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

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:

• Priority modalities of interest: non‑viral therapeutic approaches, mRNA, oligonucleotides (e.g. RNAi, siRNA, ASO, and saRNA), small molecules, protein replacement and substitution, among other novel approaches.
• Novel approaches to target delivery to...