RNA Editing for Neurodegenerative Diseases Market Size, Share and Trends 2025 to 2034

The RNA editing for neurodegenerative diseases market is witnessing rapid growth as researchers develop precise RNA-based therapies for Alzheimer’s, Parkinson’s, and other disorders. These treatments offer targeted interventions without permanent DNA changes. The market sizing and forecasts are revenue-based (USD Million/Billion), with 2024 as the base year.

Last Updated : 15 Sep 2025  |  Report Code : 6771  |  Category : Healthcare   |  Format : PDF / PPT / Excel

List of Contents

  • Last Updated : 15 Sep 2025
  • Report Code : 6771
  • Category : Healthcare

RNA Editing for Neurodegenerative Diseases Market Size and Forecast 2025 to 2034

The RNA editing for neurodegenerative diseases market is driven by breakthroughs in RNA biology, delivery systems, and precision medicine. It represents a transformative approach to treating complex neurological conditions. The rapid advancements in RNA-based therapies, the increasing prevalence of neurodegenerative diseases, supportive government framework, and surge in healthcare expenditure are expected to drive the growth of the global RNA editing for neurodegenerative diseases market over the forecast period. In this global market, North America dominated the market, fuelled by the surge in clinical trials and the increasing presence of biotech hubs.

RNA Editing for Neurodegenerative Diseases Market Size 2025 to 2034

RNA Editing for Neurodegenerative Diseases Market Key Takeaways

  • North America led the RNA editing for neurodegenerative diseases market in 2024.
  • Asia Pacific is expected to be the fastest-growing region in the upcoming years.
  • By editing modality/mechanism, the ADAR-mediated RNA base editing segment registered its dominance over the global RNA editing for neurodegenerative diseases market in 2024.
  • By editing modality/mechanism, the RNA Exon editing segment is expected to grow with the highest CAGR in the market during the studied years.
  • By product type, the single-molecule RNA editors segment held the largest revenue share in the market in 2024.
  • By product type, the vector-encoded RNA editors segment is expected to show the fastest growth over the forecast period.
  • By target neurodegenerative disease, the alzheimer’s disease segment held a dominant presence in the RNA editing for neurodegenerative diseases market in 2024.
  • By target neurodegenerative disease, the amyotrophic lateral sclerosis (ALS) segment is expected to witness the fastest growth in the market over the forecast period.
  • By end-user, the large biopharmaceutical companies segment contributed the biggest revenue share in the market in 2024.
  • By end-user, the contract research organizations (CROs) segment is expected to grow at the fastest CAGR in the market during the forecast period.

Market Overview

The RNA editing for neurodegenerative diseases market involves the development, commercialization, and deployment of therapeutic solutions and supporting technologies that utilize RNA editing techniques to treat or manage neurodegenerative disorders. These therapies leverage tools such as ADAR-mediated base editing, exon editing, programmable oligonucleotides, and RNA-guided deaminases to modulate RNA transcripts linked to diseases like Alzheimer’s, Parkinson’s, Huntington’s, ALS, and frontotemporal dementia. The market also includes delivery systems (AAV, LNP, conjugates), companion diagnostics, manufacturing services, and clinical infrastructure supporting RNA editing-based therapeutics.

How Can Artificial Intelligence Enhance RNA Editing for Neurodegenerative Diseases?

In the evolving technological landscape, Artificial intelligence emerges as a transformative force. It holds potential for growth and innovation in RNA editing for neurodegenerative diseases market by significantly improving the precision, safety, and efficiency of RNA editing.  AI algorithms assist in identifying distinct RNA editing patterns that act as biomarkers for various neurodegenerative conditions such as Alzheimer's, Amyotrophic Lateral Sclerosis (ALS), and Parkinson's disease. Through analyzing the huge datasets of RNA sequences, AI offers specific editing site changes that can correlate with disease onset or progression. The integration of Artificial Intelligence (AI) can accelerate the drug discovery process for neurodegenerative diseases by identifying and validating potential therapeutic targets and significantly improving the design of RNA editing tools. Therefore, the integration of AI is likely to accelerate the discovery, optimization, and delivery of RNA-based therapies for treating neurodegenerative diseases.

