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Novel Therapeutic Approaches for Genetic Skeletal Diseases and Targeted Delivery to Bone

Background

A leading company in rare disease therapeutics is seeking novel treatments for rare genetic skeletal diseases and genetically defined subsets of common skeletal diseases. They are also interested in delivery techniques to target bone tissues, ideally coupled with novel therapeutics.

Approaches of interest:

  • Non-viral therapeutic approaches (mRNA, oligonucleotides)
  • Small molecules, protein replacement and substitution
  • Novel bone-targeting delivery methods (peptides, lipids, glycans, antibodies, etc.)
  • Combination of novel therapeutics with bone-targeting mechanisms

Out of scope:

  • Approaches for skeletal oncology, infectious disease, or immunology
  • Drug discovery platforms and cell therapy approaches

The company is interested in opportunities from preclinical research onwards, with highest interest in those with in vivo validation and nearing IND readiness. Strong in vitro proof of concept data will also be considered. Collaboration opportunities include funded research collaborations and licensing of assets.

Email Cameron Sargent, CTTC Licensing Officer for Biotechnology and Health Science Licensing, at Cameron.Sargent@Vanderbilt.edu for more information.