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

To report on recent advancements in gene therapy and immunotherapy for various conditions.

Key Findings:
  • RGX-202 achieved primary endpoint in Duchenne muscular dystrophy trial.
  • Aglatimagene besadenovec improved disease-free survival in prostate cancer patients.
  • Rese-cel showed initial improvement in pemphigus vulgaris patients.
  • JUST-AAV demonstrated improved CNS uptake in preclinical studies.
  • Scribe's CRISPR technologies showed effective gene repression in preclinical data.
  • Cytokine-armored CAR-T therapy showed promise against glioblastoma.
  • Mezigdomide could restore T cell activity in relapsed multiple myeloma.
  • New CRISPR technology offers improved precision in RNA targeting.
Interpretation:

Limitations:
Conclusion:

Original Source(s)

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

  • May 25, 2026

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