Ultra Market Research | United Kingdom RNA-Based Rare Disease Drug Market
United Kingdom RNA-Based Rare Disease Drug Market
Report ID : 1031
Category : United-Kingdom(UK)
No Of Pages : 159
Published on: March 2025
Status: Published
Format :
Key Question Answer
Global Market Outlook
In-depth analysis of global and regional trends
Analyze and identify the major players in the market, their market share, key developments, etc.
To understand the capability of the major players based on products offered, financials, and strategies.
Identify disrupting products, companies, and trends.
To identify opportunities in the market.
Analyze the regional penetration of players, products, and services in the market.
Comparison of major players financial performance.
Evaluate strategies adopted by major players.
Recommendations
United Kingdom RNA-Based Rare Disease Drug Market
Introduction
The United Kingdom RNA-Based Rare Disease Drug Market is the emerging market that deals with developing and utilizing RNA-based drugs against rare disease. RNA-based drugs are created in order to treat genetic diseases by targeting RNA molecules involved in the disease process. RNAi and mRNA-based therapies are two such emerging drugs gaining popularity for their potential to target even untreatable genetic diseases. The industry has grown over the past few years with improvements in molecular biology, biotechnology, and the growing incidence of rare diseases. The UK has experienced substantial investment in RNA-based drug development, and some pharmaceutical firms are endeavoring to launch these cutting-edge treatments into the market. Trends in recent years have indicated growth in personalized medicine, as RNA therapies can be customized according to an individual's genetic profile. The size of the market is expanding, and the regular regulatory approvals of RNA-based medications in the UK demonstrate a favorable outlook. The situation today indicates growing collaborations and investments in the industry.
The United Kingdom RNA-Based Rare Disease Drug Market is growing due to various factors, such as progress in biotechnology, a rise in the prevalence of rare diseases, and growth in personalized medicine. With enhanced knowledge of RNA biology, RNA-based treatments have become a hopeful approach for curing genetic disorders that were once untreatable. Governments and independent investors are aware of the prospect of RNA therapeutics, causing more funding and research efforts to be directed their way. In addition, gene editing and RNA interference technologies advancements have made more accurate and powerful treatments possible. Regulatory bodies like the UK Medicines and Healthcare products Regulatory Agency (MHRA) are getting more supportive towards RNA-based medicines, which accelerates the authorization process. The increasing need for therapies that are capable of addressing the underlying causes of genetic diseases, as opposed to treating symptoms, is also driving the market. Lastly, collaborations between pharmaceutical firms and academic research institutions are driving innovation in the RNA arena.
Restraints
Even though the outlook is optimistic, the United Kingdom RNA-Based Rare Disease Drug Market also has some problems. One of the major issues is that it is expensive to develop and produce RNA-based treatments. The drugs are complicated to produce, and specialized equipment and expertise are needed, raising the total cost. In addition, the long research and development period, along with the lengthy clinical trials required to ascertain safety and efficacy, restricts the rate of drug commercialization. The other problem is delivery of RNA drugs to the target organ or tissue. Effective delivery mechanisms are still a major stumbling block, since the immune system of the body has the potential to neutralize RNA therapies prior to reaching the desired targets. Also, apprehensions regarding the long-term implications of RNA-based therapies, especially in gene editing and RNA interference technologies, may result in caution from both patients and regulators. Finally, the regulatory route for these new therapies is yet to be defined, which might result in uncertainty for manufacturers.
Opportunity
The United Kingdom RNA-Based Rare Disease Drug Market is set for tremendous growth, with numerous opportunities on the horizon. The prospect of RNA therapies offering solutions to previously untreatable genetic disorders creates a massive market for the development of new drugs. This is especially the case in the rare disease area, where few treatment options are available. Since RNA-based treatments can be tailored, the potential exists to treat a wide variety of diseases, which will further boost the market. Progress in RNA delivery systems, including lipid nanoparticles and viral vectors, is enhancing the efficacy of RNA-based treatments, which could solve the earlier delivery issues. The UK's strong healthcare infrastructure and regulatory system are also a strong base for RNA-based drug development and commercialization. Cooperation among biotech companies, universities, and government agencies will most likely speed up developments in this field. In addition, public and private investments will rise due to the increasing need for targeted treatments for orphan diseases.
