Analyses of the first three patients at 6 months post-treatment showed increases in white matter and myelin and improvements on validated functional scales

To date, 5 patients have been treated in the ongoing Phase 1/2 First-in-Human clinical trial with favorable safety and tolerability

Encouraging early data support further development of rAAV-Olig001-ASPA as a potential therapeutic approach for children diagnosed with Canavan disease

WAKEFIELD, MA, USA I July 08, 2022 I Myrtelle Inc. (“Myrtelle” or the “Company”), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced in a presentation by Armen Asatryan, MD, Chief Medical Officer, at the National Tay Sachs and Allied Diseases Annual Family Conference held in Denver, Colorado, that the Company’s recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for Canavan disease (CD) has shown encouraging early efficacy data and a favorable safety profile in the first three patients treated through their six month visit post-treatment in the Company’s open-label Phase 1/2 First-in-Human (FIH) clinical trial at Dayton Children’s Hospital (Dayton, Ohio). Myrtelle’s FIH trial utilizes the Company’s proprietary rAAV vector to directly target oligodendrocytes, the affected brain cells in CD responsible for producing myelin – the insulating material that enables proper neuronal function. In CD, the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) encoding the enzyme Aspartoacylase (ASPA). The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartate (NAA), a neurochemical that is abundant in the brain, and thereby supporting myelination. Five patients have been treated in the ongoing Phase 1/2 study with favorable safety and tolerability results to date and no study drug-related adverse events.

Analyses of the first three patients at 6-months post-treatment by Magnetic Resonance Imaging (MRI) demonstrated increases in white matter and myelin in the brain. Improvements were also exhibited in scores on the Gross Motor Function Measure (GMFM) and Mullen Scales of Early Learning (MSEL), validated functional scales. Observed improvements contrast the continuous clinical deterioration expected with the natural progression of the disease.

“The 6-month functional and anatomic data observed in the first three patients elicit hope when we compare it with the natural course of this devastating disease. Equally encouraging are positive changes in the patients’ reflexes, eye tracking, vocalizations to communicate, and overall awareness” said Robert Lober, MD, PhD, FAANS, Co-Principal Investigator and Associate Professor of Pediatrics at Wright State University Boonshoft School of Medicine and Attending Neurosurgeon at Dayton Children’s Hospital in Dayton, OH. “These improvements suggest the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease.”

Per the FIH trial protocol, the gene therapy is administered as a single total dose of 3.7 x 1013 vg delivered by intracerebroventricular (ICV) injection to target the oligodendrocytes of the brain where ASPA activity is deficient in CD patients.

“Myrtelle’s current investigational gene therapy targeting oligodendrocytes with a unique rAAV vector builds upon over two decades of science and effort with the potential, if successful, to usher in new treatment options for children with Canavan disease,” commented Armen Asatryan, MD, MPH, Chief Medical Officer of Myrtelle. “We intend to build on these encouraging initial findings and complete our current Phase 1/2 clinical study, following which we plan to engage with regulatory authorities to advance the program to the later stages of clinical development.”


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 study in Canavan disease can be found on under the identifier NCT04833907 or by emailing

SOURCE: Myrtelle