Avidity Biosciences is making strides in the treatment of Duchenne muscular dystrophy (DMD) with its drug delpacibart zotadirsen (del-zota), which aims to skip exon 44 of the dystrophin gene and has shown positive results in a phase 1/2 trial.

Duchenne muscular dystrophy is a severe inherited genetic condition characterized by progressive muscle weakening, primarily caused by mutations in the dystrophin gene, which is crucial for muscle fiber protection.

This condition affects approximately one in every 3,500 male births worldwide, highlighting the urgent need for effective treatments.

The global market for DMD treatments was valued at $3.2 billion in 2023 and is projected to grow significantly to $8.6 billion by 2032, driven by regulatory approvals for new therapies.

Recent advancements in therapeutics have led to the introduction of new drugs and the evaluation of various others, including gene therapies and inhibitors.

Currently, six clinical-stage biotech companies are at the forefront of research and development for DMD treatments, reflecting a robust pipeline of potential therapies.

One notable advancement is Italfarmaco's Duvyzat, the first nonsteroidal treatment approved by the FDA for all genetic variants of DMD, available for patients aged six and older.

Edgewise Therapeutics is also contributing to this field with its development of sevasemten, a skeletal myosin inhibitor that is currently undergoing phase 2 trials.

Capricor Therapeutics is conducting phase 3 trials for deramiocel, targeting cardiomyopathy in DMD patients, and plans to seek FDA approval.

Sarepta Therapeutics has made headlines with its FDA-approved gene therapy Elevidys, which delivers a truncated dystrophin gene for patients aged four and older.

Additionally, WAVE Life Sciences has developed WVE-N531, an exon skipping oligonucleotide that has shown promising results in trials, indicating significant dystrophin expression.