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Cas13d
- Development stage
- Preclinical
- Lead developer
- Salk Institute for Biological Studies
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Addition/Replacement → Gene Therapies
- Administration
- Intramuscular, Intravenous
01
Overview
Cas13d is a compact, RNA-targeting CRISPR-associated (Cas) endonuclease belonging to the Type VI-D CRISPR system. Unlike Cas9 or Cas12, which target DNA, Cas13d is guided by a CRISPR RNA (crRNA) to bind and cleave specific single-stranded RNA (ssRNA) sequences. Its small size (approximately 930 amino acids) makes it highly suitable for packaging into adeno-associated virus (AAV) vectors, such as MyoAAV, for therapeutic delivery. Cas13d is being developed for applications including RNA knockdown, splice modulation, and viral inhibition. Research presented at ASGCT 2026 indicates that the safety profile of Cas13d is highly dependent on the delivery route, with systemic administration showing a more favorable safety profile compared to direct intramuscular injection, which was associated with immune infiltration and muscle pathology.
- Other names
- CRISPR-Cas13dCRISPR-Cas-13dCRISPR-Cas 13dType VI-D CRISPR-associated protein
02
Targets
Target RNA (Cas13d-complementary)
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