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US 12,419,968 B2Granted
Gene editing to improve joint function
AssigneeOrthobio Therapeutics, Inc.
InventorsPeter J. Millett, Iain Alasdair Russell, Matthew J. Allen
Life of this patent
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Abstract
The present invention provides compositions and methods for treating joint disorders that are characterized by an inflammatory component. In some aspects, the compositions and methods are to prevent the progression of osteoarthritis and other arthritides and to treat osteoarthritis and other arthritides in a mammalian joint.
Claims20
A pharmaceutical composition for the treatment or prevention of a joint disease or condition, comprising:
- a therapeutically effective amount of a Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) gene-editing system, the system comprising:
- (i) an mRNA encoding a CRISPR Associated Protein 9 (Cas9) protein;
- and
- (ii) at least one guide RNA targeting an IL-1α gene, wherein:
- the at least one guide RNA comprises a sequence that is complementary to a target sequence in exon 4 of the IL-1α gene, and
- the target sequence is adjacent to a protospacer adjacent motif (PAM) sequence for the Cas9 protein.
3 more elements
A pharmaceutical composition for the treatment or prevention of a joint disease or condition, comprising:
- a therapeutically effective amount of a Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) gene-editing system, the system comprising:
- (i) an mRNA encoding a CRISPR Associated Protein 9 (Cas9) protein;
- and
- (ii) at least one guide RNA targeting an IL-1β gene, wherein:
- the at least one guide RNA comprises a sequence that is complementary to a target sequence in exon 4 of the IL-1β gene, and
- the target sequence is adjacent to a protospacer adjacent motif (PAM) sequence for the Cas9 protein.
3 more elements
Dependencies are inferred from the claim text.