(MENAFN- GlobeNewsWire - Nasdaq) Key opportunities in the RNA targeting small molecules therapeutics market include growth in RNA splicing modifications, neurodegenerative diseases, drug discovery, and pharma-biotech sectors. Expansion in AI-driven drug discovery, strategic alliances, and emerging markets offers additional potential for growth, especially in the USA.

Dublin, Nov. 12, 2025 (GLOBE NEWSWIRE) -- The "RNA Targeting Small Molecules Therapeutics Global Market Opportunities And Strategies To 2034" report has been added to ResearchAndMarkets's offering.
This report describes and explains the RNA targeting small molecules therapeutics market and covers 2019-2024, termed the historic period, and 2024-2029, 2034F termed the forecast period. The report evaluates the market across each region and for the major economies within each region.
The global RNA targeting small molecules therapeutics market reached a value of nearly $2.77 billion in 2024, having grown at a compound annual growth rate (CAGR) of 31.74% since 2019. The market is expected to grow from $2.77 billion in 2024 to $4.13 billion in 2029 at a rate of 8.28%. The market is then expected to grow at a CAGR of 11.25% from 2029 and reach $7.03 billion in 2034.
Growth in the historic period resulted from increasing incidence of neurological disorders, growing demand for precision medicine, increased healthcare expenditure and expansion of clinical trials and robust therapeutic pipeline. Factors that negatively affected growth in the historic period were the high cost of drug development and complexity of RNA-based drug discovery.
Going forward, the government initiatives for research and development in healthcare, venture capital and pharma investment in RNA, increasing incidence of genetic disorders and rising prevalence of cancer support will drive growth. Factors that could hinder the growth of the RNA targeting small molecules therapeutics market in the future include instability and degradation of RNA targets, off-target effects and toxicity risks and trade war and tariffs.
The RNA targeting small molecules therapeutics market is segmented by offering into mRNA (Messenger Ribonucleic Acid) translation modulators, RNA (Ribonucleic Acid) splicing modification, direct RNA (Ribonucleic Acid) targeting and other offerings. The RNA (Ribonucleic Acid) splicing modification market was the largest segment of the RNA targeting small molecules therapeutics market segmented by offering, accounting for 66.76% or $1.85 billion of the total in 2024. Going forward, the RNA (Ribonucleic Acid) splicing modification segment is expected to be the fastest growing segment in the RNA targeting small molecules therapeutics market segmented by offering, at a CAGR of 7.06% during 2024-2029.
The RNA targeting small molecules therapeutics market is segmented by therapeutic indication into lung fibrosis, cancer, neurodegenerative diseases, autoimmune, inflammatory and other therapeutic indications. The neurodegenerative diseases market was the largest segment of the RNA targeting small molecules therapeutics market segmented by therapeutic indication, accounting for 66.76% or $1.85 billion of the total in 2024. Going forward, the neurodegenerative diseases segment is expected to be the fastest growing segment in the RNA targeting small molecules therapeutics market segmented by therapeutic indication, at a CAGR of 6.91% during 2024-2029.
The RNA targeting small molecules therapeutics market is segmented by application into drug discovery, oncology research and disease identification. The drug discovery market was the largest segment of the RNA targeting small molecules therapeutics market segmented by application, accounting for 56.36% or $1.56 billion of the total in 2024. Going forward, the disease identification segment is expected to be the fastest growing segment in the RNA targeting small molecules therapeutics market segmented by application, at a CAGR of 12.50% during 2024-2029.
The RNA targeting small molecules therapeutics market is segmented by end user into hospitals, research laboratories, pharmaceutical and biotechnology companies and other end-users. The pharmaceutical and biotechnology companies market was the largest segment of the RNA targeting small molecules therapeutics market segmented by end user, accounting for 55.39% or $1.53 billion of the total in 2024. Going forward, the research laboratories segment is expected to be the fastest growing segment in the RNA targeting small molecules therapeutics market segmented by end user, at a CAGR of 8.57% during 2024-2029.
North America was the largest region in the RNA targeting small molecules therapeutics market, accounting for 43.99% or $1.22 billion of the total in 2024. It was followed by Western Europe, Asia-Pacific and then the other regions. Going forward, the fastest-growing regions in the RNA targeting small molecules therapeutics market will be Africa and Eastern Europe where growth will be at CAGRs of 11.60% and 10.56% respectively. These will be followed by Middle East and South America where the markets are expected to grow at CAGRs of 10.44% and 10.01% respectively.
The global RNA targeting small molecule therapeutics market is highly concentrated, with large players operating in the market. The top 6 competitors in the market made up 68.49% of the total market in 2024. F.Hoffmann-La Roche Ltd. (Genentech) was the largest competitor with a 66.76% share of the market, followed by Teva Pharmaceuticals with 0.60%, Cipla Limited, with 0.41%, Dr. Reddy's Laboratories, with 0.28%, Zydus Lifesciences (formerly Cadila Healthcare) with 0.27%, and Apotex Inc. with 0.17%.
The top opportunities in the RNA targeting small molecules therapeutics market segmented by offering will arise in the RNA (Ribonucleic Acid) splicing modification segment, which will gain $752.88 million of global annual sales by 2029. The top opportunities in the RNA targeting small molecules therapeutics market segmented by therapeutic indication will arise in the neurodegenerative diseases segment, which will gain $734.93 million of global annual sales by 2029. The top opportunities in the RNA targeting small molecules therapeutics market segmented by application will arise in the drug discovery segment, which will gain $682.43 million of global annual sales by 2029. The top opportunities in the RNA targeting small molecules therapeutics market segmented by end user will arise in the pharmaceutical and biotechnology companies segment, which will gain $763.8 million of global annual sales by 2029. The RNA targeting small molecules therapeutics market size will gain the most in the USA at $482.45 million.
Market-trend-based strategies for the RNA targeting small molecules therapeutics market include AI-powered deep learning revolutionizes RNA-targeted small molecule drug discovery, strategic alliance to develop RNA splicing modulators for challenging diseases, strategic collaboration and partnership fueling RNA targeting small molecules therapeutics, focus on startup-driven AI solutions for RNA-targeted therapeutics and use of artificial intelligence (AI) to enhance efficiency of RNA targeting small molecules.
Player-adopted strategies in the RNA targeting small molecules therapeutics market include focus on expanding its business capabilities through collaborations to expand its operational capabilities and business capabilities through innovation, improving operational efficiency, and expanding its product portfolio.
To take advantage of the opportunities, the analyst recommends the RNA targeting small molecules therapeutics companies to focus on advancing ai-driven tools for rna-targeted drug discovery, focus on advancing ai-driven tools for rna-targeted drug discovery, focus on ai integration to enhance rna small molecule discovery, focus on mrna translation modulators and other high growth segments, focus on cancer as the fastest growing therapeutic segment, expand in emerging markets, continue to focus on developed markets, focus on strategic alliances to accelerate rna therapeutics development, focus on strategic collaborations to strengthen rna therapeutics development, focus on competitive and value-based pricing strategies, focus on building credibility through scientific and clinical communication, focus on targeted outreach and stakeholder-specific messaging, focus on disease identification as the fastest growing market segment, focus on pharmaceutical and biotechnology companies as the fastest growing segment.
Major Market Trends

