Gene And Cell Therapies Targeting Cns Disorders Market

DelveInsight’s ‘Gene and Cell Therapies Targeting CNS Disorders – Market Insights and Market Forecast – 2026’ report delivers an in-depth understanding of the market trends of Gene and Cell Therapies Targeting CNS Disorders in in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.


The Gene and Cell Therapies Targeting CNS Disorders market report provides current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted Gene and Cell Therapies Targeting CNS Disorders market size from 2018 to 2026, in the 7MM . The report also covers the current Gene and Cell Therapies Targeting CNS Disorders treatment practice, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.

Geography Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study Period: 2018–2026

Gene and Cell Therapies Targeting CNS Disorders: Disease Understanding and Treatment Algorithm

Gene and Cell Therapies Targeting CNS Disorders Overview

The central nervous system (CNS) disorders are the most prevailing and destructive, yet one of the most poorly treated disease. The biggest challenge that needs to be addressed while developing therapeutics for CNS disorders is to allow satisfactory levels of blood–brain barrier (BBB) penetration. The BBB is comprised of a nonfestrated endothelium along with narrow tight junctions that lie between the brain capillary endothelial cells. BBB helps to aid in homeostasis within the cerebral microenvironment, thereby controlling the exchange of substances between the CNS and the blood. It also plays a major role in preventing macromolecules, other infectious substances, and potent neurotoxins from entering the brain. The CNS diseases can also arise due to complicated disorders comprising ischemic, hemorrhagic, neurodegenerative, inflammatory, and developmental disorders.


The array of the physiological and etiological variable that adds to such disorders and the sensitive nature of the CNS itself requires the implementation of diagnostic techniques which are both highly specific and are non-invasive. The development of precise surrogate biomarkers will help in sketching out diagnostic and prognostic criteria, for instance, patient subclassification, accuracy in therapeutic response assessments, and a better understanding of CNS pathological features.


Over 98% of the smaller molecular drugs and almost 100% large molecular therapeutics are found to be ineffective in treating CNS disorders due to the existing BBB, which strictly refrain the entry of therapeutic agents to their targeted area in the brain. The BBB has evolved as a dynamic and adaptable interface. BBB’s complicated functioning that lets only certain forms of substances to pass-through (permeate) makes the drug development process extremely problematic. From the vision of their clinical applications, there have been extensive efforts and intensive studies that are performed on the discovery of several CNS-active drugs. Still, the number of drugs that are approved for the treatment of the CNS is considerably rare.


New forms of strategies for the increase of CNS delivery, comprising of the currently available therapeutics, represent a potential pharmacological improvement in the treatment of CNS disorders. In the last two decades, several drug delivery strategies have been investigated to improve the therapeutic effect of brain disorders with varying degrees of success. These strategies would range from the tight junction modulation and drug molecule modification to focusing on the strategies which are based on endogenous transcytosis and carrier-dependent transport systems, including both the nanoparticle- and cell-based deliveries.


Cell therapies for CNS disorders are capable of offering various therapeutic potentials. First, the transplantation of exogenous cells, which comprises of various stem/progenitor cells and differentiated cells, for instance, the neural cells that are responsible for specific phenotypes, including astrocytes, oligodendrocytes, can be readily referred to as the form of cell therapy. The transplanted cells can act as a part of the newly developed network in the host’s tissue or are capable of secreting various trophic factors subsequently along with neuroprotective and neurorestorative capacities. Second, the activation of the endogenous stem cells can also aid in the foundation of the therapeutic effects of cell therapies.


Various activators of the endogenous repair mechanisms, for example, the exogenous stem cells, electrical/magnetic stimulation, and other stimulatory cues, can aggravate the innate regenerative ability of the CNS. Activation of the hibernating stem cells in the hippocampus, subventricular zone, or other significant brain areas, accelerating new cell growth in proliferative niches; promoting stem cell migration to the required region; and augmentation of differentiation in the targeted cells can afford powerful therapeutic effects. Third, immunomodulation can also be achieved by the process of cell therapy. Lastly, the development of novel drugs and screening of the disease pathology with the help of stem cell-based tools can aid in the treatment of CNS disorders.


Gene transfer technologies are responsible for improving the disease states in patients via their introduction to the brain. The delivery of therapeutic genes can effectively protect against the neurodegenerative disease or deliver growth factors, antioxidants, and anti-apoptotic molecules delay the proliferation of neoplasms via transferring anti-angiogenesis factors and therapeutic-driving proteins and downregulating the expression of dominantly acting gene products utilizing both antisense or ribozymes to the mutated mRNA or compensating for the loss-of functional mutations by the replacements of proteins. The genes and tools will have to be customized to meet all the therapeutic goals.


