Pipeline - Decibel Therapeutics

Program

Target

INDICATION

RESEARCH

IND-ENABLING

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

Gene Therapies for Congenital, Monogenic, Hearing Loss

DB-OTO* Otoferlin

OTOF-Related Hearing Loss

Program

Target

RESEARCH

IND-ENABLING

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

DB-OTO* Otoferlin OTOF-Related Hearing Loss

Modality: DB-OTO is an AAV dual vector gene therapy delivered via intracochlear injection.

Mechanism: OTOF is a large protein expressed in cochlear inner hair cells that enables communication between the sensory cells of the inner ear and the auditory nerve by regulating synaptic transmission. Newborns born with mutations in the OTOF gene have fully developed structures within the inner ear. However, these newborns have profound hearing loss because signaling between the ear and the brain is disrupted. DB-OTO is an AAV-based dual-vector gene therapy product candidate designed to selectively express functional OTOF in the inner hair cells of individuals with OTOF deficiency with the goal of enabling the ear to transmit sound to the brain and facilitate hearing.

AAV.103* GJB2

GJB2-Related Hearing Loss

Program

Target

RESEARCH

IND-ENABLING

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

AAV.103* GJB2 GJB2-Related Hearing Loss

AAV.104* Stereocilin

STRC-Related Hearing Loss

Program

Target

RESEARCH

IND-ENABLING

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

AAV.104* Stereocilin STRC-Related Hearing Loss

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

DB-ATO ATOH1

Bilateral Vestibulopathy

Program

Target

RESEARCH

IND-ENABLING

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

DB-ATO ATOH1 Bilateral Vestibulopathy

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

DB-020 Cisplatin Inactivation

Cisplatin-Induced Hearing Loss

Program

Target

RESEARCH

IND-ENABLING

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

DB-020 Cisplatin Inactivation Cisplatin-Induced Hearing Loss

Phase 1b

About the Program: DB-020 is being developed for prevention of cisplatin-induced hearing loss. Cisplatin is one of the most commonly used chemotherapeutics despite severe, dose-limiting side effects. Ototoxicity is one of the most common serious adverse effects, leading to permanent hearing loss in the majority of patients. DB-020 is optimized for local delivery to the ear, which may enable protection of hearing without impacting the beneficial effect of cisplatin.

Development Phase: A randomized, double-blind, placebo-controlled Phase 1 clinical trial in normal healthy volunteers was completed in 2019. A randomized, double-blind, placebo-controlled, multicenter Phase 1b clinical trial of DB-020 in patients undergoing treatment with cisplatin is now ongoing.

Gene Therapies for Congenital, Monogenic, Hearing Loss

PROGRAM Target

INDICATION

DISCOVERY

PRECLINICAL

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

DB-OTO* Otoferlin

OTOF-Related Hearing Loss

PROGRAM Target

DISCOVERY

PRECLINICAL

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

DB-OTO* Otoferlin OTOF-Related Hearing Loss

Modality: DB-OTO is an AAV dual vector gene therapy delivered via intracochlear injection.

AAV.103* GJB2

GJB2-Related Hearing Loss

PROGRAM Target

DISCOVERY

PRECLINICAL

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

AAV.103* GJB2 GJB2-Related Hearing Loss

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

DB-ATO ATOH1

Bilateral Vestibulopathy

PROGRAM Target

DISCOVERY

PRECLINICAL

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

DB-ATO ATOH1 Bilateral Vestibulopathy

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)

DB-020 Cisplatin Inactivation

Cisplatin-Induced Hearing Loss

PROGRAM Target

DISCOVERY

PRECLINICAL

EARLY CLINICAL

(Phase 1/2)

LATE CLINICAL

(Phase 3)

DB-020 Cisplatin Inactivation Cisplatin-Induced Hearing Loss

*Currently included in the Decibel Regeneron collaboration.

Learn about our approach