Scienza e Tecnologia
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
Gene and Cell Therapies Targeting CNS Disorders Market
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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.
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 disability-adjusted life years (DALYs) and is the 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 , which inarguably is the most complicated organ in the human body.
Secondly, the major barrier is imposed by a dearth of 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 of the smaller molecular drugs and almost large molecular therapeutics fail to offer effective care in CNS Disorders owing to their inability to cross BBB.
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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.
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 and others are investigating their therapies in Phase II and III stages of development for different CNS Disorders.
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Gene and Cell Therapies
Thus, Gene and Cell Therapies are extensively investigated, however, still the count of approved Cell Therapies-based CNS Disorder treatments remains considerably less.
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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 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.
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: 7MM (the US, EU5, and Japan )
: 2018-26
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.
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.
: Comparative and conjoint analysis of Gene and Cell Therapies Targeting CNS Disorders Emerging therapies
: SWOT analysis, Conjoint Analysis, Porter's Five Forces, PESTLE analysis, BCG Matrix analysis methods.
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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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