AIDS, Biocompatible polymeric nano-microparticles as a potential carrier for DNA.

Synthetic carriers, including liposomes, polymers and polymeric particles are capable to deliver exogenous genetic materials into cells in a cellular specific or non specific manner.

Summary of Invention The invention relates to novel core-shell nanoparticles, processes for preparing them, and their use as carriers able to reversibly bind and deliver pharmacologically active substances, in particular nucleic acids, including natural and modified (deoxy) ribonucleotides (DNA, RNA), oligo(deoxy)nucleotides (ODNs) and proteins, into cells. Solution to which problem (s) DNA vaccines are known to induce immune responses and protective immunity in many animal models of infectious diseases. In human clinical trials, certain DNA vaccines have been shown to induce immune responses, but multiple immunizations of high doses of DNA were required. Therefore, in order to provide protective efficacy in humans, the potency of DNA vaccines needs to be increased. Many types of synthetic carriers, including liposomes, polymers and polymeric particles have been studied to deliver exogenous genetic materials into cells in a cellular specific or non specific manner. Recently biodegradable or biocompatible polymeric nano-microparticles have been studied as a potential carrier for genetic materials. Advantages of biocompatible polymeric particles as gene delivery carriers include: - they are relatively inert and biocompatible; - their biological behaviour can be regulated by controlling the size and surface properties; - and preparation, storage, and handling are relatively easy. - The size and shape of the resulting formulation can also remain homogeneous and uniform, compared to the formulations based on liposomes or polycations. - Controlled delivery systems consisting of biocompatible polymers can potentially protect DNA or proteins from degradation until they are both released and delivered to the desired location at predetermined rates and durations to generate an optimal immune response. The combination of slow release and depot effect may reduce the amount of antigens used in the vaccine and eliminate the booster shots that are necessary for the success of many vaccinations. Moreover, a controlled delivery system can efficiently direct antigens into antigen- presenting cells (APCs) to generate both cellular and humoral responses. Key applications The present invention provides biocompatible polymeric carriers capable to: - reversibly bind and deliver pharmacologically active substances, such as nucleic acids and proteins intact into cells. avoid recognition by the phagocytic cells, and to last longer in the bloodstream. Stage of Development: The technology is in the pre-clinical stage with in vivo animal studies ongoing.

Preclinical

Priority Filing in UK - Nov. 3, 2003, PCT Stage, published

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