AMT-150

AMT-150 is an investigational gene therapy candidate developed by uniQure for the treatment of Spinocerebellar Ataxia Type 3 (SCA3), also known as Machado-Joseph disease. It utilizes uniQure's proprietary miQURE™ gene-silencing platform to deliver a microRNA (miRNA) via an adeno-associated virus serotype 5 (AAV5) vector. The therapy is designed to target and degrade the messenger RNA (mRNA) of the *ATXN3* gene, thereby reducing the expression of the mutant ataxin-3 protein. The accumulation of this toxic protein is the underlying cause of the progressive neurodegeneration and motor impairment characteristic of SCA3. By lowering the levels of mutant ataxin-3 throughout the central nervous system, AMT-150 aims to halt or potentially reverse the progression of the disease. It is currently in the preclinical stage of development.