Product Description
AAV.104 aims to restore hearing to individuals with a STRC deficiency, the second most common cause of autosomal recessive, non-syndromic, congenital hearing loss. STRC is a large, extracellular, structural protein expressed in outer hair cells of the cochlea. Functional outer hair cells amplify sound within the ear, a process required for normal hearing sensitivity and frequency selectivity. AAV.104 is designed to express STRC selectively in outer hair cells, thus providing STRC specifically in its natural cellular location. (Sourced from: https://www.decibeltx.com/pipeline/)
Mechanisms of Action: Gene Therapy,STRC
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Regeneron
Company Location: TARRYTOWN NY 10591
Company CEO: Leonard S. Schleifer
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
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Recent & Upcoming Milestones
Highest Development Phases
Phase 0: Hearing Loss
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