Method
US-2016206704-A1 · Jul 21, 2016 · US
Gene therapy for ocular disorders
US11090392B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-11090392-B2 |
| Application number | US-201616061530-A |
| Country | US |
| Kind code | B2 |
| Filing date | Dec 13, 2016 |
| Priority date | Dec 14, 2015 |
| Publication date | Aug 17, 2021 |
| Grant date | Aug 17, 2021 |
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- Title
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Abstract
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Compositions and methods are provided for treating ocular disorders in a subject are provided. In one aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGA3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGB3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding REP-1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.
First claim
Opening claim text (preview).
What is claimed is: 1. An adeno-associated virus (AAV) vector comprising an AAV capsid and a nucleic acid sequence comprising AAV inverted terminal repeat (ITR) sequences, a sequence encoding human cyclic nucleotide gated channel alpha 3 (CNGA3), and expression control sequences that direct expression of the CNGA3 in a host cell, wherein the sequence encoding CNGA3 comprises SEQ ID NO: 9 or SEQ ID NO: 11. 2. The AAV vector of claim 1 , wherein the CNGA3 sequence encodes the protein sequence of SEQ ID NO: 10. 3. The AAV vector of claim 1 , wherein the expression control sequences comprise a chicken β-actin (CBA) promoter with cytomegalovirus (CMV) enhancer elements. 4. The AAV vector of claim 1 , wherein the expression control sequences comprise a rhodopsin kinase promoter. 5. The AAV vector of claim 1 , wherein the ITR sequences are from AAV2. 6. A pharmaceutical composition comprising a pharmaceutically acceptable carrier and at least the AAV vector according to claim 1 . 7. The AAV vector of claim 1 , wherein the capsid is an AAV2, AAV5, AAV8, AAV9, AAV8 bp, or AAV7m8 capsid, or a variant thereof. 8. The AAV vector of claim 1 , further comprising one or more of an intron, a Kozak sequence, a polyA, and post-transcriptional regulatory elements. 9. The AAV vector of claim 1 , wherein the expression control sequence is an ocular cell-specific promoter.
Assignees
Inventors
Classifications
- A61K9/0048Primary
Eye, e.g. artificial tears · CPC title
- C07K14/47Primary
from mammals · CPC title
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy · CPC title
Ophthalmic agents · CPC title
Receptors; Cell surface antigens; Cell surface determinants {(tumour specific antigens C07K14/4748)} · CPC title
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- What does patent US11090392B2 cover?
- Compositions and methods are provided for treating ocular disorders in a subject are provided. In one aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGA3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGB3. In another aspect, an ad…
- Who is the assignee on this patent?
- Univ Pennsylvania
- What technology area does this patent fall under?
- Primary CPC classification A61K9/0048. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Tue Aug 17 2021 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 3 related publications on this page (citations in our corpus or others sharing the same primary CPC).