MYR-101

Overview

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.

Indication

No indication information available.

Associated Conditions

No associated conditions information available.

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease	2021/04/06	NCT04833907	Phase 1	Recruiting	Myrtelle Inc.

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
No FDA approvals found for this drug.

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
No EMA approvals found for this drug.

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
No TGA approvals found for this drug.

Related News

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

2 months ago

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.

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

9 months ago

• 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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