Gene Therapy For Neurodegenerative Diseases Market Size, Share, Opportunities, And Trends Type Of Neurogenerative Disease (Alzheimer's Disease, Parkinson's Disease, Huntington's Disease, Amyotrophic Lateral Sclerosis (Als), Multiple System Atrophy (Msa), Spinocerebellar Ataxias, Others), By Therapeutic Approach (Gene Replacement Therapy, Gene Silencing Therapy (Rnai), Gene Editing Therapy (Crispr-Cas9), Neurotrophic Factor Delivery, Enzyme Replacement Therapy), By Vector Type (Adeno-Associated Virus (Aav), Lentivirus, Retrovirus, Non-Viral Vectors), By End-User (Hospitals And Clinics, Research Institutions, Biotechnology Companies, Pharmaceutical Companies), And By Geography - Forecasts From 2023 To 2028

  • Published : Oct 2023
  • Report Code : KSI061615987
  • Pages : 152

The gene therapy for neurodegenerative diseases market was valued at US$81.016 million in 2021.

The gene therapy for neurodegenerative diseases market is a fast-growing sector in medical innovation, bringing fresh hope for treating diseases such as Alzheimer's, Parkinson's, and ALS. This market tries to address the fundamental causes of these chronic illnesses, which have long resisted established therapies, by leveraging the potential of gene editing. Gene therapy tries to delay or even reverse disease development by delivering therapeutic genes into afflicted cells. Advances in genetic research, improved delivery systems, and encouraging preclinical results are driving market expansion. With the world's population ageing and the frequency of neurodegenerative illnesses growing, gene therapy provides a transformational approach to addressing these difficult problems, with the potential to revolutionise the landscape of neurological care.

Gene Therapies Tailored to Specific Neurodegenerative Conditions in the Gene Therapy for Neurodegenerative Diseases Market.

Gene therapies customised to individual neurodegenerative disorders are precision medicines that target each disease's unique genetic foundations. These treatments target particular genes, proteins, or processes linked to diseases such as Alzheimer's, Parkinson's, and Huntington's disease. These personalised therapies try to repair or minimise the underlying causes of neurodegeneration by delivering therapeutic genes or changing existing ones. By concentrating on the underlying causes of the condition, this strategy improves therapy efficacy and decreases potential adverse effects. Personalised gene treatments show enormous promise for precision medicine, perhaps enabling a breakthrough in treating complicated neurodegenerative conditions with increased accuracy and better results.

Enhanced Vectors and Technologies for Efficient Gene Delivery Enhances the Gene Therapy for Neurodegenerative Diseases Market Growth.

Enhanced vectors and technologies are critical in delivering genes efficiently for enhanced therapies. Vectors, such as viral vectors or nanoparticles, are designed to precisely deliver therapeutic genes to target cells. These vectors have been optimised to improve gene transfer efficiency by increasing targeting specificity, cellular absorption, and stability. Synthetic viral vectors and genome editing technologies such as CRISPR-Cas9 allow for more precise and regulated gene insertion. This advancement guarantees that therapeutic genes are successfully integrated into the patient's DNA, encouraging long-term and effective expression. Enhanced delivery methods are critical for realising the full potential of gene therapies and, as a result, contributing to improved treatment results in a variety of medical scenarios, including neurodegenerative illnesses.

Recognition of Gene Therapy's Potential Benefits in the  Gene Therapy for Neurodegenerative Diseases Market.

Recognising the potential benefits of gene therapy represents a huge paradigm shift in medical thinking. Gene therapy is becoming more well-known, both within the scientific community and among the general public, for its ability to address the underlying causes of hereditary illnesses. Gene therapy provides promise for long-term treatments and even cures by directly changing or replenishing defective genes. Increased research funding, partnerships, and regulatory backing have resulted from this recognition, driving gene therapy into the mainstream medical discourse. The recognition of its potential advantages emphasises the revolutionary influence gene therapy might have on numerous diseases, creating optimism for future more effective, targeted, and personalised therapies.

North America is the Market Leader in the Gene Therapy for Neurodegenerative Diseases Market.

Recognising the potential benefits of gene therapy represents a huge paradigm shift in medical thinking. Gene therapy is becoming more well-known, both within the scientific community and among the general public, for its ability to address the underlying causes of hereditary illnesses. Gene therapy provides promise for long-term treatments and even cures by directly changing or replenishing defective genes. Increased research funding, partnerships, and regulatory backing have resulted from this recognition, driving gene therapy into the mainstream medical discourse. The recognition of its potential advantages emphasises the revolutionary influence gene therapy might have on numerous diseases, creating optimism for future more effective, targeted, and personalised therapies.

