Therapies database

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9 results

Therapy	Approach	Progress	Population	Additional Info
Otarmeni by RegeneronRegeneron Pharmaceuticals Compare	Gene Therapy	Stage: ApprovedStatus: Approved	Children & adults · FDA label has no age bounds; trial: 10 months–16 yearsBiallelic OTOF severe-to-profound deafness
AK-OTOF by Eli Lilly and AkouosEli Lilly and Company Compare	Gene Therapy	Stage: Phase 2Status: Active	Children & adults · Cohort-specific; no overall age limitBiallelic OTOF profound bilateral auditory neuropathy
SENS-501 by SensorionSensorion Compare	Gene Therapy	Stage: Phase 1/2Status: Active	Children · 6–31 monthsBiallelic OTOF deafness with preserved outer-hair-cell function
AK-antiVEGF by Eli Lilly and AkouosEli Lilly and Company Compare	Gene Therapy	Stage: Phase 1/2Status: Active	Adults · ≥18 yearsUnilateral progressive vestibular schwannoma with profound loss
GJB2-GT by SensorionSensorion Compare	Gene Therapy	Stage: PreclinicalStatus: Active	Children & adults · Not yet defined (preclinical)GJB2-related DFNB1A nonsyndromic deafness
CGF166 by NovartisNovartis Compare	Gene Therapy	Stage: Phase 1/2Status: Completed	Adults · 18–75 yearsNon-genetic severe-to-profound hearing loss
OTO-825 by OtonomyOtonomy Compare	Gene Therapy	Stage: PreclinicalStatus: Discontinued	Children & adults · Not yet defined (preclinical)Congenital GJB2-deficient hearing loss
EHT102 by EuHearingShanghai EuHearing Therapeutics Co., Ltd Compare	Gene Therapy	Stage: Phase 1/2Status: Active	Children · 1–17 yearsBiallelic OTOF DFNB9 severe-to-profound deafness
OTOV101N + OTOV101C by OtoVia TherapeuticsOtoVia Therapeutics Compare	Gene Therapy	Stage: Interventional/NAStatus: Unknown	Children & adults · ≥1 year; no upper limit listedBiallelic OTOF DFNB9 severe-to-total deafness

Otarmeni by Regeneron

Regeneron Pharmaceuticals · Gene Therapy · Approved

ApprovedTherapeuticStronger sources

Population

Children & adults · FDA label has no age bounds; trial: 10 months–16 years

Biallelic OTOF severe-to-profound deafness

Evidence

regulatory

Confidence

99%

Indications

Hearing LossSensorineural Hearing LossGenetic Hearing LossOTOF-Related Hearing Loss

AK-OTOF by Eli Lilly and Akouos

Eli Lilly and Company · Gene Therapy · Phase 2

ActiveTherapeuticStronger sources

Population

Children & adults · Cohort-specific; no overall age limit

Biallelic OTOF profound bilateral auditory neuropathy

Evidence

trial

Confidence

95%

Indications

Hearing LossSensorineural Hearing LossGenetic Hearing LossOTOF-Related Hearing Loss

SENS-501 by Sensorion

Sensorion · Gene Therapy · Phase 1/2

ActiveTherapeuticStronger sources

Population

Children · 6–31 months

Biallelic OTOF deafness with preserved outer-hair-cell function

Evidence

trial

Confidence

94%

Indications

Hearing LossSensorineural Hearing LossGenetic Hearing LossOTOF-Related Hearing Loss

AK-antiVEGF by Eli Lilly and Akouos

Eli Lilly and Company · Gene Therapy · Phase 1/2

ActiveTherapeuticStronger sources

Population

Adults · ≥18 years

Unilateral progressive vestibular schwannoma with profound loss

Evidence

trial

Confidence

90%

Indications

Vestibular SchwannomaHearing Loss

GJB2-GT by Sensorion

Sensorion · Gene Therapy · Preclinical

ActiveTherapeuticStronger sources

Population

Children & adults · Not yet defined (preclinical)

GJB2-related DFNB1A nonsyndromic deafness

Evidence

company

Confidence

88%

Indications

Hearing LossSensorineural Hearing LossGenetic Hearing Loss

CGF166 by Novartis

Novartis · Gene Therapy · Phase 1/2

CompletedTherapeutic

Population

Adults · 18–75 years

Non-genetic severe-to-profound hearing loss

Evidence

trial

Confidence

89%

Indications

Hearing LossSensorineural Hearing Loss

OTO-825 by Otonomy

Otonomy · Gene Therapy · Preclinical

DiscontinuedTherapeutic

Population

Children & adults · Not yet defined (preclinical)

Congenital GJB2-deficient hearing loss

Evidence

company

Confidence

87%

Indications

Hearing LossSensorineural Hearing LossGenetic Hearing Loss

EHT102 by EuHearing

Shanghai EuHearing Therapeutics Co., Ltd · Gene Therapy · Phase 1/2

ActiveTherapeutic

Population

Children · 1–17 years

Biallelic OTOF DFNB9 severe-to-profound deafness

Evidence

trial

Confidence

90%

Indications

Hearing LossSensorineural Hearing LossGenetic Hearing LossOTOF-Related Hearing Loss

OTOV101N + OTOV101C by OtoVia Therapeutics

OtoVia Therapeutics · Gene Therapy · Interventional/NA

UnknownTherapeutic

Population

Children & adults · ≥1 year; no upper limit listed

Biallelic OTOF DFNB9 severe-to-total deafness

Evidence

registry+publication

Confidence

86%

Indications

Hearing LossSensorineural Hearing LossGenetic Hearing LossDFNB9

A listing is not proof of safety or effectiveness. Approval and availability may vary by indication and country.

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