公开(公告)号：WO2023086465A1

公开(公告)日：2023-05-19

申请号：PCT/US2022/049537

申请日：2022-11-10

Applicant: MODERNATX, INC.

Inventor： CADETE PIRES, Ana ,  SUNG, Jean C. ,  CORNEBISE, Mark ,  HRKACH, Jeffrey

IPC: A61K9/51 ,  A61K31/7105 ,  C12N15/00 ,  A61P1/00 ,  A61P1/16 ,  A61P1/18 ,  A61P11/00 ,  A61P13/12 ,  A61P43/00

Abstract: The present disclosure provides LNPs comprising payload molecules, e.g., mRNA therapeutics, for the treatment of diseases or disorders, which would benefit from delivery of payload molecules to airway cells.

公开(公告)号：WO2022032154A2

公开(公告)日：2022-02-10

申请号：PCT/US2021/045038

申请日：2021-08-06

Applicant: MODERNATX, INC.

Inventor： CADETE PIRES, Ana ,  SUNG, Jean C. ,  CORNEBISE, Mark ,  HRKACH, Jeffrey

IPC: A61K9/127 ,  A61K47/18 ,  A61K47/60 ,  A61K9/51 ,  A61K31/7088 ,  A61K38/00 ,  A61K9/1273 ,  A61K9/5123 ,  C07J9/00

Abstract: The present disclosure provides LNPs comprising payload molecules, e.g., mRNA therapeutics, for the treatment of diseases or disorders, which would benefit from delivery of payload molecules to airway cells.

公开(公告)号：WO2023076605A1

公开(公告)日：2023-05-04

申请号：PCT/US2022/048231

申请日：2022-10-28

Applicant: MODERNATX, INC.

Inventor： SEEPERSAUD, Mohindra ,  CRAWFORD, Matthew ,  TATE, Daniel ,  CORNEBISE, Mark ,  MCLAUGHLIN, Christopher Karl ,  CADETE PIRES, Ana

IPC: C07J41/00 ,  A61K48/00 ,  C07J43/00

Abstract: The present invention relates to lipid amine compounds of formula (A1) which are useful in the preparation of lipid nanoparticle compositions for delivery of therapeutic or prophylactic payload into cells.

公开(公告)号：WO2022104131A1

公开(公告)日：2022-05-19

申请号：PCT/US2021/059231

申请日：2021-11-12

Applicant: MODERNATX, INC.

Inventor： SUNG, Jean C. ,  REID, David ,  BICKNELL, Alicia Anne ,  CADETE PIRES, Ana ,  HRKACH, Jeffrey

IPC: A61K9/127 ,  A61K38/17 ,  A61K48/00 ,  A61P11/00 ,  C07K14/47

Abstract: This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA or gene editing therapeutics for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs) of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects. Nucleic acid therapeutics (e.g., mRNAs) of the disclosure further decrease abnormal accumulation of ammonia associated with deficient CFTR activity in subjects.