Gene Therapy Shows Promise for Anemia

New gene therapy utilizing the EPO gene has shown the ability to cure anemia in mice.
Preclinical data on the therapy were presented at the meeting of the American Society for Gene Therapy in Boston (MA, USA).

Anemia is relatively common in patients with kidney disease, patients treated with AZT for AIDS, and patients undergoing chemotherapy. The current leading treatment is recombinant erythropoietin (EPO), but determining the accurate dosing level is difficult. The new therapy is designed to eliminate this problem, according to Oxford BioMedica plc (Oxford, UK), the developer.

The therapy consists of a viral gene delivery vector carrying the human EPO gene under the control of the company's hypoxia control element (HRE). The HRE senses low oxygen concentrations and will switch a gene on in response. When the oxygen concentration returns to normal, the HRE will switch the gene off, serving as a control mechanism for the production of EPO in situ. This way, it produces EPO when the underlying anemia results in low systemic oxygen concentrations, leading to the selective increase in the number of red blood cells. As the red blood cell count reaches normal levels and the systemic oxygen concentration reaches normal levels, the EPO gene is switched off.

Called Repoxygen, the treatment is designed to be injected into muscle. "The preclinical efficacy data for Repoxygen are excellent and we are considering the clinical development of the product, probably with a partner,” said Prof. Alan Kingsman, chief executive of Oxford BioMedica.




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