公开(公告)号：US20250090526A1

公开(公告)日：2025-03-20

申请号：US18642382

申请日：2024-04-22

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC. ,  COA THERAPEUTICS, INC.

Inventor： Suzanne JACKOWSKI ,  Charles O. ROCK ,  Richard E. LEE ,  Lalit Kumar SHARMA ,  Mi Kyung YUN ,  Chitra SUBRAMANIAN ,  Rajendra P. TANGALLAPALLY ,  Anne V. EDWARDS ,  Robert ZAMBONI ,  T. Jagadeeswar REDDY ,  Jiuyu LIU

IPC: A61K31/501 ,  A61K31/197 ,  A61K31/205 ,  A61P3/00

Abstract: The present disclosure relates to methods of treating a coenzyme A reduction, elevation, sequestration, toxicity, or redistribution (CASTOR) disease such as, for example, defects in fatty acid oxidation enzymes, methylmalonic acidemia, glutaric acidemia, propionic academia, and HMG-CoA lyase, via small molecule modulators of CoA levels. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

公开(公告)号：US12053490B2

公开(公告)日：2024-08-06

申请号：US16759263

申请日：2018-10-25

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.

Inventor： Terrence L. Geiger ,  Wenting Zheng ,  Carol E. O'Hear

IPC: A61K35/17 ,  C07K14/705 ,  C07K14/725 ,  C07K16/28 ,  C12N15/00 ,  C12N15/62

CPC classification number: A61K35/17 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70578 ,  C07K16/2896 ,  C12N15/00 ,  C12N15/62 ,  C07K16/2803 ,  C07K2317/622 ,  C07K2319/03

Abstract: The application relates to a chimeric receptor that targets CD33 and allows activation of the co-stimulatory pathway without binding the natural ligand. The application also relates to polynucleotides that encode the chimeric receptor, vectors, and host cells comprising the chimeric receptor. The application also relates to methods for preparing host cells comprising a chimeric antigen receptor in order to improve the in vivo persistence of the chimeric antigen receptor host cells.

公开(公告)号：US20240139315A1

公开(公告)日：2024-05-02

申请号：US18541691

申请日：2023-12-15

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.

Inventor： Jian ZUO ,  Fei ZHENG ,  Tetsuji YAMASHITA ,  Zoe KELLARD ,  Tal TEITZ ,  Jaeki MIN ,  Taosheng CHEN

IPC: A61K39/395 ,  A61K31/18 ,  A61K31/422 ,  A61K31/44 ,  A61K31/4706 ,  A61K31/4709 ,  A61K31/506 ,  A61K31/517 ,  A61K31/519 ,  A61K31/5377 ,  A61K31/58 ,  A61K31/713 ,  A61K38/17 ,  A61K45/06 ,  A61P27/16

CPC classification number: A61K39/3955 ,  A61K31/18 ,  A61K31/422 ,  A61K31/44 ,  A61K31/4706 ,  A61K31/4709 ,  A61K31/506 ,  A61K31/517 ,  A61K31/519 ,  A61K31/5377 ,  A61K31/58 ,  A61K31/713 ,  A61K38/1709 ,  A61K45/06 ,  A61P27/16

Abstract: Methods and compositions using an inhibitor of EGFR signaling for prevention or an inhibitor of EGFR signaling and a nucleic acid molecule encoding an atonal-associated factor for treatment of hearing loss are described.

公开(公告)号：US20240132463A1

公开(公告)日：2024-04-25

申请号：US18368497

申请日：2023-09-14

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.

Inventor： Marcus FISHER ,  Fatemeh KERAMATNIA ,  Kevin MCGOWAN ,  Jaeki MIN ,  Gisele A. NISHIGUCHI ,  Jeanine PRICE ,  Zoran RANKOVIC ,  Sourav Das ,  Charles G. MULLIGHAN ,  Yunchao CHANG

IPC: C07D401/04 ,  A61K31/45 ,  A61K45/06 ,  A61P35/00 ,  A61P35/02 ,  C07D405/14 ,  C07D409/14 ,  C07D413/14

CPC classification number: C07D401/04 ,  A61K31/45 ,  A61K45/06 ,  A61P35/00 ,  A61P35/02 ,  C07D405/14 ,  C07D409/14 ,  C07D413/14

Abstract: In one aspect, the disclosure relates to substituted N-(2-(2,6-dioxopiperidinyl-3-yl)-1,3-dioxoisoindolin-5-yl)arylsulfonamide analogs that useful as modulators of cereblon (CRBN) activity, methods of making same, pharmaceutical compositions comprising same, and methods of treating various clinical conditions and disorders using same, e.g., a disorder of uncontrolled cellular proliferation, such as a cancer, which may be associated with cereblon protein dysfunction and/or a GSPT1 dysfunction. In various further aspects, the disclosed compounds can selectively modulate the degradation of GSPT1 protein, i.e., the disclosed compounds can act as GSPT1 degraders. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present disclosure.

