公开(公告)号：WO2023069522A1

公开(公告)日：2023-04-27

申请号：PCT/US2022/047138

申请日：2022-10-19

Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION

Inventor： GREENE, Arin, K.

IPC: A61B17/32 ,  A61Q19/00 ,  A61B17/54

Abstract: Devices and methods for subcutaneously abrading an inner surface of a dermis of a subject are disclosed herein. A subcutaneous abrasion device may include a handle, an elongated shaft extending distally from the handle, and an abrasive surface disposed on a distal end portion of the elongated shaft. The abrasive surface may be configured to subcutaneously abrade an inner surface of a dermis of a subject without substantially cutting or puncturing the dermis of the subject.

公开(公告)号：WO2023039594A1

公开(公告)日：2023-03-16

申请号：PCT/US2022/076342

申请日：2022-09-13

Applicant: GANNA BIO, INC. ,  THE CHILDREN'S MEDICAL CENTER CORPORATION

Inventor： BISARIA, Namita ,  FLYNN, Ryan, Alexander ,  GOODMAN, Brian ,  LAWLOR, Ciaran

IPC: A61K47/54 ,  A61P35/00 ,  A61P37/00

Abstract: The present disclosure provides methods and compositions for modulating cell surface proteins and receptor complexes using a novel class of glycan conjugates that can be used to engage the signaling pathways within desired c cell types. Such defined cell-targeting bioactive glycoligands are directed for cell engagement and activation in therapeutic applications.

公开(公告)号：WO2023027958A1

公开(公告)日：2023-03-02

申请号：PCT/US2022/040900

申请日：2022-08-19

Applicant: CHILDREN'S MEDICAL CENTER CORPORATION

Inventor： SUI, Yao ,  AFACAN, Onur ,  GHOLIPOUR-BABOLI, Ali ,  WARFIELD, Simon K.

IPC: G01R33/565 ,  G01R33/563

Abstract: Described herein are techniques to estimate motion of a subject during a magnetic resonance image (MRI) scan. The estimation may be based on one or more slices acquired during the MRI scan, permitting automated estimation of motion of the subject during the MRI scan. Some such techniques may be performed during the MRI scan, before the completion of the MRI scan, or after the MRI scan before or after magnetic resonance data (MR data) resulting from the MRI scan has been processed to generate an image from the MR data.

公开(公告)号：WO2022240787A1

公开(公告)日：2022-11-17

申请号：PCT/US2022/028432

申请日：2022-05-10

Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION

Inventor： BAUER, Daniel E.

IPC: A61K35/28 ,  C12Q1/68 ,  A61K35/12 ,  A61K48/00 ,  A61P7/00 ,  C12N5/10 ,  C12N9/22 ,  C12Q1/6883 ,  C12Q2600/156

Abstract: Provided herein are methods and compositions for improving safety and efficacy of hemoglobinopathy treatment by assessing the presence or absence of a given SNP polymorphism that affects CRISPR/Cas mediated reinduction of fetal hemoglobin (i.e., β-globin) in a cell by disrupting BCL11A expression at the genomic level.

公开(公告)号：WO2022226396A1

公开(公告)日：2022-10-27

申请号：PCT/US2022/026117

申请日：2022-04-25

Applicant: GANNA BIO, INC. ,  THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY ,  CHILDREN'S MEDICAL CENTER CORPORATION ,  BETH ISRAEL DEACONESS MEDICAL CENTER, INC.

Inventor： FLYNN, Ryan, A. ,  GOODMAN, Brian ,  LAWLOR, Ciaran ,  BISARIA, Namita ,  CUMMINGS, Richard, D. ,  WEI, Mohui ,  BERTOZZI, Carolyn, R.

IPC: A61K47/61 ,  A61K47/54

Abstract: The present disclosure relates to glyconucleic acids, such as glycoRNA and glycoDNA described herein. Provided are glycosylated ribonucleic acid (glycoRNA)-related methods and compositions.

