The market for repeat expansion disorders treatment is expected to grow at a CAGR of 13.5% during the forecast period of 2025 to 2033. Repeat expansion disorders are a group of inherited conditions that are triggered by expansions in the DNA repeats of affected individuals. The length of the DNA repeats can range from a single nucleotide all the way up to a dodecamer or even longer. The point at which the repetitive expansions start to cause symptoms differs from one disease to another depending on what it is. These expansions in DNA sequence are now known to be the root cause of over 40 different illnesses.
The causal mutations that drive pathogenesis in a substantial number of these disorders have been recognised thanks to remarkable progress that has been made over the past three decades. The expansion of CAG, GCG, CTG, CGG, and CAAA repeats both in coding and non-coding regions in separate genes results in a wide set of disorders with processes associated to protein levels or toxicity, RNA, and/or both. These repeats can be found in either coding or non-coding sequences.
These new lines of inquiry should not only elucidate the molecular mechanisms of disease, but also highlight opportunities for therapeutic intervention in the treatment of conditions that are currently incurable. Despite of these advancements, there are challenges - just as there were when the science was just starting out - in pinpointing the precise manner in which repetitive expansion mutations lead to inherited disorders. As a result, the classification schemes that are currently in use run the risk of becoming obsolete in the near future.
In point of fact, a number of discoveries made relatively recently have shed light on the potential complexity of the molecular pathways underpinning the pathogenesis of a variety of diseases caused by repeat expansions. For instance, extensive research into diseases known as CAG-polyglutamine diseases has shown that post-translational modification of disease proteins is an important step in the progression of the disease, and there is now evidence to suggest that the autophagy pathway plays an important part in the breakdown of misfolded proteins.
Why the development of relevant treatments has not kept pace with the extraordinary trajectory of scientific advance in the understanding of mechanisms and pathways is an issue that keeps coming up in the field of recurrent disease. This disconnect applies to the vast majority of neurological disorders; nevertheless, repeat diseases present a fantastic potential for the development of reasonable therapies due to the fact that conclusive diagnosis is typically predicated upon repeat allele expansion. These discoveries have also led to some very encouraging therapeutic developments, such as the utilisation of oligonucleotide and morpholino anti-sense repeats targeted against the DM1 CTG expansion.
These factors make it difficult to identify reliable outcomemeasures to monitor the progression of the disorders.For instance, Friedreich's ataxia is a genetic, neurodegenerative movement disorder, which, according to an article that was published in the Orphanet Journal of Rare Diseases in August 2020, has a typical age of onset (the age at which the symptoms of a disease first appear) that ranges between 10 and 15 years. This age range was determined by taking the average age of patients diagnosed with Friedreich's ataxia. As a result, it takes a significant amount of time from the disease's onset until it can be properly diagnosed.
There is a greater than tenfold difference in prevalence based on geographic location, which may be due to differences in case ascertainment and diagnostic criteria. While the populations of Europe, North America, and Australia have a larger incidence, the prevalence among the Asian population has generally been lower. Targeting HD's hyperactive immune system with immunomodulatory medicines is one of the primary focuses of a number of small molecules now undergoing clinical development.
Specifically, the high burden of the disease is causing increased healthcare expenditure.Due to increased product penetration, untapped potential, and the growing prevalence of the conditions in emerging nations throughout the projection period, the market for Huntington's disease treatment is expected to exhibit the highest CAGR in Asia Pacific during the forecast period of 2025 to 2033. It is anticipated that an increasing number of measures being implemented by governments to enhance the health status of the population will fuel the expansion of the market in the region. However, there is a lot of competition in the market for treatments for Huntington's disease, which may slow down its expansion in this region.
Some of the prominent companies dominating the market includes Pfizer, Inc., CRISPR Therapeutics, Acorda Therapeutics, Capsida Biotherapeutics Inc., Larimar Therapeutics, Inc., Healx, Intellia Therapeutics, Inc., Editas Medicine, bluebird bio, Inc., Sanofi, Merck & Co., Inc., H. Lundbeck A/S, Cellectis SA, Bio-Techne, GlaxoSmithKline plc, AllerganAurobindo Pharma Ltd., Sun Pharmaceutical Industries Ltd., Torrent Pharmaceuticals Ltd., Intas Pharmaceuticals Ltd., Cipla Ltd and others.
