Gene Therapy Trial Shows Restored Hearing in Children Born Deaf
A novel gene therapy restored hearing in both ears of children with inherited deafness, improving hearing function and speech perception.
Tag: gene therapy
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Sensorion Announced Orphan Drug Designation for OTOF-GT
Sensorion (FR0012596468 – ALSEN) 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 the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to OTOF-GT, Sensorion’s lead gene therapy program, intended for the treatment of otoferlin gene-mediated hearing loss. Sensorion is on track to file a Clinical Trial Application for OTOF-GT in the first half of 2023.
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Sensorion Announces Gene Therapy Collaboration
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.
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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CIB2 Protein Found to be Part of Sound Wave Translation Process
The 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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Gene Therapy Used to Restore Hearing and Balance in Mice with Usher Syndrome
In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of Medicine showed that gene therapy was able to restore balance and hearing in genetically modified mice that mimic Usher syndrome.
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Two Studies Detail Improved Gene Therapy for Hearing Restoration in Mice
Two new papers published in Nature Biotechnology on February 6 detail research by scientists at Boston Children’s Hospital—a pediatric teaching hospital affiliated with Harvard Medical School—on an improved gene therapy that helps restore hearing to genetically deaf mice.
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Partial Hearing and Balance Restored in Deaf Mice Using New Gene-delivery Therapy
Using a novel form of gene therapy, scientists from Harvard Medical School and the Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic condition that affects both. The new model reportedly overcomes a long-standing barrier to accessing hair cells which have been notoriously difficult to treat with previous gene-delivery techniques.
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Scientists Close in on Gene Therapy for Congenital Deafness
Scientists in Boston and Switzerland believe that their mice study of the TMC1 gene will lead to a gene therapy protocol for humans in roughly 5-10 years.
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