AGTC & Otonomy Announce Strategic Collaboration to Develop & Commercialize Gene Therapy 

Applied Genetic Technologies Corporation and Otonomy, Inc. recently announced they have entered into 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 gap junction protein beta 2 gene (GJB2)— the most common cause of congenital hearing loss.

Mutations in GJB2 account for approximately 30% of all genetic hearing loss cases. Patients with this mutation can have severe-to-profound deafness in both ears that is identified in screening tests routinely performed in newborns. Under the collaboration agreement, the parties will equally share the program costs and proceeds, and can include additional genetic hearing loss targets in the future.

“There is a tremendous unmet need for effective drug therapies to treat sensorineural hearing loss and this is especially true in children born with severe or progressive hearing loss due to genetic mutations,” said Lawrence R. Lustig, MD, Chair, Department of Otolaryngology – Head & Neck Surgery, Columbia University College of Physicians and Surgeons. “Gene therapy holds great promise to restore functional and lasting hearing in these cases, with early intervention providing significant benefits in the development of language skills, socialization and overall quality of life for patients and their families.”

“We are excited to initiate this collaboration which combines our deep capabilities, technology and expertise in gene therapy development and manufacturing with Otonomy’s extensive experience in otic drug delivery and existing infrastructure for preclinical and clinical development in neurotology,” added Sue Washer, President and CEO of AGTC. “In addition to the positive data that we recently released supporting our XLRP and achromatopsia clinical trials, advancing this program in neurotology further demonstrates the versatility of our gene therapy platform and its applicability to treat a broad range of indications.”

“As leaders in neurotology, we are pleased to add our expertise to that of the highly experienced AGTC team, leveraging what each of us does best and sharing the cost and effort to bring a novel therapeutic to patients with profound need,” said David A. Weber, PhD, President and CEO of Otonomy. “The addition of this gene therapy program further extends our broad, innovative pipeline across the largest market opportunities in neurotology including acquired and genetic forms of hearing loss, tinnitus, and balance disorders such as Ménière’s disease.”

Hearing loss is the most common inherited sensory disorder. In developed countries such as the United States, genetic mutations are responsible for the vast majority of hearing loss in young children, which is estimated to affect 1 out of 500 children before speech develops. Because congenital hearing loss is one of the most prevalent chronic conditions in children, neonatal screening is routinely performed. This is typically followed by genetic testing in those cases where a deficit has been detected. Mutations have been identified in over 100 genes including the gap junction protein beta 2 gene (GBJ2), which is the most frequent cause of severe-to-profound congenital hearing loss. The GJB2 gene encodes connexin-26 which is expressed in cochlear support cells, forming gap junctions that control potassium homeostasis which is critical for the survival and function of hair cells and normal hearing. Mutations in GJB2 impair gap junctions and cochlear homeostasis leading to hair cell dysfunction and hearing loss. The goal of GJB2 gene therapy is to restore functional gap junctions and preserve hair cells to improve hearing.

AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS) and other ophthalmology and otology indications. The optogenetics program is being developed in collaboration with Bionic Sight. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products. For more information, visit

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. For more information, visit

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