Novel Technologies for Drug Delivery to Treat Genetic Cardiomyopathy

A pioneering biotechnology company specializing in rare disease therapeutics seeks innovative methods for delivering drugs to cardiac tissues to treat genetic cardiomyopathy. They aim to enhance site-specific targeting, improve biodistribution, and achieve sustained and controlled release while minimizing safety risks and immunogenicity.

 Approaches of interest:

• Novel delivery mechanisms targeting all cardiac tissue cell types
• Advanced targeting molecules for precise cardiac tissue delivery
• Delivery systems minimizing off-target effects on liver and kidneys
• Vehicles for sustained and controlled release of oligonucleotides
• Strategies to reduce toxicity and immune responses of oligonucleotide drugs

 Out of scope:

• AAV and other viral delivery approaches
• Cell therapy approaches
• Delivery approaches targeting non-cardiac tissues

 Developmental stage of interest: Preclinical research and beyond with established proof of concept data in cardiac cell types. In vivo data prioritized, but strong in vitro proof of concept will be considered.