Multifunctional Self-Assembling Polymeric Nanosystems

INV-0908

 

Delivery of nucleic acid therapies to specific diseased tissues and cells in the body is challenging due to large molecular weight, negative charge, and relatively poor stability, especially in biological fluids that are rich in degrading enzymes (DNase and RNase). 

The majority of the technologies for nucleic acid delivery utilize cationic lipids and polymers that form electrostatic complexes with the negatively-charged acid construct. These cationic systems are inefficient for gene therapy or RNA interference therapy due to lack of intracellular release and stability. Additionally, cationic lipids and polymers are extremely toxic to cells and tissues.

 

This invention relates to a multifunctional polymeric nano‑system to facilitate the delivery of nucleic acid therapy to diseased tissues and cells. This method doesn’t use electrostatic complexation but rather focuses on physical encapsulation of the nucleic acid construct in a neutral or anionic hydrogen matrix. Additionally, the multifunctional assembly has hydrophobic functionality, PEG chains, and targeting ligands to facilitate delivery to diseased tissues and cells.

This technology uses a synthesis of a polymer library using a combinatorial chemical approach using five functional blocks.

The polymers used for the preparation of the nanosized delivery systems are biocompatible.

 

- Ability to synthesize large pool of polymeric materials with “dialed in” properties for a specific application. This strategy avoids the pitfalls associated with one ideal formulation strategy

- Select “hits” based on the physicochemical and biological properties

- Optimize individual systems for in‑vitro and in‑vivo nucleic acid construct delivery

- Lack of toxicity

 

- Cancer therapy

- Treatment of Coronary restenosis, diabetes

- Treatment of neurodegenerative diseases

- Treatment of inflammatory and infectious diseases

 

- License

- Partnering

- Research Collaboration