A 6-year-old boy with Cockayne syndrome — a rare genetic disorder that causes hearing loss, vision impairment, and early death — has become the first child to receive an experimental AAV9 gene therapy developed through a parent-led initiative.
The therapy targets mutations in the CLIC5 gene, which affect hair cell stability and lead to progressive hearing and balance impairments.
Researchers found the novel gene therapy to be an effective treatment for patients with a specific form of autosomal recessive deafness.
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.
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The researchers found that the Norrie Disease Protein (NDP), which is lacking in patients with the rare disease, is essential for the maintenance and survival of hair cells in the cochlea, the cells responsible for hearing.
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Akouos, Inc announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the company’s application for orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. The positive opinion was subsequently adopted by the European Commission.
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Researchers have found a new piece of the puzzle in the quest to use gene therapy to enable people born deaf to hear. The work centers around a large gene responsible for a mutation in the inner-ear protein, otoferlin, which is linked to severe congenital hearing loss.
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A gene called GAS2 plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania.
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Sensorion announced the addition of a new gene therapy target, GJB2 coding for the Connexin 26 protein, to its development portfolio. The target is the third candidate to emerge from Sensorion’s R&D collaboration with Institut Pasteur, according to Sensorion.
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A new study from Tel Aviv University (TAU) presents an innovative treatment for deafness, based on the delivery of genetic material into the cells of the inner ear. The genetic material “replaces” the genetic defect and enables the cells to continue functioning normally.
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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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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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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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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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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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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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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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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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