Trend
One of the newest trends in the United Kingdom RNA-Based Rare Disease Drug Market is the increasing trend towards precision and personalized medicine. RNA-based treatments are particularly well-positioned for individualized treatment regimens, enabling drugs that target the precise genetic mutations that cause disease. This is particularly significant in the rare disease market, where each patient's condition can be different at a genetic level. Also, developments in delivery systems, including lipid nanoparticles and self-assembling RNA systems, are improving the capacity of RNA therapeutics to target effectively. A further trend is the rising application of mRNA-based vaccines and treatments, which received popularity after COVID-19 vaccine success. These technologies have established a strong pipeline of future RNA-based medicines, and drug companies are growing their initiatives to create RNA-based medicines for numerous rare diseases. In addition, these days there are increased collaborations between pharmaceutical giants and academic research centers to expedite the development of these revolutionized medicines.
Approved Products, Pipeline/Reg/Pre-Reg Products
Approved Products:
Onpattro (patisiran)
Tegsedi (inotersen)
Spinraza (nusinersen)
Exondys 51 (eteplirsen)
Pipeline/Reg/Pre-Reg Products:
Vyondys 53 (golodirsen)
Inclisiran
Olpasiran
Ataluren
VTS-270
Key Target Audience
Pharmaceutical Companies
Biotech Companies
Research Institutions
Genetic Disorder Treatment Providers
Healthcare Providers
Investors and Venture Capitalists
Regulatory Agencies
Healthcare Providers in Rare Disease Centers
Academics in Genetic Research
Rare Disease Advocacy Groups
Medical Professionals
Contract Research Organizations (CROs)
he RNA-Based Rare Disease Drug Market in the UK refers to the sector focused on the development of RNA-based therapies for treating genetic disorders, including RNA interference, gene editing, and messenger RNA therapies.
Factors include technological advancements in RNA therapy, increased prevalence of rare diseases, growing investments, and regulatory support for novel treatments
Challenges include high development costs, delivery issues, long research timelines, and regulatory uncertainty.
The market is expected to grow significantly, driven by innovations in personalized medicine and the ongoing development of RNA therapies for rare diseases.
Leading companies include Alnylam Pharmaceuticals, Pfizer, Moderna, BioMarin, and Vertex Pharmaceuticals, among others.
Introduction 1.1 Report Description 1.2 Research Methodology 1.2.1 Data Mining 1.2.2 Market Modeling and Forecasting 1.2.3 Data Validation 1.2.4 Industry Analysis
Market Dashboard
Market Overview 3.1 Market Definition and Scope 3.2 Market Segmentation 3.2.1 By Treatment Types 3.2.1.1 Chemotherapy 3.2.1.1.1 Induction Phase 3.2.1.1.2 Consolidation Phase 3.2.1.1.3 Maintenance Phase 3.2.1.2 Targeted Therapies 3.2.1.2.1 Tyrosine Kinase Inhibitors 3.2.1.2.2 Antibody-Drug Conjugates 3.2.1.2.3 Others 3.2.1.3 Immunotherapies 3.2.1.3.1 CAR-T Therapy 3.2.1.3.2 Monoclonal Antibodies 3.2.2 By Patient Demographics 3.2.2.1 Children 3.2.2.1.1 Infants 3.2.2.1.2 Toddlers and Young Children 3.2.2.2 Adults 3.2.2.2.1 Young Adults 3.2.2.2.2 Seniors 3.2.3 By Distribution Channels 3.2.3.1 Hospital Pharmacies 3.2.3.1.1 Tertiary Care Centers 3.2.3.1.2 Pediatric Oncology Units 3.2.3.2 Retail Pharmacies 3.2.3.2.1 Local Pharmacies 3.2.3.2.2 Chain Pharmacies 3.2.3.3 Online Pharmacies 3.2.4 By Therapeutic Applications 3.2.4.1 First-Line Treatment 3.2.4.1.1 Chemotherapy-Only Regimens 3.2.4.1.2 Targeted Therapies 3.2.4.2 Relapse or Refractory ALL 3.2.4.2.1 CAR-T Therapies 3.2.4.2.2 Experimental Treatments
United Kingdom RNA-Based Rare Disease Drug Market by Patient Demographics 6.1 Introduction 6.2 Market Size and Growth Rate by Patient Demographics (2024-2030) 6.2.1 Children 6.2.1.1 Infants 6.2.1.2 Toddlers and Young Children 6.2.2 Adults 6.2.2.1 Young Adults 6.2.2.2 Seniors
United Kingdom RNA-Based Rare Disease Drug Market by Distribution Channels 7.1 Introduction 7.2 Market Size and Growth Rate by Distribution Channels (2024-2030) 7.2.1 Hospital Pharmacies 7.2.1.1 Tertiary Care Centers 7.2.1.2 Pediatric Oncology Units 7.2.2 Retail Pharmacies 7.2.2.1 Local Pharmacies 7.2.2.2 Chain Pharmacies 7.2.3 Online Pharmacies
United Kingdom RNA-Based Rare Disease Drug Market by Therapeutic Applications 8.1 Introduction 8.2 Market Size and Growth Rate by Therapeutic Applications (2024-2030) 8.2.1 First-Line Treatment 8.2.1.1 Chemotherapy-Only Regimens 8.2.1.2 Targeted Therapies 8.2.2 Relapse or Refractory ALL 8.2.2.1 CAR-T Therapies 8.2.2.2 Experimental Treatments
