Researchers Advance Gene Therapy for CLIC5-Related Inner Ear Dysfunction
The therapy targets mutations in the CLIC5 gene, which affect hair cell stability and lead to progressive hearing and balance impairments.
Tag: gene therapy
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Gene Therapy Restores Hearing in Children with Hereditary Deafness
Researchers found the novel gene therapy to be an effective treatment for patients with a specific form of autosomal recessive deafness.
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CILcare Appoints Aurore Marie Global Head, R&D
CILcare, a CRO specializing in ear disorders, announced the appointment of Aurore Marie at the position of Global Head, R&D Operations Management. In her new role, Marie will drive support to production activities at the global level and will serve as a new member of CILcare’s Board, according to the company.
Sonova to Acquire 3.7% Ownership Stake in Sensorion
Sensorion, a clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat, and prevent within the field of hearing loss disorders, announced that Sonova Holding AG will acquire a 3.7% ownership stake in Sensorion by way of subscription to a reserved share capital increase for total gross proceeds of €5 million (USD $6.08 million).
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‘Common Inherited Genetic Variant’ Identified as Frequent Cause of Adult Deafness
While the particular manifestations of this genetically induced hearing loss varied, as did the age at which hearing problems began, its prevalence suggests that it is common (highly penetrant) and that many thousands of people might be at risk of deafness as a result, explain the researchers.
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Sensorion Appoints Géraldine Honnet as Chief Medical Officer; Juergen Heitmann as Chief Business Officer
Honnet is a medical doctor and joins the company from Généthon, where she spent nine years as Chief Medical Officer, overseeing international gene therapy clinical trials in multiple rare diseases.
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Akouos Launches Sing Registry to Track Genetic Changes in Hearing, Treatments
The Sing Registry is a new, observational research study focused on understanding the lifelong impact of genetic sensorineural hearing loss (SNHL), according to the company.
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Akouos Announces Gene Therapy to Treat Genetic Hearing Loss
AK-OTOF is intended to treat individuals with sensorineural hearing loss due to mutations in the OTOF gene, who typically have severe hearing loss in both ears from birth, by promoting the expression of normal, functional otoferlin protein in affected cells of the cochlea.
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Sensorion Appoints John Furey As Independent Board Member
Furey was most recently Chief Operating Officer (COO) for Spark Therapeutics, a global gene therapy developer. This appointment will be effective at the next meeting of the board of directors of the company, in September 2019.
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Sensorion Awarded $11 Million Research Grant for Congenital Deafness
The aim of this project is to develop a specific gene therapy program aimed at correcting an inherited monogenic form of deafness caused by a mutation in the gene encoding for the Otoferlin protein (DNBF9).
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Sensorion Awarded $11 Million Research Grant for Congenital Deafness
The aim of this project is to develop a specific gene therapy program aimed at correcting an inherited monogenic form of deafness caused by a mutation in the gene encoding for the Otoferlin protein (DNBF9).
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Sensorion Partners with Institut Pasteur on Research Framework Targeting Hearing Loss
Sensorion and Institut Pasteur have leveraged the last six months to reach a partnership framework agreement. This agreement provides for, after completion of a research program, an exclusive option to obtain licenses.
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Cold Spring Harbor Laboratory Press Releases ‘Function and Dysfunction of the Cochlea’
The book describes major advances in our understanding of the pathogenic processes underlying various forms of hearing loss and the emergence of treatments for deafness.
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Researchers Restore Hearing via Gene Therapy in Deaf Mouse Model
Researchers have managed to restore hearing in an adult mouse model of DFNB9 deafness, a hearing disorder that represents one of the most frequent cases of congenital genetic deafness
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Sensorion and Pasteur Institute to Collaborate on Gene Therapy Products
Sensorion announced a letter of intent with Pasteur Institute in Paris to exclusively negotiate a framework agreement to obtain the exclusive licenses to develop and commercialize gene therapy product candidates for restoration, treatment, and prevention of hearing loss disorders. They will collaborate on several lead programs to correct monogenic forms of hereditary hearing loss including, among others, the Usher syndrome type 1 and otoferlin deficiency, according to the announcement.
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Research Study Finds Neurogenesis Can Help Improve Cognitive Function in Mouse Model of Alzheimer’s
The investigation shows that the beneficial effects on cognition can be blocked by the hostile inflammatory environment present in the brains of patients with Alzheimer’s disease and that physical exercise can “clean up” the environment, allowing new nerve cells to survive, thrive, and improve cognition in the Alzheimer’s mice.
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Akouos Raises $50 Million in Series A Financing; Forms Scientific Advisory Board
Akouos is developing targeted adeno-associated viral vector (AAV)-based gene therapies for sensorineural hearing loss, which results from dysfunction or damage to sensory cells and/or nerve fibers of the inner ear.
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Akouos Announces $7.5 Million Seed Round, Strategic License Agreement for Hearing Disorder Gene Therapy Platform
Anc-AAVs are in silico-designed adeno-associated viral vectors (AAVs), first developed in the laboratory of Dr Luk H. Vandenberghe, assistant professor of ophthalmology at Harvard Medical School, and director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear.
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