Table 3.
Total number of AAV particles/cell in the human body
| Sponsor | Disease | Vector | Dose (vg/kg) | Total dose (quadrillioin) | AAV particles/cell |
|---|---|---|---|---|---|
| uniQure | HB | AAV5 | 2 × 1013 | 1.4 | ∼47 |
| LogicBio Therapeutics | MMA | LB-001 | 5 × 1013 | 3.5 | ∼117 |
| BioMarin | HA | AAV5 | 6 × 1013 | 4.2 | ∼140 |
| Pfizer | DMD | AAV9 | 2 × 1014 | 14.0 | ∼467 |
| Audentes | XLMTM | AAV8 | 3 × 1014 | 21.0 | ∼700 |
Based on the estimated ∼3 × 1013 (30 trillion) total number of cells in the human body.149 HB, hemophilia B; MMA, methylmalonic acidemia; HA, hemophilia A; DMD, Duchenne muscular dystrophy; XLMTM, X-linked myotubular myopathy.