公开(公告)号：US20220072152A1

公开(公告)日：2022-03-10

申请号：US17448996

申请日：2021-09-27

Applicant: Translate Bio, Inc.

Inventor： Kim Askew ,  Jou-Ku Chung ,  Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Thomas McCauley ,  Lianne Smith ,  Ann J. Barbier

IPC: A61K48/00 ,  C12N15/85 ,  C12N15/52 ,  A61K47/69 ,  A61K9/127 ,  A61K9/00 ,  A61K38/45 ,  A61P3/00

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

公开(公告)号：US12268754B2

公开(公告)日：2025-04-08

申请号：US17448996

申请日：2021-09-27

Applicant: Translate Bio, Inc.

Inventor： Kim Askew ,  Jou-Ku Chung ,  Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Thomas McCauley ,  Lianne Smith ,  Ann J. Barbier

IPC: A61K48/00 ,  A61K9/00 ,  A61K9/127 ,  A61K9/1272 ,  A61K38/45 ,  A61K47/69 ,  A61P3/00 ,  C12N15/52 ,  C12N15/85

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

公开(公告)号：US20250152739A1

公开(公告)日：2025-05-15

申请号：US19023768

申请日：2025-01-16

Applicant: TRANSLATE BIO, INC.

Inventor： Kim Askew ,  Jou-Ku Chung ,  Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Thomas McCauley ,  Lianne Smith ,  Ann J. Barbier

IPC: A61K48/00 ,  A61K9/00 ,  A61K9/1272 ,  A61K38/45 ,  A61K47/69 ,  A61P3/00 ,  C12N15/52 ,  C12N15/85

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

公开(公告)号：US11167043B2

公开(公告)日：2021-11-09

申请号：US16228596

申请日：2018-12-20

Applicant: Translate Bio, Inc.

Inventor： Kim Askew ,  Jou-Ku Chung ,  Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Thomas McCauley ,  Lianne Smith ,  Ann J. Barbier

IPC: A61K48/00 ,  C12N15/85 ,  C12N15/52 ,  A61K47/69 ,  A61K9/127 ,  A61K9/00 ,  A61K38/45 ,  A61P3/00

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

公开(公告)号：US20190192688A1

公开(公告)日：2019-06-27

申请号：US16228596

申请日：2018-12-20

Applicant: Translate Bio, Inc.

Inventor： Kim Askew ,  Jou-Ku Chung ,  Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Thomas McCauley ,  Lianne Smith ,  Ann J. Barbier

IPC: A61K48/00 ,  C12N15/85 ,  C12N15/52 ,  A61K47/69 ,  A61K9/127 ,  A61K9/00

CPC classification number: A61K48/005 ,  A61K9/0019 ,  A61K9/1272 ,  A61K38/45 ,  A61K47/6929 ,  A61K48/0025 ,  A61K48/0075 ,  A61K48/0083 ,  A61P3/00 ,  C12N15/52 ,  C12N15/85 ,  C12Y201/03003

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.