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Entrada Therapeutics ENTR-601-45: Early Exon 45 Skipping Study Shows Increased Dystrophin Production
Entrada Therapeutics has announced positive initial results from its Exon 45 Skipping Study, ELEVATE-45-201, evaluating ENTR-601-45 in people with Duchenne muscular dystrophy (Duchenne). The first group of participants showed a favorable safety profile and a small increase in dystrophin production after receiving the initial dose. The findings are an...
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Entrada Therapeutics Advances ENTR-601-44 to 18 mg/kg in Its Duchenne Exon 44 Skipping Study
Entrada Therapeutics has received clearance to test the highest dose in its Exon 44 Skipping Study for Duchenne muscular dystrophy (DMD). The independent Data Monitoring Committee (DMC) has recommended starting Cohort 3 at 18 mg/kg in the ELEVATE-44-201 clinical trial, following its review of available safety and pharmacokinetic data...
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Press Releases
New Positive Functional Data From The RGX-202 Affinity Duchenne® Trial Phase 1/2 Reported By Regenxbio
The Regenxbio press statement stated that the findings, which came from five patients in...
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PepGen Announces It Will Halt Clinical Trials of Exon 51 Skipping Study
PepGen, a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies, today announced...
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Entrada Therapeutics Receives EU Authorization to Start Exon 45 Skipping Clinical Trial in Duchenne Muscular Dystrophy
Entrada Therapeutics, today announced it has received authorization from the Health Authorities and Ethics...
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Entos Pharmaceuticals Develops Re-Dosable, Full-Length Dystrophin Gene Therapy: Fusogenix PLV Method
Entos Pharmaceuticals Inc. (Entos) is a biotech business that creates genetic therapies using its...
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Genethon Reports Results of GNT0004 Gene Therapy Clinical Trial: Permanent Decrease in CK Levels of More Than 75%
Safety, efficacy, and pharmacodynamic results show good tolerance of GNT0004 associated with transient immunological...
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Precision BioSciences PBGENE-DMD Restores Production of Nearly Full-Length Dystrophin Protein
Precision BioSciences, today announced the strategic prioritization and acceleration of PBGENE-DMD, the Company’s first-in-class...
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Satellos Reports SAT-3247 Q1 2025 Results, Highlights Recent Company Progress and Future Plans
Satellos Bioscience, today announced its financial results and corporate highlights for the first quarter...
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Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy
People aged 3 to under 8 who do not have deletions in exons 8...
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SonoThera Ultrasound-Mediated Delivery Method Can Deliver Full-Length Dystrophin to Cells
The findings of the study, which are currently being developed for SonoThera's Duchenne Muscular...
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Scribe Therapeutics and Prevail Therapeutics to Share In Vivo CRISPR-based Genome Editing Data at ASGCT 2025
Scribe Therapeutics, a genetic medicines company unlocking the potential of CRISPR to transform human...
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Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy
Capricor Therapeutics , a biotechnology company developing transformative cell and exosome-based therapeutics for the...
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Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy
Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with...
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Press Releases
Entrada Therapeutics ENTR-601-45: Early Exon 45 Skipping Study Shows Increased Dystrophin Production
Entrada Therapeutics has announced positive initial results from its Exon 45 Skipping Study, ELEVATE-45-201,...
Press Releases
Entrada Therapeutics Advances ENTR-601-44 to 18 mg/kg in Its Duchenne Exon 44 Skipping Study
Entrada Therapeutics has received clearance to test the highest dose in its Exon 44...
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Sapreme Therapeutics Reports Strong Preclinical Dystrophin Restoration With SPM-2551 in Duchenne
Sapreme Therapeutics has announced promising preclinical results for SPM-2551, an experimental treatment for Duchenne...
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Deramiocel Shows Positive 24-Month HOPE-3 Results in Duchenne
Capricor Therapeutics has reported new results from the HOPE-3 study evaluating Deramiocel in people...
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Ractigen Therapeutics Reports Positive First-in-Human Data for RAG-18 in Duchenne
Ractigen Therapeutics has presented positive first-in-human data for RAG-18 (NCT07282652), an investigational treatment for...
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Entrada Therapeutics’ ENTR-601-44 Shows Dose-Dependent Exon 44 Skipping in Early PK/PD Data
Entrada Therapeutics’ ENTR-601-44 is an investigational therapy designed for patients with Duchenne muscular dystrophy...
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NS Pharma’s Brogidirsen Shows 5-Year Clinical Data in DMD Patients Amenable to Exon 44 Skipping
NS Pharma’s brogidirsen (NS-089/NCNP-02) is an investigational therapy for Duchenne muscular dystrophy (DMD) designed...
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ELEVIDYS WMS 2026 Data: What They Show—and What They Don’t
Sarepta Therapeutics has presented new data on ELEVIDYS (delandistrogene moxeparvovec) at the 2026 World...