Sleeping Beauty
Next generation non-viral gene delivery systems for cell therapy and bioproduction
Challenge
- Cost of current CAR‑T therapies: 250 – 400k € per patient (viral gene delivery)
- Bottlenecks in manufacturing due to complex GMP virus production
- Regulatory pressure: need for safe and less costly non-viral alternatives
Solution
The Sleeping Beauty Transposase System (SB100X)
- Virus-free integration: ~80% efficiency in T cells
- Improved safety: almost random integration profile, no hotspots
- Reduced manufacturing costs: >10x cheaper than viral vectors
Main features
- Mechanism: cut and pace (no-reverse transcription)
- Cargo capacity: no upper limit +kb
- Immunogenicity: similar to plasmid-based expression vectors
- Tropism: dividing and non-dividing cells
- Integration profile: improved biosafety transposases
- Stable expression: copy number dependent, non-silenced
- Efficacy: in certain cells comparable to lentivirus
- Transcriptional activation: no promoter/enhancer activity
Development status
- Clinical phase I/II studies: e.g. ex vivo CAR‑T CARAMBA by University of Würzburg
- ex vivo engineered B cell therapy
- in vivo gene insertion therapy
IP
A developing portfolio from the foundational discovery to a high-precision genome engineering system with improved biosafety
Hyperactive variants of the transposase protein of the transposon system Sleeping Beauty
| Priority date | New variant improvement |
| 2022 | Safer integration profile |
| 2022 | ~2× activity |
| 2023 | Shorter half-life |
| 2025 | Targeting genomic safe harbors |
Publications
Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates
Mátés et al. (2009) Nature Genetics
Lab
Get in touch to explore how we can work together:
Dr. Marie Vidal, Senior BD Manager
Kseniia Choni, BD Manager
