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US 2026/0286387 A1Published application
Gene delivery vehicles comprising RNA and antibodies
AssigneeuniQure biopharma B.V.
InventorsAstrid Valles-Sanchez, Seyda Acar Broekmans, Sebastian Kieper, - Anggakusuma
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Abstract
The present invention relates to a gene delivery vehicle comprising at least one nucleic acid comprising a sequence encoding a first RNA and a sequence encoding a second RNA, wherein the second RNA comprises a guide sequence and wherein the first and second and optionally third RNA each comprise a hairpin and encoding a full-length mAb or an antigen binding fragment thereof.
Claims15
A gene delivery vehicle comprising an expression cassette
- comprising at least one nucleic acid comprising a sequence encoding a first RNA and a sequence encoding a second RNA, wherein the second RNA comprises a guide sequence and wherein the first and second RNA each comprise a hairpin,
- and encoding a full-length mAb or an antigen binding fragment thereof,
- wherein the guide sequence is substantially complementary to a target sequence in a gene associated with a disease and wherein the mAb or antigen binding fragment thereof is targeting a protein associated with that same disease.
A combination of two or more gene delivery vehicles comprising:
- a first gene delivery vehicle comprising a first expression cassette comprising at least one nucleic acid comprising a sequence encoding a first RNA and a sequence encoding a second RNA, wherein the second RNA comprises a guide sequence and wherein the first and second RNA each comprise a hairpin;
- and
- a second gene delivery vehicle comprising a second expression cassette, comprising a transgene encoding a full-length mAb or an antigen binding fragment thereof,
- wherein the guide sequence is substantially complementary to a target sequence in a gene associated with a disease and wherein the mAb or antigen binding fragment thereof is targeting a protein associated with that same disease.
1 more element
(canceled)
Dependencies are inferred from the claim text.