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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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GenAssist Announces FDA Approval of New Drug Application for GEN6050X Exon 50 Skipping Therapy
On March 06, 2025, GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company specializing in...
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PepGen Temporarily Pauses Exon 51 Skipping Study
PepGen, a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the...
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Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment...
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European Clinical Trial Map Launched
The Clinical Trials Information System's (CTIS) public website now features a new clinical trial...
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Entrada Therapeutics Receives FDA Approval to Begin Clinical Trial of ENTR-601-44 Exon Skipping Treatment
Entrada Therapeutics, announced that the United States Food and Drug Administration (FDA) has lifted...
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Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003
Solid Biosciences, a life sciences company developing precision genetic medicines for neuromuscular and cardiac...
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Santhera Announces Agreement with German GKV-SV on Reimbursement Amount for AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy in Germany
Santhera Pharmaceuticals, today announces an agreement with the German National Association of Statutory Health...
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Percheron Therapeutics Announces Terminated of ATL1102 Study for DMD
“One way or other, we don’t think ATL-1102 can remain the centerpiece of the...
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Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201
ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-44, was authorized...
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Sidra Medicine Establishes Gene Therapy Center for Rare Genetic Diseases in Qatar
The announcement was made at Arab Health 2025, where Sidra Medicine will showcase its...
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In Brazil, the Government is Expected to Implement a Risk-Sharing Model with Roche for ELEVIDYS
Elevidys, a treatment for Duchenne muscular dystrophy, is the most costly medication authorized in...
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Will Genethon and Eukarÿs Partnership Reduce the Cost of Gene Therapies?
The FDA-approved only gene therapy for Duchenne muscular dystrophy is costing far too much...
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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...
Research
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...