The global RNA targeted therapeutics market size is expected to be worth USD 1.81 Billion in 2025. It can reach a valuation of USD 15.10 Billion by 2032. The market is anticipated to peak at a CAGR of 35.4% during the forecast period (2025-2032).  

The rise in cancer cases and the acceptance of RNA therapies for major diseases can drive the market growth significantly. The large scope for RNA targeted therapeutics and a surge in clinical trials for targeting cardiometabolic disorders can bode well for the market.

However, the high development and commercialization costs for RNA targeted therapeutics can impede the market growth.

Key Market Insights

The RNA targeted therapeutics market is anticipated to expand owing to novel launches in mRNA vaccines, applications in oncology, and incentives for fueling innovation for a large patient pool.

• By type of target molecule, the CDKL5 segment is expected to score significant share in 2025. This can be attributed to the advances in antisense oligonucleotides (ASOs) and the silencing of mutant CDKL5 transcripts in preclinical models. On June 27, 2025, UCB’s Fintepla recently passed the phase III trial for the treatment of epilepsy disorders.
• By type of approach, the mRNA translation modulation segment is anticipated to attain robust share in 2025. The segment growth is driven by advances in liquid nanoparticles-delivered oligonucleotides and the research into protein production in cells.
• By region, Asia Pacific is projected to capture a significant market share in 2025 owing to the investments in biotechnology startups and collaborations between research institutes and companies. The emphasis on precision medicine and improvements in the production facilities of manufacturers can drive the growth of the RNA targeted therapeutics market.

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RNA Targeted Therapeutics Market Report Coverage

Market Dynamics

The RNA targeted therapeutics market benefited from the COVID-19 pandemic. The development of RNA vaccines for the fight against various strains of COVID-19 vouched for its efficacy and safety. This gave a boost to common types of RNA, namely ASOs, messenger RNA (mRNAs), and small-interfering RNA (siRNAs).

A surge in mergers and acquisitions and investments in RNA therapies bolstered the market growth effectively. In 2021, Pfizer spent more than USD 600 million on the acquisition of Trillium Therapeutics. Furthermore, the use of RNA interference (RNAi) therapeutics for the effective treatment of advanced cancer created growth opportunities for the market.

Market Opportunity: Popularity of mRNA Therapies to Boost Market Demand

The rise of mRNA vaccines due to collaborative efforts between biopharmaceutical companies and startups can drive the market demand significantly. Investments by venture capital firms and government agencies to attain vaccines against newer virus strains and oncological disorders can facilitate the RNA targeted therapeutics market. The Native Antigen Company generated aptamers on February 10, 2025, using Systematic Evolution of Ligands by EXponential enrichment (SELEX) with virus-like particles (VLPs) against the chikungunya virus.

Market Challenge: Pushback against Orphan Drugs Protection in Europe

The future introduction of Joint Clinical Assessment (JCA) in Europe for oncology and advanced therapy medicinal products (ATMPs) starting in 2025 and orphan products starting in 2028 can impact the protection enjoyed by orphan drugs. Orphan drugs, produced on a small budget and targeted for rare diseases, are expected to undergo various policy reforms as they become viable by the masses. The pushback by drug manufacturers against the leniency towards their evaluation and faster approval can lead to fewer approvals. The loss of market exclusivity and data protection can negatively impact the RNA targeted therapeutics market growth globally.

Analyst’s View

• The shift towards small molecule RNA binders and the development of small-interfering RNA (siRNA) for the treatment of cardiometabolic disorders can drive the RNA targeted therapeutics market growth over the forecast period.
• The CDKL5 segment is predicted to perform exceptionally in 2025 owing to the rise in central nervous system (CNS) disorders.
• Companies are focusing their efforts on making RNA targeted therapeutics commercially available and targeted at mainstream diseases.

Recent Developments

Novartis AG acquired Regulus Therapeutics on June 25, 2025, for USD 1.7 billion. The acquisition can fuel the development of farabursen, a microRNA-17 inhibitor for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

Competitor Insights

• STORM Therapeutics
• Eloxx Pharmaceuticals
• PTC Therapeutics
• Abivax
• Arrakis Therapeutics
• AC Immune
• eFFECTOR Therapeutics
• Skyhawk Therapeutics
• H3 Biomedicine
• Ribometrix

Market Segmentation

• By Type of Target Molecule
  • Cap Binding Complex
  • CDKL5
  • Dystrophin Protein
  • Erα
  • G542X
  • MNK1/MNK2
  • PAX6 gene
  • SMN2
• By Type of Approach
  • Indirect RNA Targeting: Epitranscriptomics
  • mRNA Translation Modulation
  • RNA Splicing Modulation

Regional Insights

• North America
  • U.S.
  • Canada
• Latin America
  • Brazil
  • Argentina
  • Mexico
  • Rest of Latin America
• Europe
  • Italy
  • Germany
  • U.K.
  • Spain
  • Russia
  • France
  • Rest of Europe
• Asia Pacific
  • South Korea
  • India
  • China
  • Japan
  • Australia
  • ASEAN
  • Rest of Asia Pacific
• Middle East
  • GCC Countries
  • Israel
  • Rest of Middle East
• Africa
  • South Africa
  • North Africa
  • Central Africa