Gene and Cell Therapies Expected to Offer New Treatment Paradigm in CNS Disorders; Market Expected to Increase at a CAGR of 45% During the Study Period 2018-2026 in the 7MM

Targeting the root cause of various CNS Disorders, Gene and cell therapy hold promise as the next wave of innovation in the pharmaceutical industry

LAS VEGAS, July 26, 2021 /PRNewswire/ -- DelveInsight's 'Gene and Cell Therapies Targeting CNS Disorders – Market Insights and Market Forecast – 2026' report offers a panoramic view of the current treatment practices, emerging cell and gene therapies to treat CNS disorders, market share of the individual therapies, current and forecasted Gene and Cell Therapies Targeting CNS Disorders market in the 7MM (the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan) for the study period  2018–2026. 

Some of the focal points extracted from the Gene and Cell Therapies Targeting CNS Disorders Market report: 

• Novartis' Zolgensma was the first gene replacement therapy approved for the treatment of Spinal Muscular Atrophy (SMA) in pediatric patients.
• Currently, pharmaceutical companies such as Novartis, Hope Biosciences, Roche, Brainstorm Cell Therapeutics, Helixmith, Q therapeutics, Neuroplast, Axovant,  Libella Gene Therapeutics, among others are working to add assets to the Gene and Cell Therapies Targeting CNS Disorders market. 
• The current emerging landscape of Gene and Cell Therapies targeting CNS Disorders comprises therapies in Phase II and Phase III stages of clinical development for various indications such as Alzheimer's disease, Huntington Disease, Multiple sclerosis (MS), Spinal muscular atrophy (SMA), Amyotrophic lateral sclerosis (ALS), Spinal Cord Injury, Parkinson's disease, and others. 
• As per the analysis, the outlook of the Gene and Cell Therapies targeting the CNS Disorders market is expected to transform in a positive direction owing to the launch of emerging therapies including Engensis (Helixmith), Q cells (Q therapeutics), Neuro-Cells (Neuroplast), Axo Lenti PD (Axovant), AAV-hTERT (Libella Gene Therapeutics), among others during the forecasted period 2021–2026. 
• The Gene and Cell Therapies targeting CNS Disorders market size was valued at USD 988.8 million in 2020.
• The United States reported having the maximum Gene and Cell Therapies targeting CNS Disorders market share accounting for ~85% followed by EU5 in 2020. 
• The expansion of the Gene and Cell Therapies Targeting CNS Disorders market is set to face major obstruction from antisense oligotherapy. The classic example is Spinraza (Biogen), an antisense oligonucleotide that is the key contender to Zolgensma (Novartis) for SMA treatment. 
• The high cost of Gene and Cell Therapies is one of the significant roadblocks in the market expansion. 

Know more about the emerging therapy expected to penetrate the market significantly @ Gene and Cell Therapies Targeting CNS Disorders Market Insights, Pipeline Therapies, and Emerging Trends 

The report is created to project true opportunities in the Gene and Cell Therapies Targeting CNS Disorders market landscape, to help clients gain a competitive advantage over competitors, and to plan strategic moves to yield maximum ROI. 

CNS Disorders Burden

Central Nervous System (CNS) Disorders are one of the most prevailing and destructive disorders that are often poorly understood and have limited treatment options. It contributes to the highest disability-adjusted life years (DALYs) and is the second leading cause of death in the United States. The past three decades witnessed a substantial rise in the diagnosis of neurological disorders, which are further expected to increase in coming years. 

CNS diseases can arise as a concomitant condition due to complicated disorders like ischemic, hemorrhagic, neurodegenerative, inflammatory, developmental disorders, damage from an infection, a degenerative condition, stroke, a brain tumor, or can be inherited. 

Burden of death and disability because of CNS disorders is a major cause of concern primarily driven by global population growth and aging; a lack of understanding of the pathophysiology of the CNS disorders, underlying risk factors that may lead to CNS disorders and so on. 

Unmet Needs in the Market 

At present, there is no curative option available in the healthcare and pharmaceutical market, and the drug development of curative and effective therapies is hindered by several challenges.

First and foremost, the mechanisms of CNS diseases are quite difficult to understand because of a lack of complete understanding of the brain functioning, which inarguably is the most complicated organ in the human body. 

Secondly, the major barrier is imposed by a dearth of standard targets for the treatment of CNS disorders. Target or biomarker identification, in this case, appears quite challenging owing to apparent defects in synaptic communication and functional connectivity and a lack of knowledge on genetic causes that are behind the CNS disorders. Studies of the pathophysiology of CNS disorders have failed to generate any fruitful results owing to the heterogeneity of the diseases. 

As per the estimates, more than 98% of the smaller molecular drugs and almost 100% large molecular therapeutics fail to offer effective care in CNS Disorders owing to their inability to cross BBB. 

