Ark makes breakthrough in targeted gene therapy delivery
Avoids harmful consequences of therapeutic gene being inserted in wrong place
Ark Therapeutics Group plc announceds that its scientists in Finland have discovered a novel gene therapy delivery technology which selectively inserts DNA into the specific therapeutic site in the genome (targeted integration). This technology is the subject of an Ark PCT filing.
The existing generation of integrating vectors in clinical trials, based mostly on retroviral, adeno-associated virus and lentiviral technologies, are not site-specific and carry the risk of a random gene insertion into an undesired and potentially harmful position on the chromosomes. The risks associated with non-specific integrating vectors became apparent in the X-linked severe combined immune deficiency disease (X-SCID) gene therapy trial, in 2002. The trial used a retrovirus, which was found to have inserted next to the leukaemia inducing oncogene, as a vector. Although the treatment was beneficial, three out of eleven patients treated in the study developed a leukaemia-like disease as a result of undesired random insertion. Ark's novel technology could herald a breakthrough in molecular medicine because it removes the potentially harmful consequences of a beneficial therapeutic gene being inserted into the wrong place, and could thus greatly improve the predictability and safety of gene therapy.
The technology is based on a fusion protein that catalyses the targeted integration of the treatment gene into a benign site on the human chromosomes. The process whereby the DNA carrying vector inserts the treatment gene in the chromosome is achieved by a specific DNA-binding nuclease-integrase fusion protein. The fusion protein breaks the DNA chain specifically in the benign locus of 28s ribosomal RNA gene (28s rDNA) and inserts the therapeutic gene at this site. Controlling the site where the insertion occurs has been so far very difficult, even with hybrid vectors or designed zinc finger proteins. Ark's novel vector technology removes the risk of such potentially harmful random insertions by this molecular chromosome locus targeting approach.
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Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.
Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.
