![]() Early onset profound hearing loss in KO mice and lack of information about the cochlear cell type that requires Clrn1 expression pose challenges to therapeutic investigation. Clrn1 knockout (KO) mice develop hair cell defects by postnatal day 2 (P2) and are deaf by P21-P25. “We appreciate the opportunity to partner with CWRU and Akouos to commercialize this technology that will bring hope to patients impacted by hearing loss due to USH3A,” said Neil Wyant, managing director of UH Ventures, the innovation and commercialization arm of UH.Usher syndrome type III (USH3) characterized by progressive loss of vision and hearing is caused by mutations in the clarin-1 gene ( CLRN1). Licensing manager in Case Western Reserve’s Technology Transfer Office. Hearing loss caused by Usher syndrome,” said Stephanie Weidenbecher, senior Opportunity for us to translate this to the first potential therapy to prevent “Partnering with Akouos provides an excellent Usher syndrome, and deepen our understanding of the genetics driving this and The field of precision genetic medicines for deaf-blind disorders, such as Alagramam and Case Western Reserve aims to advance ![]() Individuals worldwide with genetically-driven hearing loss or deaf-blindĭisorders, it is imperative that companies, academic research institutions, andĪdvocacy organizations work together and accelerate toward creative solutions. “With no FDA-approved medicines available for the millions of Search of hearing loss treatment,” said Manny Simons, founder, president andĬEO of Akouos. Pending demonstration of safety and efficacy in future clinical trials.Īkouos, we value working in close collaboration with pioneering investigatorsĪnd institutions to advance precision medicines for individuals and families in Technology in the United States, following additional development efforts and That restore and preserve hearing, licensed the rights to commercialize the The gene therapy may stop the progression of hearing loss and prevent deafness in people with USH3A, a form of hereditary hearing loss linked to defects in the sensory “hair” cells in the inner ear.Ī precision genetic medicine company founded in 2016 to develop gene therapies “Further, lessons learned from this technology couldīe applied to gene therapy as a potential treatment for other forms of Hearing and quality of life for children and adults diagnosed with the geneticĭisorder,” Alagramam said. Loss associated with Usher syndrome type 3A (USH3A) than previously reported,Īnd it revealed the potential for the technology to deliver preservation of Technology allowed us to develop an even more precise animal model of hearing Maniglia Chair for Research and Education, professor of otolaryngology, genetics and genomic sciences, and neurosciences at the Case Western Reserve School of Medicine and director of research at the Ear, Nose & Throat Institute at UH. The technology, jointly owned by Case Western Reserve and University Hospitals (UH), is based on research from the lab of Kumar Alagramam, the Anthony J. Percent of children who are deaf, and another 3% to 6% of children who are Hereditary deaf-blindness cases. The condition accounts for 3% to 6% To 17 per 100,000 people nationally and accounts for about half of all Usher syndrome is subdivided into various subtypes and each subtypeĪccording to NIH, Usher syndrome affects about four Loss is due to the degeneration of light-sensitive tissue (retina) at the back “sensorineural,” or caused by abnormalities of the inner ear. Total hearing loss and vision loss that worsens over time, according to the Usher syndrome is characterized by partial or ![]() Case Western Reserve University and Boston-based Akouos have entered into an exclusive licensing agreement to develop a patented gene therapy with the potential to treat hearing loss associated with a type of Usher syndrome, the most common deaf-blind disorder. ![]()
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