Antisense and RNAi Therapeutics

Antisense and RNAi Therapeutics

Global Antisense and RNAi Therapeutics Market to Reach US$3.6 Billion by 2030

The global market for Antisense and RNAi Therapeutics estimated at US$2.1 Billion in the year 2023, is expected to reach US$3.6 Billion by 2030, growing at a CAGR of 8.1% over the analysis period 2023-2030. Antisense RNA Technology, one of the segments analyzed in the report, is expected to record a 7.8% CAGR and reach US$1.8 Billion by the end of the analysis period. Growth in the RNA Interference Technology segment is estimated at 8.4% CAGR over the analysis period.

The U.S. Market is Estimated at US$552.3 Million While China is Forecast to Grow at 11.7% CAGR

The Antisense and RNAi Therapeutics market in the U.S. is estimated at US$552.3 Million in the year 2023. China, the world`s second largest economy, is forecast to reach a projected market size of US$881.2 Million by the year 2030 trailing a CAGR of 11.7% over the analysis period 2023-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 4.6% and 7.3% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 5.0% CAGR.

Global Antisense and RNAi Therapeutics Market - Key Trends and Drivers Summarized

Why Are Antisense and RNAi Therapeutics Revolutionizing Modern Medicine?

Antisense and RNA interference (RNAi) therapeutics have emerged as groundbreaking technologies in the field of biotechnology and pharmaceuticals, but why are these therapies considered revolutionary? At the heart of these approaches is their ability to specifically target and regulate gene expression, allowing for precise intervention in disease processes that were previously untreatable with traditional drugs. Antisense oligonucleotides (ASOs) and RNAi therapies work by targeting messenger RNA (mRNA), the intermediary between DNA and protein production, effectively silencing harmful genes responsible for various diseases. This mechanism enables the development of treatments for genetic disorders, cancers, viral infections, and neurodegenerative diseases, among others. Unlike conventional small-molecule drugs, which often have broader effects and can cause significant side effects, antisense and RNAi therapeutics offer high specificity, reducing the risk of off-target effects. As our understanding of genomics grows, these therapies represent a significant leap forward in personalized medicine, enabling tailored treatments that address the underlying causes of diseases at the molecular level.

What Challenges and Innovations Are Shaping the Development of Antisense and RNAi Therapeutics?

The development of antisense and RNAi therapeutics has been driven by both scientific innovation and the need to overcome numerous challenges. One of the primary hurdles in early research was the stability of oligonucleotides in the human body, as these molecules are prone to degradation by enzymes in the bloodstream, reducing their effectiveness. However, advancements in chemistry have led to the development of chemically modified oligonucleotides, such as phosphorothioate backbones and locked nucleic acids (LNAs), which enhance their stability and improve their ability to bind to target mRNA. Another challenge has been the efficient delivery of these therapies to the target tissues or cells, as oligonucleotides need to cross cellular membranes to reach the cytoplasm where mRNA resides. Lipid nanoparticles (LNPs) and conjugation strategies, such as attaching targeting ligands to the oligonucleotides, have been successfully employed to improve delivery, particularly for liver-targeted therapies. The rise of RNAi therapeutics, specifically small interfering RNA (siRNA) and microRNA (miRNA), has also seen breakthroughs in the delivery field. These therapies are typically delivered via lipid-based carriers or other advanced delivery systems designed to protect the RNA molecules and ensure they reach the desired tissues. Innovations in RNA chemistry have reduced the immune response that was initially a major concern with these therapies, improving their safety profiles. The continued refinement of delivery technologies, combined with advances in gene editing and mRNA-targeting technologies, has enabled the development of RNAi therapies for conditions previously considered untreatable, such as genetic disorders like Huntington’s disease and certain types of cancers.

How Are Antisense and RNAi Therapeutics Transforming the Treatment of Rare and Genetic Diseases?

