公开(公告)号：US20240335539A1

公开(公告)日：2024-10-10

申请号：US18682232

申请日：2022-08-10

Applicant: The Trustees of the University of Pennsylvania

Inventor： Marco Ruella ,  Yong Gu Lee

IPC: A61K39/00 ,  A61P35/00 ,  C07K14/47 ,  C07K14/725 ,  C07K16/28 ,  C12N15/86

CPC classification number: A61K39/464412 ,  A61K39/4631 ,  A61K39/4637 ,  A61P35/00 ,  C07K14/4747 ,  C07K14/7051 ,  C07K16/2803 ,  C12N15/86 ,  C07K2317/622 ,  C07K2319/02 ,  C07K2319/30 ,  C12N2740/15043

Abstract: The present disclosure provides modified cell(s), i.e., immune cell(s) or precursor cell(s) thereof, wherein the cell(s) are engineered to express a) a chimeric antigen receptor (CAR), and b) a variant of a B-cell lymphoma 2 (Bcl-2) family protein, wherein the variant confers resistance to a cytotoxic inhibitor of the Bcl-2 family protein. Also provided are methods and uses of the modified cells, e.g., for treating at least one sign and/or symptom of cancer. Related nucleic acids, vectors, and pharmaceutical compositions are also provided.

公开(公告)号：US20240327823A1

公开(公告)日：2024-10-03

申请号：US18416749

申请日：2024-01-18

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Udit Parekh ,  Yan Wu ,  Kun Zhang

IPC: C12N15/10 ,  A61K35/44 ,  C12N5/071 ,  C12N15/86

CPC classification number: C12N15/1065 ,  A61K35/44 ,  C12N5/069 ,  C12N15/86 ,  C12N2506/45 ,  C12N2740/15043 ,  C12N2740/15052

Abstract: Understanding the complex effects of genetic perturbations on cellular state and fitness in human pluripotent stem cells (hPSCs) has been challenging using traditional pooled screening techniques which typically rely on unidimensional phenotypic readouts. Here, Applicants use barcoded open reading frame (ORF) overexpression libraries with a coupled single-cell RNA sequencing (scRNA-seq) and fitness screening approach, a technique we call SEUSS (ScalablE fUnctional Screening by Sequencing), to establish a comprehensive assaying platform. Using this system, Applicants perturbed hPSCs with a library of developmentally critical transcription factors (TFs), and assayed the impact of TF overexpression on fitness and transcriptomic cell state across multiple media conditions. Applicants further leveraged the versatility of the ORF library approach to systematically assay mutant gene libraries and also whole gene families. From the transcriptomic responses, Applicants built genetic co-perturbation networks to identify key altered gene modules. Strikingly, we found that KLF4 and SNAI2 have opposing effects on the pluripotency gene module, highlighting the power of this method to characterize the effects of genetic perturbations. From the fitness responses, Applicants identified ETV2 as a driver of reprogramming towards an endothelial-like state.

公开(公告)号：US20240325441A1

公开(公告)日：2024-10-03

申请号：US18033483

申请日：2021-10-26

Applicant: CHINEO MEDICAL TECHNOLOGY CO., LTD.

Inventor： Weiyue GU

IPC: A61K35/17 ,  A61K39/00 ,  A61P31/20 ,  C07K14/705 ,  C07K14/725 ,  C07K16/28 ,  C12N5/0783 ,  C12N15/86

CPC classification number: A61K35/17 ,  A61K39/4611 ,  A61K39/4631 ,  A61K39/464412 ,  A61P31/20 ,  C07K14/7051 ,  C07K14/70521 ,  C07K16/2818 ,  C07K16/2827 ,  C12N5/0636 ,  C12N15/86 ,  A61K2239/15 ,  C07K2317/622 ,  C07K2319/03 ,  C07K2319/33 ,  C12N2510/00 ,  C12N2740/15043

Abstract: A modified immune cell and a use thereof in immunotherapy, the immune cell being a PD-1+T cell from peripheral blood. The immunotherapy is used in cancer treatment or treatment and prevention of diseases related to viral infections.

公开(公告)号：US20240309319A1

公开(公告)日：2024-09-19

申请号：US18576712

申请日：2022-07-06

Applicant: Washington University

Inventor： Andrew Yoo ,  Lucia Capano ,  Seongwon Lee

IPC: C12N5/0793 ,  C12N15/113 ,  C12N15/86 ,  G01N33/50

CPC classification number: C12N5/0619 ,  C12N15/113 ,  C12N15/86 ,  G01N33/5058 ,  C12N2310/141 ,  C12N2501/01 ,  C12N2501/13 ,  C12N2501/385 ,  C12N2501/60 ,  C12N2501/65 ,  C12N2501/999 ,  C12N2506/1307 ,  C12N2510/00 ,  C12N2740/15043 ,  C12N2830/003

Abstract: Among the various aspects of the present disclosure is the provision of cells that mirror, recapitulate, mimick, or substantially express endogenous tau isoforms and methods of making and using same.

