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Lysosomal Disease Treatment Market Size, Share & Industry Trends Analysis Report By Disease Type (Gaucher's Diseases, Fabry Diseases, Mucopolysaccharidosis, Pompe's Syndrome), By Type of Therapy By End User, By Regional Outlook and Forecast, 2022 - 2028

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    Report

  • 230 Pages
  • August 2022
  • Region: Global
  • Marqual IT Solutions Pvt. Ltd (KBV Research)
  • ID: 5659023
Description Jump to:
The Global Lysosomal Disease Treatment Market size is expected to reach $11 billion by 2028, rising at a market growth of 6.3% CAGR during the forecast period.

Lysosomal diseases (LSDs) are a complex group of metabolic disorders that are often inherited in an autosomal recessive manner. There are currently 45 known diseases. They are caused by malfunctions in various lysosomal processes, most often by abnormalities in genes that produce catabolic enzymes responsible for the breakdown of macromolecules. As the faulty enzyme accumulates substrate, cell malfunction and clinical symptoms escalate. Other types of lysosomal dysfunction that can lead to lysosomal storage include defects in post-translational processing of lysosomal enzymes, errors in enzyme trafficking or targeting, and dysfunction of non-enzymatic transmembrane and solubilize.



Lysosomal hydrolase mutations, which are implicated in the breakdown of cellular macromolecules, are the primary cause of LSDs, accounting for more than two-thirds of cases. As a result, there are many different types of lipids, glycogen, mucopolysaccharides, and glycoproteins among the storage products in LSDs that are biochemical in origin. The LSDs are all inherited as autosomal recessive characteristics, except the X-linked Fabry, Danon, and Hunter diseases.

The LSDs can affect the majority of organs, either alone or as part of a multisystem illness, and are clinically very heterogeneous. Even though lysosomal enzymes are housekeeping genes and expressed in the majority of cells, the tissues on which they act are distributed much less uniformly, and this determines which tissues in any individual are affected.

COVID-19 Impact Analysis

The COVID-19 public health crisis has affected almost every industry. The COVID-19 outbreak has caused a significant decrease in demand for the lysosomal disease treatment size of the market across several sectors, especially the health and pharmaceutical sector, as coronavirus crises sweep the globe and force healthcare providers to devote a large percentage of their funds to fighting COVID-19. The risk of infection was the primary factor contributing to the delays in treatment that patients with lysosomal storage disorder encountered in hospitals. The COVID-19 outbreak harms the market's value in 2022 and beyond because it will delay the development of the worldwide market for lysosomal disease treatments.

Market Growth Factors

Incidence of Lysosomal Disorders Are Rising

The primary driving forces in the enzyme replacement therapy market are the expanding research and development for lysosomal disease diagnosis and drug development for treatment. The increasing incidence of lysosomal disease will be a key factor contributing to the acceleration of the market's growth rate. According to a report published by the National Center for Biotechnology Information, the prevalence of Fabry disease in communities of white, male individuals ranged from 1:17,000 to 1:117,000 in January 2020. Atypical presentations were associated with around 1:6000-1:40,000 females and 1:1000-1:3000 males, whereas 1:22,000-1:40,000 males had the classic Fabry disease mutations.

Financial Incentives for Developing Orphan Drugs to Recoup R&D Expenses

The increases in healthcare spending, which helps to improve its infrastructure, is a crucial element affecting the market's growth rate for therapies for lysosomal diseases. The market dynamics will be further impacted by various government organizations' efforts to strengthen the healthcare infrastructure by boosting funding. Additionally, the market for lysosomal disease therapies will grow as a result of the rising acceptance rate of early diagnosis and the increasing number of government programs to raise awareness. Along with this, expanding government benevolent programs and an increase in disposable money is expected to accelerate market expansion.

Marketing Restraining Factor:

The Treatment of Stem Cells Is Fraught with Issues

Many of the issues that can arise soon after the transplant are caused by the bone marrow being destroyed just before it by drugs or radiation. Some may be unintended consequences of the conditioning procedures themselves. The patient can get support from the transplant team to manage side effects. Most can be addressed to make the patient feel better, and some can be prevented. This is not a comprehensive list, so please inform the physician or the transplant team of any issues or adjustments that experience.



