公开(公告)号：US12257318B2

公开(公告)日：2025-03-25

申请号：US18431504

申请日：2024-02-02

Applicant: ReCode Therapeutics, Inc.

Inventor： Mirko Hennig ,  Daniella Ishimaru ,  David J. Lockhart ,  Michael Torres ,  Jackson Eby ,  Dmitri Boudko ,  Brandon A. Wustman

IPC: A61K48/00 ,  A61K9/00 ,  A61K9/12 ,  A61K9/127 ,  A61K9/1271 ,  A61K9/51 ,  A61K38/17

Abstract: Compositions of polynucleotide(s), pharmaceutical compositions thereof, and methods of use thereof are disclosed. A polynucleotide may encode for a cystic fibrosis transmembrane conductance regulator (CFTR) protein or a functional fragment thereof. The polynucleotide may be assembled with a lipid composition for targeted delivery to a cell or an organ, such as a lung cell or a lung of a subject. Methods for enhancing an expression or activity of CFTR protein in a cell are provided. Methods for treating a subject having or suspected of having a CFTR-associated condition are also provided.

公开(公告)号：US20250019409A1

公开(公告)日：2025-01-16

申请号：US18283523

申请日：2022-03-23

Applicant: ReCode Therapeutics, Inc.

Inventor： Mirko Hennig ,  Daniella Ishimaru ,  David J. Lockhart ,  Michael Torres ,  Jackson Eby ,  Dmitri Boudko ,  Brandon A. Wustman

IPC: C07K14/47 ,  A61K9/00 ,  A61K9/51 ,  A61K38/00 ,  A61K48/00 ,  C12N15/88

Abstract: Compositions of polynucleotide(s), pharmaceutical compositions thereof, and methods of use thereof are disclosed. A polynucleotide may encode for a cystic fibrosis transmembrane conductance regulator (CFTR) protein or a functional fragment thereof. The polynucleotide may be assembled with a lipid composition for targeted delivery to a cell or an organ, such as a lung cell or a lung of a subject. Methods for enhancing an expression or activity of CFTR protein in a cell are provided. Methods for treating a subject having or suspected of having a CFTR-associated condition are also provided.

公开(公告)号：US20240269325A1

公开(公告)日：2024-08-15

申请号：US18431504

申请日：2024-02-02

Applicant: ReCode Therapeutics, Inc.

Inventor： Mirko Hennig ,  Daniella Ishimaru ,  David J. Lockhart ,  Michael Torres ,  Jackson Eby ,  Dmitri Boudko ,  Brandon Wustman

IPC: A61K48/00 ,  A61K9/00 ,  A61K9/12 ,  A61K9/127 ,  A61K9/51 ,  A61K38/17

CPC classification number: A61K48/005 ,  A61K9/0078 ,  A61K9/12 ,  A61K9/1271 ,  A61K9/5123 ,  A61K38/1709 ,  A61K48/0075

Abstract: Compositions of polynucleotide(s), pharmaceutical compositions thereof, and methods of use thereof are disclosed. A polynucleotide may encode for a cystic fibrosis transmembrane conductance regulator (CFTR) protein or a functional fragment thereof. The polynucleotide may be assembled with a lipid composition for targeted delivery to a cell or an organ, such as a lung cell or a lung of a subject. Methods for enhancing an expression or activity of CFTR protein in a cell are provided. Methods for treating a subject having or suspected of having a CFTR-associated condition are also provided.

公开(公告)号：US20240216515A1

公开(公告)日：2024-07-04

申请号：US18283406

申请日：2022-03-21

Applicant: ReCode Therapeutics, Inc. ,  The Board of Regents of the University of Texas System

Inventor： Michael Torres ,  Daniel J. Siegwart ,  Jackson Eby ,  Rumpa Bhattacherjee ,  Brandon Wustman ,  Vladimir Kharitonov ,  Qiang Cheng ,  Tuo Wei

IPC: A61K47/54 ,  A61K47/14 ,  A61K47/24 ,  A61K47/28 ,  C12N15/113

CPC classification number: A61K47/543 ,  A61K47/14 ,  A61K47/24 ,  A61K47/28 ,  A61K47/545 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/531

Abstract: Described herein are compositions, kits, and methods for potent delivery to a ceil of a subject. The cell can be of a particular cell type, such as a basal cell. In some cases, the cell can be a lung cell of a particular cell type. Also described herein are pharmaceutical compositions comprising a therapeutic or prophylactic agent assembled with a lipid composition. Tire lipid composition can comprise an ionizable cationic lipid, and a selective organ targeting lipid. The lipid composition can further comprise a phospholipid. Further described herein are high-potency intravenous dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.

公开(公告)号：US20240309376A1

公开(公告)日：2024-09-19

申请号：US18532995

申请日：2023-12-07

Applicant: ReCode Therapeutics, Inc.

Inventor： Michael Torres ,  Dmitri Boudko ,  Ella Meleshkevitch ,  Melissa Coquelin

IPC: C12N15/113 ,  A61K9/00 ,  A61K9/51 ,  A61P11/00

CPC classification number: C12N15/113 ,  A61K9/0078 ,  A61K9/5123 ,  A61P11/00 ,  C12N2310/11 ,  C12N2310/531

Abstract: Compositions of polynucleotide(s), pharmaceutical compositions thereof, and methods of use thereof are disclosed. A polynucleotide may be or encode a synthetic transfer ribonucleic acid (tRNA). The polynucleotide may be assembled with a lipid composition for delivery to a cell or an organ, such as a lung cell or a lung of a subject. Methods for enhancing an expression or activity of cystic fibrosis transmembrane conductance regulator (CFTR) protein in a cell are provided. Methods for treating a subject having or suspected of having a CFTR-associated condition are also provided.