• Myrtelle augments pipeline with gene therapy for monogenic hearing loss, designed to repair and restore damaged cells and promote neuron survival
  • Recent groundbreaking proof-of-concept animal studies show recovery of auditory function
  • Technology also presents potential for an exciting platform for future hearing restoration therapies

WAKEFIELD, Mass.–(BUSINESS WIRE)–Myrtelle Inc. (“Myrtelle” or the “Company”), 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 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.”

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. Recently, a successful meeting was held with German health authorities and the Paul Ehrlich Institute to discuss clinical trial authorization (CTA) requirements for starting a first-in-human study. CTA-enabling nonclinical work is currently planned to start later this year to meet these requirements.

RHI is a private, preclinical stage gene therapy company focusing on diseases affecting human hearing. The DFNB8 program targets a common form of genetic hearing loss caused by a mutation in the TMPRSS3 gene. TMPRSS3 mutation is the most common form of genetic deafness in the adult cochlear implant population. DFNB8 patients experience progressive hearing loss usually starting in late teens and early 20s which can lead to complete deafness. RHI’s initial preclinical research has demonstrated positive proof-of-concept data enabling entrance to the IND development phase.

“We are pleased to partner with Myrtelle on this exciting program. Myrtelle’s team and their drug development experience are an outstanding fit for the TMPRSS3 AAV-based gene therapy program for DNFB8-related genetic hearing loss. 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 and look forward to advancing the program into the clinic with Myrtelle with the goal of developing a novel therapy to positively impact unmet medical needs of the genetic hearing loss community,” said Jim Ayala, CEO/Founder Rescue of Hearing.

Myrtelle Inc. is a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer for its lead gene therapy program in Canavan Disease. For more information, please visit the Company’s website at: www.myrtellegtx.com.

Rescue Hearing Inc (RHI) is a private, preclinical stage gene therapy company focused on the genetic forms of hearing loss. RHI’s initial product (RHI100) has produced positive proof of concept data and is entering the IND development phase. RHI100 targets a common form of genetic hearing loss caused by a mutation in the TMPRSS3 gene. TMPRSS3 mutation is the most common form of genetic deafness in the cochlear implant population. RHI has two additional gene therapy assets in development. For more information, please visit the company’s website at: www.rescuehearing.com.

Individuals with mutations in TMPRSS3 present with two phenotypes: DFNB10-associated hearing impairment that is pre-lingual and DFNB8-associated hearing impairment that is typically late-onset and post-lingual. TMPRSS3 mutations can be divided into mild or severe; the combination of two severe mutations causes profound pre-lingual hearing loss, whereas milder mutations lead to less severe post-lingual hearing loss.


Jordana Holovach
Head of Communications and Community
Myrtelle Inc.