Disease Type Outlook

Based on the Disease Type, the Lysosomal Disease Treatment Market is segmented into Gaucher's Diseases, Fabry Diseases, Pompe’s Syndrome, Mucopolysaccharidosis, and Others. The Gaucher’s disease segment acquired the highest revenue share in the lysosomal disease treatment market in 2021. It is due to the rising prevalence of Gaucher's disease is the main market growth driver. Additionally, this segment's growth is fueled by potent approved medications and new treatments that are under development.

Type of Therapy Outlook

On the basis of Type of Therapy, the Lysosomal Disease Treatment Market is divided into Enzyme Replacement Therapy, Stem Cell Therapy, Substrate Reduction Therapy, and Others. The stem cell therapy segment registered a substantial revenue share in the lysosomal disease treatment market in 2021. LSDs are a very appealing target for stem cell therapy due to the capacity to perform cross-correction through secretion-reuptake of lysosomal hydrolases, and allogeneic transplantation of bone marrow was the first specific therapeutic method to be applied to LSDs.

End User Outlook

By End-user, the Lysosomal Disease Treatment Market is bifurcated into Hospitals, Clinics, and Others. The hospital segment garnered the highest revenue share in the lysosomal disease treatment market in 2021. due to an increase in people seeking hospital care for lysosomal illnesses. In response to the rising demand for clinical practices to be streamlined cost-effectively, hospitals are using these technologies. A hospital is a facility with skilled medical staff, administrative staff, and medical equipment where people can receive medical care.

Regional Outlook

Region-wise, the Lysosomal Disease Treatment Market is analyzed across North America, Europe, Asia Pacific, and LAMEA. The North America segment acquired the largest revenue share in the lysosomal disease treatment market in 2021. Due to the lysosomal disease's high prevalence in North America. The development of new medical therapies, advancements in diagnostic technology, and growing public awareness of lysosomal disorders is fueling this market's expansion. Countries have used a variety of strategies that matched their particular needs to address this particular issue of high costs.

The market research report covers the analysis of key stake holders of the market. Key companies profiled in the report include Merck & Co., Inc., Johnson & Johnson (Janssen Global Services, LLC), Eli Lilly And Company, Pfizer, Inc., Takeda Pharmaceutical Company Limited, Sanofi S.A., Novartis AG, AstraZeneca PLC (Alexion Pharmaceuticals, Inc.), BioMarin Pharmaceutical Inc. and Sigilon Therapeutics, Inc.

Scope of the Study

Market Segments Covered in the Report:

By Disease Type
  • Gaucher's Diseases
  • Fabry Diseases
  • Mucopolysaccharidosis
  • Pompe’s Syndrome
  • Others
By Type of Therapy
  • Enzyme Replacement Therapy
  • Substrate Reduction Therapy
  • Stem Cell Therapy
  • Others
By End User
  • Hospitals
  • Clinics
  • Others
By Geography
  • North America
    • US
    • Canada
    • Mexico
    • Rest of North America
  • Europe
    • Germany
    • UK
    • France
    • Russia
    • Spain
    • Italy
    • Rest of Europe
  • Asia Pacific
    • China
    • Japan
    • India
    • South Korea
    • Singapore
    • Malaysia
    • Rest of Asia Pacific
  • LAMEA
    • Brazil
    • Argentina
    • UAE
    • Saudi Arabia
    • South Africa
    • Nigeria
    • Rest of LAMEA

Key Market Players

List of Companies Profiled in the Report:

  • Merck & Co., Inc.
  • Johnson & Johnson (Janssen Global Services, LLC)
  • Eli Lilly And Company
  • Pfizer, Inc.
  • Takeda Pharmaceutical Company Limited
  • Sanofi S.A.
  • Novartis AG
  • AstraZeneca PLC (Alexion Pharmaceuticals, Inc.)
  • BioMarin Pharmaceutical Inc.
  • Sigilon Therapeutics, Inc.

Unique Offerings from the Publisher

  • Exhaustive coverage
  • The highest number of market tables and figures
  • Subscription-based model available
  • Guaranteed best price
  • Assured post sales research support with 10% customization free

Table of Contents

Chapter 1. Market Scope & Methodology
1.1 Market Definition
1.2 Objectives
1.3 Market Scope
1.4 Segmentation
1.4.1 Global Lysosomal Disease Treatment Market, by Disease Type
1.4.2 Global Lysosomal Disease Treatment Market, by Type of Therapy
1.4.3 Global Lysosomal Disease Treatment Market, by End User
1.4.4 Global Lysosomal Disease Treatment Market, by Geography
1.5 Methodology for the research
Chapter 2. Market Overview
2.1 Introduction
2.1.1 Overview
2.1.1.1 Market composition & scenarios
2.2 Key Factors Impacting the Market
2.2.1 Market Drivers
2.2.2 Market Restraints
Chapter 3. Global Lysosomal Disease Treatment Market by Disease Type
3.1 Global Gaucher's Diseases Market by Region
3.2 Global Fabry Diseases Market by Region
3.3 Global Mucopolysaccharidosis Market by Region
3.4 Global Pompe’s Syndrome Market by Region
3.5 Global Others Market by Region
Chapter 4. Global Lysosomal Disease Treatment Market by Type of Therapy
4.1 Global Enzyme Replacement Therapy Market by Region
4.2 Global Substrate Reduction Therapy Market by Region
4.3 Global Stem Cell Therapy Market by Region
4.4 Global Others Market by Region
Chapter 5. Global Lysosomal Disease Treatment Market by End User
5.1 Global Hospitals Market by Region
5.2 Global Clinics Market by Region
5.3 Global Others Market by Region
Chapter 6. Global Lysosomal Disease Treatment Market by Region
6.1 North America Lysosomal Disease Treatment Market
6.1.1 North America Lysosomal Disease Treatment Market by Disease Type
6.1.1.1 North America Gaucher's Diseases Market by Country
6.1.1.2 North America Fabry Diseases Market by Country
6.1.1.3 North America Mucopolysaccharidosis Market by Country
6.1.1.4 North America Pompe’s Syndrome Market by Country
6.1.1.5 North America Others Market by Country
6.1.2 North America Lysosomal Disease Treatment Market by Type of Therapy
6.1.2.1 North America Enzyme Replacement Therapy Market by Country
6.1.2.2 North America Substrate Reduction Therapy Market by Country
6.1.2.3 North America Stem Cell Therapy Market by Country
6.1.2.4 North America Others Market by Country
6.1.3 North America Lysosomal Disease Treatment Market by End User
6.1.3.1 North America Hospitals Market by Country
6.1.3.2 North America Clinics Market by Country
6.1.3.3 North America Others Market by Country
6.1.4 North America Lysosomal Disease Treatment Market by Country
6.1.4.1 US Lysosomal Disease Treatment Market
6.1.4.1.1 US Lysosomal Disease Treatment Market by Disease Type
6.1.4.1.2 US Lysosomal Disease Treatment Market by Type of Therapy
6.1.4.1.3 US Lysosomal Disease Treatment Market by End User
6.1.4.2 Canada Lysosomal Disease Treatment Market
6.1.4.2.1 Canada Lysosomal Disease Treatment Market by Disease Type
6.1.4.2.2 Canada Lysosomal Disease Treatment Market by Type of Therapy
6.1.4.2.3 Canada Lysosomal Disease Treatment Market by End User
6.1.4.3 Mexico Lysosomal Disease Treatment Market
6.1.4.3.1 Mexico Lysosomal Disease Treatment Market by Disease Type
6.1.4.3.2 Mexico Lysosomal Disease Treatment Market by Type of Therapy
6.1.4.3.3 Mexico Lysosomal Disease Treatment Market by End User
6.1.4.4 Rest of North America Lysosomal Disease Treatment Market
6.1.4.4.1 Rest of North America Lysosomal Disease Treatment Market by Disease Type
6.1.4.4.2 Rest of North America Lysosomal Disease Treatment Market by Type of Therapy
6.1.4.4.3 Rest of North America Lysosomal Disease Treatment Market by End User
6.2 Europe Lysosomal Disease Treatment Market
6.2.1 Europe Lysosomal Disease Treatment Market by Disease Type
6.2.1.1 Europe Gaucher's Diseases Market by Country
6.2.1.2 Europe Fabry Diseases Market by Country
6.2.1.3 Europe Mucopolysaccharidosis Market by Country
6.2.1.4 Europe Pompe’s Syndrome Market by Country
6.2.1.5 Europe Others Market by Country
6.2.2 Europe Lysosomal Disease Treatment Market by Type of Therapy
6.2.2.1 Europe Enzyme Replacement Therapy Market by Country
6.2.2.2 Europe Substrate Reduction Therapy Market by Country
6.2.2.3 Europe Stem Cell Therapy Market by Country
6.2.2.4 Europe Others Market by Country
6.2.3 Europe Lysosomal Disease Treatment Market by End User
6.2.3.1 Europe Hospitals Market by Country
6.2.3.2 Europe Clinics Market by Country
6.2.3.3 Europe Others Market by Country
6.2.4 Europe Lysosomal Disease Treatment Market by Country
6.2.4.1 Germany Lysosomal Disease Treatment Market
6.2.4.1.1 Germany Lysosomal Disease Treatment Market by Disease Type
6.2.4.1.2 Germany Lysosomal Disease Treatment Market by Type of Therapy
6.2.4.1.3 Germany Lysosomal Disease Treatment Market by End User
6.2.4.2 UK Lysosomal Disease Treatment Market
6.2.4.2.1 UK Lysosomal Disease Treatment Market by Disease Type
6.2.4.2.2 UK Lysosomal Disease Treatment Market by Type of Therapy
6.2.4.2.3 UK Lysosomal Disease Treatment Market by End User
6.2.4.3 France Lysosomal Disease Treatment Market
6.2.4.3.1 France Lysosomal Disease Treatment Market by Disease Type
6.2.4.3.2 France Lysosomal Disease Treatment Market by Type of Therapy
6.2.4.3.3 France Lysosomal Disease Treatment Market by End User
6.2.4.4 Russia Lysosomal Disease Treatment Market
6.2.4.4.1 Russia Lysosomal Disease Treatment Market by Disease Type
6.2.4.4.2 Russia Lysosomal Disease Treatment Market by Type of Therapy
6.2.4.4.3 Russia Lysosomal Disease Treatment Market by End User
6.2.4.5 Spain Lysosomal Disease Treatment Market
6.2.4.5.1 Spain Lysosomal Disease Treatment Market by Disease Type
6.2.4.5.2 Spain Lysosomal Disease Treatment Market by Type of Therapy
6.2.4.5.3 Spain Lysosomal Disease Treatment Market by End User
6.2.4.6 Italy Lysosomal Disease Treatment Market
6.2.4.6.1 Italy Lysosomal Disease Treatment Market by Disease Type
6.2.4.6.2 Italy Lysosomal Disease Treatment Market by Type of Therapy
6.2.4.6.3 Italy Lysosomal Disease Treatment Market by End User
6.2.4.7 Rest of Europe Lysosomal Disease Treatment Market
6.2.4.7.1 Rest of Europe Lysosomal Disease Treatment Market by Disease Type
6.2.4.7.2 Rest of Europe Lysosomal Disease Treatment Market by Type of Therapy
6.2.4.7.3 Rest of Europe Lysosomal Disease Treatment Market by End User
6.3 Asia Pacific Lysosomal Disease Treatment Market
6.3.1 Asia Pacific Lysosomal Disease Treatment Market by Disease Type
6.3.1.1 Asia Pacific Gaucher's Diseases Market by Country
6.3.1.2 Asia Pacific Fabry Diseases Market by Country
6.3.1.3 Asia Pacific Mucopolysaccharidosis Market by Country
6.3.1.4 Asia Pacific Pompe’s Syndrome Market by Country
6.3.1.5 Asia Pacific Others Market by Country
6.3.2 Asia Pacific Lysosomal Disease Treatment Market by Type of Therapy
6.3.2.1 Asia Pacific Enzyme Replacement Therapy Market by Country
6.3.2.2 Asia Pacific Substrate Reduction Therapy Market by Country
6.3.2.3 Asia Pacific Stem Cell Therapy Market by Country
6.3.2.4 Asia Pacific Others Market by Country
