Alnylam Pharmaceuticals

Dicerna Pharmaceuticals

This biopharmaceutical company develops ribonucleic acid interference therapeutics to treat rare, chronic, and infectious diseases for healthcare providers and global pharmaceutical partners.

Ultragenyx

This biopharmaceutical company develops and commercialises therapies for patients with serious rare and ultra-rare genetic diseases.

Dicerna

This biotechnology firm develops ribonucleic acid interference therapies for patients with rare genetic, cardiovascular and liver diseases through a proprietary gene-silencing technology platform.

Airna

This company develops RNA editing therapeutics to treat patients with rare genetic and common cardiometabolic diseases.

Mirum Pharmaceuticals

This biopharmaceutical company develops and commercialises therapies for rare liver diseases, primarily treating children and adults with cholestatic and metabolic conditions.

ADARx Pharmaceuticals

This clinical-stage biotechnology company develops RNA-targeting therapeutics for patients with genetic, cardiometabolic, complement-mediated, and central nervous system diseases.

Isis Pharmaceuticals

This biotechnology firm develops antisense medicines to treat patients with rare and cardiovascular diseases by targeting RNA-based genetic pathways.

Skyhawk Therapeutics

This company develops small molecules that modulate RNA for patients with rare neurological, oncological, and autoimmune diseases.