CureDuchenne

06/02/2026

As an early funder of Entrada Therapeutics, we are pleased to share their latest newsletter to the Duchenne community. This update includes news that after review of all the data in the first dose (5 mg/kg) cohort of the ELEVATE-45-201 study for individuals amenable to skipping exon 45, an independent data monitoring committee has recommended dose escalation up to 10mg/kg for a second cohort. The company is on track to report data from Cohort 1 in mid-2026.

See the community letter and press release here: https://cureduchenne.org/research/entrada-shares-updates-on-duchenne-programs-including-initiation-of-cohort-2-in-uk-and-eu-trial-skipping-exon-45/

06/02/2026

As an early investor in Edgewise Therapeutics we are delighted by the news that Servier has entered into a definitive agreement to acquire sevasemten and Edgewise's muscular dystrophy business. Servier is a global pharmaceutical company with extensive experience developing and delivering therapies to patients worldwide and operates in over 140 countries.

We invested in Edgewise in 2019 because we believed in the potential of their science and team. Today's announcement is a powerful validation that our venture philanthropy model works - helping accelerate promising science, attract investment, and move potential therapies closer to patients.

Sevasemten has the potential to become the first approved treatment for Becker muscular dystrophy, and the pivotal cohort of the GRAND CANYON trial is fully enrolled with topline data expected in Q4 2026.

Congratulations to the Edgewise team on this important milestone, and welcome to Servier. We look forward to continuing our work together on behalf of the Becker and Duchenne communities.

Read more on our blog:
https://cureduchenne.org/news/edgewise-acquisition-by-servier-marks-major-milestone-for-becker-and-duchenne-muscular-dystrophy-communities/

06/01/2026

You can now watch the Regexbio webinar from May 19, 2026.

HERE: https://cureduchenne.org/webinars/regenxbio-webinar-2/

06/01/2026

You can now watch the Tevard Biosciences webinar from May 28, 2026.

HERE:

Unlocking the therapeutic power of suppressor tRNAs for DMD

06/01/2026

You can watch the Sarepta Therapeutics webinar from May 19, 2026.

HERE:

ELEVIDYS Gene Therapy Update and ENDEAVOR Cohort 8 Awareness

06/01/2026

Thank you Larry luxner and Rare Disease Advisor for joining us at FUTURES and sharing stories of impact and progress.

Read more about next-generation technologies that received early funding from CureDuchenne, like Dyne Therapeutics' exon 51 skipping program, and why early funding remains so critical for the Duchenne community: https://www.rarediseaseadvisor.com/news/dyne-bla-exon-51-skipping-therapy-cureduchenne-2026-conference-philanthropy/

05/29/2026

We’re encouraged to see decades of research and investment helping move promising therapies closer to families who urgently need them. This latest BioSpace article features insights from CureDuchenne Chief Scientific Officer Dr. Michael Kelly and highlights several companies advancing toward FDA submissions, including a number of companies CureDuchenne has proudly invested in over the years.

Read the full article using the link below!

https://www.biospace.com/drug-development/regenxbio-novartis-dyne-near-fda-submissions-in-muscular-dystrophies

Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.

05/29/2026

Advancing Duchenne Care Through Global Collaboration 🌎🇨🇱

CureDuchenne was honored to participate in the first Latin American Duchenne Symposium in Chile, joining experts and advocates dedicated to improving care and outcomes across the region.

Read more in our blog using the link below!

https://cureduchenne.org/blog/duchenne-care-chile/

05/27/2026

As an investor in Avidity, we recognized early that the platform technology will be useful in many other diseases beyond Duchenne, including Alzheimer’s and Parkinson’s. This underscores the far-reaching impact that investing in innovative science can have for patients and families across multiple communities

Read the full article using the link below!

https://endpoints.news/novartis-says-aviditys-science-could-apply-beyond-muscular-dystrophies/

05/26/2026

CureDuchenne was an early funder of Dyne Therapeutics, which today announced they have submitted a BLA to the FDA for z-rostudirsen, for the treatment of individuals with Duchenne amenable to skipping exon 51. This application is for Accelerated Approval based on dystrophin as a surrogate endpoint, and includes positive data from Dyne’s Phase 1/2 DELIVER clinical trial, which showed significant increase in dystrophin production with functional improvement across multiple clinical endpoints. If approved under FDA Priority Review, z-rostudirsen could be available in Q1 2027.

In parallel, Dyne has launched a Phase 3 confirmatory trial of z-rostudirsen, and is advancing 4 additional exon-skipping programs for exons 53, 45, 44, and 55.

Read more: https://cureduchenne.org/research/dyne-submits-biologics-license-application-bla-to-the-fda-for-exon-51-skipping-z-rostudirsen/

Dyne Therapeutics