DMD Awareness Week: Duchenne Candidates to Watch in 2026

According to Parent Project Muscular Dystrophy, Duchenne muscular dystrophy (DMD), which causes progressive muscle weakness, constitutes the most common form of muscular dystrophy in children. The condition primarily affects boys, with around 1 in 5,000 boys currently living with DMD. Although no cure for DMD is available, a number of new transformative treatment options are currently under development, including gene therapies, cell therapies, RNA therapies, and other advanced approaches.

In honor of DMD Awareness Week, observed annually from February 13 to 19 by the patient and clinician communities, NeurologyLive® is taking a look at some notable therapeutic candidates currently in development for the disease.

1. Deramiocel (Capricor Therapeutics)

January 20, 2026 — In a new company update, Capricor Therapeutics announced that the FDA has formally requested the complete clinical study report and supporting data from the phase 3 HOPE-3 trial (NCT05126758) of deramiocel, as the agency continues to review of the company’s biologics license application (BLA). The investigational cell therapy, which remains under review, is aiming to become the first cell-based product for patients with DMD cardiomyopathy.

Capricor stated in its company update that preparation of the HOPE-3 clinical study report is already underway and that the requested materials are expected to be submitted to the FDA in February 2026. The company noted that it anticipates that this submission will support continued review of the BLA and may lead to the assignment of a new PDUFA action date.

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2. Del-Zota (Avidity Biosciences)

September 15, 2025 — Newly announced data from the EXPLORE44 (NCT05670730) and EXPLORE44-open label extension (OLE) trials showed that treatment with Avidity’s del-zota led to reversal of disease progression and improvements in a number of efficacy-related outcomes in patients with DMD treated for up to 1 year. The company remains on track to submit a BLA for accelerated approval at the end of the year.

EXPLORE44 was a placebo-controlled, double-blind, phase 1/2 trial of 26 patients with DMD with mutations amenable to exon 44 skipping (DMD44). Of these, 17 directly enrolled into the EXPLORE44-OLE, where both ambulatory and nonambulatory patients received 5 mg/kg of del-zota every 6 weeks over a 24-month period. While not all patients completed all the assessments, 1-year functional data from the pooled dosing cohorts for del-zota–treated patients showed improvement relative to DMD44 natural history cohorts.

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3. RGX-202 (REGENXBIO)

January 15, 2026 — Among 4 patients treated with the pivotal dose of Regenxbio’s RGX-202, an investigational adeno-associated virus (AAV) vector–based gene therapy intended to treat Duchenne muscular dystrophy (DMD), in the phase 1/2 portion of the AFFINITY DUCHENNE clinical trial (NCT05693142), all 4 surpassed expected disease trajectory on the North Star Ambulatory Assessment (NSAA) with utilization of the Collaborative Trajectory Analysis Project (cTAP) disease progression model at 18 months post treatment.¹

Specifically, Regenxbio noted that the patients treated with the gene therapy product showed an average improvement of 7.4 points compared with cTAP. The company also pointed out that at 12 months post treatment, the 4 treated patients had improved an average of 6.6 points compared with cTAP.

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4. PBGENE-DMD (Precision Biosciences)

May 28, 2025 — At the 2025 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, Precision Biosciences shared new preclinical data surrounding its first-in-class in vivo gene editing method PBGENE-DMD, which is based on the ARCUS platform, in humanized mouse models. In mice, adeno-associated-delivered PGBENE-DMD restored functional dystrophin expression across cardiac and skeletal muscles, leading to sustained muscle improvement over 9 months. Based on the totality of the preclinical data, the company is planning to file an investigational new drug application (IND) and/or clinical trial application in 2025, with clinical data expected in 2026.

For greater insights on the data presented at ASGCT 2025, as well as the mechanism and rationale behind PBGENE-DMD, NeurologyLive sat down with Jeff Smith, PhD, cofounder and chief research officer at Precision. Smith explained how the ARCUS-based approach precisely excises a “hot spot” mutation region, potentially benefiting up to 60% of patients with DMD by restoring near full-length dystrophin production.

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5. GNT0004 (Genethon)

March 18, 2025 — Interim results from the ongoing, international, all-in-one GNT-016-MDYF trial indicated that GNT0004 (Genethon), an investigational gene therapy designed to target skeletal and cardiac muscles, led to sustained reductions in creatine kinase (CK) levels and stabilization or improvement in functional outcomes among young boys with DMD.

Data from Part 1 of the study, which included a dose-escalation phase, showed that patients receiving Dose 2 (3×10¹³ vg/kg) experienced a persistent 68% reduction in CK levels over 2 years, suggesting long-term stabilization of muscle integrity. Additionally, clinical measures such as the NSAA and stride velocity (SV95C) demonstrated improvements compared with an external control group from a natural history study.

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6. DT-DEC01 (Dystrogen Therapeutics)

March 18, 2025 — Findings from a pilot single-site, open-label study showed that DT-DEC01 (Dystrogen Therapeutics), an investigational dystrophin expressing chimeric (DEC) cell therapy, was safe in non-ambulatory patients with DMD for a 24-month period. Coupled with improvements on functional tests, the findings further support the development of the agent as a treatment for DMD, regardless of gene mutation or disease progression.

Led by Maria Siemionow, MD, PhD, DSc, professor and director of Microsurgery Research at the University of Illinois, the analysis featured 24-month data on 3 patients, aged 11-16, who received doses of 2x10⁶, 4x10⁶, and 6x10⁶ cells per kg body weight, respectively, without immunosuppression. Presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas, Texas, treatment with the cell therapy results in zero study-related adverse events (AEs), serious AEs, or donor-specific antibodies (DSA) up to 24 months.

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7. SGT-003 (Solid Biosciences)

February 18, 2025 — Newly reported 90-day findings from the phase 1/2 INSPIRE DUCHENNE trial (NCT06138639) showed that treatment with SGT-003 (Solid Biosciences), an investigational gene therapy, was safe and resulted in significant microdystrophin expression in boys with DMD. The study is still enrolling, with at least 10 total participants expected to be dosed by early in the second quarter of 2025, and approximately 20 total participants by the fourth quarter of this year.

Using a cutoff date of February 11, 2025, the new data comprised clinical and efficacy findings from the first 3 patients dosed in the study, as well as safety data from the first 6 patients in the study. The first 3 participants, at the time of dosing, are two 5-year-old boys and one 7-year-old boy, while the second group consisted of a 6-year-old boy and two 7-year-old boys. In the study, patients received a one-time administration of SGT-003 at a dose of 1E14vg/kg and were followed throughout.

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REFERENCE
1. About Duchenne. Parent Project Muscular Dystrophy. Website. Accessed February 13, 2025. https://www.parentprojectmd.org/about-duchenne/