RNAi THERAPEUTICS AND TECHNOLOGY MARKET: OVERVIEW
As per Roots Analysis, the global RNAi therapeutics and technology market is estimated to grow from USD 1.2 billion in the current year to USD 9.2 billion by 2035, at a CAGR of 17% during the forecast period, till 2035.
The market sizing and opportunity analysis has been segmented across the following parameters:
Target Therapeutic Areas
- Genetic Disorders
- Metabolic Disorders
- Oncological Disorders
- Genetic Disorders
- Hematological Disorders
- Ophthalmic Disorders
- Other Disorders
Route of Administration
- Subcutaneous
- Intravenous
- Ophthalmic
- Intradermal
Type of RNAi Molecule
Geographical Regions
- North America
- Europe
- Asia-Pacific and Rest of the World
Sales Forecast
- Onpattro(R)
- Givlaari(TM)
- Leqvio(R)
- Oxlumo(TM)
- Fitusiran
- Vutrisiran
- SYL-1001
- Vigil-EWS
- Nedosiran
RNAi THERAPEUTICS AND TECHNOLOGY MARKET: GROWTH AND TRENDS
RNAi therapeutics refer to the novel class of innovative drugs that harness the RNA interference (RNAi) property of genes for the regulation of the expression system naturally inside the cells. 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 targeted degradation of specific (unwanted) mRNA molecules. From an application perspective, the gene specificity of RNAi is the primary reason for it being used in the development of therapies. In theory, RNAi-based therapeutics are capable of treating a myriad of disease 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.
It is interesting to note that in, 2018, Patisiran became the first RNAi-based therapy to receive approval from the FDA to treat patients suffering from chronic diseases. Since then, six novel RNAi-based therapies, namely, Givosiran (2019), Lumasiran (2020), Inclisiran (2021), Vutrisiran (2022) and Nedosiran (2023), have received approval from the FDA. Notably, more than 200 RNAi therapeutics are currently being evaluated across different clinical trials for the treatment of a broad array of disease indications. Additionally, several industrial players are increasing their research efforts for the development of RNA interference technology, aiming to enhance efficacy and reduce effector molecule concentrations based on the anti-sense approach. Driven by ongoing research, it is estimated that the market will grow significantly during the forecast period.
RNAI THERAPEUTICS AND TECHNOLOGY MARKET: KEY INSIGHTS
The report delves into the current state of the RNAi therapeutics and technology market and identifies potential growth opportunities within industry. Some key findings from the report include:
- Presently, more than 200 RNAi drug candidates are being investigated across various stages of development for the treatment of a myriad of disease indications; six such drug products have already received approval.
- Nearly 80% of the drug candidates are being developed using siRNA molecules; over 40% of the RNAi drugs are designed for delivery via the subcutaneous route.
- More than 45 companies, including several startups, are engaged in the development of RNAi therapeutics; the R&D efforts are being led by players located in the US.
- The market is characterized by the presence of well-established players; the relatively less experienced players also possess a robust pipeline of early-stage candidates.
- The rising interest in this rapidly evolving domain can be validated by the partnerships inked in recent years; the majority of the deals were signed for research and development purposes.
- In order to tap the lucrative opportunity, venture capital firms / strategic investors have actively supported the ongoing initiatives; over USD 8.5 billion has been invested across 110+ instances in the past few years.

- Notably, significant clinical activity, in terms of registered clinical trials, was observed; over 60% of the clinical studies are being conducted in parallel comparison of two groups of treatments.
- A number of scientists from renowned universities, presently involved in several clinical development efforts of RNAi therapeutics, have emerged as key opinion leaders.
- More than 55 innovative drug development and delivery technology platforms are presently being leveraged by industry stakeholders for the discovery, development and delivery of RNAi therapeutics.
- The healthy growth in published scientific literature over the last few years signifies the focused efforts being led by researchers to identify and develop novel technologies and therapeutic interventions.
- Over 2,100 patents have been granted / filed by academia and industry stakeholders in the last six years, exclusively for RNAi therapeutics and associated delivery technologies.
