Myrtelle to Host Symposium on Gene Therapies Targeting Oligodendrocytes and Implications in Brain Function and Disease States at the American Society of Gene and Cell Therapy 26th Annual Meeting in Los Angeles on Friday, May 19, 2023

Wakefield, Mass., May 10, 2023 – Myrtelle Inc. (“Myrtelle” or the “Company”), a clinical-stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, will host a symposium on the expanding understanding of oligodendrocytes in brain function and disease states at the American Society of Gene and Cell Therapy 26th Annual Meeting on Friday, May 19, 2023, at 12:00 pm PT in Los Angeles.  The symposium will highlight historical and emerging views of oligodendrocytes in normal brain function and a growing array of disease states, including leukodystrophies and disorders of myelin production such as multiple sclerosis.  Oligodendrocytes perform numerous key functions in the brain – including the production of myelin, the insulating material that enables proper neuronal function – and are now appreciated as having roles in a range of diseases. Myrtelle is developing a unique toolkit, including a proprietary class of recombinant adeno-associated virus (rAAV) vectors that directly target these cells.  As a leading edge of its unique strategy, the Company is conducting a First in Human (FIH) open-label Phase 1/2 clinical trial using one of its proprietary rAAV oligodendrocyte-targeting vectors for Canavan disease (CD), a fatal genetic brain disorder in children in which oligodendrocytes are involved and affected.

Symposium speakers include:

  • Ethan Hughes, Ph.D., Associate Professor at University of Colorado Anshutz Medical Campus will deliver a presentation on the “Diverse Roles of Oligodendrocytes” based on his studies on the interactions of oligodendrocyte lineage cells with neurons in the adult cerebral cortex.
  • Wouter Peelaerts, Ph.D., Assistant Professor of Neuroscience at KU Leuven whose work in modalities for delivering therapeutic gene therapy will be presented in a talk on “Targeting Oligodendrocytes in Neurodegenerative Disease.” 
  • Christopher Janson, M.D., Principal Investigator on Myrtelle’s Canavan Disease Gene Therapy Trial and Assistant Professor of Neurology and Neuroscience at Wright State University Boonshoft School of Medicine and Director of Human Gene Therapy Center at Wright State Neuroscience Institute in Dayton, Ohio, will provide an update on Myrtelle’s “Phase 1/2 First-in-Human Gene Therapy Clinical Trial ​in Patients with Canavan Disease.” 
  • Richard Layer, Ph.D., Senior Vice President, Head of Research at Myrtelle, will speak on “New Directions and Implications of Oligodendrocyte Targeted Gene Therapies.”

“Oligodendrocytes are integral to the overall health of the brain and yet they have been overlooked as potential targets for neurodegenerative diseases.” said Dr. Mark Pykett, CEO of Myrtelle.   “Given the intractable nature of many neurological diseases, these cells represent important brain components worthy of interrogation in the quest for novel interventions, including gene therapies.  We now have the toolbox for delivering therapeutic constructs to these cells and are excited by what we are learning about the impact of oligodendrocytes in many disease states and brain function, led by initial observations in our gene therapy work in Canavan disease.  We look forward to the symposium and generating further interest and insight into the unique oligodendrocyte story.”


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 diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Company’s website at:


Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of Aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available.

More information on Myrtelle’s clinical trial in Canavan disease can be found on under the identifier NCT04833907 or by emailing

Media Contact:

Jordana Holovach

Head of Communications and Community

Myrtelle Inc.

781-621-2797 Ext. 102