Delivery Solutions for Therapeutic Nucleic Acids
Despite the vast potential of nucleic acid therapeutics, the number of approved drugs or successful therapy remains small. One of the many reasons for these disappointing results lies in in vivo delivery issues. Thus the development of efficient and safe in vivo delivery systems is key toward the successful in vivo activity of new therapeutic molecules such as siRNA.
Among the different delivery methods developed over the past two decades, recombinant virus have been originally used as carriers exploiting their natural abilities to achieve efficient and stable in vivo gene expression. However, the major limitation of viral approaches is related to safety considerations. Thus, non-viral reagents offer a safer alternative to viruses and an adequate solution to transport small oligonucleotide molecules and siRNA.
Non-viral delivery technologiesIn order to significantly improve the delivery of nucleic acids, Polyplus-transfection is developing non-viral carriers with a focus on polyethylenimine (PEI), a linear cationic polymer widely used in vivo. Since PEI based reagents enable delivery of many nucleic acid including, plasmid DNA, oligonucleotides and siRNA without triggering an immune response, they are widely used in animal models and in the clinic.
A specific formulation called in vivo-jetPEI™ has been developed and is extensively used for topical and systemic injection routes on a wide range of animal models. In vivo-jetPEI™ is available for preclinical studies and clinical trial applications.
In order to improve the delivery of siRNA, Polyplus-transfection has developed the STICKY SIRNA™. These molecules are a new class of small interfering RNAs which are able to form stable complexes in the presence of a cationic polymer such as in vivo-jetPEI™. Thus they are able to survive the journey in the living organism and induce efficient RNA interference in vivo. This innovation is applicable to therapeutic siRNAs for a wide variety of pathologies.
Polyplus’ Research department is working actively toward the improvement of delivery systems to fulfill customer’s requirements and needs. Polyplus is also open to collaborations and partnerships to support customers in their development of nucleic acid based therapeutics.
