Canavan Disease

Myrtelle’s first-mover clinical trial for Canavan disease
received FDA clearance to begin Phase 1/2 in December
of 2020. The clinical program is the first gene therapy to
target oligodendrocytes, with Orphan Drug Designation
pending. Our first patient was dosed in April 2021.

Redefining the Way We Target and Treat Canavan Disease

Through our core technologies, assets, patents and expertise, we have built a platform to address Canavan disease. Myrtelle’s next-generation Canavan gene therapy (rAAV-Olig001-ASPA) is the first gene therapy designed to target the oligodendrocytes, which are critical for myelination and nerve function. Improvement of ASPA function in these cells reduces concentrations of N-Acetylaspartate (NAA), allowing myelination to occur.

Our Timeline

PRECURSORS

Non-clinical studies investigate the ability to deliver the ASPA gene in-vivo.

HUMAN CLINICAL TRIALS

First ever human clinical study of ASPA gene delivery begins outside of the US, with liposome-based delivery developed by Dr. Paola Leone and colleagues.

USA CLINICAL STUDY

Human clinical study of liposome-based ASPA gene delivery extended to the USA.

AAV2

First ever human clinical study of ASPA gene replacement with AAV2 vector begins based on technologies developed by Dr. Paola Leone and colleagues.

NATURAL HISTORY

Canavan natural history data assembled by Dr. Paola Leone and colleagues.

TARGETING

Novel AAV derived enabling cell-type specific targeting.

rAAV-OLIGO

Novel oligodendro-cyte-targeting AAVs (rAAV-Oligo) characterized.

DATA AGGREGATION

Non-clinical data with rAAV-Oligo developed to support human clinical study.

IND

USA IND opened for r-AAV-Olig001-ASPA gene therapy clinical study.

FIRST PATIENT DOSED

First patients treated in first-ever clinical study of ASPA gene replacement specifically targeting oligodendrocytes.

Canavan Disease:
At a Glance

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Overview

Canavan disease (CD) is a rare genetic neurological disorder characterized by spongy degeneration of the white matter in the brain.

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Genetic Origins

Canavan is caused by mutations in the gene encoding the oligodendrocyte-specific enzyme aspartoacylase (ASPA), a critical enzyme for healthy white matter development and function.

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Incidence

Estimates of the incidence of Canavan range from as high as 1:6,400 to 1:100,000 live births in various populations around the world.

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Disease Onset

Affected infants may appear normal at birth, but usually develop symptoms by six months of age.

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Symptoms

Motor function is severely impaired at birth and invariably worsens over time. Additional symptoms include, poor head control, eye tracking difficulty, excessive irritability, abnormally large head size, delays in motor and developmental milestones. Progressive symptoms include, seizures, spasticity, difficulties in swallowing and overall muscle deterioration. Life-threatening complications usually occur by 10 years of age.

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Treatment & Cure

There are no cures or disease modifying drugs for CD. The only drugs available to date are palliative, including antiepileptic and antispasmodic medications.

Understanding Gene
Therapy for Canavan
Disease

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