U.S. Antisense and RNAi Therapeutics Market (By Technology: RNA Interference, Antisense RNA; By Application: Ocular, Genetic; By Route of Administration) - Industry Analysis, Size, Share, Growth, Trends, Revenue, Regional Outlook and Forecast 2024-2033

The U.S. antisense and RNAi therapeutics market size was estimated at around USD 2.78 billion in 2023 and it is projected to hit around USD 16.01 billion by 2033, growing at a CAGR of 19.13% from 2024 to 2033.

Key Pointers

• By Route of Administration, the intravenous injections segment held the largest revenue share in 2023.
• By Route of Administration, the subcutaneous injection segment is poised for the fastest segment CAGR between 2024 and 2033.
• By Technology, the antisense technology segment generated the maximum market share of 71% in 2023.
• By Technology, the RNA interference (RNAi) segment is poised for rapid growth from 2024 to 2033.
• By Application, the neurodegenerative disorder segment contributed the largest market share of 71% in 2023.

U.S. Antisense and RNAi Therapeutics Market Growth Factors

The growth of the U.S. antisense and RNAi therapeutics market is driven by an advancement in molecular biology and nucleic acid therapeutics have significantly enhanced the understanding and application of antisense and RNA interference (RNAi) technologies. These scientific breakthroughs have led to the development of more efficient and targeted therapeutic interventions, driving market expansion. Additionally, increasing investment from pharmaceutical companies, biotechnology firms, and government agencies in research and development activities has accelerated the pace of innovation in this sector. Furthermore, the rising prevalence of genetic disorders, cancer, and other chronic diseases underscores the urgent need for novel treatment modalities, thereby fueling market growth. Moreover, favorable regulatory policies and streamlined approval processes for antisense and RNAi therapeutics have facilitated market entry and commercialization, encouraging further investment and development.

U.S. Antisense and RNAi Therapeutics Market Trends:

• Increasing Research and Development: There is a growing emphasis on research and development efforts focused on antisense and RNAi therapeutics, driven by the need for innovative treatment modalities for various diseases.
• Expansion of Clinical Trials: The number of clinical trials exploring antisense and RNAi therapies in diverse therapeutic areas such as oncology, genetic disorders, and infectious diseases is on the rise, reflecting increased interest and investment in this field.
• Advancements in Delivery Systems: Efforts to enhance the delivery of antisense and RNAi therapeutics to target tissues and cells are ongoing, with the development of novel delivery systems aimed at improving efficacy and reducing off-target effects.
• Personalized Medicine Approaches: There is a growing trend towards personalized medicine approaches in the development of antisense and RNAi therapeutics, with a focus on targeting specific genetic mutations or molecular pathways tailored to individual patient profiles.
• Regulatory Advancements: Regulatory agencies such as the FDA are actively engaged in providing guidance and support for the development and approval of antisense and RNAi therapeutics, contributing to a more conducive regulatory environment for market growth.
• Market Expansion Beyond Genetic Disorders: While initially focused on genetic disorders, the application of antisense and RNAi therapeutics is expanding to other therapeutic areas such as cancer, infectious diseases, and neurodegenerative disorders, broadening the market potential.

Route of Administration Insights

In 2023, the intravenous injections segment secured the largest market share, largely due to the favorable pharmacokinetics and high bioavailability associated with this mode of administration. Intravenous injections facilitate swift delivery of antisense and RNAi therapeutics throughout the body by circumventing gastrointestinal and liver barriers. By administering antisense and RNAi therapeutics intravenously, there is enhanced penetration into tumor tissues, enabling selective accumulation within tumor microenvironments. This heightened efficacy against cancer cells minimizes systemic toxicity, thus amplifying the demand for intravenous injections and driving growth within this segment.

Meanwhile, the subcutaneous injection segment is poised for the fastest compound annual growth rate (CAGR) between 2024 and 2033. Offering a convenient and pain-free administration route, subcutaneous injections deliver sustained therapeutic effects. Additionally, this method ensures stability, solubility, and tissue-specific targeting of advanced antisense and RNAi therapeutics, including lipid nanoparticles and polymer-based carriers, in patients.

Technology Insights

In 2023, antisense technology emerged as the dominant force in this market, capturing the highest share at 71%. This technology plays a pivotal role in regulating protein and gene expression, thereby aiding in the treatment of chronic cardiovascular, respiratory, and neurological diseases. The growth of antisense technology is propelled by factors such as increasing investments in healthcare and strategic M&A activities undertaken by key companies. For example, in 2022, Wave Life Sciences Ltd. collaborated with GSK to enhance Wave's PRISM platform through the advancement of RNA-based modalities. Such collaborative efforts are expected to drive market expansion.

Meanwhile, the RNA interference (RNAi) segment is poised for rapid growth from 2024 to 2033. RNAi technology offers a promising mechanism for gene regulation, allowing for the inhibition of disease-causing genes. RNAi is gaining traction as a therapeutic option for various ailments, including viral infections, cancer, and inflammatory disorders. Additionally, the growth of this segment is fueled by government grants and incentives aimed at fostering innovation in the field.

Application Insights

In 2023, the neurodegenerative disorder segment dominated the market with a share of 71%. This growth is primarily driven by the high prevalence of neurodegenerative disorders in the United States, including Alzheimer's disease and Parkinson's disease. According to the National Institute of Environmental Science, approximately 6.2 million U.S. residents were estimated to have Alzheimer's disease in 2022, while about a million people have recently been diagnosed with Parkinson's disease in the U.S. The significant burden posed by these disorders can be alleviated through targeted approaches using antisense and RNAi therapeutics. Therefore, the broad applications of antisense and RNAi technology in treating neurodegenerative disorders are fueling the growth of this segment.

Meanwhile, the genetic disorders segment is projected to grow at a compound annual growth rate (CAGR) of 20.03% from 2024 to 2033, driven by the importance of RNAi therapeutics in treating genetic disorders. RNAi therapy exhibits high efficiency and specificity in treating genetic diseases, effectively targeting disease-specific genetic variants with the assistance of single nucleotide polymorphism. Moreover, advancements in nucleic acid research and targeted delivery mechanisms are expected to accelerate the growth of this segment over the forecast period.

U.S. Antisense and RNAi Therapeutics Market Key Companies

• Sarepta Therapeutics, Inc.
• Ionis Pharmaceuticals, Inc.
• Benitec Biopharma, Inc.
• Arbutus Biopharma
• Alnylam Pharmaceuticals, Inc.
• Microsynth AG
• Aragen Bioscience
• Moderna
• Nutcracker Therapeutics
• Deep Genomics

Recent Developments

In March 2023, Alnylam Pharmaceuticals, Inc. revealed its expansion plans with Medison Pharma to introduce innovative therapies for patients in international markets. This expansion involves broadening their existing partnership to a multi-regional agreement, encompassing Poland, Czech Republic, Hungary, Slovakia, Lithuania, Estonia, and Latvia, in addition to Israel.

U.S. Antisense and RNAi Therapeutics Market Segmentations:

By Technology

• RNA Interference
• Antisense RNA

By Application

• Ocular
• Cardiometabolic & Renal Disorders
• Neurodegenerative Disorders
• Genetic Disorders

By Route of Administration

• Intravenous Injections
• Intrathecal Injections
• Subcutaneous Injections