Myrtelle Inc., a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization (CDMO), have formed a manufacturing partnership that will advance Myrtelle’s novel gene therapy for monogenic hearing loss, Myr-201, into clinical trials for patients with autosomal recessive deafness 8 (DFNB8).

Forge will provide research-grade and GMP-Pathway plasmid manufacturing services as well as cGMP adeno-associated viral (AAV) process development and scale-up manufacturing services for Myrtelle’s program, Myr-201. The program will utilize Forge’s platform process including its proprietary HEK 293 suspension Ignition Cells and pEMBR adenovirus helper plasmid. All development and cGMP manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy cGMP facility in Columbus, OH.

“We are thrilled to be collaborating with Myrtelle to help them accelerate this novel gene therapy for monogenic hearing loss to patients,” said Timothy Miller, CEO, president, and co-founder of Forge Biologics. “This collaboration showcases the full end-to-end capabilities Forge is providing to gene therapy clients. We believe that employing our plasmid manufacturing, coupled with our suite of cGMP AAV manufacturing capabilities and expertise, will help support Myrtelle’s gene therapy technology and skills, enabling this novel therapy to reach those who need it the most.”

Myrtelle entered into a worldwide exclusive licensing agreement with Rescure Hearing Inc. (RHI), a private, preclinical stage gene therapy company focusing on diseases affecting human hearing to develop a novel gene therapy for DFNB8 genetic hearing loss that includes low-dose recombinant adeno-associated virus (rAAV) gene therapy delivery of a therapeutic TMPRSS3 (transmembrane protease, serine 3) gene by local administration directly to the inner ear. 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. 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.

Mark Pykett, CEO of Myrtelle, said, “This collaboration is an important milestone in Myrtelle’s evolution as we seek to advance our novel gene therapy for monogenic hearing loss which has the potential to become a life-changing genetic medicine. Partnering with Forge Biologics, a leader in gene therapy manufacturing, and leveraging their AAV expertise, technology, and exceptional facility will support Myrtelle’s development efforts as we move into Phase 1/2 clinical trials with the goal of bringing this transformational therapy to patients in need.”