Policy & Regulation
Myrtelle Enters into a Worldwide Exclusive License Agreement with Rescue Hearing to Develop and Commercialize Gene Therapy for the Treatment of Hearing Loss
20 May 2022 - - US-based clinical stage gene therapy company Myrtelle Inc has entered into a worldwide exclusive licensing agreement with Rescue Hearing Inc. to develop a novel gene therapy for DFNB8 genetic hearing loss, the company said.

The low-dose recombinant adeno-associated virus 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 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.

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 meeting was held with German health authorities and the Paul Ehrlich Institute to discuss clinical trial authorization 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.

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.

Rescue Hearing Inc 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.
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