Drug intelligence / Profile preview

SaCas9

Development stage
Preclinical
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration
Intravenous, Intramuscular
01

Overview

SaCas9 is a CRISPR-associated protein 9 derived from *Staphylococcus aureus*. It is a type II CRISPR system component that functions as an RNA-guided DNA endonuclease. Compared to the widely used SpCas9 (from *Streptococcus pyogenes*), SaCas9 is significantly smaller (approximately 1,053 amino acids), which allows it to be more efficiently packaged into adeno-associated virus (AAV) vectors for in vivo gene editing applications. In therapeutic research, SaCas9 is being utilized to treat genetic disorders such as Duchenne muscular dystrophy (DMD) by targeting and correcting mutations like large exon duplications. Modified versions, such as the SaCas9 (D10A) nickase, are engineered to create single-strand breaks (nicks) rather than double-strand breaks, which can enhance editing precision and reduce unwanted insertions/deletions (indels) or off-target effects.

Other names
Staphylococcus aureus Cas9SaCas9 nickaseSaCas-9 nickaseSaCas 9 nickaseSaCas9 (D10A)
02

Targets

DNA

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