AC Immune SA, a Swiss-based, clinical-stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases, today announced a research partnership with leading scientists in the Perelman School of Medicine at the University of Pennsylvania (Penn) focused on studying the pathological mechanisms of TDP-43 misfolding and aggregation.
TDP-43, the transactive response (TAR) DNA binding protein, is a transcription factor found in most human tissues. It is a recently identified target of growing interest for neuroOrphan indications such as frontotemporal lobar degeneration (FTLD) and amyotrophic lateral sclerosis (ALS). It also plays an important role in other significant neurodegenerative indications such as Alzheimer’s disease (AD).
The collaboration is aimed at deciphering the governing principles of how pathological forms of TDP-43 spread from neuron to neuron, leading to a better understanding of the TDP-43 pathologies in order to support the Company’s mission to develop novel therapeutic and diagnostic approaches against FTLD and other neurodegenerative diseases.
AC Immune will contribute a two-year research grant to the laboratory of Dr. John Trojanowski and Dr. Virginia M.-Y. Lee. Dr. Trojanowski’s research focuses on neurodegeneration and has led to the novel view that major disease-related proteins including Tau, alpha-synuclein and TDP-43, are co-deposited in most neurodegenerative diseases and how these copathologies impact the clinical representation of these diseases. Dr. Lee is a world-renowned leader in research on Tau, alpha-synuclein and the Abeta precursor protein, studying their pathobiological roles in neurodegenerative diseases.
Prof. Andrea Pfeifer, CEO of AC Immune SA, commented:
“We are very proud to be working with Penn and Dr. Trojanowski and Dr. Lee, two very well-respected experts in neurodegenerative diseases. The field is still investigating the precise causes of neurodegenerative diseases and how to prevent, treat and even cure them, including neuroOrphan indications such as FTLD. Increasing our knowledge of the role of TDP-43 in disease pathology will mark valuable progress in this effort. AC Immune has generated several antibodies with unique binding profiles to TDP-43, and we are looking forward to results from preclinical proof-of-concept studies in Q3 2019.”