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Nissan Chemical Corporation, a forerunner in chemical innovations, is seeking novel ASO and siRNA therapeutics for CNS and liver diseases. They are especially interested in therapeutics facing challenges with toxicity, stability, and efficacy, which may leverage their proprietary platform incorporating 2'-O-[2-(N-methylcarbamoyl)ethyl] (MCE)-modified nucleotides to overcome these challenges.
Approaches of interest
- Rare and neurodegenerative CNS diseases (e.g., ALS, inherited ataxias), rare metabolic and liver fibrosis diseases (e.g., MASH, PBC, PSC), and conditions caused by hepatocyte-secreted proteins (e.g., complement-related disorders, hemophilia)
- CNS therapeutics delivered locally (intrathecal or intracerebroventricular injection) or systemically with BBB-penetrating technologies
- Liver therapeutics administered systemically and acting within hepatocytes
- Therapeutics that work through gene silencing are highest priority
- Novel gene and protein targets for diseases of interest
Out of scope
- Ultra-rare CNS and liver diseases (less than 1000 patients globally)
- Therapeutics acting via gene upregulation
- Other nucleic acid therapeutics (aptamers, miRNA)
- Novel chemically modified nucleic acid platforms
Developmental stage of interest: Basic research to late preclinical, provided there is in vivo proof of concept.
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.