公开(公告)号：US20220226289A1

公开(公告)日：2022-07-21

申请号：US16759715

申请日：2018-10-26

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

Inventor： Joseph Paul Taylor

IPC: A61K31/4184 ,  A61P21/00

Abstract: Methods and formulations are provided for treating spinobulbar muscular atrophy in a subject in need thereof. By administering a therapeutically effective amount of a selective androgen receptor modulator or a small molecule such as 1-[2-(4-methylphenoxy)ethyl]-2-[(2-phenoxyethyl)sulfanyl]-1H-benzimidazole or a derivative, prodrug, or pharmaceutically acceptable salt thereof, one or more symptoms of spinobulbar muscular atrophy can be ameliorated. The effective amount can be effective to prevent or delay loss of body weight, a loss of mobility, and/or a loss of physical strength in the subject; to prevent or delay neurogenic atrophy and/or to prevent a loss of spinal cord motor neurons in the subject; to restore the frequency of type I myofibers to normal levels for a healthy subject; and/or to reverse testicular atrophy in the subject.

公开(公告)号：US20220195007A1

公开(公告)日：2022-06-23

申请号：US17602892

申请日：2020-04-10

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

Inventor： Mireya Paulina VELASQUEZ ,  Stephen GOTTSCHALK ,  Janice RIBERDY ,  Albert ZHOU ,  Byoung RYU ,  Abishek VAIDYA ,  Giedre KRENCIUTE

IPC: C07K14/725 ,  A61P35/02 ,  C07K14/705 ,  C12N15/86 ,  C12N5/0783

Abstract: The present disclosure provides chimeric antigen receptors (CARs), particularly CARs that have enhanced antitumor properties and/or can be regulated by safety switches. Also provided are polypeptides of the CARs and other related molecules, polynucleotides, vectors, and cell compositions comprising the same. Pharmaceutical compositions comprising the polypeptides, polynucleotides, vectors, or cells of the present disclosure, and their uses in treating a cancer in a subject are also provided.

公开(公告)号：US20210252058A1

公开(公告)日：2021-08-19

申请号：US17056181

申请日：2019-05-17

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC. ,  BAYLOR COLLEGE OF MEDICINE

Inventor： Stephen GOTTSCHALK ,  Brooke PRINZING ,  Giedre KRENCIUTE

IPC: A61K35/17 ,  C07K14/705 ,  C07K14/725 ,  A61P35/00 ,  C07K16/28

Abstract: The application relates to a chimeric antigen receptor that targets Eph receptors and allows activation of co-stimulatory pathways. The application also relates to polynucleotides that encode the chimeric antigen receptor, vectors, and host cells comprising the chimeric antigen receptor. The application also relates to methods for preparing host cells comprising a chimeric antigen receptor in order to improve the in vivo effector function of the chimeric antigen receptor host cells.

公开(公告)号：US20210115404A1

公开(公告)日：2021-04-22

申请号：US17067016

申请日：2020-10-09

Applicant: St. Jude Children's Research Hospital, Inc. ,  National University of Singapore

Inventor： Dario CAMPANA ,  Yu-Hsiang Chang

IPC: C12N5/0783 ,  A61K38/00 ,  C07K14/725 ,  C07K14/705 ,  A61K35/17 ,  C12N5/00

Abstract: The invention provides a chimeric receptor comprising NKG2D, DAP10 and CD3 zeta. Also disclosed is a composition comprising this chimeric receptor and methods for making and using it to enhance the cytotoxicity and antitumor capacity of NK cells. The invention also encompansses methods for use of NKG2D-DAP10-CD3 zeta polypeptides, vectors and cells in methods for treating cancer and other proliferative disorders, as well as infectious diseases.

