MYR-101

DB17075

biotech investigational

Deskripsi

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.L43567 The FDA has granted MYR-101 Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations for the treatment of patients with Canavan disease.L43572

Struktur Molekul 2D

Struktur tidak tersedia

Peta Jejaring Molekuler
Legenda: ObatTargetGenEnzim(Panah → menunjukkan arah efek / relasi)TransporterCarrier

Profil Farmakokinetik

Waktu Paruh (Half-Life) -
Volume Distribusi -
Klirens (Clearance) -

Absorpsi

Data absorpsi tidak tersedia.

Metabolisme

Data metabolisme tidak tersedia.

Rute Eliminasi

Data eliminasi belum tersedia.

Interaksi Obat

0 Data
Tidak ada data.

Contoh Produk & Brand

Produk: 0 • International brands: 0
Belum ada data produk/brand untuk obat ini pada database. Jalankan import DrugBank untuk mengisi field produk/brand.

Sekuens Gen/Protein (FASTA)

Sekuens dimuat saat dibutuhkan agar halaman tetap ringan.
© 2025 Digital Pharmacy Research - Universitas Esa Unggul