公开(公告)号：US20210261502A1

公开(公告)日：2021-08-26

申请号：US17172640

申请日：2021-02-10

申请人： Translate Bio, Inc.

发明人： Michael Heartlein ,  Frank DeRosa ,  Shrirang Karve

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

摘要： 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：US11090264B2

公开(公告)日：2021-08-17

申请号：US16258191

申请日：2019-01-25

申请人： Translate Bio, Inc. ,  Ethris GmbH

发明人： Michael Heartlein ,  Braydon Charles Guild ,  Frank DeRosa ,  Carsten Rudolph ,  Christian Plank

IPC分类号： A61K9/127 ,  A61K9/00 ,  C12N15/88 ,  A61K31/7088 ,  A61M15/00 ,  A61M16/14 ,  A61K48/00

摘要： Compositions comprising mRNA formulated for pulmonary administration and related method for delivery of the mRNA and/or encoded protein to a non-lung cell or tissue. The compositions and methods may be used to prevent or ameliorate the symptoms of diseases associated with the mRNA encoded protein.

公开(公告)号：US20210220449A1

公开(公告)日：2021-07-22

申请号：US17055251

申请日：2019-05-14

申请人： Translate Bio, Inc.

发明人： Shrirang Karve ,  Frank DeRosa ,  Zarna Bhavsar ,  Michael Heartlein

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

摘要： 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

申请人： Translate Bio, Inc.

发明人： Yi Zhang ,  Shrirang Karve ,  Frank DeRosa ,  Michael Heartlein

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

摘要： 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

申请人： Translate Bio, Inc.

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

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

摘要： 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：US20210145860A1

公开(公告)日：2021-05-20

申请号：US17076195

申请日：2020-10-21

申请人： Translate Bio, Inc.

发明人： Richard Wooster ,  Anusha Dias ,  Dustin Cooper ,  Christian Cobaugh ,  Frank DeRosa ,  Tim Efthymiou ,  Jeffrey S. Dubins

IPC分类号： A61K31/7105 ,  A61K47/54 ,  A61K47/60 ,  A61K47/69

摘要： The present invention provides, among other things, methods and compositions for selective degradation of proteins. In some aspects, messenger RNAs (mRNAs) are described that encode a ubiquitin pathway moiety and a binding peptide that binds a target protein, wherein the mRNA is encapsulated within a lipid nanoparticle. Also provided herein are mRNAs that encode at least two binding peptides, wherein a first binding peptide binds a ubiquitin pathway moiety and a second binding peptide binds a target protein, and wherein the mRNA is encapsulated within a lipid nanoparticle.

公开(公告)号：US20210046192A1

公开(公告)日：2021-02-18

申请号：US16936909

申请日：2020-07-23

申请人： Translate Bio, Inc.

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

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

摘要： 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

申请人： Translate Bio, Inc.

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

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

摘要： 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

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

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

摘要： 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：US10428349B2

公开(公告)日：2019-10-01

申请号：US16280772

申请日：2019-02-20

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Michael Heartlein ,  Daniel Crawford ,  Shrirang Karve

IPC分类号： A61K31/70 ,  C07H21/04 ,  C12N15/63 ,  C12N15/85 ,  C12N15/67 ,  A61K38/18 ,  A61K38/45 ,  A61K38/44 ,  A61K38/17 ,  C07K14/705 ,  C07K14/505 ,  C12N9/10 ,  A61K48/00 ,  C12P21/00 ,  C07H21/02 ,  A61K31/7125 ,  A61K31/7115 ,  C12N9/02 ,  A01K67/00

摘要： 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.