The treatment of a focal tumor or a cluster of cells within a smaller region of the brain can be achieved easily compared to more widespread abnormalities, and different types of diseases might require short-term or stable transgene expression. Several improvements in efficacy, stability, regulation, and safety of gene transfer to the brain are required in the case of gene therapy. First, large transgene capacities are required for incorporating the gene of interest along with appropriate promoters and regulators. Secondly, high transduction efficiency and titers are required for expressing genes in specified populations of neural cells after stereotactic administration. Third, the stability of transgene expression is essential in various applications. Fourth, the transgene’s satisfactory levels are important and might involve the inclusion of sequences within the vector for regulating the transcription levels of the transgene. Fifth, cell specifity of gene transfers within the CNS depends on using targeted vectors, cell-specific promoters, and routing through neuronal projections in the brain. Finally, for effective application of viral vector-mediated gene transfer for therapy, lack of toxicity and immune response will be essential, except for brain tumors where these responses may be part of the therapeutic paradigm.

Drug Chapters

The drug chapter segment of the Gene and Cell Therapies Targeting CNS Disorders report encloses the detailed analysis of Gene and Cell Therapies Targeting CNS Disorders marketed drugs and late stage (Phase-III, Phase-II/III, Phase-II, and Phase-I/II) pipeline drugs. It also helps understand the Gene and Cell Therapies Targeting CNS Disorders clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.


The currently approved Zolgensma (Novartis), also known as Onasemnogene abeparvovec, is a gene therapy medication offered to patients suffering from spinal muscular atrophy. The therapy was recently approved in 2019 for children of less than two years of age. It can be used as a one-time injection, administered into the vein with almost two months of regular corticosteroids. It is used in children suffering from spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Onasemnogene abeparvovec has been developed to treat spinal muscular atrophy, a disease linked to a mutation in the SMN1 gene on chromosome 5q and diagnosed predominantly in young children that cause progressive loss of muscle function and frequently death. The medication is administered as an intravenous infusion.


CNS disorders possess significant disease burden. Still, the current treatment options available for most CNS disorders fail to provide curative treatment. Also, for most CNS disorders, the development of effective drug candidates is challenging as clinical trials are often lengthy and expensive. At present, the biggest competition to cell and gene therapy is antisense oligotherapy. The classic example is Spinraza (Biogen), an antisense oligonucleotide that is the key contender to Zolgensma (Novartis) for SMA treatment.

Products detail in the report…


Gene and Cell Therapies Targeting CNS Disorders emerging drugs


NurOwn (BrainStorm Cell Therapeutics) is is mesenchymal stromal stem cells based therapy secreting neurotrophic factors (MSC-NTF) cells. It is designed to target disease pathways in neurodegenerative disorders by effectively delivering NTFs directly to the site of damage. MSC-NTF cells are generated using autologous, mesenchymal stem cells (MSCs) that have been extracted from bone marrow, expanded and differentiated ex-vivo. The therapy is currently under clinical evaluation in phase III for the treatment of Amyotrophic lateral sclerosis (ALS) and in phase II for treatment of Multiple Sclerosis, Chronic Progressive.


Neuro-Cells (Neuroplast) is an autologous cell preparation that aims to decrease inflammation and reduce cell death. Neuro- Cells is produced in a certified and patented closed system manufacturing process from bone marrow and is a registered Advanced Therapy Medicinal Product (ATMP). Based on these mechanisms, Neuroplast focuses on developing a stem cell therapy, which can inhibit the inflammatory processes following damaged/dying neural tissue cells and sees its Neuro-Cells as a disease-modifying therapy (DMT). Currently it is being evaluated in phase I and phase II/III clinical studies in treatment of Spinal Chord Injuries (SCI).

Products detail in the report…

Gene and Cell Therapies Targeting CNS Disorders Market Outlook

The Gene and Cell Therapies Targeting CNS Disorders market outlook of the report builds a detailed comprehension of the historical, current, and forecasted Gene and Cell Therapies Targeting CNS Disorders market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand for better technology.


This segment gives a thorough detail of Gene and Cell Therapies Targeting CNS Disorders market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need for the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.


According to DelveInsight, the Gene and Cell Therapies Targeting CNS Disorders market in the 7MM is expected to change in the study period 2018–2026.


CNS disorders are a broad category of conditions in which the brain does not function as it should, limiting health and the ability to function. The condition may be inherited due to damage from an infection, a degenerative condition, stroke, a brain tumor, or arise from unknown or multiple factors. It contributes to the highest disability-adjusted life years and is the second leading cause of death in the United States. Despite the significant disease burden, the current treatment options available for most CNS disorders fail to provide curative treatment. Also, for most CNS disorders, the development of effective drug candidates is challenging as clinical trials are often lengthy and expensive. Several national initiatives have been launched in past decades to understand the brain better, yet large pharmaceutical companies are divesting from their neuroscience research programs.


