- Gene therapy involves fixing faulty DNA by using viral delivery systems to deliver corrective genes into the body.
- Gene therapy studies have shown positive results in conditions ranging from blood diseases, to blindness—and now deafness.
- Work is currently underway at various academic institutions and biopharma companies, including Novartis, whcih is focused on developing a gene therapy for deafness caused by disease or trauma.
Recently, scientists at the Ecole Polytechnique Federale de Lausane and the Boston Children's Hospital conducted collaborative preclinical gene therapy research on newborn mice. The results, which were promising, were published in the journal Science Translational Medicine and showed that by targeting the TIMC1 gene, they could potentially fix hereditary deafness. While the technique is still being refined, researchers expect clinical trials to start within the next five to 10 years.
Meanwhile, Novartis has already moved into early-stage clinical trials with its gene therapy research for deaf patients. The ongoing study will eventually recruit a total of 45 patients, with results due in 2017.
The current enthusiam for gene therapy R&D is a departure from the attitude 10 years ago after a slew of gene therapy-related expensive failures and safety conundrums that prevailed during the 1990's and 2000's. The major change has been refining the gene-delivery systems—and knowing when to stop, which is an important consideration given the costs associated with gene therapy.