Program

Target

INDICATION
RESEARCH
IND-ENABLING
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

Gene Therapies for Congenital, Monogenic, Hearing Loss
OTOF-Related Hearing Loss
Program

Target

RESEARCH
IND-ENABLING
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

GJB2-Related Hearing Loss
Program

Target

RESEARCH
IND-ENABLING
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

STRC-Related Hearing Loss
Program

Target

RESEARCH
IND-ENABLING
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

Gene Therapies for Hair Cell Regeneration
AAV.201 ATOH1+ Reprogramming Factor
Bilateral Vestibulopathy
Program

Target

RESEARCH
IND-ENABLING
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

AAV.201 ATOH1+ Reprogramming Factor Bilateral Vestibulopathy
Bilateral Vestibulopathy
Program

Target

RESEARCH
IND-ENABLING
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

Cochlear Hair Cell Regeneration
Sensorineural Hearing Loss
Program

Target

RESEARCH
IND-ENABLING
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

Cochlear Hair Cell Regeneration Sensorineural Hearing Loss
Otoprotection Therapeutic
Cisplatin-Induced Hearing Loss
Program

Target

RESEARCH
IND-ENABLING
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

Phase 1b

Gene Therapies for Congenital, Monogenic, Hearing Loss

PROGRAM Target
INDICATION
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

OTOF-Related Hearing Loss
PROGRAM Target
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

GJB2-Related Hearing Loss
PROGRAM Target
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

AAV.104*
Autosomal Recessive Hearing Disorder
PROGRAM Target
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

AAV.104* Autosomal Recessive Hearing Disorder

Gene Therapies for Hair Cell Regeneration

PROGRAM Target
INDICATION
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

AAV.201 ATOH1+ Reprogramming Factor
Bilateral Vestibulopathy
PROGRAM Target
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

AAV.201 ATOH1+ Reprogramming Factor Bilateral Vestibulopathy
Bilateral Vestibulopathy
PROGRAM Target
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

Cochlear Hair Cell Regeneration
Sensorineural Hearing Loss
PROGRAM Target
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

Cochlear Hair Cell Regeneration Sensorineural Hearing Loss

Otoprotection Therapeutic

PROGRAM Target
INDICATION
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

Cisplatin-Induced Hearing Loss
PROGRAM Target
DISCOVERY
PRECLINICAL
EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

*Currently included in the Decibel Regeneron collaboration.