IP Library › Granted Patent US 12,741,036
Granted Patent B2
US 12,741,036 · App. 17/624,755 · Granted Sep 22, 2026

Viral vector therapy

Inventors: Cedric Francois (Prospect, KY); Lukas Scheibler (Telluride, CO)
Assignee: Apellis Pharmaceuticals, Inc.
A61K48/0083A61P7/00A61P21/00A61P33/00C07K16/18C12N15/86C07K2317/76C12N2750/14143
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Quick Facts
Patent No.
US 12,741,036
App. No.
17/624,755
Granted
Sep 22, 2026
Kind
B2
Abstract

Methods and compositions for combined therapy with viral vectors and complement inhibitors are described.

Claims (24)

1 . 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:

2 . The method of claim 1 , wherein the efficacy of the gene therapy is improved in the subject over a specified time period relative to a control subject receiving or who has received the gene therapy and is not administered the complement inhibitor.

3 . The method of claim 1 , wherein the gene therapy comprises a viral vector.

4 . The method of claim 3 , wherein the viral vector is an adeno-associated viral (AAV) vector.

5 . The method of claim 3 , wherein the viral vector comprises a transgene.

6 . The method of claim 1 , wherein the complement inhibitor decreases an immune response against the gene therapy, relative to a control subject receiving the gene therapy and not administered the complement inhibitor.

7 . The method of claim 3 , wherein transduction of the viral vector is improved, relative to a control subject receiving the gene therapy and not administered the complement inhibitor.

8 . The method of claim 7 , wherein transduction is assessed by measuring level of transgene expression.

9 . The method of claim 3 , wherein complement-mediated clearance of the viral vector is decreased relative to a control subject receiving the gene therapy and not administered the complement inhibitor.

10 . The method of claim 1 , wherein the complement inhibitor decreases the activity of a C3 protein.

11 . 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:

12 . 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:

13 . The method of claim 1 , further comprising administering the gene therapy to the subject.

14 . The method of claim 12 , wherein the complement inhibitor decreases the activity of C3 protein.

15 . The method of claim 12 , wherein the viral vector is an adeno-associated viral (AAV) vector.

16 . The method of claim 11 , wherein complement activation is reduced in the subject over a specified time period relative to a control subject receiving or who has received the gene therapy and is not administered the complement inhibitor.

17 . The method of claim 11 , wherein the complement inhibitor decreases the activity of C3 protein.

18 . The method of claim 11 , wherein the gene therapy comprises a viral vector.

19 . The method of claim 18 , wherein the viral vector is an adeno-associated viral (AAV) vector.

20 . The method of claim 18 , wherein the viral vector comprises a transgene.

Assignments (3)
RELEASE OF PATENT SECURITY AGREEMENT RECORDED AT REEL 067398 AND FRAME 0261 Recorded May 15, 2026
From: SIXTH STREET LENDING PARTNERS, IN ITS CAPACITY AS ADMINISTRATIVE AGENT
To: APELLIS PHARMACEUTICALS, INC.
Reel/Frame 075652/0212 →
SECURITY INTEREST Recorded May 13, 2024
From: APELLIS PHARMACEUTICALS, INC.
To: SIXTH STREET LENDING PARTNERS
Reel/Frame 067398/0261 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 23, 2022
From: FRANCOIS, CEDRIC; SCHEIBLER, LUKAS
To: APELLIS PHARMACEUTICALS, INC.
Reel/Frame 059380/0875 →
Continuity (5)
Provisional Application 62935569 · Nov 14, 2019
Provisional Application 62875925 · Jul 18, 2019
Provisional Application 62871700 · Jul 8, 2019
Provisional Application 62871058 · Jul 5, 2019
Related Publication 20220249706A1 · Aug 11, 2022
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