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)
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.