公开(公告)号：US12233084B2

公开(公告)日：2025-02-25

申请号：US17523060

申请日：2021-11-10

Applicant: ModernaTX, Inc.

Inventor： Stephen Hoge ,  William Issa ,  Edward J. Miracco ,  Jennifer Nelson ,  Amy E. Rabideau ,  Gabor Butora

IPC: A61K31/7105 ,  A61P31/04 ,  A61P31/12 ,  A61P33/00 ,  A61P35/00 ,  C07K14/505 ,  C12N15/10 ,  C12P19/34 ,  G01N33/68

Abstract: The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by inproved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

公开(公告)号：US12233060B2

公开(公告)日：2025-02-25

申请号：US16067994

申请日：2017-01-06

Applicant: THE INSTITUTE OF CANCER RESEARCH: ROYAL CANCER HOSPITAL ,  BREAST CANCER NOW

Inventor： Christopher James Lord ,  Chris Williamson ,  Samuel Jones

IPC: A61K31/4965 ,  A61K31/497 ,  A61K31/506 ,  A61K31/519 ,  A61K31/7105 ,  A61P35/00

Abstract: The present invention relates to Ataxia-Telangiectasia Mutated and Rad3-related protein kinase (ATR) inhibitors for use in methods of treating BAF-complex deficient cancer. The present invention further provides methods for identifying ATR inhibitors for use in the treatment of BAF complex gene mutant or deficient cancers. Medical uses and methods relating to the treatment of synovial sarcoma using ATR inhibitors are also provided.

公开(公告)号：US20250057779A1

公开(公告)日：2025-02-20

申请号：US18721678

申请日：2022-12-22

Applicant: POLITECNICO DI TORINO ,  UNIVERSITA' DEGLI STUDI DI TORINO

Inventor： Valeria Chiono ,  Clara Mattu ,  Letizia Nicoletti ,  Camilla Paoletti ,  Silvia Maria Angela Arpicco ,  Barbara Stella

IPC: A61K9/51 ,  A61K9/127 ,  A61K31/7105

Abstract: Hybrid nanoparticles consisting of a polymeric component encapsulating cationic liposomes and/or lipoplexes comprising a cationic lipid and a helper lipid are described, wherein the polymeric component constitutes at least the 80% by weight with respect to the total material constituting the nanoparticles.

公开(公告)号：US12227549B2

公开(公告)日：2025-02-18

申请号：US17359902

申请日：2021-06-28

Applicant: CureVac SE

Inventor： Mariola Fotin-Mleczek ,  Ingmar Hoerr

IPC: C07H21/02 ,  A61K31/7088 ,  A61K31/7105 ,  A61K45/06 ,  A61K48/00 ,  C07H21/04 ,  C07K14/505 ,  C12N15/113 ,  A61K38/00

Abstract: The present invention relates to an RNA encoding a therapeutic protein. In particular, the present invention relates to RNA suitable for use as a medicament. The present invention concerns such novel RNA as well as compositions and kits comprising the RNA. Furthermore, the present invention relates to the RNA, compositions or kits as disclosed herein for use as a medicament. The present invention also provides the use of the RNA, compositions or kits as disclosed herein for increasing the expression of said encoded protein, in particular in gene therapy.

公开(公告)号：US12226430B2

公开(公告)日：2025-02-18

申请号：US18501825

申请日：2023-11-03

Applicant: SNIPR Technologies Limited

Inventor： Jasper Clube ,  Morten Sommer ,  Christian Grøndahl ,  Eric Van Der Helm ,  Ruben Vazquez-Uribe

IPC: A61K31/711 ,  A01N63/00 ,  A01N63/50 ,  A01N63/60 ,  A61K31/7105 ,  A61K35/74 ,  A61K45/06 ,  A61K48/00 ,  C12N1/20 ,  C12N7/00 ,  C12N9/16 ,  C12N15/10 ,  C12N15/113 ,  C12N15/70 ,  C12N15/74 ,  C12N15/90 ,  A61K35/00

Abstract: The invention relates to methods, uses, systems, arrays, engineered nucleotide sequences and vectors for inhibiting bacterial population growth or for altering the relative ratio of sub-populations of first and second bacteria in a mixed population of bacteria. The invention is particularly useful, for example, for treatment of microbes such as for environmental, medical, food and beverage use. The invention relates inter alia to methods of controlling microbiologically influenced corrosion (MIC) or biofouling of a substrate or fluid in an industrial or domestic system.

