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circVec

Development stage: Preclinical
Lead developer: Targovax
Modality: miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Natural RNA Aptamers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Vaccines → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Spiegelmers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, Unmodified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Vaccine mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Silencing → Gene Therapies, Long Non-coding RNA (lncRNA) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Protein Replacement mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, miRNA Inhibitors → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapy Plasmids → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration: Intravenous, Intramuscular

Overview

circVec is a proprietary circular RNA (circRNA) gene expression platform developed by Circio Holding ASA. It is designed to significantly enhance the potency, durability, and tissue specificity of gene therapies compared to conventional linear mRNA-based vectors. The technology utilizes a modular genetic cassette for efficient biogenesis of multifunctional circRNA inside target cells, enabling up to 10–20 times higher protein expression and over 15 times longer RNA half-life than standard mRNA approaches. This increased efficiency allows for reduced dosing, improved safety profiles, and lower costs in gene therapy applications. The lead therapeutic focus for circVec is on AAV-based gene therapy for Alpha-1-antitrypsin deficiency (AATD), where it employs a "remove-and-replace" strategy—expressing functional wild-type AAT protein while selectively removing the mutant form responsible for disease pathology. Preclinical data demonstrate that circVec achieves robust and durable protein expression in muscle, heart, and spleen tissues with low liver expression—a distinct pattern from mRNA vectors—potentially opening new opportunities in diseases where current gene therapies are limited by insufficient or misdirected transgene expression. Beyond monogenic diseases like AATD, the platform has potential applications in cancer vaccines (e.g., TG01 targeting KRAS mutations), cell therapy (including direct engineering of CAR-T cells via circRNA), chronic diseases, and vaccines. Ongoing collaborations aim to further improve delivery using non-viral systems such as proteolipid vehicles (PLV). As of mid-2025, development remains at preclinical/Discovery stage with plans to advance into clinical trials within the next year[1][3][4][6][7][8][9].

Targets

SERPINA1 Z (Alpha-1-antitrypsin Z variant protein)

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