Myrtelle Presents Encouraging Interim Phase 1/2 Data for Gene Therapy in Canavan Disease at 2025 Cell & Gene Meeting on the Mesa

Myrtelle Inc., a gene therapy company pioneering treatments for neurodegenerative diseases, announced that Dr. Michael Muhonen, M.D., Co-Chief Medical Officer, will present an overview of the company and interim Phase 1/2 clinical results for its investigational gene therapy rAAV-Olig001-ASPA (MYR-101) in Canavan disease at the 2025 Cell & Gene Meeting on the Mesa in Phoenix, Arizona.

The presentation marks a key milestone in Myrtelle’s mission to develop the first disease-modifying therapy for children with Canavan disease, a rare and fatal leukodystrophy caused by a single-gene defect. MYR-101 is based on the company’s oligodendrocyte-targeted AAV platform, which restores myelin-forming cell function in the brain.

Key Phase 1/2 Findings (Published in Nature Medicine)

• Favorable safety: MYR-101 showed a well-tolerated safety profile with no serious adverse events related to treatment.
• Biological activity: Patients demonstrated significant reductions in N-acetylaspartate (NAA) levels in cerebrospinal fluid, consistent with restored ASPA enzyme function.
• Myelin regeneration: Synthetic MRI (SyMRI) scans showed increased myelin volume, confirming remyelination and therapeutic benefit.
• Functional gains: Participants achieved measurable developmental improvements compared to historical controls.
• Durability: Long-term follow-up suggests sustained benefit and potential for durable outcomes.

“These data represent a major step forward for children and families affected by Canavan disease. For the first time, we’re seeing clear biological and functional evidence that gene therapy targeting oligodendrocytes can directly address the core pathology of this disease.”

• Dr. Michael Muhonen, Co-Chief Medical Officer of Myrtelle.

Regulatory Milestones

MYR-101 has been selected by the U.S. FDA for inclusion in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program — one of only four gene therapies regulated by CBER to receive this distinction.

The program provides enhanced FDA engagement to accelerate the development of promising therapies for rare diseases.

Additional designations for MYR-101 include:

• Regenerative Medicine Advanced Therapy (RMAT) designation
• Orphan Drug, Rare Pediatric Disease, and Fast Track designations (U.S. FDA)
• Orphan Drug Designation and Advanced Therapy Medicinal Product (ATMP) classification (European Medicines Agency)
• Innovative Licensing and Access Pathway (ILAP) designation (UK MHRA)

About Myrtelle

Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company holds a proprietary AAV platform and a global licensing agreement with Pfizer Inc. for its Canavan disease program.

About Canavan Disease

Canavan disease (CD) is a fatal childhood neurogenetic disorder caused by mutations in the ASPA gene, which prevent production of the enzyme aspartoacylase. This disrupts brain bioenergetics and myelin formation, leading to progressive neurodegeneration.

Early symptoms appear in infancy and include:

• Poor head control and motor delay
• Enlarged head size
• Visual tracking issues and irritability
• Low muscle tone and delayed milestones

As the disease progresses, affected children experience seizures, spasticity, and muscle deterioration, with most developing life-threatening complications before age 10.

There are currently no approved cures, only palliative treatments.

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