CureDuchenne

11/06/2025

As an early investor in Entrada Therapeutics, we are pleased to share their progress on their exon-skipping programs in Duchenne.

For Exon 44: Entrada completed enrollment for the first cohort of the ex-US ELEVATE-44-201 study for those amenable to skipping exon 44, and are on track to report data in Q2 2026. They expect to initiate a Phase 1b study in the US for ambulatory and non-ambulatory adults with Duchenne in 2026.

For Exon 45: Entrada has dosed the first participant in the ELEVATE-45-201 study for those amenable to skipping exon 45, and will report data from the first cohort in mid-2026.

For Exon 50: Entrada filed for regulatory authorization in the U.K. for the ELEVATE-50-201 study to begin the study there by the end of 2026.

Link to press release:
https://ir.entradatx.com/news-releases/news-release-details/entrada-therapeutics-reports-third-quarter-2025-financial

11/04/2025

Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026.

23 Pediatric participants have been dosed (1E14vg/kg) so far in the INSPIRE DUCHENNE trial (data cutoff 31st October). SGT-003 was generally well tolerated, there were no cases of drug-induced liver injury observed (n = 23) and day-90 biopsy data from 10 treated participants (ages 5-10) showed that all responded to treatment with mean muscle microdystrophin expression of 58% by both western blot and mass spectrometry.

The company expects to have dosed 30 participants by early 2026, and plans to meet with the FDA to discuss a potential registrational pathway in the first half of 2026.

Solid Bio also reported interim clinical data that included correlations observed between SGT-003 microdystrophin therapy and improvements in several biomarkers of muscle integrity, including serum creatine kinase, lactate dehydrogenase, aspartate aminotransferase and embryonic myosin heavy chain, that suggested an effect of treatment with SGT-003.

The company plans to host a number of educational events/webinars in the coming weeks to communicate todays results to the broader Duchenne community.

Read the press release:
https://cureduchenne.org/research/solid-plans-to-seek-fda-guidance-in-2026-for-duchenne-gene-therapy-candidate-sgt-003-s/

Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026.

11/04/2025

Sarepta has completed the ESSENCE study, which is a confirmatory trial aimed at supporting full FDA approval of the exon-skipping PMOs Amondys 45 and Vyondys 53. The study did reinforce the favorable safety profile of the therapies, but did not achieve statistical significance on the primary endpoint (4-step ascend velocity at 96 weeks).

What comes next? Sarepta says it plans to discuss with the FDA a path from accelerated approval towards full approval, utilizing this data as well as real world evidence of these therapeutic approaches collected over multiple years.

Read the press release and letter to the community:
https://cureduchenne.org/general/sareptas-amondys-45-and-vyondys-53-confirmatory-trial-fails-to-achieve-statistical-significance-on-primary-endpoint-v2/

10/30/2025

RegenXBio announced that they have completed enrollment in the Phase 1/2 portion of their RGX-202 microdystrophin gene therapy trial, with topline data expected in Q2 2026. They are continuing to enroll participants aged 1 year and above in the confirmatory trial, and have initiated manufacturing of commercial supply of drug.

Read the PRESS RELEASE & LETTER TO THE COMMUNITY :

RegenXBio announced that they have completed enrollment in the Phase 1/2 portion of their RGX-202 microdystrophin gene therapy trial

10/29/2025

In 2018, CureDuchenne made a bold investment — funding Avidity Biosciences when their Antibody Oligonucleotide Conjugates (AOCs™) were still just an idea.

Now, that idea has become a $12 billion acquisition by Novartis.
And for families living with Duchenne, that means everything.

It affirms what families, researchers, and donors have long believed: that bold science, when backed by bold belief, can change the course of Duchenne.

For those living with Duchenne muscular dystrophy, this is what progress looks like. Not just a deal, but a door, one that opens to global resources, clinical advancement, and the potential for accelerated access to life-changing therapies.

It proves that early-stage, strategic investment through venture philanthropy works and that CureDuchenne’s model is actively reshaping the therapeutic landscape for neuromuscular disease.

This acquisition validates the promise of Antibody Oligonucleotide Conjugates (AOCs™), a delivery platform first supported by CureDuchenne in 2018, when belief was all there was. Now, under Novartis’ global leadership, that same science is poised to move faster, reach farther, and touch more lives.

It’s a win for patients, a signal to scientists, and a rallying for what’s next because the journey to a cure doesn’t pause.

https://cureduchenne.org/all-news/avidity-biosciences-to-be-acquired-by-novartis/

10/28/2025

10/27/2025

What a night in Ft. Lauderdale, FL! 🌴💙
Our CureDuchenne Community Dinner was filled with laughter, connection, and even a special 23rd birthday celebration! Families came together to share stories, support one another, and remind us all what makes this community so special — the love and strength we find in each other.

It’s amazing how quickly friendships form when you’re surrounded by people who truly understand the Duchenne journey. The room was full of warmth, smiles, and a sense of belonging that you could feel from the moment you walked in.

A huge thank-you to our 2025 Cares sponsors for making these meaningful gatherings possible. Your support helps us create spaces where families, caregivers, and individuals living with Duchenne can connect, learn, and celebrate together. 🌟

Find a CureDuchenne Community event near you:
The last sessions of 2025 will be in Chicago & Phoenix:
👉 https://cureduchenne.org/events/category/community-events/

10/26/2025

When CureDuchenne invested in Avidity Biosciences in 2018, it was with strong belief in their technology and the promise of what it might be able to achieve in Duchenne.

We congratulate Avidity on all their success to date, and celebrate with them on this important new development. And we look forward to working with Novartis in the future!

Read the full press release:
https://cureduchenne.org/all-news/avidity-biosciences-to-be-acquired-by-novartis/

Avidity Biosciences to be acquired by Novartis

10/25/2025

What an incredible day in Mexico City! Thank you to everyone who made it incredible 💙
And those watching the live feed, the Chihuahua group, Enlace & ADMO in Guadalajara!!

10/25/2025

💙💙💙

10/25/2025

And we are off to a fabulous start in Mexico City! Thank you!!💙💙

10/24/2025

CureDuchenne’s Annual Austin Ladies Luncheon isn’t just an event; it’s a celebration of love, hope, and the unstoppable power of community. This year’s Moroccan-themed “Afternoon in Marrakesh” brought together more than 200 incredible women from across Austin, raising over $155,000 to help cure Duchenne muscular dystrophy. 💙

The day was made truly unforgettable thanks to the generosity and leadership of Austin’s own Revell family. In honor of their sons, Timothy and Andrew, Laura and Tim Revell have now helped raise more than $10 million, turning heartbreak into purpose and inspiring change across the country.

From everyone at CureDuchenne: thank you for making a beacon of hope for every family affected by Duchenne.

Together, we will cure Duchenne.

Read the full story: https://westaustinnews.com/events/moroccan-themed-ladies-luncheon-raises-funds-for-cureduchenne/

Find an event near you: https://cureduchenne.org/events/list/

Want to get involved or learn how to host your own fundraiser?
Reach out to jordan@cureduchenne.org