Antisense and RNAi Therapeutics Market

Market Overview

The global antisense and RNAi therapeutics market size was valued at USD 4.7 billion in 2023 and is projected to reach USD 19.8 billion by 2032, registering a CAGR of 17.4% during the forecast period (2024-2032). The increased therapeutic applications, the need for focused medicines, and technological improvements propel the growth of the antisense and RNAi therapeutics market.

Antisense and RNA interference (RNAi) treatments function by suppressing the activity of disease-related genes and have demonstrated significant promise in treating several medical ailments. Antisense therapy involves using synthetic nucleic acids known as antisense oligonucleotides (ASOs) to specifically bind to and regulate mRNA, suppressing the expression of target genes. ASOs can hinder the process of transcription, RNA processing, or transport, as well as translation. Antisense therapy has the primary benefit of enabling accurate suppression of genes essential to the condition. 

Additionally, advancements in nucleic acid technologies have created more effective and accurate medicinal treatments, which has stimulated the market's growth. In addition, the growing research and development efforts in this field have revealed novel therapeutic targets and broadened the therapeutic uses of antisense and RNAi technologies. Moreover, the increasing need for precise treatments with minimal adverse effects has led to the widespread use of antisense and RNAi medicines in other fields of medicine, such as oncology, infectious illnesses, genetic disorders, and neurological disorders. 

Highlights

• Antisense RNA generates the highest revenue share based on technology.
• Pulmonary delivery contributes significantly to the market through the route of administration.
• Neurodegenerative disorders account for the largest market share by application.

Market Dynamics

Global Antisense and RNAi Therapeutics Market Drivers

Technological Advancements in Drug Delivery Systems

One of the primary catalysts is the ongoing advancements in drug delivery techniques. These methods enable the targeted delivery of RNA molecules to specific cells in a manner that minimizes degradation, resulting in improved therapeutic effectiveness. For instance, the use of lipid nanoparticles and conjugate technology has made it possible to significantly enhance the cellular absorption and equilibrium of antisense oligonucleotides or siRNA, which were previously considered insignificant. It improves the efficiency of delivering treatments and boosts specificity in targeting specific human material, which is crucial in treating cancer, disorders, and viral infections, among other conditions.

Additionally, the market prognosis for antisense and RNAi therapies suggests that these treatments will attract significant research focus and financial backing. In July 2023, Novartis purchased DTx Pharma, a biotechnology startup specializing in short-interfering RNA therapy for neurological conditions. Moreover, these acquisitions are generating a favorable market outlook.

Increasing Prominence of Genetic Disorders and Chronic Diseases

The increasing prevalence of hereditary and chronic disorders among the general population strongly contributes to the growth of the antisense and RNAi therapies industry. Prevalent disorders such as Huntington's disease, some types of cancer, and several hereditary genetic conditions lack effective treatments, resulting in a demand for antisense and RNAi medications. The Orphanet database provides epidemiological data on 6172 distinct rare diseases, with 71.9% being hereditary and 69.9% exclusively affecting children. Out of the 5304 diseases categorized based on point prevalence, 84.5% of the diseases analyzed had a point prevalence rate of less than 1 in 1,000,000. Approximately 77.3-80.7% of the total population affected by rare diseases can be attributed to only 4.2% (n=149) of the diseases with a prevalence range of 1-5 cases per 10,000 people. 

In addition to this, Antisense and RNAi therapeutic approaches offer a novel approach as repressors of putative disease-predisposing genes. On one side, novel medications have the highest likelihood of being incorporated into clinical practice. Furthermore, due to their capacity to selectively target particular genes, they offer a novel therapeutic approach for patients suffering from illnesses that cannot be effectively treated with traditional pharmaceutical techniques. Consequently, there is a distinct requirement for ongoing investment in novel biopharmaceutical research and development initiatives.

Global Antisense and RNAi Therapeutics Market Restraints

High Manufacturing Cost of RNA Therapeutics

A significant obstacle hindering the market's quick growth is the substantial expense of producing RNA therapies, such as antisense oligonucleotides and siRNA molecules. The synthesis of these sequence-specific nucleic acid medicines is intricate and costly. The production method is capital-demanding due to the need for specialized equipment and multi-step chemical synthesis. Establishing a solid infrastructure for large-scale production of RNA therapeutics requires companies to invest significant capital. This significantly contributes to the expenses associated with drug development.

