Clinical Trials

Myrtelle’s gene therapy candidate is being studied in an open-label Phase 1/2 clinical trials in children with typical (severe) Canavan disease. We are committed to advancing a new era of treatment for those afflicted by Canavan disease.

Myrtelle, Inc., is dedicated to the development of gene therapies to treat neurodegenerative diseases and bring safe and effective treatment to patients with unmet medical needs. Our ongoing clinical trial in patients with Canavan disease is a thorough systematic scientific investigation that will provide evidence to support regulatory filings with the US Food and Drug Administration (FDA) and competent authorities in other countries. Conducting robust clinical trials is the best way to determine the safety and efficacy of an investigational therapy. Treating patients outside of a clinical trial can prolong the time it takes to accumulate the data necessary for the regulatory filings and hence delay the approval and access to the therapy for all patients. For these reasons, we are focusing our development efforts on ongoing trial and future clinical trials. At present, we do not offer treatment or intend to receive treatment requests in an Expanded Access or “Compassionate Use” setting which would be outside of ongoing or planned clinical trials.  Myrtelle may revise this policy in the future.

Our Phase 1/2 Clinical Trial for Canavan Disease

Myrtelle is conducting a groundbreaking Phase 1/2 clinical trial for Canavan Disease (CD). This First-in-Human gene therapy study utilizes a novel recombinant adeno-associated virus (rAAV) vector, known as rAAV-Olig001-ASPA, to target oligodendrocytes in the brain. These cells are responsible for producing myelin, the insulating material that enables proper neuron function. The therapy aims to deliver a functional copy of the ASPA gene, which is impacted in CD, to restore normal brain function.

Learn About Our Canavan Trial

Understanding Canavan Disease

Canavan Disease is a fatal genetic brain disorder affecting children. It is caused by mutations in the ASPA gene, leading to a deficiency in the enzyme Aspartoacylase (ASPA). This results in the accumulation of N-Acetylaspartate (NAA) in the brain, disrupting normal myelin production and brain function. Symptoms include poor head control, abnormally large head size, and delays in reaching motor milestones. As the disease progresses, seizures, spasticity, and muscle deterioration emerge, with most affected children developing life-threatening complications by approximately ten years of age.

Learn More About Canavan Disease

Patient Eligibility and Trial Participation

Patients eligible for the clinical trial must have a confirmed genetic diagnosis of Canavan Disease and be between 3 months and 5 years old. The trial involves a one-time administration of the gene therapy into the brain and requires a 5-year follow-up period. All study-related expenses, including travel and lodging, will be covered by Myrtelle. Take A Video Tour Of Dayton’s Children’s Hospital.

Inquiry Form

Myrtelle welcomes all clinical trial inquiries using this form, and we encourage eligible patients with Canavan disease and other CNS disease in our indication list above to inquire about participation in our current clinical trial, Active Trials are listed below.


    Myrtelle ASGCT Symposium Presentations

    The Expanding Role of Oligodendrocytes in Brain Function & Disease States

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    Press Release

    Myrtelle Announces Presentation of Positive 12-month Post Treatment Data in its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy

    Myrtelle Announces Positive Data at the 2023 Cell & Gene Meeting on the Mesa held by the Alliance for Regenerative Medicine

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    Press Release

    Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Orphan Drug Designation from the European Medicines Agency

    The European Medicines Agency (EMA) has granted orphan drug (OD) designation for rAAV-Olig001-ASPA, Myrtelle’s lead gene therapy product candidate for the treatment of Canavan disease.

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    Canavan Disease

    Gene Therapy – Measuring its Impact with Dr. Christopher Janson

    Dr. Christopher Janson, the Principal Investigator on Myrtelle’s Canavan Disease gene therapy trial, addresses questions frequently asked by the families in relation to the disease and gene therapy

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    Q & A

    Q&A With Christopher J. Morrison, Senior Vice President of Technical Operations

    Learn about the step-by-step process of manufacturing gene therapies at Myrtelle, including the use of the importance of cGMP standards.

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    Press Release

    Presentation on Myrtelle’s Phase 1/2 Gene Therapy Clinical Trial in Patients with Canavan Disease

    Learn about phase 1/2 of the first-in-human gene therapy clinical trial in patients with Canavan Disease

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    Understanding Gene Therapy for Rare CNS Diseases