Nevertheless, with major advancements in targeted gene and cell therapy, new avenues for the treatment of major neurological disorders through a range of versatile modalities varying from expression of exogenous to suppression of endogenous genes have opened up. The advent of safe and effective vectors for the delivery of gene therapy products is the innovation that has led to the surge of activity in the gene therapy sector. Nonviral vehicles are generally less efficient than viral vectors, but the development of nonviral delivery methods for the CNS is an intense field of research and may open up new treatment possibilities shortly. Also, gene-editing tools like ZFs, TALEs, and CRISPR/Cas systems have extended genome manipulation boundaries and promoted genome editing approaches to the level of promising strategies for counteracting genetic diseases. However, the immunogenicity induced by genome editing tools is another topic of concern due to potential inflammatory responses. These discoveries hold the potential to treat many neurodegenerative disorders such as muscular atrophy, multiple sclerosis (MS), Parkinson’s disease (PD), and Alzheimer’s disease (AD), among others.


Currently, pharmaceutical companies such as Novartis, Hope Biosciences, Roche, Brainstorm Cell Therapeutics, etc., are expanding their pipelines to incorporate cell and gene therapies to treat a range of CNS disorders. Besides, many pharmaceutical companies are also actively entering into a collaboration to bring this innovative treatment to the market in the upcoming years. For instance, Swiss pharma giant Roche has recently partnered with Cambridge, Massachusetts-based Dyno Therapeutics, to develop gene therapies for CNS disorders. Likewise, Taysha Gene Therapies collaborated with Cleveland Clinic and UT Southwestern Gene Therapy Program (UTSW) to advance next-generation mini-gene payloads for AAV Gene therapies for the treatment of genetic epilepsies and additional CNS disorders.


Novartis’ Zolgensma is the first gene replacement therapy indicated for the treatment of spinal muscular atrophy (SMA) in pediatric patients. It costs USD 2.125 million per patient, making it the world’s costliest drug. The high cost of gene therapy is one of the significant roadblocks that prevent patients from choosing gene therapy as a treatment option. However, according to Novartis, one-time treatment is more valuable than expensive long-term treatments that cost several hundred thousand dollars a year. Moreover, Novartis offers AveXis patient support program, called OneGene ProgramTM. This comprehensive, individualized support program provides a dedicated, personalized support team focused on the needs of each family throughout the Zolgensma treatment journey. AveXis has paved the path to making the therapy available to children worldwide via expanded manufacturing and paid managed access plans.


Currently, numerous CGTs at different clinical development stages are being developed for the most devastating CNS disorders such as spinal injury, Alzheimer’s disease (AD), Parkinson’s disease (PD), Huntington’s disease (HD), spinal cord injury (SCI), amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), spinal muscular atrophy (SMA), etc


Key Findings

This section includes a glimpse of the Gene and Cell Therapies Targeting CNS Disorders market in the 7MM. The market size of Gene and Cell Therapies Targeting CNS Disorders in the seven major markets was found to be USD 988.8 million in 2020.


The United States: Market Outlook

This section provides Gene and Cell Therapies Targeting CNS Disorders market size and market size by indications, therapies of Gene and Cell Therapies Targeting CNS Disorders in the United States.


The United States accounts for the largest market size of Gene and Cell Therapies Targeting CNS Disorders in comparison to the EU5(the United Kingdom, Germany, Italy, France, and Spain), Japan


EU-5 Countries: Market Outlook

The total Gene and Cell Therapies Targeting CNS Disorders market size and market size by indications, therapies of Gene and Cell Therapies Targeting CNS Disorders in EU5 (Germany, France, Italy, Spain, and the United Kingdom) are also mentioned.


Japan Market Outlook

The total Gene and Cell Therapies Targeting CNS Disordersmarket size and market size by indications, therapies of Gene and Cell Therapies Targeting CNS Disorders in Japan are also mentioned.

Gene and Cell Therapies Targeting CNS Disorders Drugs Uptake

This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2018–2026. The analysis covers Gene and Cell Therapies Targeting CNS Disorders market uptake by drugs, patient uptake by therapies, and sales of each drug.


This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allow the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.

Gene and Cell Therapies Targeting CNS Disorders Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II/III, Phase II, and Phase I/II stage. It also analyses Gene and Cell Therapies Targeting CNS Disorders key players involved in developing targeted therapeutics.


Major players include Engensis (Helixmith), Q cells (Q therapeutics), Neuro-Cells (Neuroplast), Axo Lenti PD (Axovant), AAV-hTERT (Libella Gene Therapeutics), whose key products are expected to get launched in the US market by 20XX.


Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for Gene and Cell Therapies Targeting CNS Disorders emerging therapies.

KOL Views

To keep up with current market trends, we take KOLs' and SMEs’ opinion working in the Gene and Cell Therapies Targeting CNS Disorders domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps understand and validate current and emerging therapies treatment patterns or Gene and Cell Therapies Targeting CNS Disorders market trend. This will support the clients in potential upcoming novel treatment by identifying the market's overall scenario and the unmet needs.

