New York, USA, Feb. 14, 2024 (GLOBE NEWSWIRE) -- Gene and Cell Therapies Targeting CNS Disorders Market is Predicted to Exhibit Remarkable Growth by 2032, Predicts DelveInsight
The gene and cell therapies targeting CNS disorders market is expected to grow at a significantly high rate and is mainly driven by an increase in approval of a growing number of cell and gene therapies and readily adoption on approval ability to treat a broad array of conditions, increase in some cases, expected one-time dosing approach and curative treatment options.
DelveInsight’s Gene and Cell Therapies Targeting CNS Disorders Market Insights report includes a comprehensive understanding of current treatment practices, emerging gene and cell therapies targeting CNS disorders, market share of individual therapies, and current and forecasted gene and cell therapies targeting CNS disorders market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].
Key Takeaways from the Gene and Cell Therapies Targeting CNS Disorders Market Report
- According to DelveInsight’s analysis, the market size for gene and cell therapies targeting CNS disorders reached USD 1 billion in 2021 in the US and is expected to grow with a significant CAGR by 2032.
- DelveInsight’s analysis reveals that the overall diagnosed prevalent population of amyotrophic lateral sclerosis in the 7MM was reported as ~50K in 2021.
- Prominent companies working in the domain of gene and cell therapies targeting CNS disorders, including In8bio Inc., Brainstorm Cell Therapeutics, Neurocrine Biosciences, Voyager Therapeutics, Atara Biotherapeutics, Neuroplast, UniQure Biopharma, Asklepios Biopharmaceutical, Inc., Brain Neurotherapy Bio, Inc., Myrtelle Inc., Forge Biologics, Inc, Sio Gene Therapies, Lexeo Therapeutics, Partner Therapeutics, Inc., Hoffmann-La Roche, Novartis, Freeline Therapeutics, Passage Bio, Inc., Black Diamond Therapeutics, Inc., and others, are actively working on innovative gene and cell therapies targeting CNS disorders. These novel gene and cell therapies targeting CNS disorders are anticipated to enter the gene and cell therapies targeting CNS disorders market in the forecast period and are expected to change the market.
- Some of the key gene and cell therapies targeting CNS disorders include DRI cell therapy, NurOwn, VY-AADC02, ATA188, Neuro-Cells, AMT-130, AAV2-GDNF, rAAV-Olig001-ASPA, FBX-101, AAV9-GLB1, LX2006, Sargramostim, Entrectinib, ACTUS-101, Capmatinib, FLT201, PBKR03, BDTX-4933, and others.
Discover which therapies are expected to grab the gene and cell therapies targeting CNS disorders market share @ Gene and Cell Therapies Targeting CNS Disorders Market Report
Gene and Cell Therapies Targeting CNS Disorders Overview
Gene and cell therapies targeting Central Nervous System (CNS) disorders represent a cutting-edge frontier in medical research and treatment. The intricate nature of neurological conditions, such as Alzheimer's disease, Parkinson's disease, and various neurogenetic disorders, has prompted scientists to explore innovative approaches to address these complex challenges. Gene therapies involve the introduction, modification, or silencing of specific genes to correct or modulate the underlying genetic defects contributing to CNS disorders. Additionally, cell therapies aim to replace or repair damaged cells within the nervous system by transplanting healthy cells or utilizing stem cells to encourage regeneration. These groundbreaking approaches hold tremendous potential for revolutionizing the treatment landscape of CNS disorders, offering the possibility of not only slowing disease progression but also restoring lost neurological function. While still in the early stages of development, the ongoing advancements in gene and cell therapies offer hope for more effective and personalized interventions, marking a pivotal moment in the quest to unlock novel treatment modalities for debilitating CNS disorders.
Gene and Cell Therapies Targeting CNS Disorders Epidemiology Segmentation
The gene and cell therapies targeting CNS disorders epidemiology section provides insights into the historical and current gene and cell therapies targeting CNS disorders patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
DelveInsight’s analysis reveals that the overall prevalent population of Huntington’s disease in the 7MM was reported as ~80K in 2021. Moreover, In 2022, the total diagnosed prevalent cases of Parkinson’s disease were estimated to be approximately 2.5 million cases in the 7MM. These cases are projected to increase during the forecast period (2023–2032).
