OPA1 antisense oligomers for treatment of conditions and diseases
AssigneeStoke Therapeutics, Inc.
InventorsIsabel Aznarez, Aditya Venkatesh, Gene Liau
Life of this patent
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Abstract
Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency and/or mitochondrial function deficit.
Claims58
A method of treating a disease or condition in a subject in need thereof, the method comprising administering to the subject a pharmaceutical composition that comprises:
- (a) a pharmaceutically acceptable excipient;
- and
- (b) (i) a therapeutic agent, wherein the therapeutic agent comprises an antisense oligomer that comprises the sequence set forth in any one of SEQ ID NO:
- 36, 92-96, or 166-168, and wherein the antisense oligomer comprises a 2′-O-methoxyethyl moiety;
- or
- (ii) a vector encoding an antisense oligomer that comprises the sequence set forth in any one of SEQ ID NO:
- 36, 92-96, or 166-168,
- wherein the disease or condition is Optic atrophy type I or autosomal dominant optic atrophy (ADOA).
A composition comprising
- (i) an agent, wherein the agent comprises an antisense oligomer that comprises the sequence set forth in any one of SEQ ID NO:
- 36, 92-96, or 166-168, wherein the antisense oligomer comprises a backbone modification, a sugar moiety modification, or a combination thereof;
- or
- (ii) a vector encoding the agent, wherein the agent comprises an antisense oligomer that comprises the sequence set forth in any one of SEQ ID NO:
- 36, 92-96, or 166-168.
Dependencies are inferred from the claim text.