Therapeutic Strategies to Address TDP-43 Pathology in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

Our client, a global leader in the creation and commercialization of pioneering therapies for rare genetic diseases, is looking for novel therapeutic approaches targeting TDP-43 pathology. Specifically, the team is interested in strategies that can ameliorate TDP-43 pathology in neurons for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), as well as related diseases. 

Approaches should reduce cytoplasmic inclusions of TDP-43 and increase TDP-43 nuclear function in ALS/FTD disease models. 

Approaches of Interest:

• Strategies that can reduce cytoplasmic inclusions of TDP-43 
• Approaches to increase TDP-43 nuclear function in disease 
• Oligonucleotide and peptide/protein (including antibodies) approaches are of particular interest; however, the team is open to all modalities including small molecule and gene therapy 

Out of Scope:

• Non-TDP-43 related cytoplasmic aggregation (E.g., SOD1 or FUS protein) 
• Biomarkers of disease and diagnostic solutions 

Developmental Stages of Interest:

• Research at any development stage is of interest (early studies through to Phase III and registration), providing there’s strong evidence for amelioration of TDP-43 pathology 
• Opportunities with in vivo validation are of high interest, although opportunities with strong in vitro or ex vivo validation in a relevant model are also within scope 

Submission Information

Submission of one-page, 200–300-word briefs is encouraged, along with any optional supplementary information e.g. relevant publications. The team encourages including the proposed next steps in developing the research towards commercialization. In submitting to this campaign, you confirm that your submission contains only non-confidential information. 

Opportunity for Collaboration

Our client is open to a range of collaboration opportunities, with the most appropriate outcome being decided on a case-by-case basis. Example outcomes include funded research collaborations and agreements, or licencing of assets.