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 of therapeutics for cardiomyopathy targeting all cell types within cardiac tissues 
• Advanced targeting molecules (e.g., peptides, lipids, glycans, antibodies, exosomes, polymer, and lipid nanoparticles) to precisely target cardiac tissues 
• Delivery systems that selectively target the heart while minimizing off-target effects on the liver and kidneys
• Delivery vehicles and formulations for sustained and controlled release of oligonucleotides 
• Safety and reduced immunogenicity of oligonucleotide drugs by employing strategies that minimize toxicity and immune responses 
• Targeted delivery of antisense oligonucleotides, micro-RNAs, small-interfering RNAs and small-activating RNAs to cardiac tissues 
• Novel approaches to improve the delivery of existing therapeutics to cardiac tissues

Out of Scope: 

• AAV and other viral delivery approaches 
• Cell therapy approaches 
• Delivery approaches targeting tissues other than the heart 

Developmental Stages of Interest: 

• Opportunities from preclinical research onwards which have established proof of concept data in cardiac cell types
• Opportunities with in vivo data will be prioritized, although approaches with a strong in vitro proof of concept will also be considered 

Submission Information:

Submission of 200–300-word briefs is encouraged, along with any supplementary information e.g. relevant publications, patents or slide decks. 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.