DB18582InvestigationalGene Therapies
scAAV9.U7.ACCA is a non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding... Developed by Nationwide Children’s Hospital, it is being investigated for the treatment of Duchenne muscular dystrophy.
- Also known as
- non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNA
What you can answer from here — as of April 23, 2025
Which companies are running trials of scAAV9.U7.ACCA, in which indications and phases, and which of those programs are still active?
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