Gene & Cell Therapies Targeting CNS Disorders Market to Surge by 2035 – BIS Research

What Is the Market Outlook? 

The market is maturing quickly, with gene and cell therapies transitioning from early-stage to pipeline advancement and commercialization. Technological leaps in viral vectors (AAV, lentivirus), CRISPR-based editing, and stem cell platforms are central for tackling diseases like Huntington’s, SMA, ALS, and Parkinson’s.  

How Fast Is the Market Growing? 

This rapid growth reflects a surge in approvals, R&D activity, and pipeline maturation. Significant collaborations like Novartis partnering with Voyager Therapeutics around AAV-based "Tracer" capsids underscore expanding innovation. 

How Will This Report Help You? 

1.Planning to Enter the Market? 

→ North America currently leads, with Asia‑Pacific (notably India, China, and Japan) showing accelerating uptake. 

2.Analyzing the Competitive Landscape? 

→ The report profiles key players including Novartis, BrainStorm Cell Therapeutics, Voyager Therapeutics, UniQure, StemCyte, Neuralstem, Sangamo, Biogen, and Roche. 

3.Seeking R&D Insights? 

→ Track advancements in AAV and lentiviral vectors, CRISPR gene editing, stem cell transplantation, and emerging CAR-T–inspired neuro-cell therapies. 

4.Interested in Regional Market Trends? 

→ North America maintains the highest market share, while Asia‑Pacific and Europe continue expanding with robust pipelines and more favorable regulatory conditions. 

Explore the Full TOC and Download Report Sample   

What Technologies Are Transforming the Market? 

• AAV & lentiviral vectors for targeted CNS delivery 
• CRISPR-Cas9 editing, enhanced by nanoparticle/AAV delivery across the BBB  
• Stem-cell platforms (e.g., Neuralstem’s NSI-566 for ALS, SC injury)  
• Capsid engineering, like Voyager’s Tracer system licensed to Novartis  

What’s Driving Demand & What Are the Barriers? 

Drivers: 

• Growing incidence of Alzheimer’s, Parkinson’s, ALS, Huntington’s, spinal injuries  
• Precision medicine with high efficacy potential 
• Regulatory approvals rising, e.g., Skysona for CALD, Libmeldy for MLD  

Restraints: 

• Very high therapy development and manufacturing costs 
• Complex delivery mechanisms and immunogenicity risks 
• Regulatory and reimbursement uncertainty globally  

Opportunities: 

• Personalized treatments, especially in rare inherited CNS conditions 
• Strong growth in emerging markets (Asia‑Pacific, Latin America) 
• Broader adoption of advanced delivery systems and collaborations 

How Is the Market Segmented? 

by Drug 

• Gene Therapy Drugs 

• Cell Therapy Drugs 

by Region 

• North America 

• Europe  

• Asia-Pacific 

• Rest-of-the-World 

Who Are the Key Players in the Market? 

• Novartis AG 

• BrainStorm Cell Therapeutics 

• Neuroplast 

• Rapa Therapeutics 

• Longeveron 

Strategic Moves Shaping the Future: 

• Strategic collaborations (e.g., Novartis–Voyager) targeting CNS via novel capsids  
• Expanding clinical pipelines and regulatory filings for orphan CNS disorders 
• Adoption of engineered vectors, CRISPR, stem-cell platforms 
• Scaling manufacturing and delivery networks globally, with focus on access 

Download the complete TOC now! 

Case Study: Novartis + Voyager “Tracer” Capsid Licensing 

Through a $100 M upfront, up to $1.2 B deal, Novartis secured exclusive access to Voyager’s Tracer capsid platform to accelerate CNS gene therapies for Huntington’s and SMA showing how strategic capsid innovation is propelling CNS-targeted gene therapy. 

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About BIS Research 

BIS Research is a global B2B market intelligence and advisory firm focusing on deep technology and related emerging trends, which can disrupt the market dynamics in the near future. We publish over 200 market intelligence studies annually, focusing on several deep technology verticals.   

Our strategic market analysis emphasizes market estimations, technology analysis, emerging high-growth applications, deeply segmented granular country-level market data, and other important market parameters useful in the strategic decision-making for senior management.    

BIS Research offers syndicate and custom studies and expert consultations to firms, providing them with specific and actionable insights on novel technology markets, business models, and competitive landscapes.   

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