/127 ,  A61K9/50 ,  A61K31/7088 ,  A61K47/48 ,  C07K19/00 ,  C12N15/85 ,  A61K38/18 ,  A61K38/19 ,  C07K14/47 ,  A61K9/00

CPC classification number: C07K14/535 ,  A61K9/0019 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/1277 ,  A61K9/14 ,  A61K9/5031 ,  A61K31/7088 ,  A61K38/1767 ,  A61K38/1816 ,  A61K38/1866 ,  A61K38/191 ,  A61K38/193 ,  A61K38/212 ,  A61K38/215 ,  A61K38/36 ,  A61K38/363 ,  A61K38/44 ,  A61K38/4833 ,  A61K38/4846 ,  A61K39/3955 ,  A61K47/10 ,  A61K47/54 ,  A61K47/542 ,  A61K48/00 ,  A61K48/0033 ,  A61K48/0066 ,  A61K48/0075 ,  C07K14/47 ,  C07K14/475 ,  C07K14/505 ,  C07K14/525 ,  C07K14/56 ,  C07K14/565 ,  C07K14/745 ,  C07K14/75 ,  C07K16/2887 ,  C07K16/32 ,  C07K19/00 ,  C12N9/0069 ,  C12N9/644 ,  C12N15/85 ,  C12N15/88 ,  C12N2840/00 ,  C12Y113/12007 ,  C12Y304/21005 ,  C12Y304/21022

Abstract: The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.

公开(公告)号：US20160215270A1

公开(公告)日：2016-07-28

申请号：US15023484

申请日：2014-09-24

Applicant: ID PHARMA CO., LTD.

Inventor： Hiroshi BAN ,  Akihiro IIDA ,  Mamoru HASEGAWA

IPC: C12N5/074 ,  A61K35/545 ,  C12N7/00

CPC classification number: C12N5/0696 ,  A61K35/545 ,  C12N7/00 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2506/09 ,  C12N2510/00 ,  C12N2523/00 ,  C12N2760/18843 ,  C12N2830/00 ,  C12N2840/00

Abstract: The present invention provides methods for improving the efficiency of inducing pluripotent stem cells, as well as vectors and compositions for use therein. In the induction of pluripotent stem cells which contains the step of introducing a vector that contains the KLF gene, OCT gene, and SOX gene in this order, the efficiency of pluripotent stem cell induction was successfully increased significantly by further introducing a vector that contains the KLF gene but not the OCT gene and the SOX gene. The methods of the present invention have an excellent feature in that they allow efficient induction of pluripotent stem cells under a temperature condition closer to the physiological environment, and prompt vector removal after the pluripotent stem cell induction. The present invention enables more efficient induction of pluripotent stem cells.

Abstract translation: 本发明提供了提高诱导多能干细胞的效率的方法，以及用于其中的载体和组合物。 在诱导含有KLF基因，OCT基因和SOX基因的载体的多能干细胞的诱导中，多能干细胞诱导的效率通过进一步引入含有 KLF基因，但不是OCT基因和SOX基因。 本发明的方法具有优异的特征，它们允许在更接近生理环境的温度条件下有效诱导多能干细胞，并在多能干细胞诱导后迅速进行载体移除。 本发明能够更有效地诱导多能干细胞。

公开(公告)号：US20160199513A1

公开(公告)日：2016-07-14

申请号：US15060707

申请日：2016-03-04

Applicant: Moderna Therapeutics, Inc.

Inventor： Stephane Bancel ,  Tirtha Chakraborty ,  Antonin de Fougerolles ,  Sayda M. Elbashir ,  Matthias John ,  Atanu Roy ,  Susan Whoriskey ,  Kristy M. Wood ,  Paul Hatala ,  Jason P. Schrum ,  Kenechi Ejebe ,  Jeff Lynn Ellsworth ,  Justin Guild

IPC: A61K48/00

CPC classification number: C07K14/535 ,  A61K9/0019 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/1277 ,  A61K9/14 ,  A61K9/5031 ,  A61K31/7088 ,  A61K38/1767 ,  A61K38/1816 ,  A61K38/1866 ,  A61K38/191 ,  A61K38/193 ,  A61K38/212 ,  A61K38/215 ,  A61K38/36 ,  A61K38/363 ,  A61K38/44 ,  A61K38/4833 ,  A61K38/4846 ,  A61K39/3955 ,  A61K47/10 ,  A61K47/54 ,  A61K47/542 ,  A61K48/00 ,  A61K48/0033 ,  A61K48/0066 ,  A61K48/0075 ,  C07K14/47 ,  C07K14/475 ,  C07K14/505 ,  C07K14/525 ,  C07K14/56 ,  C07K14/565 ,  C07K14/745 ,  C07K14/75 ,  C07K16/2887 ,  C07K16/32 ,  C07K19/00 ,  C12N9/0069 ,  C12N9/644 ,  C12N15/85 ,  C12N15/88 ,  C12N2840/00 ,  C12Y113/12007 ,  C12Y304/21005 ,  C12Y304/21022

Abstract: The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.

Abstract translation: 本发明涉及用于制备，制造和治疗性使用多核苷酸，初级转录物和mmRNA分子的组合物和方法。