Clinical Trial

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


  • Product: Our oligodendrocyte specific AAV with a codon-optimized, self-complementary ASPA gene administered via intracerebroventricular (ICV) delivery
  • Strategy: Delivery of the gene therapy to target oligodendrocytes is intended to potentially stop or even reverse functional decline, brain atrophy, white matter deterioration and NAA (N-Acetylaspartate) accumulation
  • Objectives: The primary clinical objective this study is the safety and tolerability of the therapy. Secondary Clinical Objectives include pharmacodynamic and measures of efficacy, including Gross Motor Function Measure (GMFM), Mullen Scales of Early Learning (MSEL), Canavan Neurological Examination (CNE) including videotaping, seizure inventory, and EEG
  • Target: Patients with a genetic diagnosis of Canavan disease (CD)
  • Administration: rAAV-Olig001-ASPA administered neurosurgically to the brain via ICV
  • Protocol: The IND protocol will treat patients using a single ICV delivery of the rAAV-Olig001-ASPA
  • Expanded Access: 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 the 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.

Myrtelle welcomes all clinical trial inquiries using the form below, and we encourage eligible patients with Canavan disease to inquire about participation in our current clinical trial, further described on this page and in the links below.

See Our Clinical Trial

Clinical Inquiry

To inquire about the Clinical Trial, please fill out and submit the below

    Understanding Gene
    Therapy for Canavan

    Download Brochure