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.
GC Biopharma's GC1130A receives EMA Orphan Drug Designation
Guangzhou Fermion Technology's FZ008-145 IND application receives Chinese regulatory approval
Newron enrols 290 patients in schizophrenia study
Vistagen granted European patent for AV-101 to treat neuropathic pain
Alora Pharmaceuticals announces Relexxii commercial launch
IGC Pharma granted patent for Alzheimer's drug formulation
NRx Pharmaceuticals signs data agreement with Columbia University for IV Ketamine trials
Cambridge Cognition launches AQUA for automated quality assurance in CNS clinical trials
Shanghai Zhimeng Biopharma's CB03 receives US FDA orphan drug designation
Genentech's Alecensa demonstrates 76% reduction in disease recurrence risk
NRx Pharmaceuticals announces presentation at 8th Annual Dawson James Conference