Gene Editing Tools & Services: A Faster Path To The Right Edit

Developing a gene-editing program can require significant time and investment before teams have the data needed to determine whether a therapeutic approach is feasible. An integrated platform can help accelerate that process by starting with the biology and matching the editing strategy to the therapeutic goal.

The platform supports five gene-editing modalities: epigenetic editing, base editing, reverse transcriptase editing, nuclease-based editing, and targeted gene insertion. Each approach addresses different therapeutic objectives, from correcting single-nucleotide variants and modulating gene expression to achieving durable gene knockout or targeted gene replacement.

AI-driven discovery and protein engineering can be combined with a portfolio of characterized enzymes. When existing enzymes do not meet a target's requirements, more than 10 billion protein sequences can be mined or generative AI can be used to identify and engineer novel candidates. Candidates are then optimized for potency, specificity, bystander profile, and off-target safety.

A proof-of-concept pathway can provide up to three gene editors for a target, along with feasibility and initial safety data, in under six months. This can help sponsors validate feasibility before making a larger program investment and support data-driven go/no-go decisions.

For organizations advancing gene-editing therapies, this integrated approach brings discovery, molecular engineering, feasibility testing, and development planning into a streamlined workflow, with options to progress toward clinical development and cGMP manufacturing.