Competitive Landscape 9.1 Company Profiles 9.1.1 Amgen Inc. (United States) 9.1.2 Novartis AG (Switzerland) 9.1.3 Pfizer Inc. (United States) 9.1.4 Gilead Sciences (United States) 9.1.5 Kite Pharma (United States) 9.1.6 Takeda Pharmaceutical Company (Japan) 9.1.7 Bristol-Myers Squibb (United States) 9.1.8 Jazz Pharmaceuticals (Ireland) 9.1.9 AbbVie Inc. (United States) 9.1.10 Sanofi (France) 9.1.11 Autolus Therapeutics (United Kingdom) 9.1.12 Cellectis (France) 9.1.13 Allogene Therapeutics (United States) 9.1.14 Celgene Corporation (United States) 9.1.15 Eli Lilly and Company (United States)
Conclusion and Recommendations
Appendix 11.1 List of Tables Table United Kingdom RNA-Based Rare Disease Drug Market Size by Treatment Types (2024-2030) Table Chemotherapy Market Size by Sub-type (2024-2030) Table Targeted Therapies Market Size by Sub-type (2024-2030) Table Immunotherapies Market Size by Sub-type (2024-2030) Table United Kingdom RNA-Based Rare Disease Drug Market Size by Patient Demographics (2024-2030) Table Children Market Size by Sub-type (2024-2030) Table Adults Market Size by Sub-type (2024-2030) Table United Kingdom RNA-Based Rare Disease Drug Market Size by Distribution Channels (2024-2030) Table Hospital Pharmacies Market Size by Sub-type (2024-2030) Table Retail Pharmacies Market Size by Sub-type (2024-2030) Table United Kingdom RNA-Based Rare Disease Drug Market Size by Therapeutic Applications (2024-2030) Table First-Line Treatment Market Size by Sub-type (2024-2030) Table Relapse or Refractory ALL Market Size by Sub-type (2024-2030)
11.2 List of Figures Figure United Kingdom RNA-Based Rare Disease Drug Market Size and Growth Rate by Treatment Types (2024-2030) Figure Chemotherapy Market Growth Rate by Sub-type (2024-2030) Figure Targeted Therapies Market Growth Rate by Sub-type (2024-2030) Figure Immunotherapies Market Growth Rate by Sub-type (2024-2030) Figure United Kingdom RNA-Based Rare Disease Drug Market Size and Growth Rate by Patient Demographics (2024-2030) Figure Children Market Growth Rate by Sub-type (2024-2030) Figure Adults Market Growth Rate by Sub-type (2024-2030) Figure United Kingdom RNA-Based Rare Disease Drug Market Size and Growth Rate by Distribution Channels (2024-2030) Figure Hospital Pharmacies Market Growth Rate by Sub-type (2024-2030) Figure Retail Pharmacies Market Growth Rate by Sub-type (2024-2030) Figure United Kingdom RNA-Based Rare Disease Drug Market Size and Growth Rate by Therapeutic Applications (2024-2030)
Tables Financial Overview of Key Companies Table Amgen Inc. RNA-Based Rare Disease Drug Financial Overview Table Novartis AG RNA-Based Rare Disease Drug Financial Overview Table Pfizer Inc. RNA-Based Rare Disease Drug Financial Overview Table Gilead Sciences RNA-Based Rare Disease Drug Financial Overview Table Kite Pharma RNA-Based Rare Disease Drug Financial Overview Table Takeda Pharmaceutical Company RNA-Based Rare Disease Drug Financial Overview Table Bristol-Myers Squibb RNA-Based Rare Disease Drug Financial Overview Table Jazz Pharmaceuticals RNA-Based Rare Disease Drug Financial Overview Table AbbVie Inc. RNA-Based Rare Disease Drug Financial Overview Table Sanofi RNA-Based Rare Disease Drug Financial Overview Table Autolus Therapeutics RNA-Based Rare Disease Drug Financial Overview Table Cellectis RNA-Based Rare Disease Drug Financial Overview Table Allogene Therapeutics RNA-Based Rare Disease Drug Financial Overview Table Celgene Corporation RNA-Based Rare Disease Drug Financial Overview Table Eli Lilly and Company RNA-Based Rare Disease Drug Financial Overview
Research Methodology
The process of market research at Ultra Market Research is an iterative in application and usually follows following path. Information from secondary used to build data models, then results from data models are validated from primary participants. Then cycle repeats where, according to inputs from primary participants, additional secondary research is done, and new Information is again incorporated into data model. The process continues till desired level of Information is not generated
To calculate the market size, the report considers the revenue generated from the sales of Ultra Market Research providers. The revenue generated from the sales of Ultra Market Research has been calculated through primary and secondary research. The report also presents the key players operating in the Ultra Market Research market across the globe identified through secondary research and a corresponding detailed analysis of the top vendors in the market. The market size calculation also includes distribution channel segmentation determined using secondary sources and verified through primary sources.
