Imagine trying to fix a single typo in a massive library of books without damaging the pages. Instead of using expensive and risky viral 'delivery trucks' to carry the correction tool, this project uses tiny, smart nanoparticles that act like GPS-guided envelopes. It also upgrades the editing tool itself to be more precise and effective across different cell types.
Current gene therapies are prohibitively expensive and risky due to viral delivery systems that cause toxicity and immunogenicity. Additionally, existing prime editors suffer from inconsistent efficiency across different cell types.
A new gene correction tool called POLYPRIME and functionalized nanoparticles for non-viral delivery of CRISPR/Cas9 complexes.
If you are a gene therapy developer dealing with high toxicity and immunogenicity of viral vectors — this project developed non-viral nanoparticles with human-derived proteins that target specific tissues with minimal adverse effects.
If you are a hematology clinic dealing with the risks of manipulating stem cells for Sickle Cell Disease — this project developed a simpler, safer, and cost-effective in vivo treatment approach.
If you are a biologic producer dealing with low editing efficiency across different cell types — this project developed POLYPRIME, a tool replacing reverse transcriptase with DNA-dependent polymerase to increase activity.
Based on available project data, the non-viral delivery systems and the avoidance of hematopoietic stem cell manipulation are designed to make treatments more cost-effective compared to current expensive viral-based systems.
The project aims to create 'off-the-shelf' recombinant biologics, which suggests a design intended for standardized production and versatility across various genetic diseases.
Based on available project data, the project has developed a specific strategy named POLYPRIME, but specific licensing terms are not provided in the summary.
The project period runs from 2022-10-01 to 2027-09-30, indicating it is currently in the development and testing phase.
It uses functionalized nanoparticles with human-derived protein moieties to target specific tissues and cells in vivo.
The consortium is well-balanced for a translation project, consisting of 6 partners across 4 countries. With a 33% industry ratio (including 2 SMEs), there is a clear bridge between the academic research led by Sorbonne Universite and commercial application, reducing the gap between lab discovery and market entry.
Contact the research office at Sorbonne Universite
Contact us to explore licensing opportunities for POLYPRIME and non-viral delivery nanoparticles.
