MYR-101
Explore a selection of our essential drug information below, or:
Identification
- Generic Name
- MYR-101
- DrugBank Accession Number
- DB17075
- Background
MYR-101 (rAAV-Olig001-ASPA) is a recombinant adeno-associated virus (rAAV) vector-based gene therapy intended to treat Canavan disease. Canavan disease is a fatal childhood genetic disorder characterized by white matter degeneration in the brain. It is caused by a mutation in the aspartoacylase gene (ASPA), which leads to a deficiency of the aspartoacylase enzyme (ASPA). ASPA is produced in oligodendrocytes, and participates in the metabolism of N-acetylaspartate (NAA). When not properly metabolized , NAA accumulates in the brain and negatively affects myelin production. MYR-101 targets oligodendrocytes and delivers functional ASPA.1 The FDA has granted MYR-101 Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations for the treatment of patients with Canavan disease.2
- Type
- Biotech
- Groups
- Investigational
- Biologic Classification
- Gene Therapies
Other gene therapies - Synonyms
- rAAV-Olig001-ASPA
Pharmacology
- Indication
Not Available
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- Pharmacodynamics
Not Available
- Mechanism of action
- Not Available
- Absorption
Not Available
- Volume of distribution
Not Available
- Protein binding
Not Available
- Metabolism
- Not Available
- Route of elimination
Not Available
- Half-life
Not Available
- Clearance
Not Available
- Adverse Effects
- Improve decision support & research outcomesWith structured adverse effects data, including: blackbox warnings, adverse reactions, warning & precautions, & incidence rates. View sample adverse effects data in our new Data Library!Improve decision support & research outcomes with our structured adverse effects data.
- Toxicity
Not Available
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.Not Available
- Food Interactions
- Not Available
Categories
- Drug Categories
- Not Available
- Classification
- Not classified
- Affected organisms
- Not Available
Chemical Identifiers
- UNII
- Not Available
- CAS number
- Not Available
References
- General References
- Business Wire: Myrtelle Completes Dosing of 8 Patients with Canavan Disease in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA [Link]
- Business Wire: Myrtelle Receives FDA Fast Track, Rare Pediatric Disease, and Orphan Drug Designations for its Proprietary Gene Therapy for the Treatment of Canavan Disease [Link]
- External Links
- Not Available
Clinical Trials
- Clinical Trials
Clinical Trial & Rare Diseases Add-on Data Package
Explore 4,000+ rare diseases, orphan drugs & condition pairs, clinical trial why stopped data, & more. Preview package Phase Status Purpose Conditions Count Start Date Why Stopped 100+ additional columns Unlock 175K+ rows when you subscribe.View sample data1, 2 Recruiting Treatment Canavan Disease 1 somestatus stop reason just information to hide
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
- Not Available
- Prices
- Not Available
- Patents
- Not Available
Properties
- State
- Not Available
- Experimental Properties
- Not Available
Drug created at October 19, 2022 23:05 / Updated at December 01, 2022 11:34