CNS Specific Antisense Oligonucleotides: Introduction

  • Antisense oligonucleotides are single-stranded oligodeoxynucleotides that can alter the RNA and modify protein expression. These oligonucleotides were discovered to influence RNA processing.
  • Subsequent chemical modifications of antisense oligonucleotides were used to address issues such as off-target toxic effects and insufficient biological activity. These modifications led to an improved mechanism of antisense oligonucleotides and clinical trial design. This paved the way toward translating their use into therapies for various genetic and neurological conditions.
  • Antisense oligonucleotides mediated therapies target the source of pathogenesis and have higher chance of success as compared to therapies that target downstream pathways. These are drugs for the treatment of central nervous system (CNS) disorders, as they have specific molecular targeting and extended pharmacological properties.

Key Drivers and Restraints of Global CNS Specific Antisense Oligonucleotides Market

  • The global CNS specific antisense oligonucleotides market is primarily driven by advances in the understanding of pharmacology of antisense oligonucleotides, rapid development of next generation antisense oligonucleotide therapies, and an increase in the prevalence of neurological and genetic disorders
  • According to an article published in Orphanet Journal of Rare Diseases, estimated incidence of spinal muscular atrophy is 1 in 6,000 to 1 in 10,000 live births

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Spinal muscular atrophy segment to account for major share of global CNS specific antisense oligonucleotides market

  • In terms of indication, the global CNS specific antisense oligonucleotides market can be classified into hereditary transthyretin amyloidosis (hATTR)/ polyneuropathy, spinal muscular atrophy, and Huntington’s disease
  • The spinal muscular atrophy segment is anticipated to dominate the global market during the forecast period, due to availability of Food and Drug Administration (FDA) approved antisense oligonucleotides therapy for spinal muscular atrophy and a rise in focus on the development of treatment of rare genetic conditions
  • In December 2016, the FDA approved Spinraza, the first drug indicated for the treatment of spinal muscular atrophy in adult and pediatric patients. It is an antisense oligonucleotide utilized to treat spinal muscular atrophy caused by mutation of in chromosome 5q.

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Hospital pharmacies institutes segment to account for major share of global market

  • Based on distribution channel, the global CNS specific antisense oligonucleotides market can be categorized into hospital pharmacies, retail pharmacies, and online pharmacies
  • The hospital pharmacies segment is expected to account for a major share of the global CNS specific antisense oligonucleotides market by 2030, owing to an increase in number of patients with genetic disorders and a rise in awareness about the need for ensuring availability of approved therapies for these patients.

North America to dominate global CNS specific antisense oligonucleotides market

  • In terms of region, the global CNS specific antisense oligonucleotides market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is projected to dominate the global CNS specific antisense oligonucleotides market during the forecast period.
  • The market in North America is anticipated to be driven by an exponential increase in the number of pharmaceutical companies conducting clinical trials for the development of promising drugs, recent FDA approvals & product launch, and a rise in awareness about treatment of neurological conditions. GeneTx Biotherapeutics LLC received orphan-drug designation for GTX-101, indicated for the treatment of Angelman syndrome.

Key players operating in global CNS specific antisense oligonucleotides market

The global CNS specific antisense oligonucleotides market is highly fragmented, with a large number of domestic players accounting for a major share of the market. Key players operating in the global CNS specific antisense oligonucleotides market are:

  • Akcea Therapeutics, Inc.
  • Ionis Pharmaceuticals Inc.
  • Biogen Inc.
  • Sarepta Therapeutics
  • Wave Life Sciences Ltd.
  • Dynacure
  • ProQR Therapeutics N.V.
  • Stroke Therapeutic Inc.

Global CNS Specific Antisense Oligonucleotides Market: Research Scope

Global CNS Specific Antisense Oligonucleotides Market, by Indication

  • Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy
  • Spinal Muscular Atrophy
  • Huntington’s Disease

Global CNS Specific Antisense Oligonucleotides Market, by Distribution Channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies

Global CNS Specific Antisense Oligonucleotides Market, by Region

  • North America
    • U.S.
    • Canada
  • Europe
    • Germany
    • France
    • U.K.
    • Italy
    • Spain
    • Rest of Europe
  • Asia Pacific
    • China
    • Japan
    • India
    • Australia & New Zealand
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East & Africa
    • GCC Countries
    • South Africa
    • Rest of Middle East & Africa

This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
  • Insights and Tools based on data-driven research
  • Actionable Results to meet all the business priorities
  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

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

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

2. Insights and Tools

The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.

3. Actionable Results

The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

4. Strategic Frameworks

The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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