Therapy Areas: Central Nervous System
Myrtelle and rAAVen Therapeutics to Develop Novel Gene Therapy Vectors
17 January 2023 - - US-based clinical stage gene therapy company Myrtelle Inc and AAV engineering company rAAVen Therapeutics have forged a partnership to develop novel recombinant adeno-associated virus vectors to advance gene therapy treatments for diseases of the central nervous system in which myelin is affected, the company said.

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.

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.

Myrtelle 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.

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.
Login
Username:

Password: