Lysosomal Storage Diseases Market by Disease Type, Type of Therapy, End User

List of Tables

The Lysosomal Storage Diseases Market is projected to reach USD 15.43 billion by 2030 from USD 8.67 billion in 2022, at a CAGR of 7.46% during the forecast period.

Global Lysosomal Storage Diseases Market

KEY MARKET STATISTICS
Base Year Value [2022]	USD 8.67 billion
Estimated Year Value [2023]	USD 9.31 billion
Forecast Year Value [2030]	USD 15.43 billion
CAGR (%)	7.46%

Lysosomal Storage Diseases Market - IMG1

Market Segmentation & Coverage:

This research report analyzes various sub-markets, forecasts revenues, and examines emerging trends in each category to provide a comprehensive outlook on the Lysosomal Storage Diseases Market.

Based on Disease Type, market is studied across Fabry Diseases, Gaucher's Diseases, Mucopolysaccharidosis, and Pompe's Syndrome. The Gaucher's Diseases is projected to witness significant market share during forecast period.

Based on Type of Therapy, market is studied across Enzyme Replacement Therapy, Stem Cell Therapy, and Substrate Reduction Therapy. The Enzyme Replacement Therapy is projected to witness significant market share during forecast period.

Based on End User, market is studied across Clinics and Hospitals. The Hospitals is projected to witness significant market share during forecast period.

Based on Region, market is studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, Thailand, and Vietnam. The Europe, Middle East & Africa is further studied across Denmark, Egypt, Finland, France, Germany, Israel, Italy, Netherlands, Nigeria, Norway, Poland, Qatar, Russia, Saudi Arabia, South Africa, Spain, Sweden, Switzerland, Turkey, United Arab Emirates, and United Kingdom. The Europe, Middle East & Africa is projected to witness significant market share during forecast period.

Market Statistics:

The report provides market sizing and forecasts across 7 major currencies - USD, EUR, JPY, GBP, AUD, CAD, and CHF; multiple currency support helps organization leaders to make well-informed decisions. In this report, 2018 to 2021 are considered as historical years, 2022 is base year, 2023 is estimated year, and years from 2024 to 2030 are considered as forecast period.

FPNV Positioning Matrix:

The FPNV Positioning Matrix is an indispensable tool for assessing the Lysosomal Storage Diseases Market. It comprehensively evaluates vendors, analyzing key metrics related to Business Strategy and Product Satisfaction. This enables users to make informed decisions tailored to their specific needs. Through advanced analysis, vendors are categorized into four distinct quadrants, each representing a different level of success: Forefront (F), Pathfinder (P), Niche (N), or Vital (V). Be assured that this insightful framework empowers decision-makers to navigate the market with confidence.

Market Share Analysis:

The Market Share Analysis offers invaluable insights into the vendor landscape Lysosomal Storage Diseases Market. By evaluating their impact on overall revenue, customer base, and other key metrics, we provide companies with a comprehensive understanding of their performance and the competitive environment they confront. This analysis also uncovers the level of competition in terms of market share acquisition, fragmentation, dominance, and industry consolidation during the study period.

Key Company Profiles:

The report delves into recent significant developments in the Lysosomal Storage Diseases Market, highlighting leading vendors and their innovative profiles. These include Abeona Therapeutics Inc., Alexion Pharmaceuticals, Inc., Amicus Therapeutics, ArmaGen, Inc., BioMarin, CHIESI USA, Inc., Denali Therapeutics Inc., Freeline Therapeutics, Homology Medicines, Inc., Horizon Pharma PLC, Idorsia Pharmaceuticals Ltd., JCR Pharmaceuticals Co., Ltd., Lysogene, Neurogene Inc., Novartis AG, Orchard Therapeutics Limited, Pfizer Inc., Protalix, Sangamo Therapeutics, Inc., Sanofi S.A., Spark Therapeutics, Inc., Swedish Orphan Biovitrum AB, Takeda Pharmaceutical Company Limited, and Ultragenyx Pharmaceutical Inc..

The report offers valuable insights on the following aspects:

1. Market Penetration: It provides comprehensive information about key players' market dynamics and offerings.

2. Market Development: In-depth analysis of emerging markets and penetration across mature market segments, highlighting lucrative opportunities.

3. Market Diversification: Detailed information about new product launches, untapped geographies, recent developments, and investments.

4. Competitive Assessment & Intelligence: Exhaustive assessment of market shares, strategies, products, certifications, regulatory approvals, patent landscape, and manufacturing capabilities of leading players.

5. Product Development & Innovation: Intelligent insights on future technologies, R&D activities, and breakthrough product developments.

The report addresses key questions such as:

1. What is the market size and forecast for the Lysosomal Storage Diseases Market?

2. Which products, segments, applications, and areas hold the highest investment potential in the Lysosomal Storage Diseases Market?

3. What is the competitive strategic window for identifying opportunities in the Lysosomal Storage Diseases Market?

4. What are the latest technology trends and regulatory frameworks in the Lysosomal Storage Diseases Market?

5. What is the market share of the leading vendors in the Lysosomal Storage Diseases Market?

6. Which modes and strategic moves are suitable for entering the Lysosomal Storage Diseases Market?

Product Code: MRR-DD0700E81E6A

1. Preface

1.1. Objectives of the Study
1.2. Market Segmentation & Coverage
1.3. Years Considered for the Study
1.4. Currency & Pricing
1.5. Language
1.6. Limitations
1.7. Assumptions
1.8. Stakeholders

2. Research Methodology

2.1. Define: Research Objective
2.2. Determine: Research Design
2.3. Prepare: Research Instrument
2.4. Collect: Data Source
2.5. Analyze: Data Interpretation
2.6. Formulate: Data Verification
2.7. Publish: Research Report
2.8. Repeat: Report Update

3. Executive Summary

4. Market Overview

4.1. Introduction
4.2. Lysosomal Storage Diseases Market, by Region

5. Market Insights

5.1. Market Dynamics
- 5.1.1. Drivers
  - 5.1.1.1. Prevalence of lysosomal storage disorders worldwide
  - 5.1.1.2. Inclination toward early diagnosis and treatment of diseases
  - 5.1.1.3. Increasing use of enzyme replacement therapies
- 5.1.2. Restraints
  - 5.1.2.1. High cost associated with the treatment of t lysosomal storage disease
- 5.1.3. Opportunities
  - 5.1.3.1. Advancements in gene editing technologies and gene therapies
  - 5.1.3.2. Emergence of personalized medicine approach to treat lysosomal storage conditions
- 5.1.4. Challenges
  - 5.1.4.1. Stringent regulatory framework for approval of lysosomal storage drugs
5.2. Market Segmentation Analysis
5.3. Market Trend Analysis
5.4. Cumulative Impact of High Inflation
5.5. Porter's Five Forces Analysis
- 5.5.1. Threat of New Entrants
- 5.5.2. Threat of Substitutes
- 5.5.3. Bargaining Power of Customers
- 5.5.4. Bargaining Power of Suppliers
- 5.5.5. Industry Rivalry
5.6. Value Chain & Critical Path Analysis
5.7. Regulatory Framework

6. Lysosomal Storage Diseases Market, by Disease Type

6.1. Introduction
6.2. Fabry Diseases
6.3. Gaucher's Diseases
6.4. Mucopolysaccharidosis
6.5. Pompe's Syndrome

7. Lysosomal Storage Diseases Market, by Type of Therapy

7.1. Introduction
7.2. Enzyme Replacement Therapy
7.3. Stem Cell Therapy
7.4. Substrate Reduction Therapy

8. Lysosomal Storage Diseases Market, by End User

8.1. Introduction
8.2. Clinics
8.3. Hospitals

9. Americas Lysosomal Storage Diseases Market

9.1. Introduction
9.2. Argentina
9.3. Brazil
9.4. Canada
9.5. Mexico
9.6. United States

10. Asia-Pacific Lysosomal Storage Diseases Market

10.1. Introduction
10.2. Australia
10.3. China
10.4. India
10.5. Indonesia
10.6. Japan
10.7. Malaysia
10.8. Philippines
10.9. Singapore
10.10. South Korea
10.11. Taiwan
10.12. Thailand
10.13. Vietnam

11. Europe, Middle East & Africa Lysosomal Storage Diseases Market

11.1. Introduction
11.2. Denmark
11.3. Egypt
11.4. Finland
11.5. France
11.6. Germany
11.7. Israel
11.8. Italy
11.9. Netherlands
11.10. Nigeria
11.11. Norway
11.12. Poland
11.13. Qatar
11.14. Russia
11.15. Saudi Arabia
11.16. South Africa
11.17. Spain
11.18. Sweden
11.19. Switzerland
11.20. Turkey
11.21. United Arab Emirates
11.22. United Kingdom

12. Competitive Landscape

12.1. FPNV Positioning Matrix
12.2. Market Share Analysis, By Key Player
12.3. Competitive Scenario Analysis, By Key Player

13. Competitive Portfolio

13.1. Key Company Profiles
- 13.1.1. Abeona Therapeutics Inc.
- 13.1.2. Alexion Pharmaceuticals, Inc.
- 13.1.3. Amicus Therapeutics
- 13.1.4. ArmaGen, Inc.
- 13.1.5. BioMarin
- 13.1.6. CHIESI USA, Inc.
- 13.1.7. Denali Therapeutics Inc.
- 13.1.8. Freeline Therapeutics
- 13.1.9. Homology Medicines, Inc.
- 13.1.10. Horizon Pharma PLC
- 13.1.11. Idorsia Pharmaceuticals Ltd.
- 13.1.12. JCR Pharmaceuticals Co., Ltd.
- 13.1.13. Lysogene
- 13.1.14. Neurogene Inc.
- 13.1.15. Novartis AG
- 13.1.16. Orchard Therapeutics Limited
- 13.1.17. Pfizer Inc.
- 13.1.18. Protalix
- 13.1.19. Sangamo Therapeutics, Inc.
- 13.1.20. Sanofi S.A.
- 13.1.21. Spark Therapeutics, Inc.
- 13.1.22. Swedish Orphan Biovitrum AB
- 13.1.23. Takeda Pharmaceutical Company Limited
- 13.1.24. Ultragenyx Pharmaceutical Inc.
13.2. Key Product Portfolio

14. Appendix

14.1. Discussion Guide
14.2. License & Pricing