Seeking Alpha wrote a piece today about Sangamo's zinc finger nuclease technology as perhaps providing the basis for the next platform technology to do targetted gene manipulation -- which could serve as the means to develop a plethora of new therapies.
Therapeutic areas cover a span from gene therapy and regenerative medicine to more conventional development of protein-based biotherapeutic drugs. Sangamo is in fact pursuing therapeutics to address diseases as disparate as diabetic neuropathy and a "functional cure" for AIDs.
SAFC has a licensing deal for the Sangamo technology to apply it to the development of cell lines - particularly CHO cell lines for the production of biotherapeutic drugs. The ability of ZFNs to knock out, knock in or modify very specific genes of interest is critcally important in this application. It allows SAFC to help pharma and biotech customers alter genes of interest to them to improve the performance of their biotherapeutic production cell lines (platform lines).
Thus cellular properties like enhanced growth, decreased cell death, enhanced proten production can all be altered and manipulated simultaneously with ZFNs. This can lead to the development of a "Super CHO" production cell line. Cell genes that can change glycosylation patterns or produced proteins can also be changed to confer greater clinical efficacy or reduced clearance of the final drug protein. Viral or cellular proteins that might co-purify with the desired protein drug can also be eliminated by use of ZFNs.
SAFC has recently introduced ZFNs to allow customers to alter the dhfr and GS selection marker in their system. ZFNs that affect and control glycosylation are coming this year as well as ZFNs for genes that affect culture productivity and growth. Platform cell lines with either the dhfr or GS knock out will be available and adapted to chemically-defined, animal component free media. These should be available about mid-year 2011.
The technology is very versatile and truly enabling in many new applications -- only a few of which are discussed here.
Posted by Bruce Lehr Feb 1st 2011.