Gene and Cell Therapies targeting CNS Disorders – Market Insights and Market Forecast – 2026

Gene and Cell Therapies targeting CNS Disorders – Market Insights and Market Forecast – 2026

  • April 2021 •
  • 244 pages •
  • Report ID: 6064447 •
  • Format: PDF
‘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.

Geographies 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 in?ammatory 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 Disordersmarket 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 the analyst, 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 Disordersmarket 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.