FDA Grants Rare Pediatric Disease Designation to Sensorion’s OTOF-GT for the Treatment of Otoferlin Gene-Mediated Hearing Loss
MONTPELLIER, FRANCE — Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, announces that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to the Company’s lead therapy gene candidate, OTOF-GT, a gene therapy intended…
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High School Student Creates Website Devoted to the Genetic Condition, TMPRSS3
A 15-year-old from Nebraska with cochlear implants and diagnosed at birth with hearing loss due to TMPRSS3 recently created a web resource devoted to TMPRSS3, the fifth most common gene causally associated with deafness. For her efforts, Lily Ardinger Stibal received the Girl Scout Gold Award, the highest honor a Girl Scout can receive. Her…
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Myrtelle and Forge Biologics Partner to Advance Hearing Loss Gene Therapy into Clinical Trials
WAKEFIELD, MASS. & COLUMBUS, OH –Myrtelle Inc., a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, have announced a manufacturing partnership that will advance Myrtelle’s novel gene therapy for monogenic hearing loss, Myr-201, into clinical trials for patients with…
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Genes Associated with Age-Related Hearing Loss Visualized in New Study
UPPSALA, SWEDEN — Researchers from Uppsala University have been able to document and visualize hearing loss-associated genes in the human inner ear, in a unique collaboration study between otosurgeons and geneticists. The findings illustrate that discrete subcellular structures in the human organ of hearing, the cochlea, are involved in the variation of risk of age-related…
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Sensorion Appoints Gene Therapy and Rare Disease Expert Nora Yang as Chief Scientific Officer
MONTPELLIER, FRANCE — Sensorion, a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, today announces the appointment of Nora Yang, Ph.D., a leader in gene therapy and rare diseases with extensive leadership experience in industry, academia, and government, as…
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New Stem Cell Technique May Help Treat Common Hereditary Deafness, Say Researchers
TOKYO, JAPAN — Researchers at Juntendo University report in the journal Human Molecular Genetics that a stem-cell technique may be used to develop a treatment of a common mutation-related type of deafness. The method involves reproducing cells occurring in the human inner ear where the researchers emphasize that ‘the pathological condition could be reproduced with…
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Akouos Receives Orphan Drug and Rare Pediatric Disease Designations for AK-OTOF for the Treatment of Otoferlin Gene-Mediated Hearing Loss
BOSTON, MASSACHUSSETTS — Akouos, Inc. (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for AK-OTOF, a gene therapy intended…
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Researchers Develop Genetic Patch to Prevent Hereditary Deafness
NIJMEGEN, NETHERLANDS — They can hear well up to about forty years old, but then suddenly deafness strikes people with DFNA9. The cells of the inner ear can no longer reverse the damage caused by a genetic defect in their DNA. Researchers at Radboud university medical center have now developed a “genetic patch” for this…
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Decibel Therapeutics and Invitae Announce Launch of Amplify™ Genetic Testing Program
BOSTON, MASSACHUSSETTS – Decibel Therapeutics, a clinical-stage biotechnology company dedicated to discovering and developing treatments to restore and improve hearing and balance, has announced a partnership with Invitae, a leading medical genetics company, to launch AmplifyTM, a no-charge genetic testing program to screen for the genetic cause of congenital hearing loss in children diagnosed with auditory neuropathy.…
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Akouos and Blueprint Genetics Announce Resonate Program to Improve Access to Genetic Testing for Auditory Neuropathy
BOSTON, MASSACHUSSETTS – Akouos, Inc. (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, and Blueprint Genetics, a Quest Diagnostics company, today announced the Resonate program. The program offers genetic testing to eligible individuals with auditory neuropathy at no cost to participants, their…
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