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Nucleic Acid-based Therapeutics Companies

Nucleic acid-based therapeutics involve the use of genetic material, such as DNA or RNA, to treat or prevent diseases. These therapies can target specific genes or cellular processes, offering potential treatments for various medical conditions, including genetic disorders, cancers, and infectious diseases.

Nucleic Acid-based Therapeutics Key Companies


Latest Nucleic Acid-based Therapeutics Companies Update



Nov 2023Moderna (US) Received FDA approval for their second-generation COVID-19 booster vaccine (mRNA-1273.214) offering broader protection against circulating variants.Advanced clinical trials for their mRNA-based therapies for HIV, cytomegalovirus, and various types of cancer.


BioNTech (Germany) Partnered with Genmab to develop mRNA-based cancer vaccines targeting specific tumor antigens.Initiated Phase 1 trials for their mRNA therapy for Duchenne muscular dystrophy.


Alnylam Pharmaceuticals (US) Received FDA approval for their Onpattro® (patisiran) for the treatment of hereditary ATTR amyloidosis, expanding their RNAi therapy portfolio.Announced positive results from Phase 2a trials for their RNAi therapy for alpha-1 antitrypsin deficiency, a genetic liver disease.


Editas Medicine (US) Dosing of the first patient in their Phase 1/2 clinical trial for their CRISPR gene editing therapy for Leber congenital amaurosis, a genetic eye disease.Collaborated with Roche to develop CRISPR-based therapies for various rare genetic diseases.


Wave Life Sciences (US) Filed an IND application with the FDA for their mRNA therapy for Duchenne muscular dystrophy, entering the race for this promising market.Collaborated with Pfizer to develop mRNA-based therapies for rare neuromuscular diseases.


List of Nucleic Acid-based Therapeutics Key Companies in the Market



  • Wave Life Sciences Ltd

  • Copernicus Therapeutics Inc

  • Imugene

  • Caperna

  • Phylogica

  • Protagonist Therapeutics

  • Benitec Biopharma

  • EGEN (Expression Genetics)

  • Benitec Biopharma

  • BioMedica (Oxford BioMedica)

  • Transgene


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