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：US20230145188A1

公开(公告)日：2023-05-11

申请号：US17275803

申请日：2019-09-13

Applicant: Translate Bio, Inc.

Inventor： Lianne Boeglin ,  Christian Cobaugh ,  Frank DeRosa ,  Michael Heartlein ,  Kim Askew

IPC: A61K48/00 ,  A61K9/127 ,  C12N15/88 ,  C12N15/52 ,  A61P3/00

CPC classification number: A61K48/005 ,  A61K9/1271 ,  C12N15/88 ,  C12N15/52 ,  A61P3/00 ,  C12Y504/99002

Abstract: The present invention provides, among other things, methods and compositions for treating methylmalonic academia (MMA) based on mRNA therapy. The compositions used in treatment of MMA comprise an mRNA comprising a methymalonyl-CoA mutase (MUT) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of MMA is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized MUT coding sequences are provided that can be administered without the need for modifying the nucleotides of the mRNA to achieve sustained in vivo function.

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.