gene therapy hearing loss treatment otonomy

Otonomy and AGTC Present Preclinical Results for GJB2 Gene Therapy Hearing Loss Program

SAN DIEGO, CALIFORNIA — Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced preclinical results from the company’s gene therapy collaboration with Applied Genetic Technologies Corporation (AGTC) that support the selection of a product candidate for development as a treatment for gap junction beta-2 (GJB2) deficiency, the most common cause of congenital hearing loss.

These results are being presented during the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting being held virtually May 12-15, 2020.

“Working with AGTC, we have identified novel and proprietary capsids that are capable of transducing support cells of the cochlea, which are the relevant target cells for treating GJB2 deficiency. The selection of a lead capsid and other components for continued development of a GJB2 gene therapy candidate is supported by cochlea explant studies in multiple rodent species as well as in vivo studies in non-human primates that are summarized in today’s ASGCT presentation.”

–David A. Weber, Ph.D., President and CEO of Otonomy

The joint poster presentation by Otonomy and AGTC demonstrate the following:

  • A gene of interest can be expressed in cochlear cells relevant to GJB2 rescue, including support cells of the organ of Corti and spiral limbus, and fibrocytes of the spiral ligament.
  • The tropism and gene expression level in support cells is higher for these novel capsids than for previously reported capsids used in the otic field.
  • Consistent gene expression can be observed in support cells throughout the cochlea for at least 12 weeks following a single local injection.
  • None of the novel AAV capsids evaluated for further development exhibited signs of cellular toxicity.

In October 2019, Otonomy and AGTC announced a strategic collaboration to co-develop and co-commercialize an AAV-based gene therapy to restore hearing in patients with sensorineural hearing loss caused by a mutation in the GJB2 gene.


About Otonomy
Otonomy is a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. The company pioneered the application of drug delivery technology to the ear in order to develop products that achieve sustained drug exposure from a single local administration. This approach is covered by a broad patent estate and is being utilized to develop a pipeline of products addressing important unmet medical needs including Ménière’s disease, hearing loss, and tinnitus. 


Source: Otonomy

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