Moreover, variations in production and the presence of contaminants can substantially affect the financial aspects of manufacturing. The exorbitant expenses ultimately lead to an escalation in the prices of drugs, so diminishing their affordability and market adoption to a certain degree. Cost obstacles in manufacturing continue to be significant limitations for the antisense and RNAi medicines business.

Global Antisense and RNAi Therapeutics Market Opportunity

Supportive Regulatory Environment For Novel Therapies

The favorable regulatory environment drives the demand for antisense and RNAi treatments. In October 2023, the Food and Drug Administration's Office of Orphan Products Development stated that monies would be provided for fiscal year (FY) 2024 to support the expenses associated with undertaking natural history studies on rare diseases and conditions. This Request for Applications (RFA) aims to promote scientific rigor and collaboration to benefit people who suffer from uncommon diseases or conditions.

Furthermore, international health regulatory bodies, such as the FDA and EMA, are implementing expedited evaluation procedures for novel treatments, including RNAi and antisense technology. Furthermore, regulatory incentives such as orphan drug status, rapid approval, and priority review vouchers have become increasingly significant since they provide pharmaceutical companies with additional motivation to invest in advancing antisense and RNAi medicines.

Regional Analysis

The global antisense and RNAi therapeutics market analysis is conducted across North America, Europe, Asia-Pacific, the Middle East and Africa, and Latin America.

North America Dominates the Global Market

North America is the most significant global antisense and RNAi therapeutics market shareholder and is estimated to grow at a CAGR of 17.2% over the forecast period.  Multiple RNA interference (RNAi) medicines are now being developed in the United States. Moreover, most businesses in the biotechnology industry have invested significantly in developing RNAi therapies. The region's prominent pharmaceutical and biotechnology sectors create diverse platforms for this industry. In May 2023, Codexis, Inc., a U.S.-based company, launched a technology platform called Enzyme-Catalyzed Oligonucleotide (ECO) Synthesis for producing RNA oligonucleotide medicines on a massive scale. This technology was introduced during the TIDES U.S. annual conference.

Furthermore, the North American market is anticipated to grow in the upcoming years due to technological advancements in the United States. Antisense and RNA interference (RNAi) treatments, vital elements of CRISPR-based systems, have been widely utilized in research and have the potential for therapeutic interventions. Major participants in the antisense and RNAi therapies industry include enterprises in gene editing technologies, biotechnology, and life sciences. Anticipated growth in product authorizations inside the nation is projected to drive up the market for antisense and RNAi therapies targeting genes linked to various genetic disorders. In 2023, the FDA approved Ionis Pharmaceuticals's medication Wainua for treating transthyretin-related hereditary amyloidosis.

Europe is anticipated to exhibit a CAGR of 17.8% over the forecast period. Regional expansion is expected to be driven by multiple companies specializing in antisense and RNAi medicines. Furthermore, these organizations are implementing other initiatives to broaden their range of products and business opportunities. In March 2023, SciNeuro Pharmaceuticals, a U.S.-based firm, formed a collaboration with Secarna Pharmaceuticals GmbH & Co., which is situated in Europe. KG aims to develop innovative antisense oligonucleotide (ASO) treatments targeting critical factors involved in Central Nervous System disorders. Consequently, the growing attention from companies operating in different countries towards the European market is anticipated to enhance the region's expansion over the projected timeframe.

The Asia-Pacific market is the fastest-growing region. The Asia Pacific market is expanding primarily because countries such as China, Japan, and India are quickly improving their generic medication manufacturing capabilities and promoting domestic innovation. Multiple indigenous biotechnology startups are investigating RNA interference (RNAi) technologies to produce therapeutic interventions. Compared to Western markets, the relatively lower production costs empower these participants to focus on providing medicines at accessible pricing. Due to this advantage, global pharmaceutical leaders are investing significant money to extend their presence in different regions.

Segmental Analysis

The global antisense and RNAi therapeutics market is segmented based on technology, route of administration, and application. 

