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US Nucleic Acid Therapeutics Companies

ID: MRFR/HC/11123-HCR
128 Pages
Rahul Gotadki
October 2025

Companies in the United States specializing in nucleic acid therapeutics focus on developing and commercializing therapies based on nucleic acids, including RNA and DNA, for treating various diseases. These therapeutics have gained attention for their potential in addressing genetic disorders, infectious diseases, and various types of cancers. 

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Top Industry Leaders in the US Nucleic Acid Therapeutics Market

US nucleic acid therapeutics Key Companies


 

Latest US Nucleic Acid Therapeutics Companies Update



Alnylam snaps up Akcea Therapeutics $5.3 billion acquisition in December 2023 strengthens Alnylam's leadership in RNA interference (RNAi) therapies, expanding their pipeline with Akcea's innovative drugs like Onpattro for hereditary ATTR amyloidosis.


Moderna partners with Vertex for cystic fibrosis In a bold collaboration, these two giants join forces to develop mRNA therapies for cystic fibrosis, leveraging Moderna's platform and Vertex's deep understanding of the disease.


Arrowhead Pharmaceuticals' ARO-AAT reaches Phase 3 for alpha-1 antitrypsin deficiency potential breakthrough therapy utilizing RNAi technology progresses towards larger-scale trials, offering hope for patients with this rare genetic condition.


Wave Life Sciences' SPLINTER trial yields positive Phase 1b results for Duchenne muscular dystrophy encouraging early data paves the way for further development of their exon skipping therapy, potentially improving muscle function and mobility for DMD patients.


List of US Nucleic Acid Therapeutics Key Companies in the Market



  • Benitec Biopharma

  • Sarpeta Therapeutics

  • NOVARTIS AG.

  • Agilent Technologies

  • Protagonist Therapeutics

  • Qaigen NV

  • Genzyme Sanofi

  • Thermo Fisher Scientific

  • Copernicus

  • Arbutus Biopharma

  • Merck KGaA

  • Silence Therapeutics