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Number of successful DFNB9 gene therapy treatments reported by end of 2024
4
Resolution / Starting Odds
10-20 • 33%
21-30 • 33%
31+ • 34%
Published studies or official reports from medical institutions
Story
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%
0-5 • 25%
6-10 • 25%
11-20 • 25%
More than 20 • 25%
Continue development • 33%
Discontinue program • 33%
Partner with another company • 33%
Under 5 • 25%
5-10 • 25%
11-20 • 25%
More than 20 • 25%
Yes • 50%
No • 50%
Successful phase 3 trial by another company • 25%
New regulatory approval • 25%
Major partnership or acquisition • 25%
Another failed trial • 25%
Yes • 50%
No • 50%
Less than 100 • 34%
100-500 • 33%
More than 500 • 33%
Seek further regulatory approvals • 33%
Begin commercial production • 33%
Partner with another company for distribution • 34%