[EN] CATIONIC LIPIDS COMPRISING AN HYDROXY MOIETY<br/>[FR] LIPIDES CATIONIQUES COMPRENANT UNE FRACTION HYDROXY
申请人:TRANSLATE BIO INC
公开号:WO2020257611A1
公开(公告)日:2020-12-24
Disclosed are lipids which are compounds of Formula I. 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.
[EN] PHENOLIC ACID LIPID BASED CATIONIC LIPIDS<br/>[FR] LIPIDES CATIONIQUES À BASE DE LIPIDE D'ACIDE PHÉNOLIQUE
申请人:TRANSLATE BIO INC
公开号:WO2021202694A1
公开(公告)日:2021-10-07
The present invention provides, in part, phenolic acid lipid compounds of Formula (I), 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.
SYNTHESIS AND STRUCTURE OF HIGH POTENCY RNA THERAPEUTICS
申请人:Arcturus Therapeutics, Inc.
公开号:US20190002906A1
公开(公告)日:2019-01-03
This invention provides expressible polynucleotides, which can express a target protein or polypeptide. Synthetic mRNA constructs for producing a protein or polypeptide can contain one or more 5′ UTRs, where a 5′ UTR may be expressed by a gene of a plant. In some embodiments, a 5′ UTR may be expressed by a gene of a member of
Arabidopsis
genus. The synthetic mRNA constructs can be used as pharmaceutical agents for expressing a target protein or polypeptide in vivo.
[EN] BIOMIMETIC NANOMATERIALS AND USES THEREOF<br/>[FR] NANOMATÉRIAUX BIOMIMÉTIQUES ET LEURS UTILISATIONS
申请人:OHIO STATE INNOVATION FOUNDATION
公开号:WO2019027999A1
公开(公告)日:2019-02-07
The present disclosure relates to biomimetic nanomaterials, compounds, compositions, and methods for delivery of therapeutic, diagnostic, or prophylactic agents (for example, a nucleic acid).
本公开涉及仿生纳米材料、化合物、组合物以及用于传递治疗、诊断或预防剂(例如核酸)的方法。
[EN] COMPOSITION AND METHODS FOR TREATMENT OF PRIMARY CILIARY DYSKINESIA<br/>[FR] COMPOSITION ET MÉTHODES DE TRAITEMENT D'UNE DYSKINÉSIE CILIAIRE PRIMITIVE
申请人:TRANSLATE BIO INC
公开号:WO2021226463A1
公开(公告)日:2021-11-11
The present invention provides, among other things, methods and compositions for treating primary ciliary dyskinesia (PCD) based on mRNA therapy. The compositions used in treatment of PCD comprise an mRNA comprising a dynein axonemal intermediate chain 1 (DNAI1) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of PCD is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized DNAI1 coding sequences are provided that can be administered without the need for modifying the nucleotides of the mRNA to achieve sustained in vivo function.