Canavan Disease
The clinical program is the first gene therapy to target
oligodendrocytes. rAAV-Olig001-ASPA has been granted
Orphan Drug Designation for the treatment of Canavan disease
in the US (designation is pending in the EU).
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
Overview
Canavan disease (CD) is a rare genetic neurological disorder characterized by spongy degeneration of the white matter in the brain.
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.
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.
Disease Onset
Affected infants may appear normal at birth, but usually develop symptoms by six months of age.
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.
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 In Canavan Disease
Understanding Gene
Therapy for Canavan
Disease
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