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：US20190216843A1

公开(公告)日：2019-07-18

申请号：US16157050

申请日：2018-10-10

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Lianne Smith ,  Michael Heartlein ,  Braydon Charles Guild

IPC: A61K31/7105 ,  A61K48/00 ,  A61K9/14 ,  A61K9/127

CPC classification number: A61K31/7105 ,  A61K9/0019 ,  A61K9/0085 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/145 ,  A61K9/146 ,  A61K48/0025 ,  A61K48/0033

Abstract: Disclosed herein are pharmaceutical compositions that comprise “blends” of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs. The blended compositions are generally characterized as being able to efficiently deliver polynucleotides to target cells and by their ability to enhance the expression of such polynucleotides and the production of functional proteins by target cells.

公开(公告)号：US10130649B2

公开(公告)日：2018-11-20

申请号：US14775818

申请日：2014-03-14

Applicant: TRANSLATE BIO, INC.

Inventor： Frank Derosa ,  Lianne Smith ,  Michael Heartlein ,  Braydon Charles Guild

IPC: A61K9/127 ,  A61K31/7105 ,  A61K9/14 ,  A61K48/00 ,  A61K9/00

Abstract: Disclosed herein are pharmaceutical compositions that comprise “blends” of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs. The blended compositions are generally characterized as being able to efficiently deliver polynucleotides to target cells and by their ability to enhance the expression of such polynucleotides and the production of functional proteins by target cells.

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：US20190127708A1

公开(公告)日：2019-05-02

申请号：US15977756

申请日：2018-05-11

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Lianne Smith

IPC: C12N7/00 ,  C12N15/86 ,  A61K35/76 ,  A61K38/43

Abstract: The present invention provides methods for producing recombinant viral particles based on the use of exogenous mRNAs to supply various helper factors for assembly of viral particles, purified recombinant viral particles produced using such methods, and methods of using such viral particles.