Introduction and marketing status of adenovirus (AAV rh74)-based gene therapy

Gene therapy based on adenovirus (AAV rh74) vector delandistrogene moxeparvovec-rokl is an innovative therapy for the treatment of Duchenne Muscular Dystrophy (DMD), developed by Sarepta Therapeutics. DMD is a rare, progressive hereditary muscle disease that mainly affects boys. The DMD gene mutation leads to a lack of the anti-damage protein dystrophin (Dystrophin) in muscle cells, ultimately causing sustained muscle damage and loss of function.

delandistrogene moxeparvovec-rokl The core mechanism is through AAV The rh74vector delivers a miniaturized dystrophin gene (micro-dystrophin) into the patient's muscle cells, thereby prompting the muscle cells to produce functional proteins and slowing down the progression of the disease. AAV rh74 was selected as a gene vector because of its high affinity for muscle tissue and low immunogenicity. It is currently a serotype commonly used in gene therapy.

The therapy was approved by the U.S. Food and Drug Administration ( an>FDA) accelerated approval for the treatment of patients aged 4 to 5 years old who have been diagnosed with DMD and patients with specific gene mutations. This marks the first gene therapy approved to treat DMD. Although it is currently only available for use in a small number of patients, its launch brings hope to patients of more age groups and types of mutations in the future.

Currently, delandistrogene moxeparvovec-rokl has not been approved for marketing in mainland China. If patients have treatment needs, they usually need to apply for use through overseas medical channels. The related costs are relatively high and need to be carefully considered after a doctor's evaluation.

References:

https://www.fda.gov/drugs/drug-approvals-and-databases/delandistrogene-moxeparvovec-rokl