Seeking Transformative Solutions to Treat Rare Skeletal Diseases

BioMarin Pharmaceutical Inc. is a world leader in developing and commercialising first- or best-in-class therapies for rare genetic diseases. BioMarin Pharmaceutical Inc. is looking for expertise in the pathology and epidemiology (highlighting a focus on clear genetic correlations) of rare skeletal diseases, alongside novel approaches for therapeutic intervention and technologies for targeted delivery to various cell types of the bone.

Approaches of Interest

• Therapeutic approaches to treat rare skeletal diseases, particularly but not limited to: osteogenesis imperfecta, Ehlers‑Danlos syndrome, pseudoachondroplasia, osteopetrosis, Stickler syndrome, Spondyloepiphyseal dysplasia congenita, and multiple hereditary exostosis 
• Unique and innovative therapeutic modalities are encouraged
• Open to different modalities including gene correction and editing, cell therapy, viral and non‑viral gene therapy, RNA interference, modified oligonucleotides, small molecules, protein replacement and substitution
• Novel approaches for the targeted delivery to cell types of the bone, including peptides, lipids, glycans, antibodies, aptamers, modified oligonucleotides, nanoparticles, extracellular vesicles, circulating factors, small molecules or delivery devices and routes of administration
• Thorough transcriptomic and/or proteomic data in various cell types of the bone in normal or diseased pathology

Out of Scope

• Approaches targeting oncology, infectious diseases or genetic skeletal muscle disorders are excluded

Developmental Stages of Interest

• Opportunities at basic research phase to Phase I clinical trial are within scope
• Opportunities with in vivo validation are of highest interest, although opportunities with compelling in vitro or in silico validation will still be considered

Submission Information

Submission of one page, 200-300 word briefs are encouraged, along with any optional supplementary information e.g., relevant publications and patents. In submitting to this campaign, you confirm that your submission contains only non-confidential information.

Opportunity for Collaboration

BioMarin Pharmaceutical Inc. is open to a range of collaboration opportunities, with the most appropriate outcome being decided on a case-by-case basis. Example outcomes include licensing assets, project/PhD funding, and research collaborations. Our client is looking to establish long-standing partnerships in the field of rare skeletal disease.