Granted Patent
B2
US 12,692,516 · App. 18/055,968 · Granted Jul 28, 2026
Tissue selective transgene expression
Inventors:
Stephanie Tagliatela (South San Francisco, CA); Andrew Young (Redwood City, CA); Szu-Ying Chen (South San Francisco, CA); Kartik Ramamoorthi (South San Francisco, CA); David Oberkofler (South San Francisco, CA)
Assignee:
Encoded Therapeutics, Inc.
C12N15/86A61K48/0058C07K14/705C12N15/113C12N15/85C12N15/8645A01K2217/052A01K2217/15A01K2227/105A01K2267/0312A61K48/00C12N2320/32C12N2750/14143C12N2830/008
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Abstract
Provided herein are compositions and methods for selective expression of a transgene. Compositions and methods for selective expression of a transgene comprise one or more human regulatory elements, which, when operably linked to a transgene, can facilitate selective expression of a transgene (for example, cell-type selective expression) in a target cell as compared to at least one or more non-target cells.
Claims (10)
1 . A viral particle comprising a viral vector, wherein the viral vector comprises an expression cassette comprising a regulatory element operably linked to a heterologous transgene, wherein the regulatory element comprises a nucleic acid sequence with at least 95% identity to SEQ ID NO: 1 or 8, and wherein the expression cassette selectively increases expression of the heterologous transgene in parvalbumin (PV) neurons compared to non-PV cells.
2 . The viral particle of claim 1 , wherein the expression cassette further comprises an enhancer, a promoter, a stability element, a UTR, or a combination thereof.
3 . The viral particle of claim 1 , wherein the regulatory element comprises a nucleic acid sequence of SEQ ID NO: 1.
4 . The viral particle of claim 1 , wherein the regulatory element comprises a nucleic acid sequence of SEQ ID NO: 8.
5 . The viral particle of claim 1 , wherein the heterologous transgene encodes (a) an amino acid sequence having at least 95% identity to any one of SEQ ID NOS: 37-43, or (b) an amino acid sequence of any one of SEQ ID NOS: 37-43.
6 . The viral particle of claim 1 , wherein the heterologous transgene encodes a DNA binding protein.
7 . The viral particle of claim 6 , wherein the DNA binding protein modulates expression of an endogenous gene.
8 . The viral particle of claim 7 , wherein the endogenous gene is SCN1A, SCN2A, SCN8A, SCN1B, SCN2B, KV3.1, KV3.2, KV3.3, or STXBP1.
9 . The viral particle of claim 1 , wherein the viral vector is an adeno-associated virus (AAV) vector or a lentiviral vector.
10 . The viral particle of claim 9 , wherein the viral vector is the AAV vector and the viral particle is an AAV viral particle, and wherein the AAV viral particle has a serotype selected from the group consisting of AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12, rh10, AAV-DJ, avian AAV, bovine AAV, canine AAV, equine AAV, primate AAV, non-primate AAV, ovine AAV, and hybrids or variants thereof.
Assignments (2)
CHANGE OF NAME
Recorded Nov 16, 2022
From: ENCODED GENOMICS, INC.
To: ENCODED THERAPEUTICS, INC.
Reel/Frame 061793/0145 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Nov 16, 2022
From: TAGLIATELA, STEPHANIE; YOUNG, ANDREW; CHEN, SZU-YING; RAMAMOORTHI, KARTIK; OBERKOFLER, DAVID
To: ENCODED GENOMICS, INC.
Reel/Frame 061793/0025 →
Continuity (5)
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