Secondary Research
The secondary research source that are typically referred to include, but are not limited to:
Company websites, annual reports, financial reports, broker reports, investor presentations and SEC filings
Internal and external proprietary databases, relevant patent and regulatory databases
National government documents, statistical databases and market reports
News articles, press releases and web-casts specific to the companies operating in the market
The source for secondary research includes but is not limited to: Factiva, Hoovers and Statista
Inner Circle Represents – Stage of Research Process
Middle Circle Represents – Source of Information
Outer Circle Represents – Information Derived from that source
Primary Research We conduct primary interviews on an ongoing basis with industry participants and commentators in order to validate data and analysis. A typical research interview fulfills the following functions:
It provides first-hand Information on the market size, market trends, growth trends, competitive landscape, future outlook etc.
Helps in validating and strengthening the secondary research findings
Further develops the analysis team’s expertise and market understanding
Primary research involves E-mail interactions, telephonic interviews as well as face-to-face interviews for each market, category, segment and sub-segment across geographies
The participants who typically take part in such a process include, but are not limited to:
Industry participants: CEOs, VPs, marketing/ distribution channel managers, market intelligence managers and national sales managers
Purchasing managers, technical personnel, distributors and resellers
Outside experts: Investment bankers, valuation experts, research analysts specializing in specific markets
Key opinion leaders specializing in different areas corresponding to different industry distribution channels
Models Where no hard data is available, we use modeling and estimates in order to produce comprehensive data sets. A rigorous methodology is adopted in which the available hard data is cross referenced with the following data distribution channels to produce estimates:
Demographic data: Population split by segment
Macro-economic indicators: GDP, etc.
Industry indicators: Expenditure, distribution channel stage & infrastructure, sector growth and facilities.
Data is then cross checked by the expert panel.
2..1 Company Share Analysis Model
Company share analysis is used to derive the size of Global market. As well as study of revenues of companies for last three to five years also provide the base for forecasting the market size and its growth rate. This model is built in following steps: 2..2 Revenue Based Modeling
Revenue based models can be built in two ways – Top-Down or Bottom-Up irrespective of industry. Market size estimated from company share analysis acts as a validation point for bottom-up approach where as it acts as starting point for top-down approach.
2.1 Research Limitations Inflation is not a part of pricing in this report. Prices of Global United Kingdom RNA-Based Rare Disease Drug Market and its derivatives vary in each region and hence similar revenue ratio does not follow for each individual region. The same price for each distribution channel has been taken into account while estimating and forecasting market revenue Globally. Regional average price has been considered while breaking down this market by application in each region. This report provides market size of Global United Kingdom RNA-Based Rare Disease Drug Market for the past year and forecasts for the next six years. Global United Kingdom RNA-Based Rare Disease Drug Market size is given in terms of revenue Market revenue is defined in USD Million. Market numbers are given on the basis of different Global United Kingdom RNA-Based Rare Disease Drug Market categories. Market size and forecasts for each major application is provided in the context of Global market. The numbers provided in this report are derived on the basis of demand for Global United Kingdom RNA-Based Rare Disease Drug Market from different application industries in different regions.
