In 2018, CureDuchenne was the only Duchenne-focused organization to invest in Avidity Biosciences, recognizing the potential of their novel RNA-targeting approach for individuals with Duchenne muscular dystrophy long before it reached clinical trials. Today, we’re proud to celebrate a milestone that reflects exactly how patient-focused progress can work when scientific innovators, regulatory agencies, and a dedicated community come together with a shared goal.
Avidity Biosciences has launched a Managed Access Program (MAP) for its investigational therapy, del-zota (delpacibart zotadirsen), designed for individuals with Duchenne muscular dystrophy amenable to exon 44 skipping. Under an FDA-authorized treatment protocol, eligible individuals will be able to receive del-zota through participating healthcare providers. Enrollment is expected to begin before the end of this year.
This initiative reflects a shared commitment to getting potential treatments to the people who need them, as safely and efficiently as possible.
From Early Belief to Real-World Impact
In 2018, CureDuchenne made an early investment in Avidity because we saw what was possible. Their AOC (Antibody Oligonucleotide Conjugates) platform offered a new way to deliver RNA therapies directly to muscle. Now that vision is closer to reaching families.
We are incredibly grateful to Avidity for their commitment to people living with Duchenne, and to the FDA for helping make this access program possible. This is a strong example of what CureDuchenne’s venture philanthropy model is designed to accelerate: early-stage innovation, careful clinical development, and regulatory pathways that prioritize both safety and efficacy.
What Comes Next
Avidity is on track to submit for FDA approval of del-zota in 2026. In the meantime, this MAP provides an important opportunity for those eligible to receive treatment earlier through the participating providers.
Additional details, including eligibility criteria, will be available at www.clinicaltrials.gov.
Why This Matters
This is how CureDuchenne’s model creates impact:
- Early investment in promising therapeutic science
- Ongoing support through clinical development
- Centering the needs of individuals with Duchenne by prioritizing safety, effectiveness, and meaningful access
We are proud to have supported Avidity from the beginning, and we are hopeful about the future of this treatment and the potential to expand the approach to reach more individuals with Duchenne. This program is a meaningful step forward and a reminder that when we work together, real progress is possible.
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