This Week’s CGT News: Duchenne Gene Therapy Hits Pivotal Mark - Scorecard - MDSpire
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This Week’s CGT News: Duchenne Gene Therapy Hits Pivotal Mark

REGENXBIO reports phase III AAV data in Duchenne; plus: adenoviral immunotherapy, preconditioning-free CAR-T, in vivo immune programming, and DNA-guided CRISPR

  • May 25, 2026

  • 7 min

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Clinical Scorecard: This Week’s CGT News: Duchenne Gene Therapy Hits Pivotal Mark

At a Glance

CategoryDetail
ConditionDuchenne muscular dystrophy
Key MechanismsAAV microdystrophin gene therapy
Target PopulationPatients with Duchenne muscular dystrophy
Care SettingClinical trial setting

Key Highlights

  • 93% of evaluable participants achieved at least 10% microdystrophin expression at week 12.
  • Editing was sustained for at least three months after the last dose.
  • No serious adverse events or clinically meaningful liver test elevations reported.

Guideline-Based Recommendations

Diagnosis

    Management

      Monitoring & Follow-up

        Risks

          Patient & Prescribing Data

          Evaluable participants in the AFFINITY DUCHENNE trial

          One-time AAV microdystrophin gene therapy

          Clinical Best Practices

            Related Resources & Content

              Original Source(s)

              This Week’s CGT News: Duchenne Gene Therapy Hits Pivotal Mark

              • May 25, 2026

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