Competitive Intelligence Analysis

We perform a Competitive and Market Intelligence analysis of the Gene and Cell Therapies Targeting CNS Disorders market by using various Competitive Intelligence tools: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

  • The report covers the descriptive overview of Gene and Cell Therapies Targeting CNS Disorders, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies
  • Comprehensive insight has been provided into the Gene and Cell Therapies Targeting CNS Disorders treatment in the 7MM.
  • Additionally, an all-inclusive account of both the current and emerging therapies for Gene and Cell Therapies Targeting CNS Disorders is provided, along with the assessment of new therapies, which will impact the current treatment landscape.
  • A detailed review of the Gene and Cell Therapies Targeting CNS Disorders market, historical and forecasted, is included in the report, covering drug outreach in the7MM.
  • The report provides an edge while developing business strategies by understanding trends shaping and driving the worldwide Gene and Cell Therapies Targeting CNS Disorders market

Report Highlights

  • In the coming years, the Gene and Cell Therapies Targeting CNS Disorders market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
  • The companies and academics are working to assess challenges and seek opportunities that could influence Gene and Cell Therapies Targeting CNS Disorders R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
  • Major players are involved in developing therapies for Gene and Cell Therapies Targeting CNS Disorders. The launch of emerging therapies will significantly impact The Gene and Cell Therapies Targeting CNS Disorders market
  • A better understanding of disease pathogenesis will also help develop novel therapeutics for Gene and Cell Therapies Targeting CNS Disorders.
  • Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competitor, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Gene and Cell Therapies Targeting CNS Disorders Report Insights

  • Therapeutic Approaches
  • Gene and Cell Therapies Targeting CNS Disorders Pipeline Analysis
  • Gene and Cell Therapies Targeting CNS Disorders Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

Gene and Cell Therapies Targeting CNS Disorders Report Key Strengths

  • 6-years Forecast
  • 7MM Coverage
  • Key Competitors
  • Highly Analyzed Market
  • Drugs Uptake

Gene and Cell Therapies Targeting CNS Disorders Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions

Market Insights:

  • What was the Gene and Cell Therapies Targeting CNS Disorders Market share (%) distribution in 2018, and how would it look like in 2026?
  • What would be the Gene and Cell Therapies Targeting CNS Disorders total market Size and market Size by therapies in the 7MM during the forecast period (2018–2026)?
  • What are the key findings pertaining to the market in the 7MM, and which country will have the largest Gene and Cell Therapies Targeting CNS Disorders market Size during the forecast period (2018–2026)?
  • At what CAGR, the Gene and Cell Therapies Targeting CNS Disordersmarket is expected to grow in the 7MM during the forecast period (2018–2026)?
  • What would be the Gene and Cell Therapies Targeting CNS Disorders market outlook in the 7MM during the forecast period (2018–2026)?
  • What would be the Gene and Cell Therapies Targeting CNS Disorders market growth till 2026, and what will be the resultant market Size in the year 2026?
  • How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?


Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

  • What are the Gene and Cell Therapies Targeting CNS Disorders marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
  • How many companies are developing Gene and Cell Therapies Targeting CNS Disorders
  • How many emerging Gene and Cell Therapies Targeting CNS Disorders are in the mid-stage and late stages of development for the treatment of CNS diseases?
  • What are the key collaborations (Industry–Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Gene and Cell Therapies Targeting CNS Disorders therapies?
  • What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Gene and Cell Therapies Targeting CNS Disorders and their status?
  • What are the key designations that have been granted for the emerging therapies for Gene and Cell Therapies Targeting CNS Disorders?
  • What are the global historical and forecasted market for Gene and Cell Therapies Targeting CNS Disorders?

Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the Gene and Cell Therapies Targeting CNS Disorders market
  • To understand the future market competition in the Gene and Cell Therapies Targeting CNS Disorders market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for Gene and Cell Therapies Targeting CNS Disorders in the 7MM.
  • Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
  • Organize sales and marketing efforts by identifying the best opportunities for Gene and Cell Therapies Targeting CNS Disorders market.
  • To understand the future market competition in the Gene and Cell Therapies Targeting CNS Disorders market.