公开(公告)号：US20240309063A1

公开(公告)日：2024-09-19

申请号：US18502333

申请日：2023-11-06

Applicant: Consejo Nacional de Investigaciones Cientificas ,  FUNDACION ARTICULAR ,  INIS BIOTECH LLC

Inventor： Ana Romo ,  Anabela Belen La Colla ,  Matias Adan Preisegger ,  Marcela Soledad Bertolio ,  Ricardo Alfredo Dewey ,  Pamela Daiana Vazquez ,  Andrea Nancy CHISARI ,  Tania Melina RODRIGUEZ ,  Benito Jorge VELASCO ZAMORA

IPC: C07K14/71 ,  A61K9/00 ,  A61K9/51 ,  A61K38/00 ,  A61P1/16 ,  A61P35/00 ,  C07K16/00 ,  C07K16/28 ,  C12N7/00 ,  G01N33/68

CPC classification number: C07K14/71 ,  A61K9/0019 ,  A61K9/5184 ,  A61P1/16 ,  A61P35/00 ,  C07K16/2863 ,  C12N7/00 ,  G01N33/6872 ,  G01N33/6893 ,  A61K38/00 ,  C07K16/00 ,  C07K2317/34 ,  C07K2319/30 ,  C12N2740/15043 ,  C12N2740/16043 ,  G01N2333/71 ,  G01N2800/102 ,  G01N2800/56

Abstract: An isoform of the TGF beta receptor II having a sequence of about of 80 amino acids and lacking a transmembrane domain. The isoform having the amino acid sequence set forth in SEQ ID No. 12. The isoform may have the amino acid sequence set forth in SEQ ID No. 2 or sequences having at least 85% sequence identity to the sequence set forth in SEQ ID No. 2. A fusion peptide is provided having an isoform of the TGF beta II receptor fused to a ligand, wherein a vector having the fusion peptide is used to treat cancer and/or hepatic fibrosis. An antibody binding the soluble isoform of the TGF beta II receptor is provided. The antibody binds the amino acid sequence shown in SEQ ID No. 12 and is used in in vitro methods.

公开(公告)号：US20240307555A1

公开(公告)日：2024-09-19

申请号：US18667761

申请日：2024-05-17

Applicant: The Broad Institute, Inc. ,  The General Hospital Corporation

Inventor： Kepler MEARS ,  Robert MANGUSO ,  Kathleen YATES ,  Kyrellos IBRAHIM ,  Peter ALLEN

IPC: A61K48/00 ,  A61K47/68 ,  A61K47/69 ,  A61P35/00 ,  C07K14/005 ,  C12N15/86

CPC classification number: A61K48/0033 ,  A61K47/6851 ,  A61K47/6901 ,  A61P35/00 ,  C07K14/005 ,  C12N15/86 ,  C07K2319/33 ,  C12N2740/15043 ,  C12N2740/15051 ,  C12N2760/18422

Abstract: The invention features pseudotyped viral particles (e.g., lentiviral or gammaretroviral particles) and compositions and methods of use thereof, where the viral particles comprise a VHH domain.

公开(公告)号：US20240307437A1

公开(公告)日：2024-09-19

申请号：US18262365

申请日：2022-01-28

Applicant: Iovance Biotherapeutics, Inc.

Inventor： Frederick G. VOGT ,  Maria FARDIS ,  Cecile CHARTIER-COURTAUD ,  Yongliang ZHANG ,  Rafael CUBAS

IPC: A61K35/17 ,  A61K31/517 ,  A61K31/675 ,  A61K31/7076 ,  A61K38/20 ,  A61K39/00 ,  C07K14/54 ,  C07K14/55 ,  C07K16/28 ,  C12N5/0783 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

CPC classification number: A61K35/17 ,  A61K31/517 ,  A61K31/675 ,  A61K31/7076 ,  A61K38/2013 ,  A61K38/208 ,  A61K38/2086 ,  A61K39/4611 ,  A61K39/46444 ,  C07K14/5434 ,  C07K14/5443 ,  C07K14/55 ,  C07K16/2875 ,  C07K16/2878 ,  C12N5/0636 ,  C12N9/22 ,  C12N15/111 ,  C12N15/86 ,  A61K2239/13 ,  A61K2239/21 ,  A61K2239/38 ,  C12N2310/20 ,  C12N2501/2302 ,  C12N2740/15043

Abstract: Provided herein are compositions and methods for the treatment of cancers using modified TILs, wherein the modified TILs include one or more immunomodulatory agents (e.g. cytokines) associated with their cell surface. The immunomodulatory agents associated with the TILs provide a localized immunostimulatory effect that can advantageously enhance TIL survival, proliferation and/or anti-tumor activity in a patient recipient. As such, the compositions and methods disclosed herein provide effective cancer therapies.

