Novel Evaluation Systems and Biomarkers for Gene Therapy in Neurodegenerative Diseases and Immunology

Daiichi Sankyo, a global pharmaceutical company, seeks novel evaluation systems and biomarkers to strengthen non-clinical gene therapy studies in neurodegenerative diseases—including ALS and Alzheimer’s disease—and to improve prediction of human immunogenicity arising from intracellularly expressed proteins in gene therapy. The company aims to identify tools with strong translational potential to inform clinical development.

Approaches of interest

• In vitro and in vivo evaluation models for ALS, replicating TDP-43 loss-of-function and gain-of-toxic-function mechanisms
• ALS biomarkers reflecting TDP-43 GoF or LoF mechanisms (e.g., STMN2 or UNC13A mis-splicing) from patient-derived samples
• AD biomarkers that predict post-onset disease progression using patient-derived samples
• Systems that accurately predict human immunogenicity from intracellularly expressed proteins (excluding secreted proteins)
   

Out of scope:

• In silico models
• Approaches involving secreted proteins circulating in blood
  
  Developmental stage of interest:  All research stages are welcome, provided proof-of-concept is demonstrated.

Email Cameron Sargent, CTTC Licensing Officer, at cameron.sargent@vanderbilt.edu for more information.

Please note: Full RFP is attached in the "More Information" section of this page. Faculty and researchers interested in applying for these opportunities based on technologies developed or disclosed at Vanderbilt must submit their proposals through the CTTC.