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

The Medicine Maker

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.

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

Objective:

Key Findings:

Interpretation:

Limitations:

Conclusion:

Original Source(s)

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

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