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The causal mutations that drive pathogenesis in a substantial number of these disorders have been recognised thanks to remarkable progress that has been made over the past three decades. The expansion of CAG, GCG, CTG, CGG, and CAAA repeats both in coding and non-coding regions in separate genes results in a wide set of disorders with processes associated to protein levels or toxicity, RNA, and/or both. These repeats can be found in either coding or non-coding sequences.
Increasing Research of Rapid Expansion Disorders Therapies is Boosting the Market
Because of the complexity of repetitive disease genetics and pathobiology, previously unknown commonalities and molecular mechanisms associated with the diseases, such as RNA toxicity, have been uncovered. The investigation of polyglutamine diseases has also shown that post-translational modification is an important step in the pathogenic cascade, and it has demonstrated that the autophagy pathway plays an important role in the degradation of misfolded proteins. These are two themes that are likely to be applicable to the entire field of neurodegeneration. Research on recurrent diseases is yielding insights that are sparking new research avenues.These new lines of inquiry should not only elucidate the molecular mechanisms of disease, but also highlight opportunities for therapeutic intervention in the treatment of conditions that are currently incurable. Despite of these advancements, there are challenges - just as there were when the science was just starting out - in pinpointing the precise manner in which repetitive expansion mutations lead to inherited disorders. As a result, the classification schemes that are currently in use run the risk of becoming obsolete in the near future.
In point of fact, a number of discoveries made relatively recently have shed light on the potential complexity of the molecular pathways underpinning the pathogenesis of a variety of diseases caused by repeat expansions. For instance, extensive research into diseases known as CAG-polyglutamine diseases has shown that post-translational modification of disease proteins is an important step in the progression of the disease, and there is now evidence to suggest that the autophagy pathway plays an important part in the breakdown of misfolded proteins.
Market to Present Significant Opportunities During the Forecast Period
Since the first discovery of a novel and obscure type of mutation as the root cause of fragile X syndrome and spinal muscular atrophy (SBMA), a significant amount of progress has been made. The research that has been done in this area has uncovered an extraordinary unanticipated complexity, which includes toxicities that are mediated at the protein, RNA, and genome levels. Research on recurrent diseases, for instance, continues to be one of the most important factors in determining how faulty RNA metabolism and autophagy contribute to human illness.Why the development of relevant treatments has not kept pace with the extraordinary trajectory of scientific advance in the understanding of mechanisms and pathways is an issue that keeps coming up in the field of recurrent disease. This disconnect applies to the vast majority of neurological disorders; nevertheless, repeat diseases present a fantastic potential for the development of reasonable therapies due to the fact that conclusive diagnosis is typically predicated upon repeat allele expansion. These discoveries have also led to some very encouraging therapeutic developments, such as the utilisation of oligonucleotide and morpholino anti-sense repeats targeted against the DM1 CTG expansion.
Clinical Trial Process for Lack of Patient Pool Presents Complexities in Designing the Therapies
It is anticipated that factors such as difficulties encountered in the process of designing and performing clinical trials for the development of a treatment for rapid expansion disorders treatment market would be among those that will impede the growth of the market during the forecast period. It is challenging to design clinical trials for rare neurodegenerative diseases for instance, Friedreich's ataxia because there are not enough patients with the disease, the disease is heterogeneous, and it has a slow and variable progression rate.These factors make it difficult to identify reliable outcomemeasures to monitor the progression of the disorders.For instance, Friedreich's ataxia is a genetic, neurodegenerative movement disorder, which, according to an article that was published in the Orphanet Journal of Rare Diseases in August 2020, has a typical age of onset (the age at which the symptoms of a disease first appear) that ranges between 10 and 15 years. This age range was determined by taking the average age of patients diagnosed with Friedreich's ataxia. As a result, it takes a significant amount of time from the disease's onset until it can be properly diagnosed.
