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ifezuntirgene inilparvovec
- Development stage
- Phase 2
- Lead developer
- uniQure
- Modality
- RNA Therapeutics → Nucleic Acid Therapeutics, Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intracerebral
01
Overview
Ifezuntirgene inilparvovec is a recombinant, replication-deficient adeno-associated virus serotype 5 (AAV5) gene therapy vector. It encodes a microRNA (miRNA), specifically miHTT, which targets exon 1 of the human huntingtin protein (HTT) mRNA. The expression cassette includes a chimeric chicken beta-actin promoter and the miHTT sequence engineered in the miR-451 backbone, with an upstream region containing an inactivated miR-144 sequence and a human growth hormone polyadenylation signal, flanked by AAV2 inverted terminal repeats. The primary therapeutic goal is to reduce mutant huntingtin protein expression for the treatment of Huntington's disease.
- Other names
- Huntington's gene therapyuniQure Huntington's gene therapy
02
Targets
HTT mRNA (HTT / Huntingtin gene)HTT (Huntingtin protein)
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