  • The rising RNA research funding and surge in clinical trials are expected to contribute to the overall growth of the RNA editing for neurodegenerative diseases market.
  • The increasing recognition of RNA editing as a safer and more reversible alternative is expected to fuel the expansion of the advanced antenna systems market during the forecast period.
  • The increasing investment in the pipeline of innovative therapies for neurodegenerative diseases is estimated to promote the growth of RNA editing for neurodegenerative diseases during the forecast period.
  • The rising aging population and increasing Central Nervous System (CNS) burden are anticipated to accelerate the market’s growth during the forecast period.

Market Scope

Report Coverage Details
Dominating Region North America
Fastest Growing Region Asia Pacific
Base Year 2024
Forecast Period 2025 to 2034
Segments Covered Editing Modality, Target Neurodegenerative Disease, Product Type, End-User, and Region
Regions Covered North America, Europe, Asia-Pacific, Latin America, and Middle East & Africa

Market Dynamics

Drivers

Prevalence of Neurodegenerative Diseases

The high incidence and prevalence of conditions such as Alzheimer's and Parkinson's create a large patient population that requires effective treatments. The market is witnessing a subsequent rise in neurodegenerative diseases globally, such as Alzheimer’s Disease, Parkinson’s Disease, Huntington’s Disease, Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), Spinocerebellar Ataxias, and other Rare CNS Disorders, which has led to an increasing demand for innovative treatments. RNA editing holds promising therapeutic potential for various neurodegenerative diseases by offering a reversible, post-transcriptional method to correct disease-causing mutations or modulate protein function without altering the genome. The use of RNA editing technologies enables highly targeted and disease-modifying therapies that address the root causes of various neurodegenerative health conditions. The global rise in the geriatric population leads to an increase in several age-related neurodegenerative diseases, which is expected to fuel the market’s expansion during the forecast period.

Restraint

High Cost of Technologies Hinders Growth in the RNA Editing for Neurodegenerative Diseases Market

The high cost associated with developing RNA editing technologies for neurodegenerative diseases is anticipated to hamper the market's growth. The high upfront cost is required for research and development activities and clinical trials. The high cost of RNA editing technologies often results in slow adoption in several low and middle-income countries. Additionally, a significant challenge for the safe and effective delivery of RNA-based therapies to the brain may hinder the growth of the global RNA editing market for neurodegenerative diseases during the forecast period.

Opportunity

How Is the Rapid Advancement in RNA Editing Tools Impacting the Market Expansion for Neurological Treatments?

The rapid advancement in RNA editing tools is projected to offer lucrative growth opportunities to the dispenser pump market in the coming years. The advancements in RNA editing tools, including CRISPR-based systems and ADAR-mediated editing, create the demand for innovative treatments of neurodegenerative diseases. CRISPR-Cas13 directly targets and modifies RNA, offering a promising and distinct therapeutic pathway distinct from DNA-focused gene editing.  CRISPR-based technology focuses on delivering RNA molecules to specific locations within neurons to repair damage and significantly promote regrowth. Moreover, the surge in investments in neuroscience research and development activities, along with rising advancements in neuroimaging, is driving the market’s growth. Therefore, the advancements in RNA editing technologies offer precise control over gene expression and hold promising therapeutic potential, fuelling the expansion of the market in the coming years.

Editing Modality / Mechanism Insights

Which Editing Modality Led the RNA Editing for Neurodegenerative Diseases Market in 2024?

In 2024, the ADAR-mediated RNA base editing segment gained dominance, driven by the therapeutic potential of ADAR-mediated RNA base editing in correcting mutations. ADAR-mediated RNA base editing is the clinically most advanced and widely adopted platform. ADAR-mediated editing provides a promising and reversible alternative to DNA editing and uses the cell's natural mechanisms, which significantly enhances its safety profile. On the other hand, the RNA exon editing segment is expected to witness the fastest growth in the market over the forecast period. RNA exon editing can correct multi-kilobase defective regions in neurodegenerative diseases. RNA exon editing can target genes too large for delivery vectors and correct disorders with high variability in mutation types.

Product Type Insights

What Causes the Single-Molecule RNA Editors Segment to Dominate the Market in 2024?

The single-molecule RNA editors (synthetic oligonucleotides) segment was dominant, with the biggest share of the global RNA editing for neurodegenerative diseases market in 2024. Synthetic oligonucleotide editors are easier to manufacture and have lower regulatory hurdles. Single-molecule RNA editing offers an innovative approach to correcting aberrant RNA sequences, thereby restoring the function of proteins in neurodegenerative diseases. On the other hand, the vector-encoded RNA editors (AAV/LV Platforms) segment is expected to register the fastest growth due to the potential therapeutics of RNA editing to correct disease-causing mutations and modulate protein function in the Central Nervous System (CNS). Vector-encoded RNA editors play an essential role in delivering RNA editing components into cells for therapeutic purposes.