Huntington Disease Treatment Market Presenting Largest Opportunities
Huntington disease treatment market among other rapid expansion disorders treatment is expected grow the fastest registering a CAGR of 20% during the forecast period 2025 to 2033. It is estimated that important drivers during the forecast period will include the planned label expansion of Ingrezza for the treatment of chorea associated with Huntington's disease, the high burden of HD in western countries, and a robust product pipeline of disease-modifying treatments. There are around 30,000 persons in North America who have HD, and the disease has a prevalence of 5.7 cases per 100,000 people. HD in children is significantly less common than in adults, accounting for only approximately 5-10% of all cases. It affects as many as 10 people out of every 1,00,000 people in Europe.There is a greater than tenfold difference in prevalence based on geographic location, which may be due to differences in case ascertainment and diagnostic criteria. While the populations of Europe, North America, and Australia have a larger incidence, the prevalence among the Asian population has generally been lower. Targeting HD's hyperactive immune system with immunomodulatory medicines is one of the primary focuses of a number of small molecules now undergoing clinical development.
North America Remains as the Global Leader and APAC to Present the Largest Opportunity
In 2021, North America held the dominant position in the market for treatments for Huntington's disease, holding a revenue share of 60%. This expansion is due to the high burden of the disease, increased healthcare expenditure, technological advancements, proactive government initiatives, and increased patient awareness about treatments available for rapid expansion disorders treatment.Specifically, the high burden of the disease is causing increased healthcare expenditure.Due to increased product penetration, untapped potential, and the growing prevalence of the conditions in emerging nations throughout the projection period, the market for Huntington's disease treatment is expected to exhibit the highest CAGR in Asia Pacific during the forecast period of 2025 to 2033. It is anticipated that an increasing number of measures being implemented by governments to enhance the health status of the population will fuel the expansion of the market in the region. However, there is a lot of competition in the market for treatments for Huntington's disease, which may slow down its expansion in this region.
Intense R&D Focus to Enhance the Market Share of the Market Players
Market is fragmented due to the scope of market that is characterized by different pharma companies leading different therapeutic segment across countries.Therefore, the competition is significantly high and is expected further intensify during the forecast period of 2025 to 2033. The key strategy adopted by Tier 1 companies is focus on R&D to develop novel therapeutic options.Some of the prominent companies dominating the market includes Pfizer, Inc., CRISPR Therapeutics, Acorda Therapeutics, Capsida Biotherapeutics Inc., Larimar Therapeutics, Inc., Healx, Intellia Therapeutics, Inc., Editas Medicine, bluebird bio, Inc., Sanofi, Merck & Co., Inc., H. Lundbeck A/S, Cellectis SA, Bio-Techne, GlaxoSmithKline plc, AllerganAurobindo Pharma Ltd., Sun Pharmaceutical Industries Ltd., Torrent Pharmaceuticals Ltd., Intas Pharmaceuticals Ltd., Cipla Ltd and others.
Historical & Forecast Period
- This study report represents an analysis of each segment from 2023 to 2033 considering 2024 as the base year. Compounded Annual Growth Rate (CAGR) for each of the respective segments estimated for the forecast period of 2025 to 2033.
- The current report comprises quantitative market estimations for each micro market for every geographical region and qualitative market analysis such as micro and macro environment analysis, market trends, competitive intelligence, segment analysis, porters five force model, top winning strategies, top investment markets, emerging trends & technological analysis, case studies, strategic conclusions and recommendations and other key market insights.
Research Methodology
- The complete research study was conducted in three phases, namely: secondary research, primary research, and expert panel review. The key data points that enable the estimation of Repeat Expansion Disorders Treatment market are as follows:
- Research and development budgets of manufacturers and government spending
- Revenues of key companies in the market segment
- Number of end users & consumption volume, price, and value.
- Geographical revenues generate by countries considered in the report
- Micro and macro environment factors that are currently influencing the Repeat Expansion Disorders Treatment market and their expected impact during the forecast period.
- Market forecast was performed through proprietary software that analyzes various qualitative and quantitative factors. Growth rate and CAGR were estimated through intensive secondary and primary research. Data triangulation across various data points provides accuracy across various analyzed market segments in the report. Application of both top-down and bottom-up approach for validation of market estimation assures logical, methodical, and mathematical consistency of the quantitative data.
