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Global Employees
69
This segment focuses on the research, development, and clinical trials of gene therapies to treat various forms of hearing loss. Akouos utilizes its proprietary adeno-associated viral (AAV) vector platform to deliver therapeutic genes to the inner ear. The lead product candidate, AK-OTOF, targets hearing loss caused by OTOF gene mutations. Research and development activities include preclinical studies, clinical trial design and execution, and manufacturing process development. The segment aims to address the significant unmet medical need for individuals with hearing loss, potentially restoring or improving hearing function. Market positioning is centered on being a pioneer in the field of genetic medicine for hearing disorders, with a competitive advantage in its targeted approach and platform technology. Future opportunities include expanding the pipeline to address other genetic causes of hearing loss and exploring partnerships for commercialization. Regulatory and clinical aspects involve navigating FDA approval processes and conducting clinical trials to demonstrate safety and efficacy. This segment is a core focus of Akouos's business strategy, with the potential to transform the treatment landscape for hearing impairment.
This segment is dedicated to the development of gene therapy for vestibular schwannoma, a benign tumor affecting the auditory nerve. Akouos is developing AK-antiVEGF, a gene therapy designed to deliver an anti-VEGF agent to the tumor site. Research and development efforts include preclinical studies to assess the efficacy and safety of the therapy, as well as clinical trial design and execution. The segment leverages the company's AAV vector platform for targeted delivery. The therapeutic area addresses the need for effective treatments for vestibular schwannoma, potentially reducing tumor growth and preserving hearing. Market positioning involves establishing a presence in the treatment of neurological disorders, with a focus on precision medicine. Future opportunities include expanding the application of the platform to other neurological conditions. Regulatory and clinical aspects involve conducting clinical trials to demonstrate safety and efficacy and obtaining regulatory approvals. This segment represents a strategic expansion of Akouos's therapeutic focus, with the potential to provide a novel treatment option for patients with vestibular schwannoma.
This segment focuses on developing gene therapy for the auditory manifestations of Usher syndrome 3A (USH3A), a rare genetic disorder causing hearing loss and vision impairment. Akouos is developing AK-CLRN1, a gene therapy designed to address the hearing loss component of USH3A. Research and development activities include preclinical studies, clinical trial design, and manufacturing process development. The segment aims to address the significant unmet medical need for individuals with USH3A, potentially improving their hearing function. Market positioning is centered on being a pioneer in the field of genetic medicine for rare diseases, with a competitive advantage in its targeted approach and platform technology. Future opportunities include expanding the pipeline to address other genetic causes of hearing loss and exploring partnerships for commercialization. Regulatory and clinical aspects involve navigating FDA approval processes and conducting clinical trials to demonstrate safety and efficacy. This segment is a core focus of Akouos's business strategy, with the potential to transform the treatment landscape for USH3A.