Download the report to understand which factors are driving gene and cell therapies targeting CNS disorders epidemiology trends @ Gene and Cell Therapies Targeting CNS Disorders Epidemiological Insights
Gene and Cell Therapies Targeting CNS Disorders Treatment Market
The potential of gene therapy in addressing neurological disorders is highly promising. Overcoming the challenge of transporting drugs through the blood-brain barrier, recent progress in gene therapy vectors and targeted promoters enables researchers to deliver molecular treatments to the brain with greater efficiency than before. In June 2018, Japan's health ministry granted conditional and time-limited approval for the clinical application of a human-derived somatic stem cell product known as STEMIRAC. During the clinical trial, patients received intravenous infusions of approximately 50 million to 200 million mesenchymal stem cells (MSCs) 40 days after their injury, aiming to facilitate the repair of the inflicted damage.
ZOLGENSMA, another approved treatment, received approval from the US FDA in May 2019 for the treatment of spinal muscular atrophy (SMA) in pediatric patients under 2 years old who have bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Subsequently, in March 2020, Novartis Pharma disclosed that the Japanese Ministry of Health, Labour, and Welfare (MHLW) approved ZOLGENSMA, followed by the European Commission (EC) providing conditional approval in May 2020.
Gene therapy holds significant promise as a potential treatment option for Parkinson's disease, presenting the theoretical potential to address both non-disease-related factors and those that modify the course of the disease. Although clinical trials have shown promising outcomes with non-disease-modifying treatments, the search for an effective gene therapy that can modify the progression of Parkinson's disease, either by slowing it down or reversing it, is still ongoing.
Learn more about the FDA-approved gene and cell therapies targeting CNS disorders @ Gene and Cell Therapies Targeting CNS Disorders Analysis
Key Gene and Cell Therapies Targeting CNS Disorders and Companies
- DRI cell therapy: In8bio Inc.
- NurOwn: Brainstorm Cell Therapeutics
- VY-AADC02: Neurocrine Biosciences/Voyager Therapeutics
- ATA188: Atara Biotherapeutics
- Neuro-Cells: Neuroplast
- AMT-130: UniQure Biopharma
- AAV2-GDNF: Asklepios Biopharmaceutical, Inc./Brain Neurotherapy Bio, Inc.
- rAAV-Olig001-ASPA: Myrtelle Inc.
- FBX-101: Forge Biologics, Inc
- AAV9-GLB1: Sio Gene Therapies
- LX2006: Lexeo Therapeutics
- Sargramostim: Partner Therapeutics, Inc.
- Entrectinib: Hoffmann-La Roche
- ACTUS-101: Asklepios Biopharmaceutical, Inc.
- Capmatinib: Novartis
- FLT201: Freeline Therapeutics
- PBKR03: Passage Bio, Inc.
- BDTX-4933: Black Diamond Therapeutics, Inc.
To know more about gene and cell therapies targeting CNS disorders in development, visit @ Gene and Cell Therapies Targeting CNS Disorders Clinical Trials
Gene and Cell Therapies Targeting CNS Disorders Market Dynamics
The gene and cell therapies targeting CNS disorders market dynamics are anticipated to change in the coming years. Cell and gene therapies represent the new frontier of medicine, providing opportunities for life extension and, in some cases, cures for conditions that were previously untreatable; larger firms hold a competitive advantage in the gene therapy industry due to their substantial resources and expertise, enabling them to navigate complex development pathways.
Furthermore, many potential gene and cell therapies targeting CNS disorders are being investigated, and it is safe to predict that the CNS treatment space will significantly impact the gene and cell therapies targeting CNS disorders market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the gene and cell therapies targeting CNS disorders market in the 7MM.
