GAINESVILLE – October 30th, 2022 – This Summer, we announced our worldwide exclusive licensing agreement with the renowned Myrtelle team. The goal is to develop novel gene therapy for DFNB8 genetic hearing loss—a form of hereditary hearing loss caused by mutations in the TMPRSS3 gene. 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.
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 led 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. We are 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 Rescue Hearing and the TMPRSS3 gene delivery system, we have more exciting products in our pipeline with major growth on the horizon. 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.
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