RP | Gene Therapy | Stage: ApprovedStatus: Approved | Children & adults · FDA label has no age bounds; trial: 10 months–16 yearsBiallelic OTOF severe-to-profound deafness | |
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EL | Gene Therapy | Stage: Phase 2Status: Active | Children & adults · Cohort-specific; no overall age limitBiallelic OTOF profound bilateral auditory neuropathy | |
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S | Small Molecule | Stage: Phase 2Status: Active | Adults · ≥18 yearsSSNHL, cisplatin, or cochlear-implant cohorts | |
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S | Gene Therapy | Stage: Phase 1/2Status: Active | Children · 6–31 monthsBiallelic OTOF deafness with preserved outer-hair-cell function | |
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RT | Cell Therapy | Stage: Phase 1/2Status: Active | Adults · 18–74 yearsCochlear-implant candidates with presbycusis or auditory neuropathy | |
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AP | Small Molecule | Stage: Phase 2Status: Active | Adults · 18–85 yearsRecent severe unilateral ISSNHL | |
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SP | Small Molecule | Stage: Phase 3Status: Active | Adults · 18–75 yearsActive Ménière’s disease | |
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AT | Small Molecule | Stage: Phase 2Status: Active | Adults · 18–45 yearsTesticular cancer receiving high-dose cisplatin | |
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CS | Small Molecule | Stage: Phase 1/2Status: Active | Adults · 45–75 yearsType 2 diabetes with speech-in-noise difficulty | |
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NS | Biologic | Stage: Phase 1/2Status: Active | Adults · 19–70 yearsSteroid-treated persistent unilateral SSNHL | |
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S | Gene Therapy | Stage: PreclinicalStatus: Active | Children & adults · Not yet defined (preclinical)GJB2-related DFNB1A nonsyndromic deafness | |
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LC | Cell Therapy | Stage: PreclinicalStatus: Active | Children & adults · Not yet defined (preclinical)Auditory neuropathy spectrum disorders | |
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BI AOK-1Boehringer Ingelheim | Regenerative Medicine | Stage: PreclinicalStatus: Active | Adults · Not yet defined (preclinical)Noise- or age-related cochlear hair-cell loss | |
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FT | Regenerative Medicine | Stage: Phase 2Status: Discontinued | Adults · 18–65 yearsStable noise- or sudden-onset SNHL | |
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AT | Biologic | Stage: Phase 3Status: Discontinued | Adults · ≥18 yearsSevere acute unilateral SSNHL | |
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FT | Regenerative Medicine | Stage: Phase 1Status: Discontinued | Adults · 18–67 yearsAcquired 40–80 dB SNHL | |
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O | Biologic | Stage: Phase 2Status: Discontinued | Adults · 21–64 yearsSpeech-in-noise hearing impairment | |
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N | Gene Therapy | Stage: Phase 1/2Status: Completed | Adults · 18–75 yearsNon-genetic severe-to-profound hearing loss | |
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OP | Small Molecule | Stage: Phase 1Status: Completed | Adults · 18–55 yearsHazardous noise or blast exposure | |
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O | Gene Therapy | Stage: PreclinicalStatus: Discontinued | Children & adults · Not yet defined (preclinical)Congenital GJB2-deficient hearing loss | |
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CT | Small Molecule | Stage: Phase 1/2Status: Completed | Adults · 18–75 yearsBilateral SNHL with speech-in-noise deficits | |
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SE | Gene Therapy | Stage: Phase 1/2Status: Active | Children · 1–17 yearsBiallelic OTOF DFNB9 severe-to-profound deafness | |
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OT | Gene Therapy | Stage: Interventional/NAStatus: Unknown | Children & adults · ≥1 year; no upper limit listedBiallelic OTOF DFNB9 severe-to-total deafness | |
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SA | Small Molecule | Stage: Phase 3Status: Completed | Adults · ≥18 yearsAcute idiopathic or noise-trauma SSNHL | |
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