公开(公告)号：WO2022197623A1

公开(公告)日：2022-09-22

申请号：PCT/US2022/020217

申请日：2022-03-14

Applicant: CHILDREN'S MEDICAL CENTER CORPORATION

Inventor： DONG, Min ,  LEE, Pyung-Gang

IPC: A61P17/00 ,  C07K14/33 ,  C12N9/52

Abstract: Disclosed herein are modified Clostridial Botulinum neurotoxin (BoNT) polypeptides with a modified protease domains of Clostridial Botulinum serotype Al or A2. Modifications include substitution amino acid mutations, isolated modified protease domains, chimeric molecules, pharmaceutical compositions, and methods of using the same are also disclosed.

公开(公告)号：WO2022192655A1

公开(公告)日：2022-09-15

申请号：PCT/US2022/019923

申请日：2022-03-11

Applicant: CHILDREN'S MEDICAL CENTER CORPORATION

Inventor： ZANONI, Ivan ,  BORRIELLO, Francesco ,  LEVY, Ofer ,  DOWLING, David, J.

IPC: A61K31/716 ,  A61K39/00 ,  A61K39/215 ,  A61K47/36 ,  A61K47/61 ,  A61P37/04

Abstract: Provided herein are adjuvantation systems comprising fungal polysaccharides for use in Beta coronavirus (e.g., MERS-CoV, SARS-CoV-1, or SARS-CoV-2) and influenza (influenza A virus and influenza B virus) vaccines and immunogenic compositions comprising the adjuvantation system and a Beta coronavirus or influenza virus antigen.

公开(公告)号：WO2022155335A1

公开(公告)日：2022-07-21

申请号：PCT/US2022/012311

申请日：2022-01-13

Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION

Inventor： HE, Zhigang ,  HEGARTY, Shane ,  TIAN, Feng ,  STANICKA, Joanna ,  ZHOU, Songlin

IPC: A61K31/047 ,  A61K31/33 ,  A61P25/00 ,  A61P25/28 ,  C12Q1/00

Abstract: As described below, the present invention features compositions and methods for neuroprotection and/or neuroregeneration of damaged or degenerating neurons. In various embodiments, the compositions and methods of the present disclosure are used to treat a neurodegenerative disease and/or nervous system injury. The methods in various embodiments include reducing or eliminating activity or expression of a target gene(s) and/or a polypeptide(s) expressed by a target gene(s) in a neuron.

公开(公告)号：WO2022150768A1

公开(公告)日：2022-07-14

申请号：PCT/US2022/011993

申请日：2022-01-11

Applicant: CHILDREN'S MEDICAL CENTER CORPORATION

Inventor： CAMARGO, Fernando ,  SHALHOUT, Sophia

IPC: A61K31/18 ,  A61K38/17 ,  A61K38/20 ,  A61P35/00 ,  C07K1/07

Abstract: Provided herein are compounds of Formula (I), or (II), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, and prodrugs thereof. Also provided are methods, uses, and kits involving the inventive compounds and pharmaceutical compositions thereof for treating and/or preventing diseases (e.g., proliferative diseases (e.g., cancers), diseases associated with dysregulation of YAP-TAZ transcriptional activity) in a subject. Provided are methods of inhibiting a transcription factor (e.g., TEAD) and/or inhibiting the transcription of a gene (e.g., a gene controlled or regulated by a transcription factor (e.g., TEAD)) in a subject, and/or inducing apoptosis.

公开(公告)号：WO2022098893A2

公开(公告)日：2022-05-12

申请号：PCT/US2021/058088

申请日：2021-11-04

Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION

Inventor： HE, Zhigang ,  YAO, Xue ,  ZHANG, Zicong ,  BROMMER, Benedikt

IPC: A61K48/00 ,  C12N15/86 ,  C07K14/01 ,  A61P25/00

Abstract: The present invention features adeno-associated virus (AAV) vectors, compositions thereof, and methods of use thereof for transducing neurons with injured axons.