Companies in #Rare Disease

City Therapeutics is a clinical-stage biotechnology company pioneering next-generation RNA interference (RNAi) medicines, founded by John Maraganore (former CEO of Alnylam Pharmaceuticals) and a team of RNAi pioneers. The company's lead platform is based on cleavage-inducing tiny guide RNAs (cityRNAs) designed to improve potency and durability over first-generation siRNA therapeutics, with three programs in the pipeline targeting cardiometabolic and rare diseases.

Eli Lilly and Company is a major global pharmaceutical company with a broad portfolio spanning diabetes/obesity, oncology, neuroscience, immunology, and rare diseases. Its GLP-1 franchise (Mounjaro/tirzepatide, Zepbound) and the oral GLP-1 candidate orforglipron are reshaping obesity care. Lilly has been the most aggressive large-pharma dealmaker of the past decade, completing more biotech buyouts than any other top-12 pharma by revenue, with 10 acquisitions in 2026 alone accounting for roughly half of pharma's total M&A capacity (~$25B+ committed).

Ipsen is a global specialty biopharmaceutical company focused on oncology, rare disease, and neuroscience, with a portfolio of in-market products including Dysport (botulinum toxin), Somatuline (lanreotide), and Onivyde (irinotecan). The company is advancing a refreshed R&D pipeline in 2026 with multiple Phase 3 readouts planned, alongside a new wave of partnership-led discovery deals.