Researchers at the University of Edinburgh have developed novel methods to produce, for the first time, recombinant Factor H (FH) in quantities sufficient for therapeutic and clinical use.
Disease haplotypes of FH play a pivotal role in the pathogenesis of age-related macular degeneration (AMD) providing a strong rational for use of protective haplotypes to treat this condition.
Currently, the short half-life, the need to derive material from plasma (with associated health risks) and the large concentrations of protein required preclude the wide therapeutic implementation of FH for treatment of AMD.
Edinburgh researchers have expressed a (proprietary) codon-optimised FH gene in cells of the yeast Pichia pastoris, producing grams of protein per litre of culture. Furthermore, by incorporating unnatural amino acids into the protein sequence, this optimised version of FH can be site-specifically conjugated (e.g. with PEG or sialic acid) to enhance half-life without compromising functionality.
Key Benefits
- Protective against development of age-related macular degeneration (AMD) providing a new blockbuster therapeutic
- Complementary to existing AMD treatments and safer than pooled plasma
- Cheaper than current AMD treatments
- Codon-optimised variant of FH generating >10-fold more protein
- Industrial-scale production of FH is achievable
- Increased half-life
- Reduced immunogenicity
Applications
- Development of a new biotherapeutic for AMD
- Recombinant FH as an adjunct therapy for AMD, e.g. in future gene-replacement or retinal stem cell-based approaches
- FH proteins for use in research labs and hospitals around the world
IP Status
Expression of this codon-optimised FH is more than ten-fold higher than current methods and is proven to be functionally active in in vitro studies. Sufficient quantities of a murine variant of FH have been produced enabling in vivo target validation studies.
A PCT patent application (ref: PCT/GB2010/002334), entitled “Factor H”, covering the production method and optimised FH sequences, has been filed claiming priority from 24th December 2009.
This technology is available for licensing and/or collaborative research to develop protein for research use and/or new blockbuster treatment for AMD.
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