Compositions for CFTR MRNA therapy
AssigneeTranslate Bio, Inc.
InventorsAnusha Dias, Khang Anh Tran, Lianne Boeglin, Frank Derosa, John Androsavich, Shraddha Sharma, Gang Sun, Neha Kaushal, Shrirang Karve
Life of this patent
Estimating the term…
Abstract
The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding a wild-type Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.
Claims20
A codon-optimized mRNA molecule comprising a coding sequence encoding Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the coding sequence encodes the amino acid sequence set forth by SEQ ID NO:
- 1 and the coding sequence is at least 90%, at least 95%, at least 98%, at least 99%, or 100% identical to SEQ ID NO:
- 2 or SEQ ID NO:
- 3.
A polynucleotide comprising a sequence encoding the mRNA of any one of SEQ ID NOs:
- 2-11.
A pharmaceutical composition for treating cystic fibrosis, comprising a codon optimized mRNA encoding the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein set forth by SEQ ID NO:
- 1 and wherein the codon optimized CFTR mRNA comprises a polynucleotide sequence at least 90%, at least 95%, at least 98%, at least 99%, or 100% identical to SEQ ID NO:
- 2 or SEQ ID NO:
- 3.
Dependencies are inferred from the claim text.