Market Segmentation
Disorders- Dentatorubral-pallido-luysian atrophy (DRPLA)
- Fragile X syndrome ofmental retardation
- Fragile X tremor ataxia syndrome (FXTAS)
- FRAXE mental retardation (FMR2)
- Spinal and Bulbar Muscular Atrophy/Kennedy's Disease
- type1 (SCA1)
- type 2 (SCA2)
- type 3 (SCA3)
- type 6 (SCA6)
- type 7 (SCA7)
- type 8 (SCA8)
- type 10 (SCA10)
- type 12 (SCA12)
- type 17 (SCA17)
- Huntington's Disease
- Oculopharyngeal musculardystrophy (OPMD)
- Myoclonic epilepsy type 1
- Myotonic dystrophy
- Type 1
- Type 2
- Spinocerebellar ataxia
- Friedreich Ataxia
- Amyotrophic lateral sclerosis (ALS)
- Frontotemporal dementia (FTD).
- Myoclonic epilepsy
- Syndromic / non-syndromicX-linked mental retardation(Gene: ARX)
- Others
- Oral
- Injectable
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
- Spinal and Bulbar Muscular Atrophy/Kennedy's Disease
- Huntington's Disease
- spinocerebellar ataxia
- Friedreich Ataxia
- amyotrophic lateral sclerosis (ALS)
- frontotemporal dementia (FTD)
Region Segment (2023 - 2033; US$ Million)
- North America
- U.S.
- Canada
- Rest of North America
- UK and European Union
- UK
- Germany
- Spain
- Italy
- France
- Rest of Europe
- Asia Pacific
- China
- Japan
- India
- Australia
- South Korea
- Rest of Asia Pacific
- Latin America
- Brazil
- Mexico
- Rest of Latin America
- Middle East and Africa
- GCC
- Africa
- Rest of Middle East and Africa
Key questions answered in this report
- What are the key micro and macro environmental factors that are impacting the growth of Repeat Expansion Disorders Treatment market?
- What are the key investment pockets concerning product segments and geographies currently and during the forecast period?
- Estimated forecast and market projections up to 2033.
- Which segment accounts for the fastest CAGR during the forecast period?
- Which market segment holds a larger market share and why?
- Are low and middle-income economies investing in the Repeat Expansion Disorders Treatment market?
- Which is the largest regional market for Repeat Expansion Disorders Treatment market?
- What are the market trends and dynamics in emerging markets such as Asia Pacific, Latin America, and Middle East & Africa?
- Which are the key trends driving Repeat Expansion Disorders Treatment market growth?
- Who are the key competitors and what are their key strategies to enhance their market presence in the Repeat Expansion Disorders Treatment market worldwide?
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Table of Contents
1. Preface
2. Executive Summary
3. Repeat Expansion Disorders Treatment Market: Competitive Analysis
4. Repeat Expansion Disorders Treatment Market: Macro Analysis & Market Dynamics
5. Repeat Expansion Disorders Treatment Market: By Disorders, 2023-2033, USD (Million)
6. Repeat Expansion Disorders Treatment Market: By Route of Administration, 2023-2033, USD (Million)
7. Repeat Expansion Disorders Treatment Market: By Distribution Channel, 2023-2033, USD (Million)
8. Repeat Expansion Disorders Treatment Market: By Drug Pipeline (Profile), 2023-2033, USD (Million)
9. North America Repeat Expansion Disorders Treatment Market, 2023-2033, USD (Million)
10. UK and European Union Repeat Expansion Disorders Treatment Market, 2023-2033, USD (Million)
11. Asia Pacific Repeat Expansion Disorders Treatment Market, 2023-2033, USD (Million)
12. Latin America Repeat Expansion Disorders Treatment Market, 2023-2033, USD (Million)
13. Middle East and Africa Repeat Expansion Disorders Treatment Market, 2023-2033, USD (Million)
14. Company Profile
List of Figures
List of Tables
Companies Mentioned
- Pfizer, Inc.
- CRISPR Therapeutics
- Acorda Therapeutics
- Capsida Biotherapeutics Inc.
- Larimar Therapeutics, Inc.
- Healx, Intellia Therapeutics, Inc.
- Editas Medicine
- Bluebird bio, Inc.
- Sanofi, Merck & Co., Inc.
- H. Lundbeck A/S
- Cellectis SA
- Bio-Techne
- GlaxoSmithKline plc
- Allergan Aurobindo Pharma Ltd.
- Sun Pharmaceutical Industries Ltd.
- Torrent Pharmaceuticals Ltd.
- Intas Pharmaceuticals Ltd.
- Cipla Ltd
