Gene Therapy

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Just 4 of the 20 cell and gene therapy trials in the study submitted the same trial evidence to both the FDA and EMA. | Image credit: Treecha - stock.adobe.com
Uniform Global Standards, Processes Could Accelerate CGT Approvals, Access

March 13th 2025

The study found that just 20% of trials submitted to both the FDA and European Medicines Agency had matching evidence.

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Genetic Hearing Loss Improves With DB-OTO Gene Therapy: CHORD Trial

February 27th 2025

In the INSPIRE DUCHENNE trial, interim 90-day biopsy data showed an average microdystrophin expression of 110% among the first 3 participants in the study. | Image credit: RFBSIP - stock.adobe.com
SGT-003 Gene Therapy Shows Potential in DMD

February 26th 2025

The authors emphasized the importance of early diagnosis and treatment with CGTs, especially for patients with genetic conditions. | Image credit: RFBSIP - stock.adobe.com
Mitigating Barriers to Cell and Gene Therapy Access

February 14th 2025

Duchenne muscular dystrophy | Image credit: hafakot - stock.adobe.com
EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD

January 28th 2025

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