Drug intelligence / Profile preview

dual-AAV.Var4 STRC

Development stage
Preclinical
Lead developer
Medizinische Hochschule Hannover
Modality
Gene Therapies
Administration
Otic
01

Overview

**dual-AAV.Var4 STRC** is a preclinical inner-ear gene-replacement therapy designed for autosomal recessive nonsyndromic hearing loss caused by pathogenic variants in the **stereocilin gene**. It uses the outer-hair-cell-tropic AAV capsid variant Var4 in a dual-AAV RNA-trans-splicing system, with each vector carrying a portion of the large STRC coding sequence. Co-delivery is intended to reconstitute full-length stereocilin expression in cochlear outer hair cells, restore stereociliary architecture, and improve auditory function. In mature Strc-deficient mice, the system produced persistent STRC expression, partial outer-hair-cell stereocilia rescue, and partial improvement in auditory brainstem response thresholds.

Other names
dual-AAV.Var4

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