Gene Therapy

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SRP-9003 is currently being evaluated in a phase 1/2 clinical trial and the phase 3 EMERGENE clinical trial. | Image credit: vladimircaribb - stock.adobe.com
FDA Grants Platform Technology Designation to Limb-Girdle Muscular Dystrophy Gene Therapy Vector

July 1st 2025

The platform technology designation is a new designation established by the FDA.

An analysis of outcomes from part 2 of the EMBARK trial included 14 patients who had received a placebo in part 1 of the study and were aged 8 to 9 years at crossover. | Image Credit: RFBSIP - stock.adobe.com
DMD Gene Therapy Shows Clinical Benefits in 8- and 9-Year-Old Patients

June 13th 2025

Participants who received RGX-202 demonstrated functional improvement and better outcomes than controls for all measures. | Image Credit: Treecha- stock.adobe.com
RGX-202 Gene Therapy Improves DMD Trajectory, Biomarkers

June 12th 2025

While significant progress has been made, innovations in vector design, immune modulation, and delivery strategies are needed to further improve gene therapy for DMD. | Image credit: RFBSIP - stock.adobe.com
Hurdles Remain Despite Progress in Gene Therapy for DMD

May 30th 2025

While patients should be closely monitored in the first 90 days post-treatment, delandistrogene moxeparvovec showed a manageable, consistent safety profile in a broad population of patients. | Image Credit: Hypnosis - stock.adobe.com
Delandistrogene Moxeparvovec Shows Long-Term Safety, Tolerability in Pooled Trial Data

May 16th 2025

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