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Cas9 mRNA

Development stage
Preclinical
Lead developer
Biomay
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

Cas9 mRNA is a messenger RNA molecule that encodes the Cas9 (CRISPR-associated protein 9) endonuclease, a key component of the CRISPR-Cas9 gene-editing system. When delivered into cells, the mRNA is translated by the cellular machinery into the Cas9 protein, which, in complex with a guide RNA (gRNA), can induce site-specific double-strand breaks in DNA. This approach is widely used in gene therapy and genome engineering to knock out genes or insert new genetic material. Biomay AG has developed optimized fed-batch in vitro transcription (IVT) processes to manufacture high-quality Cas9 mRNA at scale, significantly reducing production costs and improving purity by minimizing double-stranded RNA (dsRNA) contaminants.

Other names
Cas9-encoding mRNACas-9-encoding mRNACas 9-encoding mRNACRISPR-associated protein 9 mRNA
02

Targets

CRISPR-Cas9 genomic target DNA sequenceInnate immune RNA sensors

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