IP Library Granted Patent US 12,251,395
Granted Patent B2
US 12,251,395 · App. 17/942,698 · Granted Mar 18, 2025

RNAS for complement inhibition

Inventors: Christian Mueller (Concord, MA); Lukas Scheibler (Telluride, CO)
Assignee: Apellis Pharmaceuticals, Inc.
A61K31/713A61P43/00C07K16/18C12N15/113C12N15/86C12N2310/14C12N2310/141
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Quick Facts
Patent No.
US 12,251,395
App. No.
17/942,698
Granted
Mar 18, 2025
Kind
B2
Abstract

RNAs, such as miRNA and siRNA, and their use in treating complement-mediated disorders, are described.

Claims (7)

1. A method of inhibiting expression of C3 in a cell, the method comprising contacting the cell with an miRNA consisting of a nucleotide sequence at least 90% identical to any one of SEQ ID NOs: 76-77 or 79-85, or a portion thereof, wherein the nucleotide sequence is 19-23 nucleotides in length, or an siRNA comprising an antisense strand consisting of the nucleotide sequence.

2. The method of claim 1 , wherein the method comprises contacting the cell with a composition comprising a nucleic acid encoding the miRNA or siRNA.

3. The method of claim 2 , wherein the nucleic acid comprises any one of SEQ ID NOs: 86-87 or 89-115.

4. The method of claim 2 , wherein the composition comprises an expression vector comprising the nucleic acid.

5. The method of claim 4 , wherein the expression vector is a viral vector.

6. The method of claim 5 , wherein the viral vector is an adeno-associated viral (AAV) vector.

7. The method of claim 1 , wherein the cell is a hepatocyte.

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 7, 2023
From: MUELLER, CHRISTIAN; SCHEIBLER, LUKAS
To: APELLIS PHARMACEUTICALS, INC.
Reel/Frame 062906/0901 →
Continuity (3)
Division 16853732 · Apr 20, 2020
Provisional Application 62836412 · Apr 19, 2019
Related Publication 20230302038A1 · Sep 28, 2023
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