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DFNB9 gene therapy trial expanded to over 50 children by end of 2024?
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Gene Therapy in China Restores Hearing in Five Deaf Children
Jun 5, 2024, 09:20 AM
A pioneering gene therapy trial in China has successfully restored hearing in both ears of children who were born deaf. The trial, which involved five children, provided a normal copy of a mutated gene, specifically targeting a condition called DFNB9 caused by Otof mutations. This condition accounts for 2-8% of all congenital hearing loss. As a result of the therapy, the children can now listen and dance to music. The study was published in Nature Medicine and The Lancet.
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Success • 33%
Partial Success • 33%
Failure • 33%
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Continue development • 33%
Discontinue program • 33%
Partner with another company • 33%
Successful phase 3 trial by another company • 25%
New regulatory approval • 25%
Major partnership or acquisition • 25%
Another failed trial • 25%
Seek further regulatory approvals • 33%
Begin commercial production • 33%
Partner with another company for distribution • 34%