公开(公告)号：US20210353761A1

公开(公告)日：2021-11-18

申请号：US17239131

申请日：2021-04-23

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K47/54 ,  A61K48/00 ,  A61K47/28 ,  A61K47/69 ,  A61K47/60 ,  A61P11/12 ,  A61K9/00 ,  A61K9/127 ,  A61K38/17

Abstract: The present invention provides, among other things, compositions and methods of formulating nucleic acid-containing nanoparticles comprising no more than three distinct lipids components, one distinct lipid component being a sterol-based cationic lipid. In some embodiments, the present invention provides compositions and methods in which the lipid nanoparticles further comprise helper lipids and PEG-modified lipids. The resulting formulation comprises a high encapsulation percentage for nucleic acids.

公开(公告)号：US11124804B2

公开(公告)日：2021-09-21

申请号：US16540791

申请日：2019-08-14

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Daniel Crawford ,  Shrirang Karve

IPC: C07H21/02 ,  A61K31/7115 ,  A61K31/7125 ,  C12N15/85 ,  C07K14/505 ,  C12N9/10 ,  C12N9/02 ,  C07K14/705 ,  A61K48/00 ,  A61K38/17 ,  A61K38/44 ,  A61K38/45 ,  A61K38/18 ,  C12N15/67 ,  C12P21/00 ,  A01K67/00

Abstract: The present invention provides, among other things, multimeric coding nucleic acids that exhibit superior stability for in vivo and in vitro use. In some embodiments, a multimeric coding nucleic acid (MCNA) comprises two or more encoding polynucleotides linked via 3′ ends such that the multimeric coding nucleic acid compound comprises two or more 5′ ends.

公开(公告)号：US20210261502A1

公开(公告)日：2021-08-26

申请号：US17172640

申请日：2021-02-10

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Shrirang Karve

IPC: C07C305/06 ,  A61K47/58 ,  A61K47/54 ,  A61K9/127 ,  C07C317/18 ,  A61K31/7105

Abstract: Disclosed are dendritic anionic lipids which are compounds of Formula (I): wherein R and R1 are non-polar groups, L is a linking moiety, and Dm is a dendritic moiety of m generations, each as defined herein. These dendritic anionic lipids are useful for delivery and expression of m RNA 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：US20210220449A1

公开(公告)日：2021-07-22

申请号：US17055251

申请日：2019-05-14

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Zarna Bhavsar ,  Michael Heartlein

IPC: A61K38/47 ,  A61K47/54 ,  A61K31/7105 ,  A61K9/51

Abstract: The present invention provides, among other things, methods of formulating nucleic acid-containing nanoparticles with an mRNA encoding an enzyme to afford efficient delivery of payload to a cell or tissue of interest via subcutaneous administration. The resulting payload can be efficiently delivered to the liver and other organs or tissues of a treated subject.

公开(公告)号：US20210213140A1

公开(公告)日：2021-07-15

申请号：US17058557

申请日：2019-05-29

Applicant: Translate Bio, Inc.

Inventor： Yi Zhang ,  Shrirang Karve ,  Frank DeRosa ,  Michael Heartlein

IPC: A61K47/69 ,  A61K9/127 ,  A61K47/60

Abstract: 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：US20210186890A1

公开(公告)日：2021-06-24

申请号：US17129666

申请日：2020-12-21

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Ashish Sarode ,  Natalia Vargas Montoya ,  Priyal Patel

IPC: A61K9/51 ,  A61K31/7105 ,  B82Y5/00 ,  B82Y40/00

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of combining a solution of preformed lipid nanoparticles with lipid nanoparticles encapsulating mRNA, resulting in a composition of increased potency of mRNA encoded protein expression in vivo.

公开(公告)号：US20210046192A1

公开(公告)日：2021-02-18

申请号：US16936909

申请日：2020-07-23

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Michael Heartlein ,  Natalia Vargas Montoya ,  Priyal Patel ,  Ashish Sarode

IPC: A61K48/00 ,  A61K9/107 ,  A61K47/34 ,  A61K47/32

Abstract: The present invention provides an improved compositions and processes for preparing mRNA-loaded lipid nanoparticles (mRNA-LNPs). In some embodiments, the present invention provides mRNA-LNPs with exceptional stability and is particularly useful in cases where LNPs comprising low or no PEG-modified lipids are desired.

公开(公告)号：US10808241B2

公开(公告)日：2020-10-20

申请号：US15906864

申请日：2018-02-27

Applicant: Translate Bio, Inc.

Inventor： Jonathan Abysalh ,  Daniel Crawford ,  Frank DeRosa ,  Shrirang Karve ,  Anusha Dias ,  Michael Heartlein

IPC: C12N15/10 ,  B01L3/00 ,  C12Q1/6806

Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, steps of forming an mRNA slurry, stirring the slurry, and vacuum or pressure filtering the slurry.

公开(公告)号：US10780052B2

公开(公告)日：2020-09-22

申请号：US15676570

申请日：2017-08-14

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K48/00 ,  A61K51/00 ,  C07H21/02 ,  C12N15/88 ,  A61K9/127 ,  A61K38/17 ,  A61P25/00

Abstract: The present invention provides, among other things, methods and compositions for effective delivery of messenger RNA (mRNA) to the central nervous system (CNS). In particular, the present invention provides methods and compositions for administering intrathecally to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome, such that the administering of the composition results in the intracellular delivery of mRNA in neurons in the brain and/or spinal cord. The present invention is particularly useful for the treatment of CNS diseases, disorders or conditions, such as spinal muscular atrophy.

公开(公告)号：US20200038515A1

公开(公告)日：2020-02-06

申请号：US16599928

申请日：2019-10-11

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K47/54 ,  A61K48/00 ,  A61K47/28 ,  A61K47/69 ,  A61K47/60 ,  A61P11/12 ,  A61K9/00 ,  A61K9/127 ,  A61K38/17

Abstract: The present invention provides, among other things, compositions and methods of formulating nucleic acid-containing nanoparticles comprising no more than three distinct lipids components, one distinct lipid component being a sterol-based cationic lipid. In some embodiments, the present invention provides compositions and methods in which the lipid nanoparticles further comprise helper lipids and PEG-modified lipids. The resulting formulation comprises a high encapsulation percentage for nucleic acids.