Can Electroporation Revive Gene Therapy?
As regulators increase their scrutiny of viral vectors for gene therapy and set the bars for safety ever higher, some industry observers have begun questioning whether gene therapy will ever become a commercial reality. The difficulties in the field may turn out to be a life-saver for MaxCyte Inc. Its managers think their flow electroporation technology could be a platform for transforming patients' cells and getting them to produce a desired protein--without viruses, and the risks they bring.
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Is gene therapy ever going to become a routine, reliable method of disease treatment? It appears that the odds of success in anything like the near term are slimming. Now one of the first companies to get involved in gene therapy is finally giving up on it: San Diego-based Vical Inc., founded in 1987, recently announced its intention to use its naked DNA technology to develop a vaccine for cytomegalovirus (CMV).
To carry on work focusing on the oxygen-delivering capacity of red blood cells, EntreMed Inc, (best known for its anti-angiogenic molecules) created spin-off TheraMed Inc. TheraMed has developed a prototype device and is experimenting with different sorts of reagents, which it believes will enable the three basic sorts of blood cells--platelets, red cells and white blood cells--to be transformed into delivery vehicles for oxygen, drugs, and genes.
Data from the first 275 patients in the trial are promising, but outside experts say they are wary of another hasty Emergency Use Authorization.