Cross-Sector Collaborations to Advance Gene Therapy Bolsters the Gene Therapy for Neurodegenerative Diseases Market Size.

Cross-sector partnerships are critical to the advancement of gene therapy. These collaborations harness varied viewpoints and resources by bringing together professionals from academia, industry, healthcare, and research institutes. They speed up research, boost technical innovation, and make it easier to turn promising discoveries into actual medicines. Collaborations between scientists, physicians, biotech businesses, and regulatory agencies speed up the development process, assuring solid research, rigorous clinical studies, and timely regulatory approval. This multidisciplinary approach promotes synergy, allowing for speedier progress in understanding disease causes, improving treatment methods, and eventually providing patients with safe and effective gene therapies. Collaborations across sectors are critical to realising gene therapy's full promise to revolutionise healthcare.

Key Developments:

  • In January 2023, uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with critical medical needs, and Apic Bio, an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, announced a global licencing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. The agreement grants uniQure global rights to develop and commercialise APB-102, adding to its portfolio of gene treatments for neurological illnesses. The US Food and Drug Administration approved APB-102's investigational new drug (IND) application and awarded Orphan Drug and Fast Track designations.
  • In June 2023, Sangamo Therapeutics, Inc., a genomic medicine business, and Voyager Therapeutics, Inc. announced a formal licence agreement for a possible prion disease therapy. Sangamo has produced zinc finger transcriptional regulators (ZF-TRs) using its patented epigenetic regulatory technology, which it thinks may selectively and potently limit the production of the prion protein, the pathogenic driver of prion disease. Sangamo's ZF-TRs have been proven in animal models to drastically lower prion protein production in the brain, lengthen life span, and inhibit harmful prion aggregation formation. Voyager has granted Sangamo access to a capsid generated from Voyager's proprietary TRACERTM platform under the terms of the agreement. In animal studies, this capsid has been demonstrated to successfully pass the blood-brain barrier when administered intravenously.
  • In 2020,  Eli Lilly and Company and Prevail Therapeutics Inc. announced a definitive agreement under which Lilly will acquire Prevail for $22.50 per share in cash (or an aggregate of approximately $880 million) payable at closing, plus one non-transferable contingent value right ("CVR") worth up to $4.00 per share in cash (or an aggregate of approximately $160 million), for a total consideration of up to $26.50 per share in cash (or an aggregate of approximately $1.040 billion). The CVR is payable (subject to specific restrictions and limitations) following the first regulatory approval of a Prevail pipeline product, as detailed below. Prevail is a biotechnology business that is working to produce possibly disease-modifying AAV9-based gene treatments for individuals suffering from neurodegenerative illnesses. 

Company Products:

  • Lysosomal Storage Disorders: While most lysosomal storage diseases affect diverse organs, some can also impact the neurological system and cause neurodegenerative symptoms. To slow disease development, Amicus' gene treatments may address these underlying genetic reasons.
  • Zinc Finger Protein Technology: For gene editing and control, Sangamo uses its patented zinc finger protein (ZFP) technology. This method allows for precise changes to particular genes linked to neurodegenerative disorders.
  • Viral Vector Delivery: Voyager often uses viral vectors, particularly adeno-associated viral (AAV) vectors, as gene therapy delivery vehicles. These vectors are designed to deliver therapeutic genes into afflicted brain cells.
  • Clinical Pipeline: UniQure has been focusing on gene treatments for neurological illnesses including Huntington's. Their clinical pipeline includes experimental treatments aimed at addressing the underlying genetic origins of various illnesses.

Key Segment:

  • By Type Of Neurogenerative Disease
    • Alzheimer's Disease
    • Parkinson's Disease
    • Huntington's Disease
    • Amyotrophic Lateral Sclerosis (Als)
    • Multiple System Atrophy (Msa)
    • Spinocerebellar Ataxias
    • Others       
  • By Therapeutic Approach
    • Gene Replacement Therapy
    • Gene Silencing Therapy (Rnai)
    • Gene Editing Therapy (Crispr-Cas9)
    • Neurotrophic Factor Delivery
    • Enzyme Replacement Therapy
  • By Vector Type
    • Adeno-Associated virus (Aav)
    • Lentivirus
    • Retrovirus
    • Non-Viral Vectors 
  • By End-User
    • Hospitals And Clinics
    • Research Institutions
    • Biotechnology Companies
    • Pharmaceutical Companies 
  • By Geography
    • North America
      • United States
      • Canada
      • Mexico
    • South America
      • Brazil
      • Argentina
      • Others
    • Europe
      • United Kingdom
      • Germany
      • France
      • Italy
      • Spain   
      • Others
    • Middle East and Africa
      • Saudi Arabia
      • UAE
      • Others
    • Asia Pacific
      • Japan
      • China
      • India
      • South Korea
      • Taiwan
      • Thailand
      • Indonesia
      • Others

