Novel Technologies for Drug Delivery to Treat Genetic Cardiomyopathy

A pioneering biotechnology company focusing on rare disease therapeutics is exploring innovative methods for delivering drugs to cardiac tissues to treat genetic cardiomyopathy. Solutions should address the following: enhanced site-specific targeting, improved biodistribution profile, sustained and controlled release, and de-risked safety and immunogenicity. Approaches enabling the delivery of antisense oligonucleotides, micro-RNAs, small-interfering RNAs and small-activating RNAs are of the highest priority.

Approaches of Interest:

• Novel delivery mechanisms...