RegenXBio shared positive updates from their Phase 1/2 AAV micro-dystrophin gene therapy clinical trial for individuals with Duchenne aged 4-11 years old.
The 3 individuals in the lowest dose cohort all demonstrated reductions in CK levels, and expression of micro-dystrophin averaging 44% at 3 months.
Enrollment is also complete for a second cohort, which will receive a higher dose of the experimental therapy. RegenXBio plans to share initial strength and functional data for both dose levels and initiate a pivotal trial in the second half of this year.
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The post REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial appeared first on CureDuchenne.