1. INTRODUCTION

1.1. Market Overview

1.2. Market Definition

1.3. Scope of the Study

1.4. Market Segmentation

1.5. Currency

1.6. Assumptions

1.7. Base, and Forecast Years Timeline

2. RESEARCH METHODOLOGY  

2.1. Research Data

2.2. Sources

2.3. Research Design

3. EXECUTIVE SUMMARY

3.1. Research Highlights

4. MARKET DYNAMICS

4.1. Market Drivers

4.2. Market Restraints

4.3. Porters Five Forces Analysis

4.3.1. Bargaining Power of Suppliers

4.3.2. Bargaining Power of Buyers

4.3.3. Threat of New Entrants

4.3.4. Threat of Substitutes

4.3.5. Competitive Rivalry in the Industry

4.4. Industry Value Chain Analysis

5.  GENE THERAPY FOR NEURODEGENERATIVE DISEASES MARKET, BY TYPE OF NEUROGENERATIVE DISEASE

5.1. Introduction

5.2. Alzheimer's Disease

5.3. Parkinson's Disease

5.4. Huntington's Disease

5.5. Amyotrophic Lateral Sclerosis (ALS)

5.6. Multiple System Atrophy (MSA)

5.7. Spinocerebellar Ataxias

5.8. Others                            

6. GENE THERAPY FOR NEURODEGENERATIVE DISEASES MARKET, BY THERAPEUTIC APPROACH

6.1. Introduction

6.2. Gene Replacement Therapy

6.3. Gene Silencing Therapy (RNAi)

6.4. Gene Editing Therapy (CRISPR-Cas9)

6.5. Neurotrophic Factor Delivery

6.6. Enzyme Replacement Therapy                       

7. GENE THERAPY FOR NEURODEGENERATIVE DISEASES MARKET, BY VECTOR TYPE

7.1. Introduction

7.2. Adeno-Associated Virus (AAV)

7.3. Lentivirus

7.4. Retrovirus

7.5. Non-Viral Vectors                                            

8. GENE THERAPY FOR NEURODEGENERATIVE DISEASES MARKET BY END-USER

8.1. Introduction

8.2. Hospitals and Clinics

8.3. Research Institutions

8.4. Biotechnology Companies

8.5. Pharmaceutical Companies    

9.  GENE THERAPY FOR NEURODEGENERATIVE DISEASES MARKET, BY GEOGRAPHY

9.1. Introduction

9.2. North America

9.2.1. United States

9.2.2. Canada

9.2.3. Mexico

9.3. South America

9.3.1. Brazil

9.3.2. Argentina

9.3.3. Others

9.4. Europe

9.4.1. United Kingdom

9.4.2. Germany

9.4.3. France

9.4.4. Italy

9.4.5. Spain 

9.4.6. Others

9.5. Middle East and Africa

9.5.1. Saudi Arabia

9.5.2. UAE

9.5.3. Others

9.6. Asia Pacific

9.6.1. Japan

9.6.2. China

9.6.3. India

9.6.4. South Korea

9.6.5. Indonesia 

9.6.6. Taiwan

9.6.7. Others

10. COMPETITIVE ENVIRONMENT AND ANALYSIS

10.1. Major Players and Strategy Analysis

10.2. Emerging Players and Market Lucrativeness

10.3. Mergers, Acquisitions, Agreements, and Collaborations

10.4. Vendor Competitiveness Matrix

11. COMPANY PROFILES

11.1. Voyager Therapeutics

11.2. uniQure N.V.

11.3. Axovant Gene Therapies

11.4. Novartis AG

11.5. Amicus Therapeutics

11.6. REGENXBIO Inc.

11.7. Sarepta Therapeutics

11.8. GenSight Biologics

11.9. Krystal Biotech

11.10. Avexis (Acquired by Novartis)    


Voyager Therapeutics

Uniqure N.V.

Axovant Gene Therapies

Novartis Ag

Amicus Therapeutics

Regenxbio Inc.

Sarepta Therapeutics

Gensight Biologics

Krystal Biotech

Avexis (Acquired By Novartis)