RNAi Therapeutics Market– Creating a New Range of Therapeutics to Treat a Multitude of Disorders / Diseases
The RNA interference (RNAi) technique was discovered close to two decades ago. As of today, it is considered among the upcoming and versatile therapeutic mechanisms known to medical science. Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids. Cells use this process to silence and / or inhibit gene expression, via the targeted degradation of specific (unwanted) mRNA molecules. From an application perspective, the gene specificity of RNAi is the primary reason why it is being considered for therapy development. In theory, RNAi based therapeutics are capable of treating indications, such as age-related macular degeneration (AMD), hepatitis C and various forms of cancer, which are actually hard to treat, using conventional pharmacological options.
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Since 2011, there has been an increase in the number of candidate therapies in the clinical pipeline as companies are developing novel delivery methods and anti-immuno-stimulatory strategies based on sound scientific rationale. The current clinical dataflow is set to be a major attractor for rich biotech schemes as RNAi is the next hot area in drug development. In 2019, the projected market is worth USD 160 million. We expect the overall RNAi therapeutics market to be worth USD 3.9 billion by 2025 and this value is projected to reach USD 9.2 billion by 2030.
RNAi technology has emerged as a powerful tool to analyze various genes in a variety of organisms. This technology can be used in functional genomics as it is highly specific and can silence a particular gene from a multiple gene family. Since, RNAi depends on sequence homology, it enables the selection of unique / conserved regions of target genes for silencing.
RNAi technology offers the promise of being able to treat diseases that are characterized by abnormal gene functions. It targets a disease at post-transcriptional level and thus, is a highly selective technology. The most common therapeutic areas that have captured the focus of both research institutions and companies are neurogenerative disorders, oncology and viral diseases.
It is worth mentioning that, Onpattro, a RNAi based therapeutic drug, recently received marketing approval for the treatment of Hereditary ATTR Amyloidosis. Currently, there are many clinical trials going on, to study the safety and efficacy of siRNAs molecules for the treatment of various infectious diseases. Viral diseases that can be potentially treated using RNAi include hepatitis B, hepatitis C, HIV and influenza. In addition, RNAi technology has the potential to transform the treatment regimen for various cancers. Due to its reduced cytotoxic effects, this technology is regarded safe. Further, it is highly specific and only involves the knockdown of its target gene.
Further, genome wide screening can be achieved through gene knockdowns or by using RNAi for the identification of genes that influence a particular phenotype. The use of RNAi in genome screening provides a vast amount of data per experiment as it interacts with thousands of genes. The basic process of genome wide screening experiments involves the use of a RNAi library, stable cell types, transfection with RNAi agents, signal detection and analysis and identification of genes for therapeutic purposes.
Rnai Therapeutics market size – The “RNAi Therapeutics Market (2nd Edition), 2019-2030: Focus On siRNA, miRNA, shRNA and DNA” report features an extensive study of the current market landscape and future opportunities associated with RNAi therapeutics.
With two approved drug and several therapy candidates in late stages of clinical development, the field of RNAi therapeutics presents significant opportunity for interested biopharmaceutical developers. This drug class has the potential to treat a variety of clinical conditions, including oncological disorders, genetic diseases, hepatic diseases, respiratory disorders and infectious diseases.
One of the major challenges in this domain is related to the delivery of RNAi therapeutics. However, in recent years, several types of novel delivery systems have been developed and are being investigated for therapeutic nucleic acid delivery. The key aim in this context is to increase the efficiency of drug delivery to the target site. Examples of the various delivery systems, which are either already being used, or under evaluation, include lipid nanoparticle delivery, polypeptide delivery and conjugated delivery system. In summary, RNAi-based therapeutics are expected to soon become one of the prominent therapeutic options within mainstream healthcare.
RNA interference is a post transcriptional gene silencing process that is believed to have the potential to treat undruggable diseases. Unlike other drugs, which target a disease at the protein level, RNAi therapeutics target the mRNA that codes for proteins. Several companies, such as Alnylam Pharmaceuticals, Silence Therapeutics and Quark Pharmaceuticals have realized the potential of this market and are actively developing RNAi drugs to target various disease indications. The future of this market looks highly promising; however, careful analysis of the market indicates some challenges that need to be addressed. These factors are likely to determine the success and failure of the market and are anticipated to help in carving strategies for overcoming the weaknesses and threats to the market.
Strengths
RNAi based therapeutics have demonstrated potential to treat previously undruggable targets, including rare genetic disorders, owing to its ability to perform sequence specific degradation of mRNA strands. Further, RNAi therapeutics are capable of treating different disease indications by targeting a common gene. For instance, Alnylam Pharmaceuticals has two RNAi therapeutics in its pipeline, Onpattro and Vutrisiran, that target the transthyretin (TTR) mutation. Although both the drug candidates, target the same gene, they are intended for different indications, namely hATTR amyloidosis and ATTR amyloidosis, respectively. It is worth mentioning that this market is characterized by the presence of a rich and growing pipeline, of which some drug candidates are likely to be approved in the near future.
Weaknesses
One of the major challenges associated with the development of novel drug classes is the delivery of therapeutic agent to the target tissue. The challenge of delivering RNAi payload to target cells without metabolic clearance and immunogenicity is faced by players engaged in this domain. In order to elicit the therapeutic response properly, key considerations for a delivery technology include crossing RNAi payload through the biological barrier, selectively hitting the target, release of siRNA, high tolerance and high therapeutic index (ratio of therapeutic effect to toxicity). Often, size and charge of siRNA serves as an obstacle in the delivery to target cell or tissue. The current delivery vehicles used by developers include lipid-based nanoparticles, polymer-based nanoparticles, conjugated systems and delivery of naked siRNA.
Oppertunities
Some disorders are caused due to multiple gene mutations along with other factors, such as lifestyle and environmental factors; these include cardiovascular disorders, genetic disorders and autoimmune disorders, Presently, not many effective treatments for such disorders are available in the market. However, RNAi based therapeutics have the potential to target multiple targets and thus, can effectively cure such diseases and provide relief to patients. It is worth noting that companies such as Alnylam and Arbutus Biopharma are developing RNAi based drugs that target disorders that are caused due to lifestyle habits and environmental factors; molecules of both the companies are currently in discovery stage.
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Threats
Apart from RNAi, other technologies that use the gene silencing approach are also being developed. These technology platforms include CRISPR interference and antisense technology. RNAi technology regulates gene at mRNA level, while CRISPR interference controls expression at transcriptional level. This new technology could become a potential competitor of the RNAi as it provides additional advantages, such as inducing pluripotency in stem cells. Another competitor of the RNAi technique is antisense technology, which has significant number of approved therapies, such as Vitravene, Macugen and Exondys 51. This technology uses the antisense
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