- In 2035, close to 60% of the demand for siRNA drugs is likely to be generated in North America for the treatment of patients suffering from various genetic disorders.
- In order to efficiently monetize their proprietary offerings, drug developers are actively exploring diverse commercialization strategies applicable across different stages of a product's launch cycle.
- The market opportunity associated with RNAi therapeutics is anticipated to witness an annualized growth rate of 17%, over the coming decade.

- The overall opportunity is likely to be well distributed across different routes of administration, market players and countries / regions.
RNAI THERAPEUTICS AND TECHNOLOGY MARKET: KEY SEGMENTS
Genetic Disorders Occupy the Largest Share of the Global RNAi Therapeutics and Technology Market
Based on the target therapeutic area, the market is segmented into oncological disorders, genetic disorders, metabolic disorders, hematological disorders, ophthalmic disorders and other disorders. At present, the genetic disorders segment holds the maximum share of the global RNAi therapeutics and technology market. This trend is likely to change in the forthcoming years.
By Route of Administration, RNAi Therapeutics and Technology focused on Intradermal Route of Administration is the Fastest Growing Segment of the Global RNAi Therapeutics and Technology Market During the Forecast Period
Based on the route of administration, the market is segmented into subcutaneous, intramuscular, ophthalmic and intradermal routes. Currently, the RNAi therapeutics and technology market is dominated by subcutaneous route of administration segment, capturing the highest proportion of the global RNAi therapeutics and technology market. It is worth highlighting that the global RNAi therapeutics and technology market for intradermal route of administration segment is likely to grow at a relatively higher CAGR.
siRNA Segment Occupies the Largest Share of the RNAi Therapeutics and Technology Market by Type of RNAi Molecule
Based on the type of RNAi molecule, the market is segmented into siRNA and shRNA. At present, siRNA segment by type of RNAi molecule holds the maximum share of the global RNAi therapeutics and technology market. Notably, shRNA segment is likely to grow at a relatively higher CAGR.
North America Accounts for the Largest Share of the Market
Based on the geographical regions, the market is segmented into North America, Europe, and Asia-Pacific and Rest of the World. The majority share is expected to be captured by players based in North America and Europe.
Example Players in the RNAi Therapeutics and Technology Market
- AgelessRx
- ANOVA Institute for Regenerative Medicine
- Betterhumans
- BioAge Labs
- BioXcellerator
- Cambrian Biopharma
- Gero.ai
- Mayo Clinic
- Rejuvenate Bio
- UT Health San Antonio
RNAI THERAPEUTICS AND TECHNOLOGY MARKET: RESEARCH COVERAGE
- Market Sizing and Opportunity Analysis: The report features an in-depth analysis of the global RNAi therapeutics and technology market, focusing on key market segments, including [A] key therapeutic areas, [B] route of administration, [C] type of RNAi molecule, [D] geographical regions and [E] leading players.
- RNAi Therapeutics Market Landscape: A comprehensive evaluation of the RNAi pipeline candidates, based on several relevant parameters, such as [A] phase of development (marketed, clinical, and preclinical / discovery stage) of pipeline candidates, [B] target disease indication(s), [C] key therapeutic areas (oncological disorders, infectious diseases, genetic disorders, ophthalmic diseases, respiratory disorders, hepatic disorders, metabolic disorders, cardiovascular disorders, dermatological disorders and other disorders), [D] type of RNAi molecule (siRNA, miRNA, shRNA, shRNA and DNA), [E] target gene, [F] route of administration and [G] special drug designations (if any).
- RNAi Therapeutics Developers Market Landscape: A comprehensive evaluation of the companies engaged in this domain, based on several relevant parameters, such as [A] year of establishment, [B] company size and [C] location of headquarters.
- Company Competitiveness Analysis: A comprehensive competitive analysis of RNAi therapeutic developers, examining factors, such as [A] developer strength and [B] product portfolio strength.
- Drug Profiles: In-depth profiles of key RNAi therapeutic drugs, focusing on [A] current development status of the drug, [B] developer details, [C] mechanism of action, [D] route of administration, [E] affiliated technology / platform (if available), [F] dosage and [G] and recent clinical trial results.
