Scientists from Sangamo BioSciences Inc. and collaborators has presented the data from clinical, preclinical and research-stage programmes focused on the development of ZFP Therapeutics for HIV/AIDS, monogenic diseases and stem cell applications at the 15th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) at Philadelphia.
“Sangamo's zinc finger DNA-binding protein (ZFP) technology is enabling development of new and improved gene and cell therapy approaches,” said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president, research and development. “Our ZFP Nuclease (ZFN) technology provides an extremely efficient and precise process for editing any DNA sequence. This enables us to disrupt specific genes or to precisely add DNA sequences that allow a patient's own gene to be corrected and its proper function restored while preserving the natural regulation of the gene.”
Sangamo has also developed technology that allows a therapeutic gene to be inserted into a specific 'safe harbour' site. Our ability to target changes to precise locations rather than randomly into the genome, avoids the challenges of traditional gene-addition approaches that can result in unintended mutations. The increased number of related presentations at this meeting demonstrates the growing adoption of ZFN-based gene editing by the field.”
Presentations from Sangamo included preliminary clinical data from ongoing phase I clinical trials in HIV/AIDS as well as data from preclinical and research-stage human therapeutic programmes. Therapeutic areas included ZFP-based approaches for monogenic diseases such as haemophilia, haemoglobinopathies and Huntington's disease as well as adoptive T-cell therapies for oncology.
“Visibility of ZFPs in the scientific agenda at the ASGCT meeting illustrates the broad range of potential applications for ZFP Therapeutics,” said Edward Lanphier, Sangamo's president and CEO. “Our technology can be used to modify any gene with singular specificity and high efficiency. As our technology functions at the DNA level, it can potentially be applied to any disease-related gene making it a versatile platform for the generation of novel therapeutic approaches for the treatment of unmet medical needs.”
Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing.