RNA Targeting Small Molecules Therapeutics Market by Type of Target Molecule, Type of Approach, Target Indication, Target Therapeutic Area, Route of Administration, and Key Geographical Region: Industry Trends and Global Forecasts, 2021-2030

Overview

Small molecule products presently dominate the overall pharmaceutical market, representing around 80% of the total pipeline candidates. In fact, in 2020, the USFDA approved 55 drugs, of which, over 65% were small molecule therapeutics. It is worth noting that majority of these drugs were conventionally developed to target proteins. However, reports suggest that merely 2% of the human genome is responsible for coding for proteins. Hence, the pharmaceutical industry is now focused on evaluating previously untapped targets, such as non-coding DNA and RNA; the latter molecules are expected to correspond to ~70% of the unknown portion of the human genome. In recent past, RNA’s limitless potential as a target for small molecules, against a wide range of indications (both oncological and non-oncological), has been recognized. Specifically, a variety of small molecules that affect RNA function have been discovered. Further, many RNA riboswitch regulatory elements (capable of binding to a variety of small molecule metabolites and control gene expression) and small molecule antibiotics (having the ability to bind to ribosomal RNA and interfere with translation) have been identified. Consequently, drug developers are experimenting with RNA modification, RNA translation, RNA splicing, indirect RNA targeting (epitranscriptomics), and other ways to target RNA with small molecules. 

Currently, over 100 small molecule therapeutics, targeting naïve RNA, are under development. Several experiments and clinical trials focused on RNA targeting antisense oligonucleotides (many of which have received regulatory approval), as well as preclinical studies for synthetic RNAs, which can redirect cellular RNA interference (RNAi) machinery or activate CRISPR-based systems, have demonstrated strong proof of principle for RNA targeting drugs. Moreover, a number of industry players (mostly small firms and start-ups) have developed their proprietary technologies for direct and indirect RNA targeting. Additionally, in the past five years, more than USD 800 million was invested in companies engaged in the development of RNA targeting small molecule therapeutics (primarily those developing candidates against oncological disorders). Many big pharma players, such as Pfizer, Novartis and Takeda Pharmaceutical, have also actively supported the initiatives taken by such drug developers, by making lucrative investments. Driven by encouraging clinical trial results, strategic collaborations and initiatives undertaken to expand expanding intellectual capital, this emerging market is expected to grow at a steady pace in the foreseen future.

Scope of the Report

The “RNA Targeting Small Molecules Therapeutics Market by Type of Target Molecule (Cap Binding Complex, CDKL5, Dystrophin Protein, Erα, G542X, MNK1/MNK2, PAX6 gene, SMN2), Type of Approach (Indirect RNA Targeting- Epitranscriptomics, mRNA Translation Modulation, RNA Splicing Modification), Target Indication (Aniridia, Breast Cancer, Castrate-resistant Prostate Cancer, Colorectal Cancer, Crohn Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Dravet Syndrome, Epilepsy, HIV Infections, Impaired Renal Function, Non-Small Cell Lung Cancer, Rheumatoid Arthritis, Spinal Muscular Atrophy, Ulcerative Colitis), Target Therapeutic Area (Autoimmune Disorders, Genetic Disorders, Infectious Disorders, Inflammatory Disorders, Neurological Disorders, Oncological Disorders, Ophthalmic Disorders, Rare Disorders, Renal Disorders, Respiratory Disorders), Route of Administration (Oral and Subcutaneous Injection) and Region (North America, Europe, Asia, Middle East and North Africa, Latin America and Rest of the World): Industry Trends and Global Forecasts, 2021-2030” report features an extensive study of the current landscape, offering an informed opinion on the likely adoption of RNA targeting small molecule therapeutics in the pharmaceutical industry, over the next decade. The report features an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain. 

Amongst other elements, the report includes:

• A detailed overview of the current market landscape of RNA targeting small molecule therapeutics, including information on phase of development (clinical and preclinical), target disease indication(s), therapeutic area (non-oncological disorders and oncological disorders), target molecule (BMI1, cap binding complex, eLF4A, MNK1/MNK2, VEGF, others), type of approach (indirect RNA targeting – epitranscriptomics, mRNA translation modulation, RNA splicing modification) and route of administration (intravenous, subcutaneous, and oral). In addition, it highlights the developers of these novel therapeutics, along with information on their year of establishment, company size (in terms of employee count), location of headquarters and number of proprietary drug candidates.
• An in-depth analysis of the company competitiveness and investment landscape, including a bubble analysis comparing the key players engaged in the domain of RNA targeting small molecules, based on several relevant parameters (such as company experience, pipeline strength, partnership activity, funding activity, patents filed and company size) and a multivariate representation illustrating the start-ups and investment activity based on various multiple parameters (such as funding type, therapeutic area and amount invested).
• Elaborate profiles of key players engaged in the development of RNA targeting small molecule therapeutics. Each profile features a brief overview of the company, recent developments and an informed future outlook.
• An analysis of the recent collaborations and partnerships that have been inked between various stakeholders in this domain, during the period between 2017 and 2021, covering R&D agreements, clinical trial agreements, acquisitions, licensing agreements and product development agreements.
• An analysis of the investments made, during the period between 2015 and 2021, including grant, seed financing, venture capital financing, initial public offering (IPO), debt financing and other equity, in companies that are engaged in the development of RNA targeting small molecule therapeutics.

One of the key objectives of the report was to estimate the existing size and the future opportunity associated with the RNA targeting small molecules therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2021-2030. The report also provides details on the likely distribution of the current and forecasted opportunity across [A] type of target molecule (cap binding complex, CDKL5, dystrophin protein, Erα, G542X, MNK1/MNK2, PAX6 gene, SMN2) [B] type of approach (indirect RNA targeting- epitranscriptomics, mRNA translation modulation, RNA splicing modification) [C] target indication (aniridia, breast cancer, castrate-resistant prostate cancer, colorectal cancer, Crohn disease, cystic fibrosis, Duchenne muscular dystrophy, Dravet syndrome, epilepsy, HIV infections, impaired renal function, non-small cell lung cancer, rheumatoid arthritis, spinal muscular atrophy, ulcerative colitis) [D] target therapeutic area (autoimmune disorders, genetic disorders, infectious disorders, inflammatory disorders, neurological disorders, oncological disorders, ophthalmic disorders, rare disorders, renal disorders, respiratory disorders), [E] route of administration (oral and subcutaneous injection) and [E] region (North America, Europe, Asia, Middle East and North Africa, Latin America and Rest of the World). In order to account for future uncertainties and to add robustness to our forecast model, we have provided three forecast scenarios, portraying the conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.

The opinions and insights presented in the report were also influenced by discussions held with multiple stakeholders in this domain. 

The report features detailed transcripts of interviews held with the following individuals (in alphabetical order):

• Clara Assouline (Business Development, Anima Biotech)
• Dominique Cheneval (President/Co-Founder, Novation Pharmaceuticals)

All actual figures have been sourced and analyzed from publicly available information forums. Financial figures mentioned in this report are in USD, unless otherwise specified.

Key Questions Answered

• Who are the key players engaged in the development of RNA targeting small molecule therapeutics?
• What are the current investment trends in this field?
• Which are the key therapeutic areas for which RNA targeting small molecule therapies are being developed?
• What are the different technological approaches adopted by various players for the development of RNA targeting small molecule therapeutics?
• What type of partnership models are most commonly adopted by stakeholders engaged in this domain?
• What is the relative competitiveness of different RNA targeting small molecule therapeutics developers engaged in this market?
• How is the current and future market opportunity likely to be distributed across key market segments?