1. Key Insights

2. Report Introduction

3. Executive Summary

4. Gene and Cell Therapies Targeting CNS Disorders: Overview

4.1. Introduction to CNS Disorders

4.2. Role of Cell and Gene Therapy in CNS Disorders

4.3. Multiple Sclerosis

4.3.1. Types of Multiple Sclerosis

4.3.2. Pathogenesis

4.3.3. Diagnosis

4.3.4. Treatment

4.3.5. Cell/Gene-based therapy

4.4. Parkinson’s disease

4.4.1. Pathogenesis

4.4.2. Diagnosis

4.4.3. Treatment

4.4.4. Cell-based therapy

4.4.5. Gene-based therapy

4.5. Spinal cord injury

4.5.1. Pathogenesis

4.5.2. Diagnosis

4.5.3. Treatment

4.5.4. Stem cell effects on Spinal Cord Injury

4.6. Amyotrophic Lateral Sclerosis

4.6.1. Pathogenesis

4.6.2. Diagnosis

4.6.3. Treatment

4.7. Huntington’s disease

4.7.1. Pathogenesis

4.7.2. Diagnosis

4.7.3. Treatment

4.8. Alzheimer’s disease

4.8.1. Stages of Alzheimer’s disease

4.8.2. Pathogenesis

4.8.3. Diagnosis

4.8.4. Treatment

4.9. Spinal Muscular Atrophy

4.9.1. Types of SMA

4.9.2. Pathophysiology

4.9.3. Diagnosis

4.9.4. Treatment

4.9.5. Gene therapy for SMA

5. Landscape Assessment

5.1. Regulatory Scenario of Cell and Gene Therapies in the 7MM

5.2. United States

5.3. The European Union

5.3.1. United Kingdom

5.3.2. Germany

5.3.3. France

5.3.4. Italy

5.3.5. Spain

5.4. Japan

5.5. Reimbursement Scenario of Cell and Gene Therapies in the 7MM

5.5.1. United States

5.5.2. United Kingdom

5.5.3. Germany

5.5.4. France

5.5.5. Italy

5.5.6. Spain

5.5.7. Japan

5.6. Mergers and Acquisitions

5.7. Industry News and Policies by Regions

5.7.1. Industry News

5.7.2. Policies and Government Initiatives for Cell and Gene Therapies

6. Marketed Products

6.1. Zolgensma: Novartis

6.1.1. Product Description

6.1.2. Indication

6.1.3. Dosage and Administration

6.1.4. Mechanism of action

6.1.5. Regulatory Milestones

6.1.6. Safety and Efficacy of Zolgensma

6.1.7. Adverse effects of Zolgensma

6.1.8. Product Profile

7. Emerging Therapies for Amyotrophic Lateral Sclerosis

7.1. NurOwn: BrainStorm Cell Therapeutics

7.1.1. Product Description

7.1.2. Product Developmental Activities

7.2. Engensis: Helixmith

7.2.1. Product Description

7.2.2. Research and Development

7.2.3. Product Developmental Activities

7.3. HYNR-CS inj: Corestem

7.3.1. Product Description

7.3.2. Research and Development

7.4. Q Cells: Q therapeutics

7.4.1. Product Description

7.4.2. Research and Development

7.4.3. Product Developmental Activities

7.5. VM202: Helixmith

7.5.1. Product Description

7.5.2. Research and Development

7.5.3. Product Developmental Activities

7.6. RAPA-501 Autologous T cells: Rapa Therapeutics

7.6.1. Product Description

7.6.2. Research and Development

8. Emerging Therapy for Multiple sclerosis

8.1. NurOwn: BrainStorm Cell Therapeutics

8.1.1. Product Description

8.1.2. Product Developmental Activities

9. Emerging Therapy for Spinal Cord Injury

9.1. Neuro-Cells: Neuroplast

9.1.1. Product Description

9.1.2. Research and Development

9.1.3. Product Developmental Activities

9.2. Umbilical Cord Blood Mononuclear Cell: StemCyte

9.2.1. Product Description

9.2.2. Research and Development

9.2.3. Product Developmental Activities

9.3. FAB117-HC: Ferrer Internacional

9.3.1. Product Description

9.3.2. Research and Development

9.4. Human spinal cord stem cells: Neuralstem

9.4.1. Product Description

9.4.2. Research and Development

9.4.3. Product Developmental Activities

9.5. FAB117-HC: Ferrer Internacional

9.5.1. Product Description

9.5.2. Research and Development

10. Emerging therapies for Alzehimer

10.1. itMSCs: Stemedica Cell Technologies

10.1.1. Product Description

10.1.2. Research and Development

10.2. AAV-hTERT: Libella Gene Therapeutics

10.2.1. Product Description

10.2.2. Research and Development

10.3. ST-501: Sangamo Therapeutics

10.3.1. Product Description

10.3.2. Product Developmental Activities

11. Emerging Therapies for Parkinson Disease

11.1. VY-AADC: Hoffmann-La Roche

11.1.1. Product Description

11.1.2. Research and Development

11.1.3. Product Developmental Activities

11.2. Lomecel-B: Longeveron

11.2.1. Product Description

11.2.2. Research and Development

11.2.3. Product Developmental Activities

11.3. OXB-102: Sio Gene Therapies

11.3.1. Product Description

11.3.2. Research and Development

11.3.3. Product Developmental Activities

11.4. PR001: Prevail Therapeutics (Eli Lilly and Company)

11.4.1. Product Description

11.4.2. Research and Development

11.4.3. Product Developmental Activities

11.5. Neural Stem Cell-Derived Neurons: NeuroGeneration

11.5.1. Product Description

11.5.2. Research and Development

11.6. AAV2-GDNF: Brain Neurotherapy Bio (AskBio)

11.6.1. Product Description

11.6.2. Research and Development

11.6.3. Product Developmental Activities

12. Emerging Therapy for Huntington

12.1. Intra-striatal rAAV5-miHTT: UniQure Biopharma

12.1.1. Product Description

12.1.2. Research and Development

12.1.3. Product Developmental Activities

13. Company Profiles

13.1. Novartis

13.1.1. Business Overview

13.1.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.1.3. Recent Developments

