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US 12,741,036 B2Granted
Viral vector therapy
AssigneeApellis Pharmaceuticals, Inc.
InventorsCedric Francois, Lukas Scheibler
Life of this patent
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Abstract
Methods and compositions for combined therapy with viral vectors and complement inhibitors are described.
Claims20
A method of improving efficacy of a gene therapy in a subject receiving or who has received the gene therapy, the method comprising administering a complement inhibitor to the subject, thereby improving efficacy of the gene therapy;
- wherein the complement inhibitor comprises a compstatin analog, wherein the compstatin analog comprises a clearance reducing moiety (CRM) comprising a PEG of about 40 kD, and wherein the compstatin analog has the following structure:
A method of reducing complement activation in a subject who has received or is receiving gene therapy, the method comprising:
- administering a gene therapy to the subject;
- and
- administering a complement inhibitor to the subject,
- thereby reducing complement activation in the subject;
- wherein the complement inhibitor comprises a compstatin analog, wherein the compstatin analog comprises a CRM comprising a PEG of about 40 kD, and wherein the compstatin analog has the following structure:
2 more elements
A method of increasing transduction of a viral vector comprising a transgene in a subject receiving gene therapy, the method comprising:
- administering a complement inhibitor to the subject, wherein expression of the transgene in the subject is increased relative to a control subject receiving the gene therapy but not administered the complement inhibitor;
- wherein the complement inhibitor comprises a compstatin analog, wherein the compstatin analog comprises a CRM comprising a PEG of about 40 kD, and wherein the compstatin analog has the following structure:
Dependencies are inferred from the claim text.