Based on technology, the market is segmented into RNA Interference (siRNA and miRNA) and Antisense RNA. 

Antisense RNA technology segment dominated in 2023. Antisense RNA technology dominates the Antisense and RNAi Therapeutics industry because of its well-established technique and extensive track record in clinical use. Antisense RNA functions by binding to specific mRNA sequences using synthetic RNA molecules, thereby inhibiting the activity of disease-causing target genes.

Furthermore, several antisense treatments that use RNA interference technology have previously been approved by the FDA and are already available on the market. This confirms their efficacy and promotes additional investment in this field. Therefore, this technology dominates the RNA-based therapeutics industry and establishes the groundwork for advancements in this domain.

The RNA interference technology segment is projected to witness the fastest CAGR over the forecast period. This technique is employed to study gene functions in model species and cell cultures and target specific gene sequences associated with cancer. In addition, RNAi technology is widely used to treat bacterial illnesses, viruses, and parasites and to relieve pain.

Based on the route of administration, the market is segmented into Intravenous Route, Subcutaneous Route, intrathecal route, Pulmonary Delivery, Intraperitoneal Injection, and Others.

The pulmonary delivery segment dominated in 2023. There are multiple reasons for using localized administration to treat respiratory disorders since it dramatically enhances the effectiveness of the treatment. Hence, the capacity to reach the lungs and transport medicinal substances is highly efficient in treating ailments including respiratory syncytial virus, pulmonary arterial hypertension, and lung cancer. As the workload continues to grow, there is a demand to enhance the efficiency and precision of therapy. As a result, the RNAi technology market places significant emphasis on pulmonary delivery, which guides investment and innovation efforts.

Subcutaneous injections are the fastest growing. Subcutaneous injections are delivered in the adipose tissue located directly below the skin. This approach provides versatility in choosing the location for administering the infusion, encompassing possibilities such as the abdomen, thighs, and posterior aspect of the arms. In addition, subcutaneous infusion systems can use smaller needle sizes, potentially decreasing discomfort experienced during the infusion procedure. The anticipated benefits of subcutaneous administration are projected to propel the expansion of this segment in the near future.

Based on the application, the market is segmented into oncology, cardiovascular diseases (CVDs), respiratory disorders, renal diseases, neurodegenerative disorders, genetic disorders, infectious diseases, and others.

The neurodegenerative disorders segment dominated in 2023. Industry players actively seek progress in RNA interference (RNAi) and antisense treatments to tackle neurodegenerative illnesses. In June 2023, Arrowhead Pharmaceuticals stated its plan to get regulatory approval in Australia to begin a study on an experimental RNA interference-based treatment for amyotrophic lateral sclerosis (ALS), a neurological condition. These efforts by prominent companies to address neurodegenerative disorders are expected to drive growth in this sector.

Genetic disorders are projected to witness the fastest CAGR over the forecast period. The domain of oligonucleotide therapeutics is seeing significant growth, as indicated by the escalating quantity of research investigations. Companies employ several strategies to create antisense and RNAi treatments for genetic illnesses. In June 2023, Charles River Laboratories International, Inc. partnered with Curigin, a Korean biotechnology company, to develop oncolytic RNA interference (RNAi) gene therapy.

Recent Developments

• April 2024 – OliX Pharmaceuticals Inc., a prominent developer of RNA-interfering therapeutics for the treatment of severe diseases such as fibro-inflammatory and metabolic diseases, announced that it has received approval from the Alfred Human Research Ethics Committee to initiate the Phase 1 clinical trial of its drug candidate OLX75016 (a derivative of OLX702A) for the treatment of NASH.
• November 2023 – Benitec Biopharma Inc. (NASDAQ: BNTC) declared that the first participant has administered a dose in the BB-301 Phase 1b/2a Clinical Treatment Study. Benitec is a biotechnology company focusing on gene therapy and developing innovative genetic medicines using its unique "Silence and Replace" DNA-directed RNA interference (ddRNAi) platform. BB-301 is the initial gene therapy candidate of the Company that utilizes the Silence and Replace method. It is currently in development for treating Dysphagia associated with Oculopharyngeal Muscular Dystrophy.