13.1.4. Financial Health

13.2. Ionis Pharmaceuticals

13.2.1. Business Overview

13.2.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.2.3. Recent Developments

13.2.4. Financial Health

13.3. Stemedica Cell Technologies

13.3.1. Business Overview

13.3.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.3.3. Recent Developments

13.4. Wave Life Sciences Ltd.

13.4.1. Business Overview

13.4.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.4.3. Financial Health

13.5. UniQure Biopharma B.V.

13.5.1. Business Overview

13.5.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.5.3. Financial Health

13.6. StemCyte, Inc.

13.6.1. Business Overview

13.6.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.7. Sio Gene Therapies

13.7.1. Business Overview

13.7.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.7.3. Financial Health

13.8. Rapa Therapeutics LLC

13.8.1. Business Overview

13.8.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.9. Q therapeutics

13.9.1. Business Overview

13.9.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.9.3. Recent Developments

13.10. Ferrer Internacional S.A.

13.10.1. Business Overview

13.10.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.11. Cyto Therapeutics

13.11.1. Business Overview

13.11.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.12. Corestem, Inc.

13.12.1. Business Overview

13.12.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.12.3. Financial Health

13.13. Brainstorm Cell Therapeutics

13.13.1. Business Overview

13.13.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.13.3. Recent Developments

13.13.4. Financial Health

13.14. Brain Neurotherapy Bio Inc.

13.14.1. Business Overview

13.14.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.14.3. Recent Developments

13.15. Biogen

13.15.1. Business Overview

13.15.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.15.3. Financial Health

13.16. Voyager Therapeutics

13.16.1. Business Overview

13.16.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.16.3. Recent Developments

13.16.4. Financial Health

13.17. Hope Biosciences

13.17.1. Business Overview

13.17.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.17.3. Recent Developments

13.18. Libella Gene Therapeutics

13.18.1. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.18.2. Recent Developments

13.19. Hoffmann-La Roche

13.19.1. Business Overview

13.19.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.19.3. Financial Health

13.20. Neuralstem Inc. (Seneca Biopharmaceuticals, Inc.)

13.20.1. Business Overview

13.20.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.20.3. Recent Developments

13.20.4. Financial Health

13.21. Neuroplast B.V.

13.21.1. Business Overview

13.21.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.21.3. Recent Developments

13.22. Longeveron

13.22.1. Business Overview

13.22.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.22.3. Recent Developments

13.22.4. Financial Health

13.23. Prevail Therapeutics

13.23.1. Business Overview

13.23.2. Gene and Cell Therapies Targeting CNS Disorders Portfolio

13.23.3. Recent Developments

13.23.4. Financial Health

14. Gene and Cell Therapies Targeting CNS Disorders: 7 Major Market Analysis

14.1. Key Findings

14.2. Market Size of Gene and Cell Therapies Targeting CNS Disorders in 7MM

14.3. Market Size of Gene and Cell Therapies Targeting CNS Disorders in 7MM by Indications

15. Market Outlook

15.1. United States Market Size

15.1.1. Total Market Size of Gene and Cell Therapies Targeting CNS Disorders in the United States

15.1.2. Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in United States

15.1.3. Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in United States

15.2. EU-5 Market Size

15.2.1. Germany

15.2.2. France

15.2.3. Italy

15.2.4. Spain

15.2.5. United Kingdom

15.3. Japan

15.3.1. Total Market size of Gene and Cell Therapies Targeting CNS Disorders in Japan

15.3.2. Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in Japan

15.3.3. Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in Japan

16. Attribute Analysis

17. Key Opinion Leaders

18. Market Drivers

19. Market Barriers

20. SWOT Analysis

21. Unmet Needs

22. Appendix

22.1. Bibliography

23. Report Methodology

24. DelveInsight Capabilities

25. Disclaimer

26. About DelveInsight

List of Table

Table 1: Summary of Gene and Cell Therapies Targeting CNS Disorders – Pipeline Insight (2018–2026)