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Gene and Cell Therapy Market

Just like any sector, Gene and cell therapy market also went into the stage of quiescent with the stoppage of trials and shifting of focus towards COVID-19 therapeutics and prophylactics development, however, the R&D restored its pace, and the year 2020 witnessed significant developments and approvals with the majority in oncology. 

Gene and Cell Therapies Targeting CNS Disorders Market Analysis

At present, numerous Cell and Gene Therapies (CGTs) are under different stages of clinical development indicated for 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), and others. 

The major players such as BrainStorm Cell Therapeutics, Helixmith, Corestem, Q therapeutics, and others are investigating their therapies in Phase II and III stages of development for different CNS Disorders. 

Drop by @ Gene and Cell Therapies Targeting CNS Disorders to learn more about the recent strides in R&D in CGTs being developed to treat CNS Disorders 

Gene and Cell Therapies Targeting CNS Disorders Pipeline Therapies 

Have a walkthrough of the detailed profile of the budding therapies in the Gene and Cell Therapies Targeting CNS Disorders Market 

Gene and Cell Therapies possess the potential to offer significant therapeutic benefits to patients living with different CNS Disorders. 

• The transplantation of exogenous cells, which comprises various stem/progenitor cells and differentiated cells, for instance, the neural cells that are responsible for specific phenotypes, including astrocytes, oligodendrocytes, is referred to as the form of cell therapy. 
• The activation of endogenous stem cells can also aid in the foundation of the therapeutic effects of cell therapies.
• Cell Therapies can also offer Immunomodulation 
• 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 have the ability to improve patient outcomes ascribed to their direct induction into the brain. 

Thus, Gene and Cell Therapies are extensively investigated, however, still the count of approved Cell Therapies-based CNS Disorder treatments remains considerably less. 

Know which Pharma player has the potential to emerge as the leader in Gene and Cell Therapies Targeting CNS Disorders Market 

Gene and Cell Therapies Targeting CNS Disorders Market: Way Ahead

The past two decades witnessed several drug delivery strategies ranging 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,  under investigation to improve the therapeutic effect of therapies. 

Rampant advances in genome analysis, healthcare technologies, and assembly of larger patient cohorts have facilitated the successful genetic analyses of polygenic brain disorders. 

Novartis' Zolgensma itself costs around USD 2.125 million per patient, earning the tag of the world's costliest drug, however, the drug maker argues that the one-time therapy has a clear upper hand over the other therapies that build up the cost burden on the patients with time. 

Get in touch with our KOLs @ Gene and Cell Therapies Targeting CNS Disorders Market Landscape and know more about the factors driving and hampering the market growth

Scope of the Report 

Coverage: 7MM (the US, EU5, and Japan)

Study Period: 2018-26

Key Companies in the Gene and Cell Therapy Market: Novartis, BrainStorm Cell Therapeutics, Corestem, Q Therapeutics, Helixmith, Rapa Therapeutics, BrainStorm Cell Therapeutics, Neuroplast, StemCyte, Ferrer Internacional, Neuralstem, Ferrer Internacional, Stemedica Cell Technologies, Libella Gene Therapeutics, Sangamo Therapeutics, Hoffmann-La Roche, Longeveron, Sio Gene Therapies, Eli Lilly and Company, NeuroGeneration, Brain Neurotherapy Bio (AskBio), UniQure Biopharma, and others. 

Key Gene and Cell Therapies Targeting CNS Disorders Pipeline Therapies: Zolgensma, NurOwn, Engensis, HYNR-CS inj, Q Cells, VM202, RAPA-501 Autologous T cells, NurOwn, Neuro-Cells, Umbilical Cord Blood Mononuclear Cell, FAB117-HC, Human spinal cord stem cells, FAB117-HC, itMSCs, AAV-hTERT, ST-501, VY-AADC, Lomecel-B, OXB-102, PR001, Neural Stem Cell-Derived Neurons, AAV2-GDNF, Intra-striatal rAAV5-miHTT, and others. 

Analysis: Comparative and conjoint analysis of Gene and Cell Therapies Targeting CNS Disorders Emerging therapies

Tools used: SWOT analysis, Conjoint Analysis, Porter's Five Forces, PESTLE analysis, BCG Matrix analysis methods.

Case Studies

KOL's Views

Analyst's Views

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Table of Contents

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The key therapies currently in the development phase for different CNS indications are Amyotrophic lateral sclerosis: NurOwn (BrainStorm Cell Therapeutics), Engensis (Helixmith), Q cells (Q therapeutics), VM202 (Helixmith); Spinal cord injury: Neuro-Cells (Neuroplast), Umbilical Cord Blood Mononuclear Cell (StemCyte); Parkinson's disease: Axo Lenti PD (Axovant); Alzheimer Disease: AAV-hTERT (Libella Gene Therapeutics), VY-AADC (Voyager Therapeutics). 

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