Myrtelle Enters Into a Worldwide Exclusive License Agreement With Rescue Hearing To Develop and Commercialize Gene Therapy for the Treatment of Hearing Loss (Business Wire)

Myrtelle Enters Into a Worldwide Exclusive License Agreement With Rescue Hearing To Develop and Commercialize Gene Therapy for the Treatment of Hearing Loss

A clinical stage gene therapy company focused on developing transformative treatments for rare genetic diseases, today announced that it has entered into a worldwide exclusive licensing agreement with UF Innovate | Accelerate at The Hub alum Rescue Hearing Inc. (RHI) 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 and off-target effects that can arise with high-dose gene therapy administration delivered systemically. This strategy, currently being developed for Myrtelle’s central nervous system (CNS) programs, 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.

“We are excited to partner with RHI on this important potential gene therapy for patients with DFNB8 genetic hearing loss. The program will leverage Myrtelle’s core capabilities and augment our product opportunities to build on the proof-of-concept demonstrated by RHI and move toward the clinic to advance a novel therapeutic approach for patients with DFNB8 genetic hearing,” said Mark Pykett, Myrtelle CEO, adding, “The potential significance of this new therapeutic hearing loss strategy for patients and families is high.”

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