MedPath

MYR-101

Generic Name
MYR-101
Drug Type
Biotech
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. The FDA has granted MYR-101 Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations for the treatment of patients with Canavan disease.

Clinical Trials
Related News

Myrtelle to Share First-Year Insights from FDA's START Pilot Program for Rare Disease Therapies at ASGCT 2025

• Myrtelle Inc. will present at ASGCT's Annual Meeting on May 15, 2025, highlighting its experience as one of only three gene therapy developers selected for the FDA's START Pilot Program. • The START Program enables accelerated development of rare disease treatments through enhanced regulatory communication, directly benefiting Myrtelle's gene therapy (rAAV-Olig001-ASPA) for fatal childhood Canavan disease. • Myrtelle's innovative therapy delivers a functional ASPA gene using a proprietary oligodendrocyte-targeting AAV vector, potentially restoring brain development in children with Canavan disease who currently have only palliative treatment options.

FDA's START Program Selects First Rare Disease Therapies for Accelerated Development

• The FDA's START program, designed to expedite rare disease therapy development, has chosen its first candidates for enhanced guidance and support. • Denali Therapeutics' DNL12 for mucopolysaccharidosis IIIA, Neurogene's NGN-401 for Rett syndrome, and Larimar Therapeutics' nomlabofusp for Friedreich's ataxia are among the selected therapies. • Grace Science's GS-100 gene therapy for NGLY1 deficiency is also included, marking a significant step forward in addressing this life-threatening condition with no approved treatments. • The START program aims to provide comprehensive support in clinical trial design and data generation, facilitating efficient development of potentially life-saving therapies.

Gene and Cell Therapies Show Promise in Treating CNS Disorders

• The market for gene and cell therapies targeting CNS disorders was valued at approximately USD 1 billion in 2021 and is projected to grow significantly by 2034. • Several companies are developing gene and cell therapies for CNS disorders, with some therapies already approved for conditions like spinal muscular atrophy (SMA). • Clinical trials are showing positive trends, such as uniQure's AMT-130 for Huntington's disease demonstrating encouraging results in mean CSF NfL levels.

Myrtelle's rAAV-Olig001-ASPA Gene Therapy Shows Promise in Canavan Disease Trial

• Myrtelle's rAAV-Olig001-ASPA (MYR-101) gene therapy significantly reduced N-Acetylaspartate (NAA) levels in Canavan disease patients in a phase 1/2 trial. • MRI scans revealed increases in brain white matter and myelin volume in treated patients, suggesting potential structural improvements in the brain. • Patients demonstrated functional improvements on validated scales, contrasting the expected deterioration in untreated Canavan disease, offering hope for a treatment option. • The FDA has granted rAAV-Olig001-ASPA RMAT designation, orphan drug designation (ODD), rare pediatric disease designation, and fast track designation.
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