Target Neurodegenerative Disease Insights

Why Did the Alzheimer’s Disease Segment Hold the Largest Share in the RNA Editing for Neurodegenerative Diseases Market During 2024?

The alzheimer’s disease segment led the RNA editing for neurodegenerative diseases market in 2024, owing to the rising cases of alzheimer’s disease globally and increasing research funding into RNA editing as a potential therapeutic target. Several preclinical studies indicated that RNA editing can be a therapeutic pathway for Alzheimer's disease. Numerous biotechnology companies are increasingly investing in developing RNA editing platforms for Alzheimer's disease. On the other hand, the amyotrophic lateral sclerosis (ALS) segment is anticipated to register rapid expansion in the predicted timeframe. The early RNA editing breakthroughs and orphan drug incentives mainly drive the growth of the segment. Amyotrophic lateral sclerosis (ALS) is a nervous system disease that affects or destroys nerve cells in the brain and spinal cord, which often causes loss of muscle control. RNA editing offers a promising approach for treating Amyotrophic Lateral Sclerosis (ALS). RNA editing in ALS is an emerging and promising area of therapeutic development, supported by the discovery of dysregulated RNA editing (specifically A-to-I editing).

End-User Insights

What Has Led the Large Biopharmaceutical Companies to Dominate the RNA Editing for Neurodegenerative Diseases Market in 2024?

In the RNA editing for neurodegenerative diseases market, the large biopharmaceutical companies segment led the market with the highest share in 2024. Large biopharmaceutical companies lead in funding, clinical development, and commercialization. Large biopharmaceutical companies such as Airna, Korro Bio, and Wave Life Sciences are widely involved in developing RNA-targeting therapies. These companies are increasingly focusing on advancing their own programs in neurodegenerative diseases and are receiving significant funding to move into clinical trials. On the other hand, the contract research organizations (CROs) are predicted to expand fastest in the coming years. The segment with the fastest growth is driven by the surge in outsourcing that accelerates RNA editing trials. The RNA editing for neurodegenerative diseases market is rapidly expanding, with CROs playing a crucial role by providing specialized expertise in areas such as target identification, clinical trial management, and drug development for the innovative therapies in neurodegenerative diseases like Alzheimer's and Parkinson's disease.

Regional Insights

What Made North America Dominate the RNA Editing for Neurodegenerative Diseases Market in 2024?

In 2024, North America held a dominant presence in the RNA editing for neurodegenerative diseases market. This region holds a strong position in the RNA editing for neurodegenerative diseases market, with the presence of thriving biopharmaceutical companies, an accelerating emphasis on healthcare spending, surging R&D investments by key players, and an increasing number of clinical trials. The increasing prevalence of neurodegenerative diseases, such as Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and Amyotrophic Lateral Sclerosis (ALS), along with the rapid advancement in RNA editing technologies like CRISPR-Cas13 and ADAR-mediated editing, is expected to accelerate the market’s revenue growth during the forecast period. The United States is a major contributor to the growth of the RNA editing for neurodegenerative diseases market. The presence of modern research institutions and biopharmaceutical and pharmaceutical companies in the country is anticipated to boost commercialization in the country. The country has a well-established healthcare infrastructure, increasing investment in advanced therapies, rising cases of neurodegenerative diseases, demand for personalized medicine, high per capita healthcare expenditure, and expanding applications of RNA editing technologies.

  • In May 2025, Stanford researchers developed a technology that delivers RNA to damaged neurons and stimulates regrowth, paving the way for potential treatments targeting neurodegenerative diseases like Amyotrophic Lateral Sclerosis (ALS) and spinal muscular atrophy. New CRISPR technology could help repair damaged neurons (Source: https://news.stanford.edu)

Which Factors are Going to Play a Crucial Role in the Asia Pacific Expansion?

