Myrtelle Inc. targets Canavan disease with an exclusive worldwide licensing agreement with Pfizer Inc. for an investigational rAAV gene therapy.

WAKEFIELD, Mass.–(BUSINESS WIRE)–Myrtelle Inc. today announced that earlier this year it entered into an exclusive worldwide licensing agreement with Pfizer Inc. for an investigational recombinant adenovirus (rAAV) gene therapy to bring potential new medicine to patients with Canavan disease (CD), a fatal childhood genetic neurological disorder characterized by the degeneration of the white matter in the brain.

Following the licensing agreement, Myrtelle is now conducting a first-in-human Phase I/II clinical study for CD under an open U.S. Investigational New Drug (IND). The investigational gene therapy utilizes a proprietary vector that is designed to directly target oligodendrocytes. Oligodendrocytes are the cells in the brain responsible for producing myelin – the insulating material that enables proper neuronal function. The oligodendrocyte-targeted investigational gene therapy is intended to restore ASPA function, enable metabolism of the abundant brain chemical N-Acetylaspartic Acid (NAA), and support myelination. Treatment of children with CD is underway at Dayton Children’s Hospital (Dayton, Ohio).

“Canavan is a devastating disease of young children which typically results in short life expectancy, with only palliative treatments available,” said Dr. Mark Pykett, CEO of Myrtelle Inc. “We are proud to continue work to develop this potentially transformative gene therapy for patients with Canavan disease and their families.”


Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. Our lead program is a novel investigational gene therapy treatment for Canavan disease. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurological diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan program. For more information visit:


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

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

Jordana Holovach
Head of Communications and Community
Myrtelle Inc.
781-621-2797 Ext. 102