RNA Therapeutics

Pendrea’s RNA Therapeutics program uses NanoJackets for delivery of all types of RNA.  The Company is particularly involved in the development of mRNA therapies and Immuno-Oncology therapies using its NanoJackets.

 

mRNA Therapies

NanoJackets with active targeting moieties on their surface deliver mRNA to the interior of targeted cells resulting a highly efficient mRNA expression.  The mRNA-NJ system is made of biologically compatible materials that avoids the toxicities associated with LNPs (Lipid Nano Particles) and therefor provides a unique and important alternative to LNP based mRNA therapeutics.  mRNA-NJs can be actively targeted for delivery for mRNA to organs and tumor types outside the liver.  Data shows the mRNA is protected from degradation by the NanoJacket while in the bloodstream and is properly configured for correct protein confirmation upon expression.  The mRNA encapsulated in the NJs can code for therapeutic and prophylactic vaccines as well as therapeutic proteins.

 

Immuno-Oncology

Pendrea’s immuno-oncology program uses NanoJackets to deliver RNA agents that alter the immune system:

  • siRNA-NJs  --  down regulate immunological products that represses the immune system in cancer

  • miRNA-NJs  –  reprograms immuno-signaling pathways to promote immuno surveillance

  • mRNA-NJs  –  produces immunological products such as cancer vaccines and therapeutics

 

Results with Pendrea’s RNA NanoJackets  include:

  • siRNA-NJs  --  98% knockdown of target protein expression, and 80% tumor regression in breast cancer

  • miRNA-NJs  --  an actively targeted micro RNA formulation achieved 4 times higher concentration in the lung than a chemically modified micro RNA 

  • mRNA-NJs  --  high levels of protection of mRNA from enzymatic degradation and confirmed mRNA functionality via expression after mRNA release from NJs in cells.