公开(公告)号：US12138348B2

公开(公告)日：2024-11-12

申请号：US17676586

申请日：2022-02-21

Applicant: CureVac SE

Inventor： Fabian Johannes Eber ,  Benyamin Yazdan Panah ,  Stefanie Sewing ,  Thomas Ketterer ,  Thorsten Mutzke ,  Tilmann Roos ,  Michael Sonntag ,  Michael Wiggenhorn ,  Katharina Kolland

IPC: C07H21/04 ,  A61K9/16 ,  A61K9/19 ,  A61K31/7105 ,  A61K48/00

Abstract: The present invention is directed to a storage-stable formulation of long-chain RNA. In particular, the invention concerns a dry powder composition comprising a long-chain RNA molecule. The present invention is furthermore directed to methods for preparing a dry powder composition comprising a long-chain RNA molecule by spray-freeze drying. The invention further concerns the use of such a dry powder composition comprising a long-chain RNA molecule in the preparation of pharmaceutical compositions and vaccines, to a method of treating or preventing a disorder or a disease, to first and second medical uses of such a dry powder composition comprising a long-chain RNA molecule and to kits, particularly to kits of parts, comprising such a dry powder composition comprising a long-chain RNA molecule.

公开(公告)号：US20240366678A1

公开(公告)日：2024-11-07

申请号：US18653793

申请日：2024-05-02

Applicant: Mammoth Biosciences, Inc.

Inventor： Mark Alan DEWITT ,  Siming XU ,  William Douglass WRIGHT ,  Stepan TYMOSHENKO ,  Benjamin Julius RAUCH ,  Wiputra Jaya HARTONO ,  Aaron DELOUGHERY ,  Lucas Benjamin HARRINGTON

IPC: A61K35/28 ,  A61K31/7105 ,  A61K38/46 ,  A61K48/00 ,  C07K14/00 ,  C12N5/0789 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  C12N15/90

Abstract: Provided herein are compositions, systems, and methods for engineering hematopoietic stem cells that engineered by using polypeptides, such as effector proteins, and guide nucleic acids. Also, provided herein are the engineered hematopoietic stem cells, methods of manufacturing such hematopoietic stem cells, and methods of treating a disease or disorder using such hematopoietic stem cells.

公开(公告)号：US20240366522A1

公开(公告)日：2024-11-07

申请号：US18291710

申请日：2022-07-25

Applicant: ModernaTX, Inc.

Inventor： Michael H. Smith ,  Nimil Sood ,  Chang Tian ,  Daniel W. Doherty

IPC: A61K9/51 ,  A61K31/7105

Abstract: Provided are lipid nanoparticle compositions, and processes for their preparation, which are useful for the delivery of therapeutic or prophylactic agents to airway epithelium in patients.

公开(公告)号：US12133923B2

公开(公告)日：2024-11-05

申请号：US18413653

申请日：2024-01-16

Applicant: ReCode Therapeutics, Inc.

Inventor： Mirko Hennig ,  Ali Ahmed Alfaifi ,  Sakya Sing Mohapatra ,  Daniella Ishimaru ,  Vladimir Grigor'evich Kharitonov ,  Julia Jung-un Baek ,  Shuang Li ,  David J. Lockhart ,  Brandon A. Wustman ,  Yufeng Wang ,  Joseph S. Cefalu

IPC: A61K9/51 ,  A61K8/86 ,  A61K9/00 ,  A61K31/7105 ,  A61K47/14 ,  A61K47/18 ,  A61K47/22 ,  A61K47/24 ,  A61K47/28 ,  A61K48/00 ,  C07C69/708 ,  C07C223/02 ,  C07C321/14 ,  C07D317/32

Abstract: Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for primary ciliary dyskinesia (PCD), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject.

公开(公告)号：US12133894B2

公开(公告)日：2024-11-05

申请号：US18390700

申请日：2023-12-20

Applicant: HDT Bio Corp.

Inventor： Amit Khandhar ,  Steven Reed ,  Malcolm Duthie ,  Jesse Erasmus ,  Darrick Carter ,  Bryan J. Berube

IPC: A61K47/69 ,  A61K9/107 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7105 ,  A61K31/711 ,  A61K31/713 ,  A61K39/215 ,  A61K39/39 ,  A61K47/02 ,  A61K47/44 ,  A61K49/18 ,  A61P31/14 ,  C07K14/165 ,  C12N15/86 ,  C12N15/88 ,  A61K39/00 ,  A61K47/10 ,  A61K47/26 ,  A61K47/54 ,  B82Y5/00 ,  B82Y25/00 ,  B82Y30/00 ,  B82Y40/00

Abstract: The disclosure provides nanoemulsion compositions and methods of making and using thereof to deliver a bioactive agent such as a nucleic acid to a subject. The nanoemulsion composition comprises a hydrophobic core based on inorganic nanoparticles in a lipid nanoparticle that allows imaging as well as delivering nucleic acids. Methods of using these particles for treatment and vaccination are also provided.