• AI-Powered Deep Learning Revolutionizes RNA-Targeted Small Molecule Drug Discovery
• Strategic Alliance To Develop RNA Splicing Modulators For Challenging Diseases
• Strategic Collaboration And Partnership Fueling RNA Targeting Small Molecules Therapeutics
• Focus on Startup-Driven AI Solutions for RNA-Targeted Therapeutics
• Use Of Artificial Intelligence (AI) To Enhance Efficiency Of RNA Targeting Small Molecules

Competitive Landscape And Company Profiles

• F.Hoffmann-La Roche Ltd. (Genentech)
• Teva Pharmaceuticals
• Cipla Limited
• Dr. Reddy's Laboratories Ltd
• Zydus Lifesciences (formerly Cadila Healthcare)

Other Major And Innovative Companies

• Apotex Inc.
• Skyhawk Therapeutics
• STORM Therapeutics
• Arrakis Therapeutics Inc.
• Anima Biotech Inc.
• Ribometrix Inc.
• Accent Therapeutics Inc.
• Promega Corporation
• Bayer AG
• Arrowhead Pharmaceuticals Inc.
• Ionis Pharmaceuticals Inc.
• Alnylam Pharmaceuticals
• Abivax SA
• Remix Therapeutics
• Therapeutics Inc.

Key Mergers And Acquisitions

• Novartis Acquired Regulus Therapeutics Inc.

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