-
公开(公告)号:US20240408020A1
公开(公告)日:2024-12-12
申请号:US18815955
申请日:2024-08-27
Applicant: Translate Bio, Inc.
Inventor: Braydon Charles Guild , Michael Heartlein , Frank DeRosa , Jerry Chi Zhang
IPC: A61K9/19 , A61K9/127 , A61K9/51 , A61K31/7088 , A61K38/45 , A61K38/46 , A61K38/47 , A61K38/50 , C07C323/25 , C07C323/27 , C07C323/44 , C07D233/64 , C07J41/00 , C07J43/00 , C12N15/88
Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
-
公开(公告)号:US20240299489A9
公开(公告)日:2024-09-12
申请号:US17449679
申请日:2021-10-01
Applicant: Translate Bio, Inc.
Inventor: Michael Heartlein , Frank DeRosa , Alan Kimura , Jonathan Abysalh , Anusha Dias , Shrirang Karve , Zarna Patel
CPC classification number: A61K38/1709 , A61K9/0078 , A61K9/127 , A61K48/0075 , A61P11/12 , C12N15/88
Abstract: The present invention provides, among other things, methods of treating cystic fibrosis, comprising a step of administering to a subject in need of treatment a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA encoding the CFTR protein comprises a polynucleotide sequence at least 80% identical to SEQ ID NO: 1, wherein the mRNA is at a concentration of at least 0.4 mg/mL, and wherein the step of administering comprises inhalation.
-
公开(公告)号:US12076438B2
公开(公告)日:2024-09-03
申请号:US17066919
申请日:2020-10-09
Applicant: Translate Bio, Inc.
Inventor: Christian Cobaugh , Richard Wooster , Frank DeRosa , Anusha Dias , Shrirang Karve
CPC classification number: A61K9/127 , A61K9/0019 , A61K9/007 , A61K9/0078 , A61K38/1709 , A61K38/19 , A61K38/193 , A61K38/208 , A61K45/06 , A61P35/00
Abstract: The present invention provides, among other things, methods and compositions for cancer treatment. The methods and compositions disclosed herein are particularly effective in reducing the size/volume of a tumor and inhibiting tumor growth.
-
公开(公告)号:US20240263217A1
公开(公告)日:2024-08-08
申请号:US18565849
申请日:2022-06-03
Applicant: TRANSLATE BIO, INC.
Inventor: Frank DEROSA , Anusha DIAS , Xiaobo GU
IPC: C12Q1/6825 , C12N15/10
CPC classification number: C12Q1/6825 , C12N15/10
Abstract: The invention relates to a method of quantifying capping efficiency in a sample from an in vitro transcription reaction mixture comprising a plurality of mRNA transcript, characterized by a step of contacting the mRNA transcripts with an oligonucleotide complementary to a sequence of nucleotides in the 5′ untranslated region of the mRNA transcripts to form an mRNA:DNA hybrid between the oligonucleotide and the sequence of nucleotides of the mRNA transcripts in order to release the first five, six, or seven nucleotides of the mRNA transcripts using nuclease (e.g., RNAse H) digestion.
-
公开(公告)号:US11964051B2
公开(公告)日:2024-04-23
申请号:US17054886
申请日:2019-05-15
Applicant: Translate Bio, Inc.
Inventor: Frank DeRosa , Shrirang Karve , Yi Zhang , Michael Heartlein
CPC classification number: A61K9/1272 , A61K9/0019 , A61K9/0078 , A61K38/1816 , C07H13/06
Abstract: Disclosed are cationic lipids which are compounds of Formula (I′). Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.
-
公开(公告)号:US11884692B2
公开(公告)日:2024-01-30
申请号:US17811022
申请日:2022-07-06
Applicant: Translate Bio, Inc.
Inventor: Frank DeRosa , Anusha Dias , Michael Heartlein , Shrirang Karve
CPC classification number: C07H1/06 , C07H21/00 , C07H21/02 , C12N15/1017
Abstract: The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that the precipitated mRNA is captured by a membrane; and (c) eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution. In some embodiments, a purification process involving membrane filtration suitable for the present invention is tangential flow filtration.
-
公开(公告)号:US20240018514A1
公开(公告)日:2024-01-18
申请号:US18065952
申请日:2022-12-14
Applicant: Translate Bio, Inc.
Inventor: Jonathan Abysalh , Daniel Crawford , Frank DeRosa , Anusha Dias , Michael Heartlein
IPC: C12N15/10 , C12P19/34 , C12N9/12 , G01N27/447
CPC classification number: C12N15/1096 , C12Y207/07006 , G01N27/44778 , C12N9/1247 , C12N15/1068 , C12P19/34
Abstract: The present invention provides methods for large-scale production of a composition enriched for full-length mRNA molecules using an SP6 RNA polymerase and compositions produced using such methods and uses thereof.
-
公开(公告)号:US20230372440A1
公开(公告)日:2023-11-23
申请号:US18247976
申请日:2021-10-06
Applicant: TRANSLATE BIO, INC.
Inventor: Frank DEROSA , Hardip R. GOPANI , Shrirang KARVE
CPC classification number: A61K38/1709 , A61K9/19 , A61K47/22 , A61K47/26 , A61K9/1272 , A61K47/183 , C12N15/88
Abstract: The present invention provides an improved process for preparing a stable lyophilized composition comprising lipid nanoparticles encapsulating mRNA, which results in high mRNA integrity suitable for both short- and long-term storage for therapeutic use. In some embodiments, the present invention provides a method of preparing a stable lyophilized composition comprising the steps of adding ascorbic acid to an aqueous solution comprising one or more lipid nanoparticles, freezing the aqueous solution to obtain a frozen solution, drying the frozen solution to obtain a lyophilized composition, and increasing and holding the temperature of the lyophilized composition to a temperature of between 15° C. to 30° C.
-
公开(公告)号:US20230357140A1
公开(公告)日:2023-11-09
申请号:US18246261
申请日:2021-09-22
Applicant: TRANSLATE BIO, INC.
Inventor: Shrirang KARVE , Frank DEROSA
IPC: C07C311/32 , A61K48/00 , A61K9/127 , A61K9/107 , A61K9/00
CPC classification number: C07C311/32 , A61K48/0033 , A61K9/1271 , A61K9/1075 , A61K9/007
Abstract: The present invention provides, in part, TES-based lipid compounds of Formula (Ia), and sub-formulas thereof: or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.
-
公开(公告)号:US20230302039A1
公开(公告)日:2023-09-28
申请号:US18049210
申请日:2022-10-24
Applicant: Translate Bio, Inc. , Ethris GmbH
Inventor: Michael Heartlein , Braydon Charles Guild , Frank DeRosa , Carsten Rudolph , Christian Plank , Lianne Smith
IPC: A61K31/713 , A61K31/7105 , A61K48/00 , A61K31/7115 , A61K9/00 , A61K9/51 , A61K47/69 , C07K14/705 , A61K9/127 , C12N9/14
CPC classification number: A61K31/713 , A61K31/7105 , A61K48/005 , A61K31/7115 , A61K9/0078 , A61K9/5123 , A61K47/6935 , C07K14/705 , A61K9/0073 , A61K9/1271 , C12N9/14 , C12N15/63
Abstract: Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.
-
-
-
-
-
-
-
-
-