ASPO Session to Cover Sensorion Audiogene Trial of Pediatric Therapy for Hearing Loss
Sensorion will present early clinical trial results of its gene therapy SENS-501 for treating severe pediatric hearing loss at the 2025 ASPO meeting.
Sensorion will present early clinical trial results of its gene therapy SENS-501 for treating severe pediatric hearing loss at the 2025 ASPO meeting.
The trial demonstrated pharmacologic hearing restoration within 30 days of AK-OTOF gene therapy administration in the first participant.
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.
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.
Read MoreHonnet 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.
Read MoreThe Sing Registry is a new, observational research study focused on understanding the lifelong impact of genetic sensorineural hearing loss (SNHL), according to the company.
Read MoreAK-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.
Read MoreFurey 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.
Read MoreThe 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).
Read MoreThe 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).
Read MoreSensorion 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.
Read MoreThe book describes major advances in our understanding of the pathogenic processes underlying various forms of hearing loss and the emergence of treatments for deafness.
Read MoreResearchers 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
Read MoreSensorion 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.
Read MoreThe 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.
Read MoreAkouos 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.
Read MoreAnc-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.
Read MoreThe findings were published June 29 in “Nature Communications.“ The study is reportedly the first to illuminate in detail how a particular protein, which is known as CIB2, allows hearing to work.
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