公开(公告)号：US20250049868A1

公开(公告)日：2025-02-13

申请号：US18262759

申请日：2022-01-25

Applicant: FORDAYS CO., LTD.

Inventor： Keisuke KIRIYAMA ,  Kunio MATSUMOTO ,  Keita SUTOH ,  Katsuya SAKAI ,  Ryu IMAMURA ,  Hiroki SATO

IPC: A61K36/064 ,  A61K9/00 ,  A61K31/7105 ,  A61K47/46 ,  A61P43/00

Abstract: A drug and a health food for suppressing gene mutations using nucleic acids and RNA. The drug or the health food contains a yeast extract and one or more components derived from a plant, fish, and a dairy product. The yeast extract is present as an active ingredient and particularly contains the RNA as an active ingredient. It does not inhibit DNA damage, but promotes an action of repairing damaged DNA or promotes an action of killing cells containing damaged DNA to suppress gene mutations.

公开(公告)号：US20250049729A1

公开(公告)日：2025-02-13

申请号：US18704387

申请日：2022-10-25

Applicant: Duke University

Inventor： Ashutosh Chilkoti ,  Soumen Saha ,  Parul Sirohi ,  Angus Hucknall ,  Sonal Deshpande

IPC: A61K9/51 ,  A61K31/7105 ,  A61K31/711 ,  A61K39/215

Abstract: Disclosed herein are lipid nanoparticles including a POEGMA-lipid conjugate that can effectively encapsulate and deliver therapeutics without the immune consequences suffered by PEG-based counterparts. An example lipid nanoparticle includes an ionizable lipid, a phospholipid, a sterol, a POEGMA-lipid conjugate, and a therapeutic. Also disclosed herein are pharmaceutical compositions that include the POEGMA-based lipid nanoparticles, methods of treating a disease or disorder, and methods of delivering a therapeutic to a cell.

公开(公告)号：US20250041441A1

公开(公告)日：2025-02-06

申请号：US18751931

申请日：2024-06-24

Applicant: Beam Therapeutics Inc.

Inventor： Cory Dane Sago ,  Gregory Lawrence Hamilton

IPC: A61K47/69 ,  A61K31/7105 ,  A61K31/711 ,  A61K47/24 ,  A61K47/26 ,  A61P1/16 ,  C07C219/06 ,  C07D295/13

Abstract: The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.

公开(公告)号：US20250025505A1

公开(公告)日：2025-01-23

申请号：US18717782

申请日：2022-12-08

Applicant: BioNTech SE

Inventor： Ugur SAHIN ,  Petra OEHM ,  Benjamin RENGSTL ,  Katharina REINHARD ,  Liane Monika PREUSSNER ,  Özlem TÜRECI

IPC: A61K35/17 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7105 ,  A61K39/00 ,  A61K45/06 ,  A61P35/00 ,  C07K16/28 ,  C12N5/0783

Abstract: The present disclosure relates to chimeric antigen receptor (CAR) expressing immune effector cells showing highly specific and sensitive recognition of CLDN6 expressing cancer cells as well as a high potential for treating cancer in humans.

公开(公告)号：US20250025419A1

公开(公告)日：2025-01-23

申请号：US18762242

申请日：2024-07-02

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Zarna Patel ,  Yi Zhang ,  Ashish Sarode ,  Rebecca L. Goldman ,  Frank DeRosa ,  Michael Heartlein

IPC: A61K9/127 ,  A61K9/14 ,  A61K9/51 ,  A61K31/7105

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA at a low concentration.