公开(公告)号：US20240299449A1

公开(公告)日：2024-09-12

申请号：US18260377

申请日：2022-01-05

Applicant: The Regents of the University of California

Inventor： Matthew Nix ,  Arun Wiita

IPC: A61K35/17 ,  A61K39/00 ,  A61P35/02 ,  C07K14/725 ,  C07K16/28 ,  C12N15/86

CPC classification number: A61K35/17 ,  A61K39/4611 ,  A61K39/4613 ,  A61K39/4631 ,  A61K39/464411 ,  A61P35/02 ,  C07K14/7051 ,  C07K16/2851 ,  C12N15/86 ,  A61K2239/13 ,  A61K2239/21 ,  A61K2239/28 ,  A61K2239/48 ,  C07K2317/22 ,  C07K2317/24 ,  C07K2317/31 ,  C07K2317/565 ,  C07K2317/569 ,  C07K2317/92 ,  C07K2319/03 ,  C12N2740/15043

Abstract: Provided herein are anti-CD72 nanobodies and methods of using such nanobodies for diagnostic and therapeutic purposes.

公开(公告)号：US12083172B2

公开(公告)日：2024-09-10

申请号：US16953221

申请日：2020-11-19

Applicant: Duke University

Inventor： Herbert K. Lyerly ,  Takuya Osada ,  Zachary C. Hartman

IPC: A61K39/00 ,  A61K38/45 ,  A61K39/395 ,  C07H21/04 ,  C07K16/28 ,  C12N7/00

CPC classification number: A61K39/001106 ,  A61K38/45 ,  A61K39/001102 ,  A61K39/001104 ,  A61K39/3955 ,  C07H21/04 ,  C07K16/2818 ,  C12N7/00 ,  C12Y207/10001 ,  A61K2039/53 ,  C07K2317/76 ,  C12N2740/15034 ,  C12N2740/15043 ,  A61K39/0011 ,  A61K2300/00

Abstract: Methods of reducing the likelihood of a cancer or precancer developing resistance to a cancer therapeutic or prevention agent are provided herein. The methods include administering a vaccine comprising a polynucleotide encoding a polypeptide whose expression or activation is correlated with development of resistance of the cancer or precancer to the cancer therapeutic or prevention agent to a subject. The vaccine may include a polynucleotide encoding a HER2 polypeptide or a truncation, deletion or substitution mutant thereof. Methods of using the vaccine including the polynucleotide encoding the HER2 polypeptide to treat a cancer or precancer are also provided. The vaccines may be administered with a cancer therapeutic or prevention agent or a checkpoint inhibitor immunomodulatory agent.

公开(公告)号：US20240290421A1

公开(公告)日：2024-08-29

申请号：US18206981

申请日：2023-06-07

Applicant: Algen Biotechnologies, Inc.

Inventor： Spencer Charles Knight ,  Chun-Hao Huang

IPC: G16B20/00 ,  C12N5/09 ,  C12N9/22 ,  C12N15/10 ,  C12N15/11 ,  C12N15/86 ,  G06N20/00 ,  G16B40/20

CPC classification number: G16B20/00 ,  C12N5/0693 ,  C12N5/0694 ,  C12N9/22 ,  C12N15/1058 ,  C12N15/1089 ,  C12N15/1096 ,  C12N15/11 ,  C12N15/86 ,  G06N20/00 ,  G16B40/20 ,  C12N2310/20 ,  C12N2740/15043 ,  C12N2800/80

Abstract: The present disclosure provides methods and systems for identification of genomic regions for therapeutic targeting. A method for identifying one or more genomic regions for therapeutic targeting, which may facilitate re-programming of a cell from one phenotypic state to another, may comprise: providing single-cell RNA-seq data for a plurality of diseased cells and a plurality of normal cells of a cell type; mapping the single-cell RNA-seq data for the plurality of diseased cells and the plurality of normal cells into a latent space corresponding to a plurality of phenotypic states of the cell type; identifying, based at least in part on a topology of the latent space, the one or more genomic regions for therapeutic targeting; and electronically outputting the one or more genomic regions for therapeutic targeting.