基因疗法DB-ATO在大多数患有遗传性聋哑的儿童恢复听力,有可能取代耳垂植入。 Gene therapy DB-OTO restored hearing in most children with genetic deafness, potentially replacing cochlear implants.

根据CHORD试验的结果,称为DB-OTO的基因疗法使由于OTOF基因突变而丧失深刻遗传听力的儿童的听力得到显著、持久的改善。 A gene therapy called DB-OTO led to significant, lasting hearing improvements in children with profound genetic hearing loss due to OTOF gene mutations, according to results from the CHORD trial. 在12名参加者中,11名在几周内显示临床上有意义的听力增益,3名达到正常的听力水平。 Of 12 participants, 11 showed clinically meaningful hearing gains within weeks, with three achieving normal hearing levels. 9人达到70分B或70分B以上的主要听力临界点,有可能消除耳塞植入的需要。 Nine met the primary endpoint of hearing thresholds at 70 dB or better, potentially eliminating the need for cochlear implants. 所有被评估的儿童的言语感觉都有所改善,治疗得到良好缓解,没有发生严重的不良事件。 Speech perception improved in all assessed children, and the treatment was well-tolerated with no serious adverse events. Regeneron计划于2025年晚些时候提交美国管制申请。 Regeneron plans to submit a U.S. regulatory application later in 2025.