However, several factors may impede the growth of the gene and cell therapies targeting CNS disorders market. Despite advancements since the passage of the Orphan Drug Act, US patients with rare CNS diseases continue to experience barriers to diagnosis, care, and treatment, and setting a maximum reimbursement level for orphan drugs would greatly deter investment in research and development for rare diseases.
Moreover, gene and cell therapies targeting CNS disorders treatment poses a significant economic burden and disrupts patients’ overall well-being and QOL. Furthermore, the gene and cell therapies targeting CNS disorders market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the gene and cell therapies targeting CNS disorders market growth.
| Gene and Cell Therapies Targeting CNS Disorders Report Metrics | Details |
| Study Period | 2019–2032 |
| Gene and Cell Therapies Targeting CNS Disorders Report Coverage | 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] |
| Gene and Cell Therapies Targeting CNS Disorders Market Size in 2021 (US) | USD 1 Billion |
| Key Gene and Cell Therapies Targeting CNS Disorders Companies | In8bio Inc., Brainstorm Cell Therapeutics, Neurocrine Biosciences, Voyager Therapeutics, Atara Biotherapeutics, Neuroplast, UniQure Biopharma, Asklepios Biopharmaceutical, Inc., Brain Neurotherapy Bio, Inc., Myrtelle Inc., Forge Biologics, Inc, Sio Gene Therapies, Lexeo Therapeutics, Partner Therapeutics, Inc., Hoffmann-La Roche, Novartis, Freeline Therapeutics, Passage Bio, Inc., Black Diamond Therapeutics, Inc., and others |
| Key Pipeline Gene and Cell Therapies Targeting CNS Disorders | DRI cell therapy, NurOwn, VY-AADC02, ATA188, Neuro-Cells, AMT-130, AAV2-GDNF, rAAV-Olig001-ASPA, FBX-101, AAV9-GLB1, LX2006, Sargramostim, Entrectinib, ACTUS-101, Capmatinib, FLT201, PBKR03, BDTX-4933, and others |
Scope of the Gene and Cell Therapies Targeting CNS Disorders Market Report
- Gene and Cell Therapies Targeting CNS Disorders Therapeutic Assessment: Gene and Cell Therapies Targeting CNS Disorders current marketed and emerging therapies
- Gene and Cell Therapies Targeting CNS Disorders Market Dynamics: Attribute Analysis of Emerging Gene and Cell Therapies Targeting CNS Disorders Drugs
- Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
- Unmet Needs, KOL’s views, Analyst’s views, Gene and Cell Therapies Targeting CNS Disorders Market Access and Reimbursement
Discover more about gene and cell therapies targeting CNS disorders in development @ Gene and Cell Therapies Targeting CNS Disorders Assessment
Table of Contents
| 1. | Gene and Cell Therapies Targeting CNS Disorders Market Key Insights |
| 2. | Gene and Cell Therapies Targeting CNS Disorders Market Report Introduction |
| 3. | Gene and Cell Therapies Targeting CNS Disorders Market Overview at a Glance |
| 4. | Gene and Cell Therapies Targeting CNS Disorders Market Executive Summary |
| 5. | Disease Background and Overview |
| 6. | Gene and Cell Therapies Targeting CNS Disorders Treatment and Management |
| 7. | Gene and Cell Therapies Targeting CNS Disorders Epidemiology and Patient Population |
| 8. | Patient Journey |
| 9. | Gene and Cell Therapies Targeting CNS Disorders Marketed Drugs |
| 10. | Gene and Cell Therapies Targeting CNS Disorders Emerging Drugs |
| 11. | Seven Major Gene and Cell Therapies Targeting CNS Disorders Market Analysis |
| 12. | Gene and Cell Therapies Targeting CNS Disorders Market Outlook |
| 13. | Potential of Current and Emerging Therapies |
| 14. | KOL Views |
| 15. | Unmet Needs |
| 16. | SWOT Analysis |
| 17. | Appendix |
| 18. | DelveInsight Capabilities |
| 19. | Disclaimer |
| 20. | About DelveInsight |
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