公开(公告)号：US11963954B2

公开(公告)日：2024-04-23

申请号：US17470678

申请日：2021-09-09

Applicant: MEMORIAL SLOAN KETTERING CANCER CENTER ,  ST. JUDE CHILDREN'S RESEARCH HOSPITAL

Inventor： Jaeki Min ,  Daniel C. Scott ,  Deepak Bhasin ,  Brenda A. Schulman ,  Bhuvanesh Singh ,  Jared T. Hammill ,  R. Kiplin Guy

IPC: A61K31/4545 ,  A61K31/17 ,  A61K31/437 ,  A61K31/451 ,  A61K31/4525 ,  A61K31/454 ,  A61K31/506 ,  A61P15/16 ,  A61P31/04 ,  A61P31/12 ,  C07D211/58 ,  C07D401/04 ,  C07D401/06 ,  C07D401/12 ,  C07D405/06 ,  C07D405/12 ,  C07D409/14 ,  C07D417/04 ,  C07D417/06 ,  C07D471/04

CPC classification number: A61K31/4545 ,  A61K31/17 ,  A61K31/437 ,  A61K31/451 ,  A61K31/4525 ,  A61K31/454 ,  A61K31/506 ,  A61P15/16 ,  A61P31/04 ,  A61P31/12 ,  C07D211/58 ,  C07D401/04 ,  C07D401/06 ,  C07D401/12 ,  C07D405/06 ,  C07D405/12 ,  C07D409/14 ,  C07D417/04 ,  C07D417/06 ,  C07D471/04

Abstract: In one aspect, the invention relates to substituted 1-phenyl-3-(piperidin-4-yl)urea analogs, derivatives thereof, and related compounds, which are useful as inhibitors of the DCN1-UBC12 interaction inhibitors of DCN1-mediated cullin-RING ligase activity, methods of making same, pharmaceutical compositions comprising same, methods of treating disorders using the disclosed compounds and compositions, methods of treating disorders associated with a DCN1-UBC12 interaction dysfunction, methods of treating disorders associated with a DCN1-mediated cullin-RING ligase activity dysfunction, methods of male contraception comprising the disclosed compounds and compositions, and kits comprising the disclosed compounds and compositions. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

公开(公告)号：US20230321092A1

公开(公告)日：2023-10-12

申请号：US18076044

申请日：2022-12-06

Applicant: St. Jude Children's Research Hospital, Inc. ,  COA Therapeutics, Inc.

Inventor： Suzanne JACKOWSKI ,  Charles O. ROCK ,  Richard E. LEE ,  Lalit Kumar SHARMA ,  Mi Kyung YUN ,  Chitra SUBRAMANIAN ,  Rajendra P. TANGALLAPALLY ,  Anne V. EDWARDS ,  Robert ZAMBONI ,  T. Jagadeeswar REDDY ,  Jiuyu LIU

IPC: A61K31/501 ,  A61P3/00 ,  A61K31/197 ,  A61K31/205

CPC classification number: A61K31/501 ,  A61P3/00 ,  A61K31/197 ,  A61K31/205

Abstract: The present disclosure relates to methods of treating a coenzyme A reduction, elevation, sequestration, toxicity, or redistribution (CASTOR) disease such as, for example, defects in fatty acid oxidation enzymes, methylmalonic acidemia, glutaric acidemia, propionic academia, and HMG-CoA lyase, via small molecule modulators of CoA levels. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

公开(公告)号：US20230313140A1

公开(公告)日：2023-10-05

申请号：US17712787

申请日：2022-04-04

Applicant: St. Jude Children's Research Hospital, Inc.

Inventor： Swantje LIEDMANN ,  Ao GUO ,  Douglas R. GREEN

IPC: C12N5/0783 ,  A61K35/17 ,  A61P35/00

CPC classification number: C12N5/0638 ,  A61K35/17 ,  A61P35/00 ,  C12N2501/73 ,  C12N2510/00 ,  C12N2501/515

Abstract: Disclosed is a method for preparing T cells for adoptive T cell therapy by contacting a population of activated T cells with an Eukaryotic initiation factor-4A (eIF4A) inhibitor. Also disclosed is a kit, a population of T cells or engineered T cells produced by the method and use of the same in adoptive T cell therapy and the treatment of cancer.

公开(公告)号：US20230310559A1

公开(公告)日：2023-10-05

申请号：US18041873

申请日：2021-08-13

Applicant: St. Jude Children's Research Hospital, Inc.

Inventor： Fabio DEMONTIS ,  Mamta RAI

IPC: A61K38/47 ,  A61K31/7016 ,  C12N15/86 ,  A61P25/28

CPC classification number: A61K38/47 ,  A61K31/7016 ,  C12N15/86 ,  A61P25/28 ,  C12Y302/01001

Abstract: A method for preventing or treating a neurodegenerative disease or condition by administering to a subject in need thereof an effective amount of an amylase or maltose is provided, wherein the amylase or maltose reduces aggregation-associated or misfolded protein- associated proteotoxicity, induces transcription of chaperones and proteases, promotes degradation of proteasome substrates, or preserves protein quality under stress conditions in a subject.

公开(公告)号：US20230183748A1

公开(公告)日：2023-06-15

申请号：US17550051

申请日：2021-12-14

Applicant: St. Jude Children's Research Hospital, Inc.

Inventor： Yongqiang FENG ,  Xinying ZONG

IPC: C12N15/90 ,  A61K35/17

CPC classification number: C12N15/907 ,  A61K35/17 ,  C12N2310/20

Abstract: Disclosed are methods for modifying regulatory T cell lineage stability or induction by simultaneously modulating the activity of Foxp3 enhancers, CNS0 and CNS2 or CNS0 and CNS3. Methods for treating cancer, an autoimmune disease or condition, or a regulatory T cell-related disease or condition are also provided.

公开(公告)号：US20230181762A1

公开(公告)日：2023-06-15

申请号：US17924469

申请日：2021-05-07

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.

Inventor： Sheng ZHOU ,  Brian SORRENTINO ,  Yong CHENG

IPC: A61K48/00 ,  C12N15/86

CPC classification number: A61K48/0008 ,  C12N15/86 ,  C12N2740/15043 ,  C12N2830/48 ,  C12N2830/40

Abstract: A construct including a genomic insulator element that exhibits strong enhancer blocking activities in T lymphocytes is provided as are host cells, pharmaceutical compositions and methods of using the construct in the treatment of disease, in particular a disease to be treated with a retroviral vector-modified T lymphocyte.