One of the greatest risks of CRISPR technology is off-target effects. Vigilance rigorously determines the target space for any CRISPR system in any genome.
SilicoTx Vigilance enhances CRISPR safety via optimal CRISPR guide RNA selection
The next frontier in CRISPR precision therapies
We leverage our knowledge of CRISPR specificity to design the most accurate guide RNAs for novel therapeutics
Researchers who want to ensure a high level of accuracy for their CRISPR experiments
Biotech companies that want to ensure the safety of their CRIPSR discoveries
Regulators who examine the safety of a CRISPR treatment
We discuss the partner’s assignment and scope the timeline and deliverables of the project.
We offer a white glove service where we rigorously determine the CRISPR system and the characteristics of the guide RNAs that are most appropriate for the customer’s task.
We engage in close collaboration with the customer and embrace open communication as a cornerstone of any partnership.
After project deliverables are rendered to the customer we can provide ongoing technical, database hosting, and repository support.
Our mission is to ensure the optimal utilization and ease of use for all Silico Therapeutics CRISPR guide RNA systems.
The collaboration will use the latest advances in machine learning to create automated methods and tools for life science researchers.
The genome-editing Cas9 protein uses multiple amino-acid residues to bind the target DNA. Considering only the residues in proximity to the target DNA as potential sites to optimise Cas9’s activity, the number of combinatorial variants to screen through is too massive for a wet-lab experiment. Here we generate and cross-validate ten in silico and experimental datasets of multi-domain combinatorial mutagenesis libraries for Cas9 engineering, and demonstrate that a machine learning-coupled engineering approach reduces the experimental screening burden by as high as 95% while enriching top-performing variants by ∼7.5-fold in comparison to the null model. Using this approach and followed by structure-guided engineering, we identify the N888R/A889Q variant conferring increased editing activity on the protospacer adjacent motif-relaxed KKH variant of Cas9 nuclease from Staphylococcus aureus (KKH-SaCas9) and its derived base editor in human cells. Our work validates a readily applicable workflow to enable resource-efficient high-throughput engineering of genome editor’s activity.
This week's clinical update includes news from Allogene Therapeutics and Graphite Bio. The U.S. Food and Drug Administration (FDA) recently granted Orphan Drug Designation to Allogene's AlloCAR T candidate ALLO-605 for the treatment of relapsed or refractory multiple myeloma. Yesterday, Graphite Bio announced that the FDA has granted Fast Track Designation to its CRISPR-edited cell therapy candidate GPH101 for the treatment of sickle cell disease.
