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Drug intelligence / Profile preview
VTx-003 is an investigational vectorized antibody gene therapy being developed by VectorY Therapeutics for Huntington's disease. It uses an adeno-associated viral vector to deliver genetic instructions for therapeutic antibody expression in brain cells. The expressed antibody is intended to selectively bind mutant huntingtin and promote its clearance while sparing normal huntingtin. VectorY also describes VTx-003 as targeting TDP-43. ([vectorytx.com](https://www.vectorytx.com/vtx-003-for-huntingtons))
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