Myrtelle and rAAVen Therapeutics to Develop Novel Gene Therapy Vectors

Myrtelle Inc., (“Myrtelle”) a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and rAAVen Therapeutics, (“rAAVen”) an AAV engineering company focused on generating innovative vectors for the next generation of gene therapies, today announced a partnership to develop novel recombinant adeno-associated virus (“rAAV”) vectors to advance gene therapy treatments for diseases of the central nervous system (CNS) in which myelin is affected. Myelin, the insulating material that enables the proper function of neurons, is made by specialized cells of the nervous system called oligodendrocytes. The myelin sheath provides a protective coating that allows for signals to be transmitted between one nerve cell to the next. When myelin is damaged or does not develop normally, these impulses slow down and affect normal brain function.

The partnership will leverage rAAVen’s expertise in modifying established viral vectors for the design and production of new viral vectors as potential precision treatments for a broad spectrum of diseases in combination with Myrtelle’s expertise in CNS gene therapy research and development. rAAVen will utilize its unique platform for AAV development that combines state-of-the-art methodologies within cloning, viral vector production and next generation sequencing. Myrtelle will test the vectors in a range of myelin-based disorders and, if successful, pursue further development. Myrtelle will own the vector compositions and retain exclusive worldwide rights to commercialize the resulting gene therapies. In return, rAAVen will receive milestone and sales-based royalty payments.

“We are very excited to announce this partnership as it is the first in the U.S. for rAAVen,” said Marcus Davidsson, Ph.D., COO, and Founder of rAAVen Therapeutics. “We appreciate the opportunity to help Myrtelle achieve their goals for their novel gene therapy programs. This forward-thinking approach by both companies will accelerate the push towards our common goal of improving the lives of patients.”

Myrtelle has built enabling technologies and capabilities in gene therapy for disorders involving myelin production, including a novel class of rAAV vectors that selectively target oligodendrocytes. The Company has a lead program in clinical development for Canavan disease and an exciting pipeline of additional white matter diseases.

“We are enthusiastic about this collaboration with rAAVen, a pioneer in the development of novel methodologies that efficiently identify potent and selective new rAAV vectors,” said Rick Layer, PhD, Myrtelle’s Vice President of Pharmacology and Translational Science. “The field of neuroscience has lacked the tools to properly target and interrogate myelin-producing cells. This has hampered the development of novel gene therapies for many intractable CNS diseases. We are excited to embark with rAAVen on the work of finding solutions for these important unmet needs.”

ABOUT MYRTELLE

Myrtelle Inc. is a 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 and genetic hearing loss diseases. For more information, please visit the Company’s website at: www.myrtellegtx.com.

ABOUT rAAVen Therapeutics

rAAVen Therapeutics is an AAV engineering company focusing on generating novel vectors for the next generation of gene therapies. The company’s unique platform for AAV development in combination with state-of-the-art methodologies within cloning, viral vector production and next generation sequencing allows for a streamlined process with high success rate and increased translational value. rAAVen’s mission is to address all areas of AAV engineering to achieve increased cell specificity, targeting new cell types, increase infectivity, antibody avoidance as well as increased loading capacity. For more information, please visit the company´s website at: www.raaven.se

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