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The Sound of Success
Photo by Webb Chappell
A groundbreaking gene therapy developed by Akouos, Inc., a precision genetic medicine company founded in 2016 by Emmanuel (Manny) Simons (MBA 2012), has enabled an 11-year-old boy from Morocco to hear sounds for the first time. According to a recent New York Times article, Aissam Dam, who was born deaf, became the first person to receive gene therapy for congenital deafness during a clinical trial at the Children’s Hospital of Philadelphia. “There’s no sound I don’t like,” he said through interpreters during an interview in January, the Times reports. “They’re all good.”
Last week, Simons and researchers from the other four other trials around the world presented their findings at the winter meeting of the Association for Research in Otolaryngology. While trials will need to be conducted on many more patients, the Times article notes that the initial success of the gene therapy could lead to others that target different forms of congenital deafness.
Boston-based Akouos (Greek, for “to listen”), acquired by Eli Lilly in 2022, focuses on developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss. In a 2021 Alumni Bulletin article that featured Simons and his work, he noted that there are more than 150 genes linked to hearing loss and that disabling hearing loss affects more than 400 million people across the globe. “In some infants with profound hearing impairment, there’s a mutation in one of those genes that results in loss of a protein that inner-ear cells need to convert sound waves into electrical activity the brain can interpret as sound,” said Simons, who received a 2016 HBS Blavatnik Fellowship. “Getting a healthy copy of the critical gene to the appropriate cells is a solution, but precision inner-ear drug delivery has been a historical stumbling block. An early breakthrough for us was figuring out a way to pair gene therapy with a one-time surgical administration to deliver genes to the inner ear with unprecedented efficiency.”
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