Table 2: Regulatory Bodies in the United States, Europe and Japan

Table 3: Mergers and Acquisitions

Table 4: Government Initiatives in the United States, Europe and Japan

Table 5: Clinical Trials Description: NurOwn

Table 6: General Description: NurOwn

Table 7: Clinical Trials Description: Engensis

Table 8: General Description: Engensis

Table 9: Clinical Trials Description: HYNR-CS inj

Table 10: General Description: HYNR-CS inj

Table 11: Clinical Trials Description: Q Cells

Table 12: General Description: Q Cells

Table 13: Clinical Trials Description: VM202

Table 14: General Description: VM202

Table 15: Clinical Trials Description: RAPA-501 Autologous T cells

Table 16: General Description: RAPA-501 Autologous T cells

Table 17: Clinical Trials Description: NurOwn

Table 18: General Description: NurOwn

Table 19: Clinical Trials Description: Neuro-Cells

Table 20: General Description: Neuro-Cells

Table 21: Clinical Trials Description: Umbilical Cord Blood Mononuclear Cell

Table 22: General Description: Umbilical Cord Blood Mononuclear Cell

Table 23: Clinical Trials Description: FAB117-HC

Table 24: Clinical Trials Description: Human spinal cord stem cells

Table 25: General Description: Human spinal cord stem cell

Table 26: Clinical Trials Description: FAB117-HC

Table 27: General Description: FAB 117-HC

Table 28: Clinical Trials Description: itMSCs

Table 29: General Description: itMSCs

Table 30: Clinical Trials Description: AAV-hTERT

Table 31: General Description: AAV-hTERT

Table 32: General Description: ST-501

Table 33: Clinical Trials Description: VY-AADC

Table 34: General Description: VY-AADC

Table 35: Clinical Trials Description: Lomecel-B

Table 36: General Description: Lomecel-B

Table 37: Clinical Trials Description: OXB-102

Table 38: General Description: OXB-102

Table 39: Clinical Trials Description: PR001

Table 40: General Description: PR001

Table 41: Clinical Trials Description: Neural Stem Cell-Derived Neurons

Table 42: General Description: Neural Stem Cell-Derived Neurons

Table 43: Clinical Trials Description: AAV2-GDNF

Table 44: General Description: AAV2-GDNF

Table 45: Clinical Trials Description: Intra-striatal rAAV5-miHTT

Table 46: General Description: Intra-striatal rAAV5-miHTT

Table 47: Company Profile

Table 48: Ionis Pharmaceuticals Company Profile

Table 49: Stemedica Cell Technologies Company Profile

Table 50: Wave Life Sciences Ltd. Company Profile

Table 51: UniQure Biopharma B.V. Company Profile

Table 52: StemCyte, Inc. Company Profile

Table 53: Sio Gene Therapies Company Profile

Table 54: Rapa Therapeutics LLC Company Profile

Table 55: Q Therapeutics Company Profile

Table 56: Ferrer Internacional S.A. Company Profile

Table 57: Cyto Therapeutics Company Profile

Table 58: Cyto Therapeutics Company Profile

Table 59: Brainstorm Cell Therapeutics Company Profile

Table 60: Brain Neurotherapy Bio Inc. Company Profile

Table 61: Biogen Company Profile

Table 62: Voyager Therapeutics Company Profile

Table 63: Hope Biosciences Company Profile

Table 64: Libella Gene Therapeutics Company Profile

Table 65: Hoffmann-La Roche Company Profile

Table 66: Neuralstem Inc. Company Profile

Table 67: Neuroplast B.V. Company Profile

Table 68: Longeveron Company Profile

Table 69: Prevail Therapeutics Company Profile

Table 70: 7 Major Market Size of Gene and Cell Therapies Targeting CNS Disorders in USD Million (2018–2026)

Table 71: 7 Major Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Table 72: United States Market Size of Gene and Cell Therapies Targeting CNS Disorders in USD Million (2018–2026)

Table 73: United States Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Table 74: United States Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in USD Million (2018–2026)

Table 75: Germany Market Size of Gene and Cell Therapies Targeting CNS Disorders in USD Million (2018–2026)

Table 76: Germany Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Table 77: Germany Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in USD Million (2018–2026)

Table 78: France Market Size of Gene and Cell Therapies Targeting CNS Disorders in USD Million (2018–2026)

Table 79: France Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Table 80: France Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in USD Million (2018–2026)

Table 81: Italy Market Size of Gene and Cell Therapies Targeting CNS Disorders in USD Million (2018–2026)

Table 82: Italy Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Table 83: Italy Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in USD Million (2018–2026)

Table 84: Spain Market Size of Gene and Cell Therapies Targeting CNS Disorders in USD Million (2018–2026)

Table 85: Spain Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Table 86: Spain Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in USD Million (2018–2026)

Table 87: United Kingdom Market Size of Gene and Cell Therapies Targeting CNS Disorders in USD Million (2018–2026)

Table 88: The United Kingdom Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Table 89: United Kingdom Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in USD Million (2018–2026)

Table 90: Japan Market Size of Gene and Cell Therapies Targeting CNS Disorders in USD Million (2018–2026)

Table 91: Japan Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Table 93: Japan Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in USD Million (2018–2026)

