Cftr mrna compositions and related methods and uses
AssigneesTranslate Bio, Inc., ethris GmbH
InventorsMichael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
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Abstract
Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.
Claims31
A method of inducing CFTR expression in epithelial cells in a lung of a mammal,
- the method comprising contacting the epithelial cells in the lung of the mammal with a composition, wherein:
- the composition is a pharmaceutical composition comprising an in vitro transcribed mRNA;
- the in vitro transcribed mRNA comprises a coding sequence which encodes SEQ ID NO:
- 1.
(canceled)
A non-naturally occurring mRNA molecule comprising a coding sequence, a 5′-UTR, and a 3′-UTR, wherein the coding sequence encodes the amino acid sequence of SEQ ID NO:
- 1 and the coding sequence is at least 80% identical to SEQ ID NO:
- 3.
5-6. (canceled)
(canceled)
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17-18. (canceled)
21-25. (canceled)
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30-33. (canceled)
35-38. (canceled)
A method of making CFTR mRNA in vitro, comprising contacting an isolated polynucleotide with an RNA polymerase in the presence of nucleoside triphosphates, wherein:
- the isolated polynucleotide and RNA polymerase are not contained within a cell;
- the isolated polynucleotide is a template for the RNA polymerase;
- the isolated polynucleotide comprises a promoter operably linked to a template sequence;
- the template sequence comprises a coding sequence complement which is complementary to a sequence encoding SEQ ID NO:
- 1;
- and:
- (a) the template sequence comprises fewer cryptic promoters than the complement of SEQ ID NO:
- 2, (b) the template sequence comprises fewer direct and/or inverted repeats than SEQ ID NO:
- 2, (c) the template sequence comprises complements of fewer disfavored codons than SEQ ID NO:
- 2, or (d) the GC content of the coding sequence complement is lower than the GC content of SEQ ID NO:
- 2.
41-42. (canceled)
44-47. (canceled)
Dependencies are inferred from the claim text.