eteplirsen

(redirected from AVI-4658)

eteplirsen

A proprietary agent in clinical trials for managing Duchenne muscular dystrophy, a condition characterised by defective dystrophin—a key cytoplasmic protein that links muscle fibre cytoskeleton to extracellular matrix through the cell membrane.

Mechanism
Eteplirsen is a splice-switching oligomer that skips exon 51 of the dystrophin gene-DMD, resulting in the production of a shorter, but still functional, dystrophin.
References in periodicals archive ?
The "antisense" drug, known as AVI-4658, is a synthetic form of the genetic chemical RNA.
In this clinical trial of 19 patients, study participants aged five to 15 at Great Ormond Street Hospital and the Royal Victoria Infirmary, Newcastle, were given weekly doses of the drug, AVI-4658.
The primary objective of the trial was to assess the safety of AVI-4658 at these doses over the 26-week duration of the trial.
AVI and Ercole have collaborated since December 2006 to develop drug candidates including AVI-4658, which is in clinical testing in the United Kingdom for treatment of Duchenne muscular dystrophy (DMD).
Nasdaq:AVII), today announced dosing of the first patient in a proof-of-principle clinical trial using AVI-4658, AVI's lead drug candidate for Duchenne muscular dystrophy (DMD), based on the company's proprietary ESPRIT (Exon Skipping Pre-RNA Interference Technology) drug platform.
Food and Drug Administration has granted Fast Track status to its product candidate, AVI-4658, for the treatment of Duchenne muscular dystrophy (DMD).
Approval by the Medicines and Healthcare products Regulatory Agency (MHRA), the health authority in the United Kingdom, to commence an intramuscular clinical study with AVI-4658 in DMD boys afflicted with an exon 51 mutation.
AVI-4658 uses AVI's ESPRIT technology (Exon Skipping Pre-RNA Interference Technology), which is designed to benefit patients with certain genetic mutations.
Food and Drug Administration's (FDA) Office of Orphan Products Development has granted orphan drug designation to AVI-4658 for the treatment of Duchenne muscular dystrophy (DMD).
AVI-4658 is designed to benefit patients with mutations in the gene for dystrophin that can be neutralized by skipping exon 51.
This therapeutic approach is similar to that of AVI-4658, in development to potentially treat DMD patients with mutations that could benefit from skipping exon 51 of the dystrophin gene.
AVI's objective is to use the ESPRIT therapeutic AVI-4658 to skip exon 51, which would put the subsequent protein back in the correct reading frame, creating a shortened but functional version of dystrophin.