Antisense and RNAi therapeutics are having a profound impact on the treatment of rare and genetic diseases, where traditional drug development approaches have often fallen short. Many rare diseases are caused by specific genetic mutations that result in the production of abnormal proteins or the loss of necessary proteins, leading to severe, often life-threatening conditions. Antisense oligonucleotides are designed to bind to the mRNA produced from these mutated genes, blocking their translation into harmful proteins or correcting splicing errors that result in dysfunctional proteins. This approach has already shown success in treating diseases like spinal muscular atrophy (SMA) with drugs like nusinersen (marketed as Spinraza), which modifies the splicing of the SMN2 gene to produce functional protein in patients who would otherwise lack it. This type of therapy offers hope for many other rare genetic disorders that currently have no effective treatments. RNAi therapies are similarly transforming the landscape for genetic diseases, especially in conditions where overexpression of harmful genes is a key factor. For example, patisiran (marketed as Onpattro), an RNAi therapy, has been approved for the treatment of hereditary transthyretin-mediated amyloidosis, a rare genetic disorder that causes the buildup of amyloid proteins in organs, leading to organ failure. By targeting and silencing the mRNA responsible for the production of the abnormal transthyretin protein, patisiran provides a therapeutic effect that addresses the root cause of the disease, rather than merely managing symptoms. T

What Factors Are Driving the Growth of the Antisense and RNAi Therapeutics Market?

The growth in the antisense and RNAi therapeutics market is driven by several factors, including advances in genomics, increasing approvals of RNA-based drugs, and the expanding scope of treatable diseases. One of the primary drivers is the significant progress in genomics and gene sequencing technologies, which has provided a deeper understanding of the genetic basis of many diseases. This knowledge has enabled the identification of specific genetic targets for antisense and RNAi therapies, facilitating the development of highly targeted treatments. Additionally, regulatory agencies like the FDA have become more familiar with these novel therapies, leading to faster approval processes for promising drugs. The success of early RNA-based therapies, such as Spinraza and Onpattro, has also boosted confidence in the efficacy and safety of these treatments, encouraging further investment and innovation in the field. Another key factor is the broadening range of diseases that antisense and RNAi therapeutics can address. While these therapies were initially focused on rare and genetic disorders, their applications are expanding into more common diseases, including cancers, cardiovascular diseases, and metabolic disorders. For example, inclisiran, an RNAi therapeutic, has been developed to lower cholesterol levels by targeting the PCSK9 gene, offering a new treatment option for hypercholesterolemia. The versatility of these therapies in treating a wide variety of conditions is driving their adoption across multiple therapeutic areas. Furthermore, the ongoing innovations in delivery systems and the ability to enhance the stability and specificity of these molecules have made them more viable as commercial products, contributing to the rapid expansion of the market. The increasing investment from pharmaceutical companies, coupled with growing interest in personalized medicine, ensures that the market for antisense and RNAi therapeutics will continue to grow, offering new hope for patients with previously untreatable diseases.

Select Competitors (Total 36 Featured) -

• Acuitas Therapeutics
• Alnylam Pharmaceuticals
• Antisense Therapeutics Ltd.
• Arbutus Biopharma
• Benitec Biopharma Ltd.
• Bio-Path Holdings Inc.
• Calando Pharmaceuticals
• Dicerna Pharmaceuticals
• Enzon Pharmaceuticals Inc
• Gene Signal
• GlaxoSmithKline Plc
• Gradalis
• iCo Therapeutics
• Ionis Pharmaceuticals
• Lorus Therapeutics (Aptose Biosciences)
• Marina Biotech
• miRagen Therapeutics
• Mirna Therapeutics Inc
• Olix Pharmaceuticals
• OncoGeneX Pharmaceuticals
• Quark Pharmaceuticals
• Regulus Therapeutics
• Rexahn Pharmaceuticals
• Rxi Pharmaceuticals
• Sanofi S.A
• Santaris Pharma A/S (Roche)
• Sarepta Therapeutics
• Silence Therapeutics Plc
• Sirnaomics Inc.

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