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Capricor’s Deramiocel

Regulatory Update on Capricor’s Deramiocel for Duchenne

June 24, 2025

Capricor’s Deramiocel for Duchenne: Capricor Therapeutics, which received early funding from CureDuchenne, has indicated that the FDA has decided that an Advisory Committee meeting is not warranted at this time.  […]

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Entos Gene Therapy

CureDuchenne’s latest investment aims to address the limitations of current AAV-delivered gene therapy treatments for Duchenne muscular dystrophy (DMD).

June 18, 2025

Our recent investment in Entos Pharmaceuticals (https://entospharma.com) aims to explore the potential of their non-viral, redosable Fusogenix PLV platform to deliver full-length dystrophin protein to all muscle groups in a […]

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FDA news Rare disease

US leadership emphasizes support for rare disease drug development

June 6, 2025

CureDuchenne is pleased that one of the main themes at the recent US FDA Cell and Gene Therapy Roundtable was preserving incentives and leveraging regulatory flexibility and innovation to remove […]

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Duchenne gene therapy Entos

CureDuchenne Ventures Invests in Entos Pharmaceuticals for Development of a Redosable, Full-Length Dystrophin Gene Therapy

May 22, 2025

Newport Beach, Calif. and Edmonton, Canada (May 22, 2025) – CureDuchenne Ventures announced an initial $1M investment in Entos Pharmaceuticals Inc (Entos), a biotech company that develops genetic medicines utilizing […]

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Wave exon skipping Duchenne

Wave Life Sciences: Update on 48-week Data from FORWARD-53 Exon Skipping Study

April 8, 2025

Wave Life Science Exon Skipping in Duchenne Wave Life Sciences: Update on 48-week Data from FORWARD-53 Exon Skipping Study: An update on Wave’s Phase 2 FORWARD-53 trial of WE-N531, Wave’s […]

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Wave exon skipping

Promising New Data from Wave Life Sciences in Duchenne Exon-skipping Trial

March 26, 2025

Wave Life Sciences Exon-skipping Trial Wave Life Sciences has shared new data from its ongoing FORWARD-53 clinical trial, evaluating the investigational exon-skipping therapy WVE-N531 for individuals with Duchenne muscular dystrophy […]

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gene therapy trial

RegenXBio continues to report positive biomarker data from their AAV-microdystrophin gene therapy trial

March 24, 2025
AAV-microdystrophin gene therapy trial RegenXBio reported new interim data from 2 additional participants in the Phase 1/2 portion of the AFFINITY DUCHENNE trial of RGX-202, an AAV8-microdystrophin gene therapy. In [...]
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Exon skipping 45

Entrada receives authorization in the United Kingdom to start a Phase 1/2 Duchenne trial skipping exon 45

March 24, 2025

Entrada trial skipping exon 45 CureDuchenne provided early funding to Entrada Therapeutics, who has announced that they received authorization in the United Kingdom (UK) to initiate a Phase 1/2 multiple […]

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Capricor announces the FDA will decide on approval of their experimental cell therapy in August 2025

March 4, 2025

CureDuchenne provided early funding to Capricor Therapeutics, and we are pleased to share that the FDA has accepted its Biologics Licenses Application (BLA) seeking approval for Deramiocel to treat cardiomyopathy in […]

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CureDuchenne is a 501c3 registered tax-exempt nonprofit organization. Federal Tax ID: #20-0299958 | ©2025 CureDuchenne. All rights reserved.

Please be advised that the contents within the CureDuchenne site include a compilation of articles, advisements, and references from the community. CureDuchenne does not provide medical advice nor is CureDuchenne a medical provider. The intent of this shared content is to serve as a point of reference for informational purposes. Please refer to your health care provider(s) for your individualized, specific care management needs. The CureDuchenne website may contain links to external websites that are not provided and/or maintained by or in any way affiliated with CureDuchenne. CureDuchenne recommends that you review the terms of service and privacy policies of these external websites before entering. Please note that CureDuchenne does not guarantee the accuracy, relevance, timeliness, or completeness of any information on these external websites.

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