The Power of Gene Editing
Treating severe or life threatening genetic based diseases requires safe, durable, and effective therapies. BlueAllele harnesses key features of gene editing and gene therapy to develop powerful, tailored therapies for individual genetic diseases.
BlueAllele’s PALIDON™
PALIDON™ Repair Template Technology
BlueAllele's bi-directional repair templates PALIDON™
- Designed with reverse complement coding sequences
- Potential to significantly improve integration, leading to increased efficacy
- Broad patent filings covering PALIDON™ templates
- Designed to maximize efficiency of DNA corrections
- Provides ability to address larger patient populations
PALIDON™ Addresses Unmet Therapeutic Challenges in Gene Editing
Nuclease only (no repair template) approach is limited to specific types of patients
- Only small sections of DNA can be deleted — limiting the ability to repair genes
- Diseases with mutations across the gene cannot be addressed by one therapy
- Lack of precise DNA correction restricts use to smaller patient segments
Traditional repair templates have failed to achieve therapeutic thresholds
- Low integration efficiency
- Limitations in template design
- Lack of compatibility with all DNA repair mechanisms
Potential to Achieve Supra Therapeutic Levels of Efficacy
Subtherapeutic
- - below threshold
- - no/minimal benefit
Therapeutic
- - meets threshold
- - benefit observed
Supratherapeutic
- - exceeds threshold