Real programs. Real validation data.
Client identities are anonymized at their request, but every metric below is from a closed engagement with downstream wet-lab or preclinical confirmation.
- 4×
- Average speed vs. manual design
- 10–14 days
- Typical computational delivery
- 92%
- Wet-lab prediction accuracy
- 100%
- On-time delivery rate
Three programs across gene editing modalities
Select a case study to read the full engagement timeline, ranked deliverables, validation outcomes, and lessons learned.
Genome-Wide CRISPR Guide Design for Liver-Targeted Gene Therapy
Series A gene therapy startup needed high-specificity guide RNAs for an in vivo hepatocyte edit with zero tolerance for off-target cuts at pathogenic loci.
- 10 days
- End-to-end delivery
- 95%+
- On-target specificity (top 5)
Adenine Base Editor Guide Optimization for Sickle Cell Disease
Clinical-stage cell therapy team needed ABE guide RNAs that achieve ≥60% HbF reactivation with minimal bystander edits before ex vivo manufacturing scale-up.
- 12 days
- Computational delivery
- 68%
- Observed HbF reactivation (top guide)
Micro-Dystrophin Codon Optimization for DMD AAV Gene Therapy
Gene therapy biotech needed a codon-optimized micro-dystrophin construct that maximizes expression in skeletal muscle while fitting AAV packaging constraints and avoiding immunogenic epitopes.
- 14 days
- Computational delivery
- 3.2×
- Expression vs. prior construct
Your program could be the next case study.
Tell us about your target locus or construct. We will scope a 10 to 14 day pilot.