A Novel Preparation Method of Cationic Building Blocks for Gene-Scilencing Molecules
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Category |
Anti-gene and anti-sense drug development |
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Development Stage |
Technology up-scale and enhancement |
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Patent Status |
Patent Pending |
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Market Size |
Market research firm Datamonitor (2005) has identified 99 companies developing DNA/RNA therapies, with 229 products in development. Biotech and pharmaceutical companies engaged in the R&D of anti-gene and anti-sense drugs include Isis Pharmaceuticals, Pfizer, Elan Corp., GeneVec, AVI Biopharma, Sanofi Aventis, GlaxoSmithKline, Targeted Genetics Corp., to name a few. |
Highlights
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Cationic peptide nucleic acid (cPNA) conjugates were shown to penetrate cell membranes and blood brain barrier.
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These constructs may also serve as vehicles of drugs, allowing cell and BBB penetration.
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The proposed structures were shown to be able to escape early and late endosomic vesicles and lysosomal cell compartment, due to their positive charge
Our Innovation
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Cationic building blocks of peptide nucleic acid (PNA) were prepared.
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Several binding-enhancing substances were incorporate to the designed cationic sequences, resulting in fully charged sequences, or partially-charged PNA.
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The selective use of such binding-enhancing substances was shown to contribute significantly to the hybridization between the polymer and the targeted DNA.
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The group will also be able to produce a PNA-cassette that will allow for hybridization with particularly large DNA sequences.
Development Milestones
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Building block and enhancer and PNA production scale-up
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Establish cell membrane and BBB permeation in larger scales, in various biological systems
The Opportunity
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The proposed technology will be offered to anti-gene and anti-sense drug manufacturers. These drugs are mainly targeted at is non-responding cancer patients. According to the US National Institute of Cancer, it is estimated that 50% of cancer patients develop multidrug resistance to anticancer drugs.
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