Gene therapies have started to show their potential over the last decade, with several impressive clinical successes, and approvals by the regulatory authorities. Although extremely powerful for selected disease indications, current gene therapies also face certain challenges, either because of their limited durability in in vivo applications, or because of uncontrolled expression of the therapeutic gene.
At Genespire, we target the root cause of the disease by inserting therapeutic genes directly into the DNA of patient’s cells. When those cells divide and multiply the therapeutic gene is maintained in the progeny, and functional proteins are permanently and stably expressed for a durable and life-long therapeutic effect. We build on two first-in-class technology platforms:
– The immune shielded lentiviral vectors (ISLVs), for in vivo application in the liver;
– The advanced gene editing platform, for precise ex vivo targeted gene insertion of genes in the patient’s blood cells.
Our first targets are in genetic diseases affecting the immune system and the metabolism, with the possibility to expand beyond those areas.