The gene and cell therapies targeting CNS disorders market is estimated to be valued at US$ 8.2 Bn in 2023 and is expected to exhibit a CAGR of 30% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.

Market Overview:
Gene and cell therapies target multiple neurological disorders including Parkinson’s disease, Alzheimer's disease, stroke, amyotrophic lateral sclerosis, spinal cord injury, Huntington's disease, and traumatic brain injury. These therapies involve introducing new genes or repairing dysfunctional genes in the central nervous system to correct underlying gene mutations or replace dysfunctional or dead cells.

Market Dynamics:
The gene and cell therapies targeting CNS disorders market is driven by the increasing prevalence of neurological disorders globally. For instance, according to the World Health Organization (WHO), over 50 million people suffer from dementia worldwide. Furthermore, increasing research and development activities for development of novel therapies for CNS disorders is also expected to aid market growth over the forecast period. For example, in December 2021, Brain storm announced positive results from a phase 3 clinical trial of NurOwn in treating amyotrophic lateral sclerosis. Additionally, growing collaborations and partnerships between biotech and pharmaceutical companies to develop effective therapies is expected to create new market opportunities. For example, in January 2022, Ionis Pharmaceuticals and Biogen partnered to advance antisense therapies for neurological disorders.

SWOT Analysis
Strength: Gene and cell therapies targeting CNS disorders have the potential to transform treatment outcomes for neurological conditions. These next generation therapies are more targeted than conventional drugs and allow direct delivery of therapeutic agents to the areas of the brain affected. Several companies have developed promising gene and cell therapy candidates which have shown safety and efficacy in clinical trials.
Weakness: Gene and cell therapies for CNS disorders are complex to develop and manufacture. The delivery and targeting of therapeutic payloads to specific cells and regions in the brain poses significant technical challenges. High development costs and regulatory hurdles also make these therapies costly. Further research is still needed to fully understand disease mechanisms and improve targeting ability.
Opportunity: The market offers significant growth opportunities driven by rising R&D investments and an increasing clinical trial pipeline. An aging global population and growing prevalence of neurodegenerative conditions have increased the demand for more effective treatments. Several large pharmaceutical companies are now actively conducting research to develop novel therapies.
Threats: Safety issues and setbacks during clinical development can significantly slow down program timelines and impact commercialization prospects. Strict regulatory pathways and manufacturing complexities make these therapies high-risk ventures. Further, limited understanding of disease biology poses translational challenges. Generic competition from conventional drug therapies once approved also threatens market entry.

Key Takeaways
The Global Gene And Cell Therapies Targeting Cns Disorders Market Size is expected to witness high growth, exhibiting CAGR of 30% over the forecast period, due to increasing R&D investments by pharmaceutical companies and a growing clinical trial pipeline.

Regional analysis: North America dominates the global market currently due to presence of major players and availability of early phase clinical trial options for patients in the US. However, Asia Pacific is expected to grow at the fastest rate owing to rising healthcare expenditure, low-cost clinical trials and increasing focus of players to expand in emerging markets like China and India.

Key players operating in the Gene and Cell Therapies Targeting CNS Disorders market are Novartis, Brainstorm Cell Therapeutics, Helminth, Core stem, Q therapeutics, Helminth, Rapa Therapeutics, Brainstorm Cell Therapeutics, Neuroblast, Osteocyte, Ferrer International, Neuralstem, Ferrer International, Semeia Cell Technologies, Labella Gene Therapeutics, Sangamon Therapeutics, Hoffmann-La Roche, Longeron, Soi Gene Therapies, Prevail Therapeutics (Eli Lilly and Company), Aerogeneration, Brain Neurotherapy Bio (Ask Bio), Unique Biopharma, Neurogenic Inc., Passage Bio, AXOVANT SCIENCES GMBH, Meira Tx Limited, Asclepios Biopharmaceutical (Ask Bio), Biogen, Sarepta Therapeutics, REGENXBIO Inc., Lysogen, Voyager

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