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Central nervous system (CNS) diseases include diverse group of conditions in which the brain loses its normal functioning, thereby reducing the capacity to operate day to day life activities. These can occur due to several reasons such as hereditary, others are caused by injury, and few are caused by infections. The most difficultly in providing treatments for CNS diseases is allowing adequate amount of blood–brain barrier (BBB) penetration. Several Cell and Gene Therapies (CGTs) are in various stages of clinical development for targeting the most destructive CNS disorders, including spinal muscular atrophy (SMA), Parkinson's disease, Alzheimer's disease (AD), multiple sclerosis (MS), Huntington’s disease (HD), spinal cord injury (SCI), and amyotrophic lateral sclerosis (ALS).

Cell therapy for CNS has advanced to new level of clinical applications. Several clinical trials are investigating potential cell therapy for various CNS disorders such as stroke, traumatic brain injury, Parkinson's disease and other neurological diseases. The transplanted cells can serve as a member of the newly formed network in the host tissue and are capable of secreting different trophic factors as well as neuroprotective & neurorestorative properties. Gene transfer technologies are responsible for improving illness conditions in patients. Therapeutic genes can effectively protect against the neurodegenerative disease.

Gene and Cell Therapies Targeting CNS Disorders Market- Dynamics

Key market players emerging into licensing and inventory agreements, is expected to drive the market growth during the forecast period. For instance, in August 2020, Taysha Gene Therapies and Abeona Therapeutics Inc., announced that they entered into licence and inventory purchase agreements for ABO-202, an adeno-associated virus (AAV) gene therapy for CLN1 disease (also known as infantile Batten disease).

In addition, increasing collaboration of key market players for development of gene editing therapy is projected to spur the market growth during the forecast period. For instance, in June 2021, CRISPR Therapeutics is signing a deal with private company Capsida Biotherapeutics to cooperate on treatments for amyotrophic lateral sclerosis (ALS) and nerve disorder. The partners want to use Capsida's technology to better target on central nervous system tissues. CRISPR will create the gene editing tools for both initiatives, while Capsida will design the viral protein shells that will transport the medicines.

Moreover, the rising innovations in gene therapy to reverse neurological deficiencies for cell reprogramming is anticipated to boost the market growth during the forecast period. For instance, in July 2021, according to, The Ohio State University Wexner Medical Center and The Ohio State University College of Medicine, an innovative gene therapy approach is assisting children born with aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disease that causes significant physical and developmental impairments. The findings of this study reveal the results of a targeted delivery of gene therapy to the midbrain to treat a rare fatal neurodevelopmental condition in children with a neurogenetic disease, aromatic L-amino acid decarboxylase (AADC) deficiency which is characterized by dopamine and serotonin synthesis deficiencies.

Global Gene and Cell Therapies Targeting CNS Disorders Market – Regional Insights

North America is expected to expand at robust growth during the forecast period owing to increasing product approvals as well as launches. For instance, in April 2021, Biogen, a U.S. based biotechnology firm obtained clearance from China's National Medical Products Administration (NMPA) in April 2021 for its new medicine named TECFIDERA. TECFIDERA is an oral medication that is used to treat relapse multiple sclerosis.

Europe is projected to dominate the fastest CAGR in the global gene and cell therapies targeting CNS disorders market during the forecast period. Europe is estimated to contribute second largest share in the global gene and cell therapies targeting CNS disorders market owing to earlier adoption of biotechnology products in the region.

Global Gene and Cell Therapies Targeting CNS Disorders Market   – Taxonomy

By Therapy Type:

  • Gene Therapy
  • Cell Therapy

By Indication:

  • Alzheimer's Disease (AD)
  • Huntington's Disease (HD)
  • Parkinson's Disease (PD)
  • Amyotrophic Lateral Sclerosis (ALS) & Motor Neuron Disease
  • Multiple Sclerosis (MS)
  • Adrenoleukodystrophy & Metachromatic Leukodystrophy
  • Neuropathies
  • Batten Disease (BD)

By Region

  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East
  • Africa

Global Gene and Cell Therapies Targeting CNS Disorders Market – Competitive landscape

Key players operating in the global gene and cell therapies targeting CNS disorders market include Novartis, BrainStorm Cell Therapeutics, Corestem, Q Therapeutics, Helixmith, Rapa 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), and UniQure Biopharma.

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