Canavan Disease

Myrtelle’s clinical program for Canavan disease is the first gene therapy to specifically target oligodendrocytes. rAAV-Olig001-ASPA has been granted Orphan Drug, Fast-Track and Rare Pediatric Disease Designations for the treatment of Canavan disease in the US, with similar designations in Europe.

OUR MISSION FOR CANAVAN DISEASE

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 oligodendrocytes, cells which are critical for myelination and nerve function. Improvement of ASPA function in these cells is intended to reduce concentrations of N-Acetylaspartate (NAA) and produce other positive benefits, allowing myelination to occur.

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Timeline

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

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.

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

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

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

Novel AAV derived enabling cell-type specific targeting.

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

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

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

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

Canavan Disease at a Glance

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

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

Motor function is severely impaired and worsens over time. Symptoms include poor head control, eye tracking difficulty, excessive irritability, abnormally large head size, and 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.

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

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

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

Understanding Gene-Therapy In Canavan Disease

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Canavan disease is a fatal childhood genetic brain disease in which mutations in the ASPA gene prevent the normal activity of aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes (the affected brain cells in Canavan disease responsible for producing myelin and support overall brain health) that breaks down the neurochemical N-Acetylaspartate (NAA) into acetate and aspartate. When not properly broken down by functioning ASPA in oligodendrocytes, NAA accumulates, acetate and aspartate are depleted, and myelin production and brain function decline. Myelin is the insulation that coats axons and, in the brain, appears white, which is why it is also referred to as white matter. Myelin insulates the brain and spinal cord protecting nerve fibers and enabling normal transmission of nerve signals. Currently, there are no cures for Canavan disease. Myrtelle’s goal is to change this outcome. Myrtelle’s Phase 1/2 clinical trial is a First-in-Human gene therapy study in patients with Canavan disease.

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Robust Discovery Pipeline

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.

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Indication
Proof of Concept
Phase 1/2
Pivotal

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Understanding Gene Therapy for Rare CNS Diseases

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