Rare disease drugmaker · based in Woodbridge, United States · cureraredisease.org
Cure Rare Disease works on Duchenne Muscular Dystrophy, one of the rare diseases we track.
Cure Rare Disease is a nonprofit biotechnology company in Woodbridge, Connecticut that develops gene therapies and antisense oligonucleotide (ASO) treatments for rare and ultra-rare diseases. Its lead programs are CRD-003, a gene therapy for limb-girdle muscular dystrophy type 2I/R9, and CRD-002, an ASO for spinocerebellar ataxia type 3.
Cure Rare Disease clinical trials
We did not find active Cure Rare Disease trials for the rare diseases we track on ClinicalTrials.gov right now.
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CRD-003 received FDA orphan drug designation in September 2025 and a $7.4 million grant from the California Institute for Regenerative Medicine in December 2025. CRD-002 received orphan drug designation in May 2025. Neither program had a trial listed on ClinicalTrials.gov in October 2026.
Its earlier Duchenne muscular dystrophy study (CRD-TMH-001, NCT05514249) treated a single patient with a custom CRISPR-based gene therapy developed with researchers at Yale School of Medicine and UMass Chan Medical School. The study ended when its only participant, a 27-year-old man, died in October 2022, 8 days after treatment.
A 2023 report in the New England Journal of Medicine concluded that an innate immune reaction caused acute respiratory distress syndrome after the high-dose AAV9gene therapy.
Rare diseases Cure Rare Disease works on
Each disease page lists every open trial, not only Cure Rare Disease's, plus approved treatments.
Duchenne muscular dystrophy is an X-linkedgenetic disorder causing progressive muscle weakness and degeneration, beginning in early childhood.
How common: 1 in 3,500 to 5,000 male births
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Cure Rare Disease's own site is cureraredisease.org, and our help paying for treatment page lists drugmaker programs and charity funds.
1 shared disease, including Duchenne Muscular Dystrophy
Questions about Cure Rare Disease
Cure Rare Disease is a nonprofit biotechnology company in Woodbridge, Connecticut that develops gene therapies and antisense oligonucleotide (ASO) treatments for rare and ultra-rare diseases. Its lead programs are CRD-003, a gene therapy for limb-girdle muscular dystrophy type 2I/R9, and CRD-002, an ASO for spinocerebellar ataxia type 3. CRD-003 received FDA orphan drug designation in September 2025 and a $7.4 million grant from the California Institute for Regenerative Medicine in December 2025. CRD-002 received orphan drug designation in May 2025. Neither program had a trial listed on ClinicalTrials.gov in October 2026. Its earlier Duchenne muscular dystrophy study (CRD-TMH-001, NCT05514249) treated a single patient with a custom CRISPR-based gene therapy developed with researchers at Yale School of Medicine and UMass Chan Medical School. The study ended when its only participant, a 27-year-old man, died in October 2022, 8 days after treatment. A 2023 report in the New England Journal of Medicine concluded that an innate immune reaction caused acute respiratory distress syndrome after the high-dose AAV9gene therapy.
Cure Rare Disease has active programs across 1 rare disease: Duchenne Muscular Dystrophy. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. Cure Rare Disease is a rare disease specialist headquartered in Woodbridge, United States. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Duchenne Muscular Dystrophy.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Cure Rare Disease, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
None that we track as of November 2026. No Cure Rare Disease label in the FDA's openFDA database matches a rare disease on Trial Friend, which usually means the company has no approved product yet or its approved drugs are for common conditions.
In development: the trials list on this page shows what Cure Rare Disease is recruiting for now, and the FDA calendar lists any application once a decision date is public.