Combination therapy for c3 inhibition
US-2020282012-A1 · Sep 10, 2020 · US
RNAs for complement inhibition
US11510939B1 · US · B1
| Field | Value |
|---|---|
| Publication number | US-11510939-B1 |
| Application number | US-202016853732-A |
| Country | US |
| Kind code | B1 |
| Filing date | Apr 20, 2020 |
| Priority date | Apr 19, 2019 |
| Publication date | Nov 29, 2022 |
| Grant date | Nov 29, 2022 |
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Abstract
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RNAs, such as miRNA and siRNA, and their use in treating complement-mediated disorders, are described.
First claim
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We claim: 1. 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. A nucleic acid encoding the miRNA or siRNA of claim 1 . 3. The nucleic acid of claim 2 , comprising any one of SEQ ID NOs: 86-87 or 89-115. 4. An expression vector comprising the nucleic acid of claim 2 . 5. The expression vector of claim 4 , wherein the expression vector is a viral vector. 6. The expression vector of claim 5 , wherein the viral vector is an adeno-associated viral (AAV) vector. 7. The expression vector of claim 4 , further comprising a nucleotide sequence encoding a C3 inhibitor, wherein the C3 inhibitor is an aptamer, an anti-C3 antibody, an anti-C3b antibody, a mammalian complement regulatory protein, or mini factor H. 8. An miRNA consisting of a nucleotide sequence that is at least 90% identical to a nucleic acid sequence that targets nucleotides 617-637, 753-773, 2811-2831, 2835-2855, 3541-3561, 3849-3869, 4125-4145, 4309-4329, and/or 4394-4414 of SEQ ID NO:75, 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. 9. The miRNA or siRNA of claim 1 , wherein the nucleotide sequence is at least 90% identical to SEQ ID NO: 76. 10. A nucleic acid encoding the miRNA or siRNA of claim 9 . 11. The nucleic acid of claim 10 , comprising the nucleotide sequence of SEQ ID NO: 86. 12. An expression vector comprising the nucleic acid of claim 10 . 13. The expression vector of claim 12 , wherein the expression vector is a viral vector. 14. The expression vector of claim 13 , wherein the viral vector is an adeno-associated viral (AAV) vector. 15. The expression vector of claim 12 , further comprising a nucleotide sequence encoding a C3 inhibitor, wherein the C3 inhibitor is an aptamer, an anti-C3 antibody, an anti-C3b antibody, a mammalian complement regulatory protein, or mini factor H. 16. The miRNA or siRNA nucleotide sequence of claim 8 , wherein the nucleotide sequence targets nucleotides 617-637 of SEQ ID NO: 75, or a portion thereof.
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Classifications
Special targeting system for viral vectors · CPC title
viral genome or elements thereof as genetic vector · CPC title
Viral vectors · CPC title
- A61P43/00Primary
Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title
- A61K31/713Primary
Double-stranded nucleic acids or oligonucleotides · CPC title
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- What does patent US11510939B1 cover?
- RNAs, such as miRNA and siRNA, and their use in treating complement-mediated disorders, are described.
- Who is the assignee on this patent?
- Apellis Pharmaceuticals Inc
- What technology area does this patent fall under?
- Primary CPC classification A61P43/00. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Tue Nov 29 2022 00:00:00 GMT+0000 (Coordinated Universal Time) (B1). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 4 related publications on this page (citations in our corpus or others sharing the same primary CPC).