Myrtelle’s gene therapy candidate is being studied in an open-label single-center 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 pilot product oligodendrocyte-specific AAV with a codon-optimized, self-complementary ASPA gene administered via intracerebroventricular (ICV) delivery. The primary clinical objective of the Phase 1/2 clinical study is the safety and tolerability of the therapy. Secondary clinical objectives include pharmacodynamic measurements and assessments of efficacy using established scales, including the Gross Motor Function Measure (GMFM), Mullen Scales of Early Learning (MSEL), and Canavan Neurological Examination (CNE) including videotaping, seizure inventories, and EEGs.
See Our Current Trials
We are developing delivery methods 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. We are focusing on patients with a CNS diseases and genetic diagnosis’s like Canavan disease (CD).
Explore SolutionsOur current Phase 1/2 protocols is administered neurosurgically to the brain via ICV delivery to treats patients with a single infusion.
Learn more about our site location, Dayton’s Childerns Hospital in our video tour.
Myrtelle’s proprietary technologies target global patient populations with unmet medical needs. The enabling technologies allow for a therapeutic pipeline with hundreds of disease candidates, from large indications like MS to orphan diseases like Canavan. Primary discovery candidates have been identified.
See Full PipelineMyrtelle 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.