Companies in #Cystic Fibrosis

Arcturus Therapeutics is a messenger RNA medicines company focused on liver and respiratory rare diseases, built on the STARR self-amplifying mRNA platform and the LUNAR lipid-mediated delivery system. Its pipeline includes ARCT-032 (LUNAR-CF, an mRNA therapeutic candidate for cystic fibrosis in Phase 2), ARCT-810 (LUNAR-OTC for ornithine transcarbamylase deficiency, Phase 2), KOSTAIVE (a self-amplifying COVID-19 vaccine approved in Japan), and Phase 1 influenza (ARCT-2138) and pandemic-influenza (ARCT-2304) programs. Headquartered in San Diego, the company beat Q2 2026 earnings estimates by $0.16 per share and traded up 21.9% on August 7, 2026 on above-average volume.

ReCode Therapeutics is a clinical-stage genetic medicines company using its proprietary Selective Organ Targeting (SORT) lipid nanoparticle platform to deliver mRNA and gene correction therapeutics directly to the lungs and other organs beyond the liver. Its lead programs target cystic fibrosis and primary ciliary dyskinesia, rare genetically defined diseases with no existing treatment options.

Vertex Pharmaceuticals is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. Vertex is the leading developer of cystic fibrosis therapies (Trikafta/Kaftrio franchise), brought the first FDA-approved CRISPR-based therapy to market (Casgevy, with CRISPR Therapeutics), and has a late-stage pipeline in acute pain, sickle cell disease, beta thalassemia, and Duchenne muscular dystrophy.