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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.

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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.

Research Study Finds Neurogenesis Can Help Improve Cognitive Function in Mouse Model of Alzheimer’s

by Stefani Kim | Sep 12, 2018 | Research | 0

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.

Akouos Raises $50 Million in Series A Financing; Forms Scientific Advisory Board

by Stefani Kim | Aug 9, 2018 | Hearing Disorders, Industry News, Research | 0

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.

Akouos Announces $7.5 Million Seed Round, Strategic License Agreement for Hearing Disorder Gene Therapy Platform

by Stefani Kim | Dec 6, 2017 | Research | 0

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.

CIB2 Protein Found to be Part of Sound Wave Translation Process

by Stefani Kim | Jun 30, 2017 | Components, Research | 1

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.

Gene Therapy Used to Restore Hearing and Balance in Mice with Usher Syndrome

by Christa Nuber | Mar 28, 2017 | Vestibular Care, Vestibular Testing, Vestibular Training, Vestibular Treatment | 0

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.

Two Studies Detail Improved Gene Therapy for Hearing Restoration in Mice

by Christa Nuber | Feb 28, 2017 | Cochlear Implants, In the Ear, Industry News, Research | 0

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.

Partial Hearing and Balance Restored in Deaf Mice Using New Gene-delivery Therapy

by Karl | Jan 31, 2017 | Vestibular Care, Vestibular Testing, Vestibular Training, Vestibular Treatment | 0

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.

Tag: gene therapy

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Sensorion Announced Orphan Drug Designation for OTOF-GT

Gene Therapy for Hearing Loss Reversal

CILcare Appoints Aurore Marie Global Head, R&D

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Akouos’ AK-OTOF Receives Orphan Drug Designation

OSU Researchers Study Otoferlin Mutations

UPenn Researchers ID New Cause of Hearing Loss

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Sensorion Announces Gene Therapy Collaboration

TAU Scientists Develop Gene Therapy for Deafness

Sonova to Acquire 3.7% Ownership Stake in Sensorion

‘Common Inherited Genetic Variant’ Identified as Frequent Cause of Adult Deafness

Sensorion Appoints Géraldine Honnet as Chief Medical Officer; Juergen Heitmann as Chief Business Officer

Akouos Launches Sing Registry to Track Genetic Changes in Hearing, Treatments

Akouos Announces Gene Therapy to Treat Genetic Hearing Loss

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Sensorion Appoints John Furey As Independent Board Member

Sensorion Awarded $11 Million Research Grant for Congenital Deafness

Sensorion Awarded $11 Million Research Grant for Congenital Deafness

Sensorion Partners with Institut Pasteur on Research Framework Targeting Hearing Loss

Cold Spring Harbor Laboratory Press Releases ‘Function and Dysfunction of the Cochlea’

Researchers Restore Hearing via Gene Therapy in Deaf Mouse Model

Sensorion and Pasteur Institute to Collaborate on Gene Therapy Products