Drug intelligence / Profile preview

SpyCas9

Development stage
Unknown
Lead developer
CRISPR Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Recombinant Proteins and Enzymes
Administration
Intravenous, Parenteral
01

Overview

SpyCas9 is a Class 2 Type II CRISPR-associated endonuclease derived from *Streptococcus pyogenes* that serves as the foundational component for many programmable genome-editing technologies. It functions as an RNA-guided DNA nuclease, forming a ribonucleoprotein (RNP) complex with a single-guide RNA (sgRNA) to recognize and cleave double-stranded DNA targets. Recognition is mediated by a 20-nucleotide sequence complementary to the guide RNA and an adjacent 5'-NGG-3' protospacer adjacent motif (PAM). Upon binding, the enzyme's HNH and RuvC nuclease domains generate a double-strand break (DSB), which can be repaired by cellular mechanisms to introduce gene knockouts or precise sequence insertions. SpyCas9 is extensively utilized in clinical-stage gene therapies, such as those for sickle cell disease and transthyretin amyloidosis, and in the engineering of allogeneic cell therapies.

Other names
Streptococcus pyogenes Cas9Cas9 nucleaseCas-9 nucleaseCas 9 nucleaseSpyCas9WTSpyCas-9WTSpyCas 9WT
02

Targets

AcrIIA26 (Anti-CRISPR protein AcrIIA26)Protospacer (Genomic DNA target site (CRISPR-Cas9))AcrIIA28 (Anti-CRISPR protein AcrIIA28)

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