K013 Working together to address translational challenges for delivering advanced therapy medicinal products (ATMPs) in Huntington’s disease

<h3>Background</h3> Many potential disease-modifying treatments for Huntington’s Disease (HD) are advanced therapy medicinal products (ATMPs), ie they are based on genes, cells or tissues. Currently, most ATMPS must be delivered directly to the brain, because they do not cross the blood brain barrier or are best delivered to a restricted brain locality. Thus, clinical trials of ATMPs present various challenges along the translational pipeline, many of which are common regardless of the specific therapeutic product. Through working together, we anticipate addressing these challenges more efficiently and comprehensively. <h3>Aims</h3> To identify challenges to clinical translation and suggest pathways forwards. <h3>Methods/Results</h3> The EHDN Advanced Therapies Working Group (ATWG) and Stem Cells for HD (SC4HD) work together and comprise academics and industrial partners from 3 continents and multiple countries. Together, they aim to consider barriers to clinical translation, provide guidance and guidelines, and generate a robust clinical development plan. Over a series of in-person and virtual meetings, ATWG/SC4HD produced a consensus white paper identifying the challenges of cell therapy for HD (Rosser et al Brain 2022 Jun 3;145(5):1584-1597) and has proceeded to set up task forces to address neurosurgical, preclinical, manufacturing and clinical trial constraints. <h3>Conclusions</h3> Here we highlight challenges associated with the clinical translation of advanced therapies for HD and have set up task forces as outlined above. This provides the starting point for developing guidance and highlighting future directions, with the aim to expedite progress towards advanced therapies for clinical benefit in HD.