Aissam Dam was born with a rare form of deafness caused by a mutation in one gene.The therapy is designed to replace the mutated gene in his ears with a functional version. He received the treatment Oct. 4 through a new clinical trial at Children’s Hospital of Philadelphia.
Researchers said they aim to expand the trial to six research centers nationwide, though it will take years to identify and sign up more patients. The trial is sponsored by Eli Lilly and its subsidiary Akouos, a small biotech company.
Worldwide, Aissam’s trial is one of five planned or underway to test gene therapy for deafness. Researchers said they are hopeful early findings could lead to gene therapies for other forms of congenital deafness.
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