Asia Pacific is expected to grow at the fastest rate in the market during the forecast period. The Asia Pacific market is expanding steadily, driven by the development of healthcare infrastructure, increasing R&D investments, rising government funding, growing demand for personalized medicine, advancements in biotechnology, favorable reimbursement policies, a surge in clinical trials, and expanding regulatory approvals. The rising prevalence of neurodegenerative diseases and the rising aging population in the region create a significant medical need and a substantial market growth for innovative therapies. RNA editing for neurodegenerative diseases has the potential to precisely correct genetic mutations. The market is experiencing rapid advancements in RNA editing technologies, like CRISPR-based platforms and RNA interference (RNAi) are paving the way for more effective and targeted treatments and increasingly expanding therapeutic possibilities for neurodegenerative diseases in the region. Such a combination of factors is expected to bolster the expansion of RNA editing for neurodegenerative diseases market in the Asia Pacific region.

RNA Editing for Neurodegenerative Diseases Market Companies

RNA Editing for Neurodegenerative Diseases Market Companies
  • ADARx Pharmaceuticals
  • Amber Bio
  • Ascidian Therapeutics
  • Beam Therapeutics
  • Eli Lilly & Co.
  • GSK Plc
  • Korro Bio
  • Moderna Inc.
  • ProQR Therapeutics
  • Roche Holding AG
  • Shape Therapeutics
  • Takeda Pharmaceutical Company
  • Verve Therapeutics
  • Wave Life Sciences
  • Regeneron Pharmaceuticals

Latest Announcements by Industry Leaders

  • In May 2025, Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, announced the formation of its Scientific Advisory Board (SAB). These globally recognized leaders in RNA biology and gene expression will provide their collective expertise on scientific direction, translational priorities, and research strategy as Ascidian builds an industry-leading portfolio of RNA editing medicines. (Source: https://www.prnewswire.com)

Recent Developments

  • In June 2024, Ascidian Therapeutics’ technology edits RNA to treat inherited disorders. While the startup’s lead internal program focuses on the retinal disorder Stargardt disease, its new R&D alliance with Roche will concentrate on developing RNA-editing therapies for neurological indications. Biotechnology research has brought patients therapies made by editing DNA. Roche aims to be part of the next wave of genetic medicines with a focus on RNA. Its latest move in this strategy is a partnership with Ascidian Therapeutics, a startup with technology that edits RNA to address multiple mutations driving a disease.
    (Source: https://medcitynews.com)
  • In April 2025, Ono Pharmaceutical, a Japan-based pharmaceutical company, partnered with US-based biotechnology company Jorna Therapeutics to develop novel RNA-editing therapeutics. It allows Ono Pharma to explore nucleic acid sequences as potential drug candidates, enhancing its capabilities in the pharmaceutical sector. (Source: https://www.worldpharmaceuticals.net)

Segments Covered in the Report

By Editing Modality

  • ADAR-Mediated RNA Base Editing
  • RNA Exon Editing / Trans-Splicing
  • Programmable Oligonucleotide Editors
  • RNA-Guided Deaminases & Engineered Editing Enzymes
  • Transient Knockdown + Reprogramming Approaches

By Target Neurodegenerative Disease

  • Alzheimer’s Disease
  • Parkinson’s Disease
  • Huntington’s Disease
  • Amyotrophic Lateral Sclerosis (ALS)
  • Frontotemporal Dementia (FTD)
  • Spinocerebellar Ataxias & Other Rare CNS Disorders

By Product Type

  • Single-Molecule RNA Editors (Synthetic Oligonucleotides)
  • Vector-Encoded RNA Editors (AAV / LV Platforms)
  • Programmable RNA-Guided Editors (Engineered Deaminases)
  • Platform-Licensed Editing Programs

By End-User

  • Large Biopharmaceutical Companies
  • RNA Editing Biotech Startups
  • Academic Research Institutions
  • Contract Research Organizations (CROs)
  • Specialized Neurology Clinics & Hospitals

By Region

  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East and Africa

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Frequently Asked Questions

The major players in the RNA editing for neurodegenerative diseases market include ADARx Pharmaceuticals, Amber Bio, Ascidian Therapeutics, Beam Therapeutics, Eli Lilly & Co. (via Prevail Therapeutics), GSK Plc, Korro Bio, Moderna Inc., ProQR Therapeutics, Roche Holding AG, Shape Therapeutics, Takeda Pharmaceutical Company, Verve Therapeutics, Wave Life Sciences, and Regeneron Pharmaceuticals.

The driving factors of the RNA editing for neurodegenerative diseases market are the increasing prevalence of neurodegenerative diseases, supportive government framework, and surge in healthcare expenditure.

North America region will lead the global RNA editing for neurodegenerative diseases market during the forecast period 2025 to 2034.

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