Novel Strategies for Targeted Drug Delivery to CNS, Bone, Cartilage, Skeletal Muscle and Kidney Tissues

Our client, a global leader in the rare disease space, is seeking novel strategies for tissue-specific drug delivery to the central nervous system (CNS), bone, connective tissue/cartilage, skeletal muscle, kidney and heart tissues. Key target cell types include (but are not limited to): CNS – neurons, microglia and astrocytes, muscle – myofibers and satellite cells, kidney – podocytes and tubular epithelial cells, bone and cartilage – mesenchymal stem cells, osteo- and chondro- progenitor cells and osteoblasts. 

Approaches should be capable of delivering one or more of: viral and non-viral nanoparticles, oligonucleotides, mRNAs, biologics. Delivery of nucleic acids, proteins and antibodies are a priority. Technologies and approaches which can be applied to multiple/existing drug molecules, payloads or sites are of high interest. Submissions should outline how the approach can achieve targeting one of the listed tissues. The team is also interested in approaches that achieve improved/selective biodistribution profile, sustained release, increased efficacy or therapeutic index, and/or reduced toxicity. 

Approaches of Interest:

• Targeting molecules E.g., peptides, lipids, glycans, antibodies and fragments thereof, oligonucleotides, small molecules 
• Advanced chemistries E.g., click chemistry, linker chemistries for drug conjugates, biodegradable or stimuli-responsive prodrug chemistries 
• Delivery vehicles or formulations E.g., drug nanocarriers (inorganic, polymer, and lipid nanoparticles), exosomes, liposomes, in situ gels, micelles, micro fluids, dendrimers, emulsion particles, magnetic particles, microbubbles 
• Novel delivery strategies or devices E.g., oral and injectable formulations, magnetic and microelectromechanical systems, implantable drug delivery devices 

Out of Scope: Cell-therapy based solutions, and approaches for oncology or infectious diseases. 

Developmental Stages of Interest:

• Opportunities from early studies to phase III are within scope 
• Opportunities should have in vitro validation of targeted delivery to one of the specified tissues. Approaches with in vivo validation in large animal models are of highest interest 

Submission Information and Opportunity for Collaboration

Submission of one-page, 200–300-word briefs is encouraged, along with any optional supplementary information e.g. relevant publications. The team encourages including any proof-of-concept data, along with your proposed next steps in developing the research towards commercialization. In submitting to this campaign, you confirm that your submission contains only non-confidential information. 

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.