RITDM™ 2.0 gene editing technology benefits
Efficient & safe gene editing
RITDM 2.0 gene editing offers important therapeutic advantages, especially for in vivo gene editing because of the following key differentiators:
- Strong safety profile
- Protein delivery option, next to LNPs
- Compact editor size (280-400 amino acids)
- No use of bacterial or retroviral proteins
RITDM 2.0 provides a strong safety profile, which is required for in vivo therapies:
• RITDM 2.0 is cleavage free and thus avoids DNA damage through cutting
• Two-component system provides additional safety, as each component has sequence specificity
RITDM 2.0 can help to unlock targets that are currently inaccessible:
• Unique possibility for protein delivery, next to LNPs
• Compact size (280-400 amino acids) supporting easier delivery and deeper tissue penetration
RITDM 2.0 creates possibility for repeat dosing:
• High(er) efficiency can be reached through multiple dosing, especially for difficult to reach organs and tissues
• To compensate for tissue turn-over
• Allows for treatment at various stages in a person’s life
• Gradual increase of dosage overtime to fine-tune therapies to individual circumstances