- RNAi Therapeutics Technology Platforms Market Landscape: A comprehensive evaluation of the RNAi therapeutics technology platforms and their developers, based on several relevant parameters, such as [A] purpose of technology, [B] type of molecule (s) delivered and [C] type of cell (s) / tissue (s) targeted, [D] year of their establishment, [E] company size and [F] location of headquarters. Additionally, a brief overview of the profiles of key RNAi-based drug discovery / development technology platforms and drug delivery technologies (shortlisted on the basis of competitiveness score).
- Technology Competitiveness Analysis: A comprehensive competitive analysis of RNAi technologies, examining factors, such as [A] supplier power and [B] key technology specifications.
- Target Indications: A comprehensive analysis of target indications (segregated by various therapeutic areas) that are currently being focused on the companies engaged in the development of RNAi therapeutics.
- Clinical Trial Analysis: An insightful analysis of clinical studies, based on several parameters, such as [A] trial registration year, [B] current status, [C] phase of development, [D] type of RNAi molecule, [E] enrolled patient population and [F] regional distribution of trials.
- Key Opinion Leaders (KOLs]: An in-depth 2X2 analysis that emphasizes the key opinion leaders in this domain, shortlisted based on their contributions (in terms of involvement in various clinical studies) in this field.
- Patent Analysis: An in-depth analysis of patents filed / granted till date in the RNAi therapeutics domain, based on various relevant parameters, such as [A] type of patent, [B] publication year, [C] regional applicability, [D] CPC symbols, [E] emerging focus areas, [F] leading industry / non-industry players and [G] patent valuation.
- Publication Analysis: An insightful analysis of 3,000 peer-reviewed scientific articles in the RNAi therapeutics domain, based on various relevant parameters, such as [A] year of publication, [B] type of publication, [C] popular keywords, [D] top journals, [E] top publishers, [F] key copyright holders and [G] key funding institutes.
- Partnerships and Collaborations: An in-depth analysis of the deals inked by stakeholders in the RNAi therapeutics and technology market, based on several parameters, such as [A] year of partnership, [B] type of partnership, [C] target disease indication, [D] therapeutic area, [E] type of RNAi molecule, [F] financial details (wherever applicable), [G] focus area of collaboration and [E] most active players (in terms of number of partnerships).
- Funding and Investment Analysis: An in-depth analysis of the fundings raised by RNAi therapeutics and technology companies, based on relevant parameters, such as [A] year of funding, [B] type of funding, [C] amount invested, [D] type of RNAi molecule, [E] most active players and [F] key investors.
- Case Study 1: A detailed discussion on the various commercialization strategies that can be adopted by the companies engaged in this domain, based on [A] different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing of the drug.
- Case Study 2: An in-depth discussion on the use of miRNA as a potential biomarker, along with a list of diagnostic kits that are either available in the market, or likely to be approved in the foreseen future.
- RNAi Therapeutics Service Providers Market Landscape: A comprehensive analysis of the companies that are actively supporting the development of RNAi therapeutics market, including contract manufacturers, contract researcher organizations and other service providers based on various parameters, such as [A] type of service provider, [B] location of their headquarters and [C] type of RNAi molecule developed.
- Demand Analysis: A detailed analysis of the annual clinical and commercial demand for RNAi therapeutics, based on the target patient population in ongoing and planned clinical trials of RNAi therapeutics, sponsored by both industry and non-industry players.
- SWOT Analysis: An analysis of industry affiliated trends, opportunities and challenges, which are likely to impact the evolution of RNAi therapeutics market; it includes a Harvey ball analysis, assessing the relative impact of each SWOT parameter on industry dynamics.
KEY QUESTIONS ANSWERED IN THIS REPORT
- How many companies are currently engaged in this market?
- Which are the leading companies in this market?
- What factors are likely to influence the evolution of this market?
- What is the current and future market size?
- What is the CAGR of this market?
- How is the current and future market opportunity likely to be distributed across key market segments?
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