Myrtelle Inc., a clinical-stage gene therapy company, has entered into a worldwide exclusive licensing agreement with Rescue Hearing Inc. to develop a novel gene therapy for DFNB8 genetic hearing loss. This therapy will utilize a low-dose recombinant adeno-associated virus (rAAV) gene therapy to deliver a therapeutic TMPRSS3 (transmembrane protease, serine 3) gene directly to the inner ear. The TMPRSS3 gene is responsible for causing DFNB8 genetic hearing loss in humans.

Myrtelle’s gene therapy programs utilize direct administration of low-dose gene therapy to target key cell types involved in the disorder, avoiding immune-related and off-target effects that can arise with high-dose gene therapy administration delivered systemically. This strategy is currently being developed for Myrtelle’s central nervous system (CNS) programs and can be leveraged to other therapeutic areas outside the CNS, including adjacent and related areas such as the ear, where local gene therapy delivery is potentially advantageous for hearing loss disorders such as DFNB8.

Overall, this partnership between Myrtelle and Rescue Hearing Inc. represents a promising step forward in the development of gene therapies for rare genetic diseases, particularly those affecting hearing loss. By focusing on direct, low-dose administration of gene therapy, Myrtelle’s approach has the potential to be more effective and better tolerated by patients than traditional high-dose gene therapy methods.

We are so thrilled to announce this amazing opportunity!