Myrtelle Proves Promising Results in Gene Therapy for Canavan Disease in 6-Month Follow-Up Study!

Myrtelle Inc. has made a breakthrough in its quest to develop transformative treatments for neurodegenerative diseases. At the 6 month post-treatment time point, early data from the Company’s open-label Phase 1/2 First-in-Human (FIH) clinical trial for Canavan disease (CD) have revealed an encouraging safety profile in the 8 patients treated with its recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy. This is a major step forward in the treatment of this fatal genetic brain disorder in children.

Magnetic resonance imaging (MRI) assessments revealed remarkable improvements in myelin, white matter, grey matter, and total brain volume, as well as a reduction in the volume of cerebrospinal fluid (CSF), in treated patients compared to untreated age-matched CD patients within Myrtelle’s natural history data set.

Clinical measurements of motor and cognitive function using the Gross Motor Function Measure (GMFM) and Mullen Scales of Early Learning (MSEL) demonstrated mean absolute and percent improvements across multiple domains, without any serious drug-related adverse events. These results are encouraging, and offer hope to those suffering from CD.

Myrtelle’s FIH trial is using a revolutionary gene therapy to restore normal brain development in patients with Canavan’s Disease (CD). The therapy involves a proprietary rAAV vector that targets oligodendrocytes, the brain cells affected by CD.

This vector is designed to correct a mutation in the ASPA gene, which encodes the enzyme Aspartoacylase (ASPA). By restoring ASPA function, the vector helps to metabolize N-Acetylaspartate (NAA) and returns normal brain development in those with CD.

The findings from this study are a promising sign of positive changes in the motor, language, cognitive, and visual skills of the patients. Principal Investigator and Assistant Professor of Neurology and Neuroscience at Wright State University Boonshoft School of Medicine, Chris Janson, MD, noted improvements in functional scores as measured by the GMFM and MSEL assessment tools.

Furthermore, volumetric MRI and MRS (magnetic resonance imaging and spectroscopy, respectively) scans indicated increases in multiple brain tissue compartments and reductions in CSF volume and NAA concentrations. This suggests that the gene therapy is successful and could potentially open the door to new treatment options for CD patients.

Dr. Janson will be presenting an exciting update on the trial at the ASGCT 26th Annual Meeting in Los Angeles this May. Attendees will have the chance to gain insight into the latest developments and results of the trial, and to find out what the future holds. Don’t miss this opportunity to hear the latest news!


Myrtelle Inc. is pioneering a revolutionary approach to treating neurodegenerative diseases. With a powerful platform, extensive intellectual property, and an impressive portfolio of programs and technologies, the company is committed to developing transformative treatments.

Myrtelle has also secured an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program, a major step forward in its mission to revolutionize the treatment of neurodegenerative diseases.


Canavan Disease (CD) is an incurable genetic brain disorder, affecting young children from birth. It is caused by a mutation in the Aspartoacylase gene (ASPA), which prevents the production of the essential enzyme, Aspartoacylase (ASPA).

This results in an accumulation of the neurochemical N-Acetylaspartate (NAA) in the brain, leading to impaired bioenergetics, myelin production, and health. Initially, patients may appear normal until several months old, when symptoms such as poor head control, abnormally large head size, difficulty in eye tracking, irritability, and delayed motor milestones, like rolling, sitting, and walking, start to manifest.

As the disease progresses, seizures, spasticity, and deterioration of muscle control can occur, with the majority of affected children facing life-threatening complications by the age of 10. Although there is no cure for CD, palliative treatments are available to help manage symptoms.

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