Table 94: List of References

List of Figures

Figure 1: Cell therapy in CNS Disorders

Figure 2: Gene Therapy in CNS Disorders

Figure 3: Pathogenesis of Multiple Sclerosis

Figure 4: Cell-based therapy

Figure 5: Pathogenesis of primary spinal cord injury (SCI)

Figure 6: Causes of Spinal Cord Injury

Figure 7: Stem cell effects on Spinal Cord Injury

Figure 8: Pathogenesis of Huntington’s Disease

Figure 9: Pathogenesis of Alzheimer’s disease

Figure 10: Pathophysiology of the disease

Figure 11: Company Revenue

Figure 12: R&D Expenses

Figure 13: Ionis Pharmaceuticals Company Revenue

Figure 14: Ionis Pharmaceuticals R&D Expenses

Figure 15: Wave Life Sciences Ltd. Company Revenue

Figure 16: Wave Life Sciences Ltd. R&D Expenses

Figure 17: UniQure Biopharma B.V. Company Revenue

Figure 18: UniQure Biopharma B.V. R&D Expenses

Figure 19: Sio Gene Therapies R&D Expenses

Figure 20: Brainstorm Cell Therapeutics Company Revenue

Figure 21: Brainstorm Cell Therapeutics R&D Expenses

Figure 22: Biogen Company Revenue

Figure 23: Biogen Geographical Revenue Split

Figure 24: Biogen R&D Expenses

Figure 25: Voyager Therapeutics Company Revenue

Figure 26: Voyager Therapeutics R&D Expenses

Figure 27: Hoffman-La Roche Company Revenue

Figure 28: Hoffman-La Roche Geographical Revenue Split

Figure 29: Hoffman-La Roche R&D Expenses

Figure 30: Neuralstem Inc. Company Revenue

Figure 31: Neuralstem Inc. R&D Expenses

Figure 32: Longeveron Company Revenue

Figure 33: Prevail Therapeutics Company Revenue

Figure 34: Prevail Therapeutics R&D Expenses

Figure 35: 7 Major Market Size of Gene and Cell Therapies Targeting CNS Disorders in

Figure 36: 7 Major Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications

Figure 37: Market Size of Gene and Cell Therapies Targeting CNS Disorders in the United States

Figure 38: The United States Market Size of Gene and Cell Therapies Targeting CNS Disorders

Figure 39: The United States Market Size of Gene and Cell Therapies Targeting CNS Disorders

Figure 40: Market Size of Gene and Cell Therapies Targeting CNS Disorders in Germany

Figure 41: Germany Market Size of Gene and Cell Therapies Targeting CNS Disorders

Figure 42: Germany Market Size of Gene and Cell Therapies Targeting CNS Disorders

Figure 43: Market Size of Gene and Cell Therapies Targeting CNS Disorders in France

Figure 44: France Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Figure 45: France Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies

Figure 46: Market Size of Gene and Cell Therapies Targeting CNS Disorders in Italy, USD Million (2018–2026)

Figure 47: Italy Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Figure 48: Italy Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies in USD Million (2018–2026)

Figure 49: Market Size of Gene and Cell Therapies Targeting CNS Disorders in Spain

Figure 50: Spain Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications

Figure 51: Spain Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies

Figure 52: Market Size of Gene and Cell Therapies Targeting CNS Disorders in the United Kingdom, USD Million (2018–2026)

Figure 53: The United Kingdom Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Figure 54: The United Kingdom Market Size of Gene and Cell Therapies Targeting CNS Disorders

Figure 55: Market Size of Gene and Cell Therapies Targeting CNS Disorders in Japan

Figure 56: Japan Market Size of Gene and Cell Therapies Targeting CNS Disorders by Indications in USD Million (2018–2026)

Figure 57: Japan Market Size of Gene and Cell Therapies Targeting CNS Disorders by Therapies

Zolgensma: Novartis

NurOwn: BrainStorm Cell Therapeutics

Engensis: Helixmith

HYNR-CS inj: Corestem

Q Cells: Q therapeutics

VM202: Helixmith

RAPA-501 Autologous T cells: Rapa Therapeutics

NurOwn: BrainStorm Cell Therapeutics

Neuro-Cells: Neuroplast

Umbilical Cord Blood Mononuclear Cell: StemCyte

FAB117-HC: Ferrer Internacional

Human spinal cord stem cells: Neuralstem

FAB117-HC: Ferrer Internacional

itMSCs: Stemedica Cell Technologies

AAV-hTERT: Libella Gene Therapeutics

ST-501: Sangamo Therapeutics

VY-AADC: Hoffmann-La Roche

Lomecel-B: Longeveron

OXB-102: Sio Gene Therapies

PR001: Prevail Therapeutics (Eli Lilly and Company)

Neural Stem Cell-Derived Neurons: NeuroGeneration

AAV2-GDNF: Brain Neurotherapy Bio (AskBio)

Intra-striatal rAAV5-miHTT: UniQure Biopharma

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