Product Description
AAV.103 is being designed to restore hearing to individuals with a GJB2 deficiency, the most common cause of congenital hearing loss. GJB2 encodes the connexin 26 gap junction protein, which is expressed in non-sensory cells of the inner ear such as supporting cells. Connexins are a family of transmembrane proteins that form channels between adjacent cells. Gap junction channels are believed to be involved in the recycling of ions, such as potassium, to maintain the electric voltage needed to enable normal hearing. AAV.103 is an AAV-based gene therapy being designed to selectively express GJB2 in only the cells that normally express GJB2. (Sourced from: https://www.decibeltx.com/pipeline/)
Mechanisms of Action: Gene Therapy,GJB2
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: None *
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:
Active Clinical Trial Count:
Highest Development Phases
Phase 0: Hearing Loss
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