1. Delivery Parameters
2. Expression & CRISPR Target
3. Timeline Scrubber
Day 14
Target Brain Transduction
68.4%
Optimal CNS Coverage
Off-Target Cleavage Index
0.042 DSB/cell
Low-Moderate Genomic Risk
Hepatic Burden / Toxicity
1.2x Baseline
Below Inflammatory Threshold
CNS CSF Half-Life
18.4 hours
Sustained Bio-availability
Tissue Vector Concentration (vg/mg)
Kinetic ODE ModelOrgan Tropism & Transduction Map
Bio-DistributionHuman CNS Off-Target Genomic Loci (DSB %)
CRISPR Target ProfilingNeuroinflammatory Trigger Index
Capsid Immunogenicity
CNS Gene Therapy Delivery & Risk Safety Profile Report
Selected Vector System:
AAV-PHP.B via Intrathecal Route
AAV-PHP.B via Intrathecal Route
Dose & Promoter Specificity:
1.50e+13 vg/kg (Syn1_neuronal)
1.50e+13 vg/kg (Syn1_neuronal)
Genomic Precision & Fidelity:
SpCas9-HF1 with Mismatch Profile [0,0,1,0,2]
SpCas9-HF1 with Mismatch Profile [0,0,1,0,2]
Safety Evaluation Verdict:
SAFE - Therapeutic Window Acceptable
SAFE - Therapeutic Window Acceptable