Peringatan Keamanan

No information is available regarding the acute toxicity (LD50) and overdosage of delandistrogene moxeparvovec.

Delandistrogene moxeparvovec

DB16802

biotech approved investigational

Deskripsi

Delandistrogene moxeparvovec is an adeno-associated virus vector-based gene therapy developed by Sarepta Therapeutics. It was granted accelerated approval by the FDA on June 22, 2023, as the first gene therapy to treat Duchenne Muscular Dystrophy (DMD).L47026 DMD is an X-linked genetic disorder characterized by mutations in the dystrophin gene, leading to a deficiency in functional dystrophin protein. Dystrophin is an essential protein responsible for muscle function; thus, patients with DMD experience a progressive deterioration of muscle mass and function.A260256 DMD tends to be more prevalent in males.A260261

Delandistrogene moxeparvovec comprises a non-replicating, recombinant adeno-associated virus serotype rh74 (AAVrh74) based vector containing the microdystrophin transgene under the control of a muscle-specific promoter (MHCK7) to enhance expression in cardiac and skeletal muscles. Microdystrophin is a truncated, functional form of dystrophin.A260256, L47021

Struktur Molekul 2D

Struktur tidak tersedia

Peta Jejaring Molekuler
Legenda: Obat • Target • Gen • Enzim • (Panah → menunjukkan arah efek / relasi) • Transporter • Carrier

Profil Farmakokinetik

Waktu Paruh (Half-Life) No information is available.
Volume Distribusi There is limited information. In mice toxicology studies, vector DNA was detected in all major organs with the highest quantities detected in the liver, followed by lower levels in the heart, adrenal glands, skeletal muscle, and aorta following delandistrogene moxeparvovec administration. Low levels of delandistrogene moxeparvovec were also detected in the spinal cord, sciatic nerve and testis.[L47021] Protein expression of microdystrophin was highest in the cardiac tissue, exceeding physiologic dystrophin expression levels in healthy mice, with lower levels in the skeletal muscle and diaphragm. In some studies, microdystrophin was also detected at low levels in the liver.[L47021]
Klirens (Clearance) No information is available.

Absorpsi

No information is available.

Metabolisme

No information is available.

Rute Eliminasi

No information is available.

Interaksi Obat

0 Data
Tidak ada data.

Referensi & Sumber

Artikel (PubMed)
  • PMID: 35935842
    Deng J, Zhang J, Shi K, Liu Z: Drug development progress in duchenne muscular dystrophy. Front Pharmacol. 2022 Jul 22;13:950651. doi: 10.3389/fphar.2022.950651. eCollection 2022.
  • PMID: 37261034
    Klimchak AC, Sedita LE, Rodino-Klapac LR, Mendell JR, McDonald CM, Gooch KL, Malone DC: Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States. J Mark Access Health Policy. 2023 May 26;11(1):2216518. doi: 10.1080/20016689.2023.2216518. eCollection 2023.
  • PMID: 35205302
    Wilton-Clark H, Yokota T: Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot. Genes (Basel). 2022 Jan 28;13(2):257. doi: 10.3390/genes13020257.

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