MDS 2021 | Update: novel disease-modifying treatments in development for Huntington’s disease

Anne Rosser, PhD, FRCP, Cardiff University, Cardiff, UK, outlines novel strategies under investigation for the treatment of Huntington’s disease, including gene therapy and oral medications. AMT-130, a gene therapy intended to modify the disease course in Huntington’s, is currently being assessed in a Phase I/II, placebo-controlled, proof-of-concept study (NCT04120493). This treatment involves the delivery of microRNA via an adeno-associated virus (AAV) vector to silence the huntingtin gene and potentially toxic exon 1 protein fragment. Another dosing study investigating AMT-130 will also be launching in Europe towards the end of 2021. Prof. Rosser further discusses oral medications that are under development for patients with Huntington’s disease, including splicing agents. This interview took place during the 2021 International Congress of Parkinson’s Disease and Movement Disorders.