Today, Myrtelle announced that it has entered into a worldwide exclusive licensing agreement with Rescue Hearing Inc. to develop a novel gene therapy for DFNB8 genetic hearing loss. The low-dose recombinant adeno-associated virus (rAAV) gene therapy is intended to deliver a therapeutic TMPRSS3 (transmembrane protease, serine 3) gene by local administration directly to the inner ear. Mutation in the TMPRSS3 gene is the underlying cause of DFNB8 genetic hearing loss in humans. Across its gene therapy programs, Myrtelle utilizes direct administration of low-dose gene therapy to target key cell types involved in the disorder, thereby avoiding immune-related side effects.
Hearing loss is a huge problem, with over 360 million people affected worldwide. But there may be hope on the horizon, thanks to a new worldwide exclusive license agreement between Rescue Hearing and Myrtelle. Myrtelle is developing an innovative new treatment for hearing loss. This could be great news for investors, life science investors, and CIOs alike! Myertelle’s technology shows real promise in restoring hearing, and Rescue Hearing is well-positioned to bring it to market. Stay tuned for more updates as this story develops!
Mutation in the TMPRSS3 gene is the underlying cause of DFNB8 genetic hearing loss in humans. RHI utilizes direct administration of low-dose gene therapy to target key cell types involved in the disorder, thereby avoiding immune-related and off-target effects that can arise with high-dose gene therapy administration delivered systemically. This strategy can be leveraged to other therapeutic areas outside the central nervous system, including adjacent and related areas such as the ear where local gene therapy delivery is potentially advantageous for hearing loss disorders such as DFNB8. The TMPRSS3 gene provides instructions for making a protein called transmembrane protease, serine 3. This protein is found in cells throughout the body, but its precise function is unknown. However, researchers believe that it may play a role in modulating cellular responses to environmental factors, such as viral infections. Mutations in the TMPRSS3 gene are thought to disrupt the normal function of this protein. As a result, affected individuals are less able to mount an effective response to viral infections. In addition, these mutations are thought to interfere with the development and function of certain cells in the inner ear, leading to hearing loss. The RHI’s approach of direct administration of low-dose gene therapy holds promise for not only treating DFNB8 genetic hearing loss, but also for other disorders caused by TMPRSS3 gene mutations.
Blog Title: Restoration Hearing Announces Exciting New Data in the Treatment of Deafness
Blog Introduction: We are excited to announce new data in the treatment of deafness. Preclinical studies in the mouse model of DFNB8-mediated deafness have demonstrated that delivery of a wild type TMPRSS3 gene was able to promote hair cell and neuron survival and improve hearing function. The groundbreaking proof-of-concept data generated by our scientific team lead by Dr. Hinrich Staecker (University of Kansas Medical Center), Dr. Zheng Yi Chen (Mass Eye and Ear Infirmary), and Dr. Xue Zhong Liu (University of Miami Health System) provide a strong foundation for further development. The RHI team is proud to have brought the TMPRSS3 program to this exciting stage.
The implications of this data are far-reaching. According to the World Health Organization, 466 million people worldwide suffer from some degree of deafness or hearing loss. Of these, 34 million are children. In the United States alone, it is estimated that 15% of the population—or 48 million people—report some trouble hearing. This number is only expected to grow as the population ages; by 2060, there will be an estimated 22 million Americans over the age of 65 with disabling hearing loss.
There are many causes of deafness, from inherited genetic conditions to viruses to exposure to loud noise. However, the vast majority of cases are due to damage to the inner ear hair cells, which can occur due to any number of reasons, including aging, disease, and trauma. Once these hair cells are damaged, they cannot be repaired or replaced—leading to irreversible hearing loss.
The new data released by our team offers hope for those suffering from deafness caused by damage to their inner ear hair cells. The TMPRSS3 gene delivery system was able to promote hair cell and neuron survival and improve hearing function in a mouse model of DFNB8-mediated deafness—a form of hereditary hearing loss caused by mutations in the TMPRSS3 gene. These results suggest that this approach has the potential to one day be used in humans suffering from similar forms of deafness caused by damage to their inner ear hair cells.
This is just the beginning for Restoration Hearing and the TMPRSS3 gene delivery system for treating deafness caused by damage to inner ear hair cells. We are grateful for the support of our investors as we continue working towards our goal of bringing this potentially life-changing therapy to those who need it most.