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Audiogene: A New Hope in Gene Therapy for Treating Profound Hearing Loss in Children

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Mason Walker
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Audiogene: A New Hope in Gene Therapy for Treating Profound Hearing Loss in Children

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Audiogene - A Breakthrough in Gene Therapy

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In a significant leap in genetic therapies for deafness, France has approved a clinical trial for Audiogene. This new gene therapy drug, developed by a French consortium that includes the biotech firm Sensorion, aims to treat hereditary deafness in children aged between 6 and 31 months who are suffering from profound hearing loss.

The Promise of SENS-501

The drug, known as SENS-501, is designed to correct genetic abnormalities in inner ear cells, thereby restoring hearing. It works by injecting a copy of the normal otoferlin gene into the child's impaired inner ear. This innovative treatment targets DFNB9, a type of hereditary deafness caused by mutations in the OTOF gene.

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From Lab to Clinical Trials

The Audiogene clinical trial marks a significant milestone in the field of gene therapy for the treatment of hereditary deafness. As part of the first step in this trial, two doses of SENS-501 will be tested to determine the optimal dose for subsequent trials. This procedure will be performed under general anesthesia by an ENT surgeon.

Using the AAV as a Viral Vector

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A critical aspect of this therapy is the delivery of the gene into the inner ear cells. This is achieved using an adeno-associated virus (AAV) as a viral vector, capable of crossing the cell membrane. This represents a technological innovation in the treatment, providing a new way to treat genetic deafness.

A Collaborative Effort

The Audiogene project is a result of the RHU AUDINNOVE program, an initiative that brings together a consortium of scientists, physicians, and research teams. The French National Research Agency, through the RHU AUDINNOVE program, supports this groundbreaking work.

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Direct Injection into the Inner Ear

In a similar process to cochlear implantation surgery, the SENS-501 gene therapy drug will be directly injected into the child's inner ear. This innovative injection system was developed in partnership with the company EVEON, further demonstrating the collaborative efforts behind this therapy.

A New Hope for Children with Genetic Deafness

The Audiogene trial brings new hope to children with otoferlin defects and people suffering from genetic deafness. If successful, this treatment could potentially offer an alternative to cochlear implantation, restoring inner ear cell function and hearing. The launch of the trial positions France as a key player in the field of genetic therapies for deafness, marking a significant advancement in patient care.

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