GlaxoSmithKline plc (ADR) (NYSE:GSK) and its partner San Raffaele Telethon Institute for Gene Therapy have started marketing a gene therapy for hyper rare cases of immune deficiency. The immune deficiency is triggered by ADA-SCID.
The delivery of the therapy to regulators comes in the backdrop of some prominent setbacks that have begun to cloud what is otherwise a pretty bullish sector of biotech.
The drug is named GSK2696273, it is a gene therapy that uses a viral vector to insert working copies of ADA gene into stem cells that are extracted from the marrow of patients. The extracted cells are then reintroduced into the patient where the gene multiplies thus revitalizing the immune system.
The therapy is submitted to European regulators. The regulators will be reviewing the data derived from 18 patients with some of the patients being under therapy for 13 years. All the patients are alive. However some have had to take an enzyme replacement therapy. This enzyme replacement therapy is what most patients depend on.
If GlaxoSmithKline gets approval in Europe, then it could have widespread implications. As the disease is very rare, the treatment facility in Italy may become the only one in the world.
The application marks a rare good news for GlaxoSmithKline plc (ADR) (NYSE:GSK), which has been having a bleak period for some time now. The company has been struggling to restore its revenue numbers and analysts have become sceptical about the prospects of a turnaround. The move by GlaxoSmithKilne also shows the increase in gene therapy tough the prospects of failure remains. Investors though are wary of failure in gene therapy research as the cost involved is high.
Both big partners and investors have been turning to gene therapy in increasing numbers. There is hope that this field of biotech s finally arrives and is ready to deliver new treatments that are effective and safe.