Industries

Rare Disease Therapeutics

Rare disease therapeutics address conditions affecting fewer than 200,000 patients using gene therapy, enzyme replacement, and RNA modulation. Orphan drug designation and premium pricing support returns despite small patient pools, making this industry highly motivated to partner with academic researchers who own rare disease cohorts, biobanks, and foundational mechanistic IP.

Details

Companies

Alexion AstraZenecaSarepta TherapeuticsBioMarin PharmaceuticalUltragenyxGenzyme SanofiHorizon TherapeuticsAmicus TherapeuticsRhythm PharmaceuticalsEntrada TherapeuticsPraxis Precision Medicine

Market Size

$230B

Target Researchers

Molecular geneticists, enzymologists, and translational researchers specializing in enzyme replacement therapy, gene therapy, antisense oligonucleotides, substrate reduction, and natural history characterization of ultra-rare conditions

Use Cases

Orphan gene therapy IP licensingASO chemistry platform accessDisease natural history research partnershipsPatient registry collaborationBiomarker development for IND-enabling workERT enzyme manufacturing partnerships