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dual-AAV mini-dystrophin (Cfa intein)

Development stage: Preclinical
Lead developer: Instituto de Biologia Experimental e Tecnológica (iBET)
Modality: Gene Therapies

Overview

**Dual-AAV mini-dystrophin Cfa intein** is a research-stage, dual-vector adeno-associated virus gene-therapy construct designed to address the limited packaging capacity of AAV for dystrophin replacement. Two AAV vectors each deliver a portion of a mini-dystrophin coding sequence fused to complementary fragments of the engineered **Cfa split intein**. Following co-transduction of muscle cells, Cfa-mediated protein trans-splicing is intended to join the two translated mini-dystrophin fragments into a reconstituted therapeutic protein. The construct was described in an ASGCT 2026 abstract as one of several split-intein approaches evaluated in murine myotubes and human induced pluripotent stem cell-derived skeletal-muscle models. It is not presented as a sponsor-named clinical candidate, and the exact AAV capsid, mini-dystrophin sequence, dose, and clinical route were not specified.

Other names: dual-AAV mini-dystrophin Cfa intein

Targets

DMD (Dystrophin)F-actin (Filamentous actin)

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