公开(公告)号：US20240197825A1

公开(公告)日：2024-06-20

申请号：US17761893

申请日：2020-09-17

Applicant: Translate Bio, Inc.

Inventor： Richard Wooster ,  Frank DeRosa ,  Lianne Boeglin ,  Priyaanka Nanduri ,  Anusha Dias ,  Khang Anh Tran

IPC: A61K38/17 ,  A61K48/00

CPC classification number: A61K38/177 ,  A61K48/0033 ,  A61K48/005

Abstract: The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding an engineered or mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.

公开(公告)号：US20230407358A1

公开(公告)日：2023-12-21

申请号：US17800157

申请日：2021-02-18

Applicant: Translate Bio, Inc.

Inventor： Dustin Cooper ,  Frank DeRosa ,  Anusha Dias ,  Jeffrey S. Dubins ,  Xiaobo Gu ,  Khang Anh Tran

IPC: C12P19/34 ,  C12N9/12 ,  C12N15/10

CPC classification number: C12P19/34 ,  C12Y207/07006 ,  C12N9/1247 ,  C12N15/1068

Abstract: The present invention provides methods for preparing optimized DNA sequences as templates for in vitro transcription of mRNA. These DNA sequences are optimized to avoid premature termination of transcription by RNA polymerase. The invention also provides methods for preparing optimized DNA sequences that include one or more termination signal at their 3′ end to reduce or prevent non-templated “runoff” transcription.