公开(公告)号：US12133860B2

公开(公告)日：2024-11-05

申请号：US17266069

申请日：2019-08-08

Applicant: THERAMIR LTD

Inventor： Marianna Prokopi

IPC: A61P35/00 ,  A61K31/7105 ,  A61K45/06 ,  C12N15/113

Abstract: The present invention relates to a panel of miRNAs comprising miR-16-5p, miR-23a-5p, miR-125b-5p, miR-145-5p, miR-146a-3p, miR-181c-5p, miR-218-5p, miR-495-3p, let-7b-5p, and further comprising one or more miRNAs selected from the group consisting of miR-30a-5p, miR-30b-5p, miR-30c-5p, miR-30d-5p, miR-30e-5p, miR-194-5p, miR-302a-3p, miR-302a-5p, miR-335-3p, miR-335-5p, miR-367-3p, miR-373-3p, and miR-885-5p, and to extracellular vesicles (EVs), e.g. of stem cell origin, loaded with the panel of miRNAs. The present invention further provides the panel or EVs for use in a method of treating LCP-1 positive cancer in a patient.

公开(公告)号：US12128062B2

公开(公告)日：2024-10-29

申请号：US17258544

申请日：2019-08-14

Applicant: Ohannes Kevork Melemedjian

Inventor： Ohannes Kevork Melemedjian

IPC: C12N15/11 ,  A61K31/16 ,  A61K31/19 ,  A61K31/7004 ,  A61K31/7105 ,  A61K47/54 ,  C12N15/113

CPC classification number: A61K31/7105 ,  A61K31/16 ,  A61K31/19 ,  A61K31/7004 ,  A61K47/543 ,  A61K47/549 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/346

Abstract: Methods of alleviating or prophylactically blocking pain through the alteration of certain metabolic pathways in a subject experiencing pain or expecting to experience pain are disclosed. In particular, methods of increasing the amount of the neutral amino acid transporter ASCT2 in neurons are disclosed. Increased glutamine uptake by neurons leads to reduced extrusion of lactate and protons into the extracellular space and by extension, reduced sensitization of the neurons. Reduced sensitization of the neurons lessens the feeling of pain in the subject.

公开(公告)号：US12128061B2

公开(公告)日：2024-10-29

申请号：US16147844

申请日：2018-09-30

Applicant: Vycellix, Inc.

Inventor： Evren Alici

IPC: A61K31/7105 ,  A61K9/127 ,  A61K31/7115 ,  A61K38/20 ,  A61P35/00 ,  C07K16/28 ,  C07K16/40 ,  C12N15/117

CPC classification number: A61K31/7105 ,  A61K9/1271 ,  A61K31/7115 ,  A61K38/2013 ,  A61P35/00 ,  C07K16/28 ,  C07K16/40 ,  C12N15/117 ,  C12N2310/17 ,  C12N2310/531 ,  C12N2320/30 ,  C12N2320/31 ,  C12N2320/32 ,  A61K31/7115 ,  A61K2300/00

Abstract: The present invention comprises Rig I agonists for enhancing the effects of cytotoxic cells and stem Cells. The Rig I agonists can be used in vivo as small molecule therapeutics or in vitro to enhance cells for adoptive cell transfer. Applications include cancer therapy, immune system enhancement, chronic viral infection and treatment of viral induced inflammation and enhancement of virus based therapies.

公开(公告)号：US12122848B2

公开(公告)日：2024-10-22

申请号：US17365723

申请日：2021-07-01

Applicant: THE LUNDQUIST INSTITUTE FOR BIOMEDICAL INNOVATION AT HARBOR-UCLA MEDICAL CENTER

Inventor： Omid Khorram ,  Tsai-Der Chuang

IPC: C07K16/40 ,  A61K31/4188 ,  A61K31/4245 ,  A61K31/4439 ,  A61K31/47 ,  A61K31/7105 ,  A61P15/00 ,  A61P19/04

CPC classification number: C07K16/40 ,  A61K31/4188 ,  A61K31/4245 ,  A61K31/4439 ,  A61K31/47 ,  A61K31/7105 ,  A61P15/00 ,  A61P19/04

Abstract: The present disclosure relates generally to compositions and methods for treating fibroid in a patient in need thereof, which entails administering to the patient an effective amount of an agent that inhibits the expression or activity of TDO2 or IDO1.

公开(公告)号：US20240342207A1

公开(公告)日：2024-10-17

申请号：US18294680

申请日：2022-08-02

Applicant: BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM

Inventor： Liuqing YANG ,  Chunru LIN ,  Sergey D. EGRANOV ,  Yajuan LI

IPC: A61K31/7105 ,  A61K48/00 ,  A61P3/00 ,  C12N15/11

CPC classification number: A61K31/7105 ,  A61K48/0075 ,  A61P3/00 ,  C12N15/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3517

Abstract: This disclosure provides modified nucleic acids and compositions thereof that are able to mimic the human HULC lncRNA and/or the mouse Pair lncRNA and enhance phenylalanine hydroxylase enzyme (PAH)-substrate binding affinity, PAH-cofactor binding affinity, and/or PAH enzymatic conversion of phenylalanine to tyrosine. Also provided are methods of using the modified nucleic acids and compositions thereof to treat subjects having or suspected of having phenylketonuria and hyperphenylalaninemia.