5 DMD Researches You Should Follow up in 2025

It took thirty years after the dystrophin gene was discovered in 1986 for the first treatment for Duchenne muscular dystrophy to be available on the market. With the approval of eight treatments in as many years, significant progress has been made in the fight against the neuromuscular condition that can be fatal.

Despite significant advancements over the past ten years for DMD patients, there is still a significant unmet demand. In order to meet the need, a number of businesses, such as Wave, Dyne, and Avidity, are developing experimental treatments that aim for increased effectiveness and wider accessibility.

2025 could be a breakout year for the Duchenne muscular dystrophy field. While several therapies have entered the market in recent years, there is still a significant unmet need for children living with DMD. But with several companies advancing next-generation treatments and the industry now having established regulatory pathways, experts say new options are on the horizon.

A number of businesses, such as Dyne, Avidity, and Wave Life Sciences, are currently working to respond to this need. Given the development of a new generation of exon skipping therapies and the gene therapies offered by Regenxbio and Solid Bio, there is good reason to think that further DMD treatments may soon be available. [Read More: Next Generation Exon Skipping Therapies for DMD]

Capricor Therapeutics: Deramiocel

The FDA is now reviewing a biologics license application for Capricor’s allogeneic cell treatment for DMD cardiomyopathy. [Read More: Capricor Therapeutics Submits Application for Treatment of DMD Cardiomyopathy]

If approved, Deramiocel would be the first drug to specifically treat DMD cardiomyopathy. Nearly all patients develop heart problems by adulthood, making it the primary cause of death from the disease. Based on agency input, Capricor has applied for full FDA approval, which is unusual in the DMD market because most drugs are approved through the FDA’s accelerated approval program. [Read More: What is Deramiocel?]

Dyne Therapeutics: Dyne-251

In order to obtain fast approval for its next-generation exon 51 skipping therapy, Dyne-251, based on FDA input, Dyne is now recruiting for a pivotal trial. Dystrophin expression will be used as a surrogate endpoint. [Read More: What is exon skipping?]

Wave Life Sciences: WVE-N531

After evaluating patients’ needs, Wave Life Sciences CEO Paul Bolno said that the way to a more successful treatment was improved cell distribution for higher dystrophin production and consistent expression.

The Phase II study’s six-month interim analysis revealed that Wave’s WVE-N531 (an exon 53 skipping) consistently displayed 9% dystrophin expression in all patients. This resulted in healthier-looking muscle and reduced blood levels of creatine kinase, a biomarker for muscle injury, according to Bolno. Wave anticipates presenting the complete 48-week results in the first quarter of 2025, which will include the impact on clinical metrics.

Avidity Biosciences: Del-Zota

By focusing on exon 44, for which there are presently no approved medications, Avidity may be able to provide treatment for the 6% of DMD patients who are susceptible to exon 44 skipping.

According to Michael Kelly, chief scientific officer of CureDuchenne, delpacibart zotadirsen (del-zota) enhanced dystrophin production in DMD patients to a staggering 25% of normal function in a Phase I/II trial. This is some of the best exon skipping evidence observed from any medication. Additionally, Del-Zota brought creatine kinase down to almost normal levels.

The business intends to submit a BLA in mid-2026, and more results from the Explore44 open-label extension study is anticipated this year. Del-Zota has already received fast-track classification and orphan drug designation from the FDA.

Regenxbio: RGX-202

With RGX-202, which is presently undergoing a pivotal research phase , Regenxbio is vying for Sarepta’s Elevidys in the gene therapy market.

Thirty patients who are one year of age or older are being recruited for the experiment, making the total population younger than Elevidys’ existing label. According to Kelly, first findings from a small group of patients published in November 2024 were “extremely encouraging,” showing improvements in strength and time function tests along with a solid safety profile. In 2026, Regenxbio anticipates submitting a BLA.

The size of the DMD gene, which is just too big for the AAV delivery vehicle, is a major obstacle for gene therapy in this field, according to Kelly. As a result, a shortened form is used, but its end product is unable to function as well as the full-sized protein. Compared to Elevidys, Regenxbio’s architecture permits a somewhat larger version of the gene.

Kelly is eager to see the results of a previous trial extended to a greater number of patients. If effective, he thinks it will give parents searching for a DMD gene therapy another option and chance.

Learn More: Cures of Duchenne (List of All Researches)

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