公开(公告)号：US20210023081A1

公开(公告)日：2021-01-28

申请号：US16957996

申请日：2018-12-26

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 ,  A61K31/205 ,  A61K31/197 ,  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：US10829737B2

公开(公告)日：2020-11-10

申请号：US15857268

申请日：2017-12-28

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC. ,  NATIONAL UNIVERSITY OF SINGAPORE

Inventor： Dario Campana ,  Yu-Hsiang Chang

IPC: C12N5/0783 ,  A61K38/00 ,  C07K14/725 ,  C07K14/705 ,  A61K35/17 ,  C12N5/00

Abstract: The invention provides a chimeric receptor comprising NKG2D, DAP10 and CD3 zeta. Also disclosed is a composition comprising this chimeric receptor and methods for making and using it to enhance the cytotoxicity and antitumor capacity of NK cells. The invention also encompasses methods for use of NKG2D-DAP10-CD3 zeta polypeptides, vectors and cells in methods for treating cancer and other proliferative disorders, as well as infectious diseases.

公开(公告)号：US20200016189A1

公开(公告)日：2020-01-16

申请号：US16584338

申请日：2019-09-26

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

Inventor： Stanislav S. ZAKHARENKO

IPC: A61K31/713 ,  C12N15/113 ,  C12Q1/6883 ,  A61K9/00

Abstract: The invention is directed to a method for treating the 22q11 deletion syndrome (22q11 DS) and schizophrenia (SCZ) by replenishment of decreased levels of miR-338-3p in thalamic neurons.

公开(公告)号：US20190376037A1

公开(公告)日：2019-12-12

申请号：US16550548

申请日：2019-08-26

Applicant: National University of Singapore ,  St. Jude Children's Research Hospital, Inc.

Inventor： Dario Campana ,  David Shook ,  Masaru Imamura

IPC: C12N5/0783 ,  A61K35/17 ,  A61K38/20 ,  C07K14/54 ,  C07K14/705

Abstract: The present invention provides, in certain aspects, a natural killer (NK) cell that expresses all or a functional portion of interleukin-15 (IL-15), and methods for producing such cells. The invention further provides methods of using a natural killer (NK) cell that expresses all or a functional portion of interleukin-15 (IL-15) to treat cancer in a subject or to enhance expansion and/or survival of NK cells.

公开(公告)号：US20180135016A1

公开(公告)日：2018-05-17

申请号：US15857315

申请日：2017-12-28

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC. ,  NATIONAL UNIVERSITY OF SINGAPORE

Inventor： Dario Campana ,  Yu-Hsiang Chang

IPC: C12N5/0783 ,  C07K14/705 ,  C12N5/00 ,  A61K35/17 ,  C07K14/725

CPC classification number: C12N5/0646 ,  A61K35/17 ,  A61K38/00 ,  C07K14/705 ,  C07K14/7051 ,  C07K14/7056 ,  C07K2319/00 ,  C07K2319/70 ,  C12N5/0087 ,  C12N2510/00

Abstract: The invention provides a chimeric receptor comprising NKG2D, DAP10 and CD3 zeta. Also disclosed is a composition comprising this chimeric receptor and methods for making and using it to enhance the cytotoxicity and antitumor capacity of NK cells. The invention also encompasses methods for use of NKG2D-DAP10-CD3 zeta polypeptides, vectors and cells in methods for treating cancer and other proliferative disorders, as well as infectious diseases.

公开(公告)号：US09856322B2

公开(公告)日：2018-01-02

申请号：US14303331

申请日：2014-06-12

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

Inventor： Dario Campana ,  Chihaya Imai

IPC: C07K16/28 ,  C07K14/705 ,  C12N5/22 ,  C12N5/0783 ,  A61K39/00 ,  C07K14/725

CPC classification number: C07K16/2896 ,  A61K39/0011 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70578 ,  C07K16/2803 ,  C07K16/2866 ,  C07K16/2878 ,  C07K2317/569 ,  C07K2317/622 ,  C07K2319/00 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/30 ,  C12N5/0646 ,  C12N2501/2302 ,  C12N2501/2315 ,  C12N2501/599 ,  C12N2502/99 ,  C12N2510/00

Abstract: The present invention relates to a chimeric receptor capable of signaling both a primary and a co-stimulatory pathway, thus allowing activation of the co-stimulatory pathway without binding to the natural ligand. The cytoplasmic domain of the receptor contains a portion of the 4-1BB signaling domain. Embodiments of the invention relate to polynucleotides that encode the receptor, vectors and host cells encoding a chimeric receptor, particularly including T cells and natural killer (NK) cells and methods of use.