Novartis' investment in Ato1 research
Hearing Journal:
June 2014 - Volume 67 - Issue 6 - pp 26,27
Moving Closer to a Gene Therapy for Hearing Loss
Parker, Mark A. PhD
Author Information Dr. Parker is director of audiology at Steward St. Elizabeth's Medical Center and assistant professor of otolaryngology at Tufts University School of Medicine in Boston.
Gene therapy, by which specific sequences of DNA are used to treat human disease, is in its infancy as a clinical treatment but has a unique application for hearing loss. Relevant to our field, a single gene called atonal homolog-1 (Atoh1 or Math1) causes cochlear supporting cells to develop into functioning hair cells and may eventually be used as a hearing loss treatment. In fact, the therapeutic use of Atoh1 to restore sensorineural hearing loss is currently being investigated in many laboratories and private biotechnology companies. Novartis has invested about $500 million and created a new division aimed at developing the first generation of Atoh1 therapies.