6.3.3 Asia Pacific Lysosomal Disease Treatment Market by End User
6.3.3.1 Asia Pacific Hospitals Market by Country
6.3.3.2 Asia Pacific Clinics Market by Country
6.3.3.3 Asia Pacific Others Market by Country
6.3.4 Asia Pacific Lysosomal Disease Treatment Market by Country
6.3.4.1 China Lysosomal Disease Treatment Market
6.3.4.1.1 China Lysosomal Disease Treatment Market by Disease Type
6.3.4.1.2 China Lysosomal Disease Treatment Market by Type of Therapy
6.3.4.1.3 China Lysosomal Disease Treatment Market by End User
6.3.4.2 Japan Lysosomal Disease Treatment Market
6.3.4.2.1 Japan Lysosomal Disease Treatment Market by Disease Type
6.3.4.2.2 Japan Lysosomal Disease Treatment Market by Type of Therapy
6.3.4.2.3 Japan Lysosomal Disease Treatment Market by End User
6.3.4.3 India Lysosomal Disease Treatment Market
6.3.4.3.1 India Lysosomal Disease Treatment Market by Disease Type
6.3.4.3.2 India Lysosomal Disease Treatment Market by Type of Therapy
6.3.4.3.3 India Lysosomal Disease Treatment Market by End User
6.3.4.4 South Korea Lysosomal Disease Treatment Market
6.3.4.4.1 South Korea Lysosomal Disease Treatment Market by Disease Type
6.3.4.4.2 South Korea Lysosomal Disease Treatment Market by Type of Therapy
6.3.4.4.3 South Korea Lysosomal Disease Treatment Market by End User
6.3.4.5 Singapore Lysosomal Disease Treatment Market
6.3.4.5.1 Singapore Lysosomal Disease Treatment Market by Disease Type
6.3.4.5.2 Singapore Lysosomal Disease Treatment Market by Type of Therapy
6.3.4.5.3 Singapore Lysosomal Disease Treatment Market by End User
6.3.4.6 Malaysia Lysosomal Disease Treatment Market
6.3.4.6.1 Malaysia Lysosomal Disease Treatment Market by Disease Type
6.3.4.6.2 Malaysia Lysosomal Disease Treatment Market by Type of Therapy
6.3.4.6.3 Malaysia Lysosomal Disease Treatment Market by End User
6.3.4.7 Rest of Asia Pacific Lysosomal Disease Treatment Market
6.3.4.7.1 Rest of Asia Pacific Lysosomal Disease Treatment Market by Disease Type
6.3.4.7.2 Rest of Asia Pacific Lysosomal Disease Treatment Market by Type of Therapy
6.3.4.7.3 Rest of Asia Pacific Lysosomal Disease Treatment Market by End User
6.4 LAMEA Lysosomal Disease Treatment Market
6.4.1 LAMEA Lysosomal Disease Treatment Market by Disease Type
6.4.1.1 LAMEA Gaucher's Diseases Market by Country
6.4.1.2 LAMEA Fabry Diseases Market by Country
6.4.1.3 LAMEA Mucopolysaccharidosis Market by Country
6.4.1.4 LAMEA Pompe’s Syndrome Market by Country
6.4.1.5 LAMEA Others Market by Country
6.4.2 LAMEA Lysosomal Disease Treatment Market by Type of Therapy
6.4.2.1 LAMEA Enzyme Replacement Therapy Market by Country
6.4.2.2 LAMEA Substrate Reduction Therapy Market by Country
6.4.2.3 LAMEA Stem Cell Therapy Market by Country
6.4.2.4 LAMEA Others Market by Country
6.4.3 LAMEA Lysosomal Disease Treatment Market by End User
6.4.3.1 LAMEA Hospitals Market by Country
6.4.3.2 LAMEA Clinics Market by Country
6.4.3.3 LAMEA Others Market by Country
6.4.4 LAMEA Lysosomal Disease Treatment Market by Country
6.4.4.1 Brazil Lysosomal Disease Treatment Market
6.4.4.1.1 Brazil Lysosomal Disease Treatment Market by Disease Type
6.4.4.1.2 Brazil Lysosomal Disease Treatment Market by Type of Therapy
6.4.4.1.3 Brazil Lysosomal Disease Treatment Market by End User
6.4.4.2 Argentina Lysosomal Disease Treatment Market
6.4.4.2.1 Argentina Lysosomal Disease Treatment Market by Disease Type
6.4.4.2.2 Argentina Lysosomal Disease Treatment Market by Type of Therapy
6.4.4.2.3 Argentina Lysosomal Disease Treatment Market by End User
6.4.4.3 UAE Lysosomal Disease Treatment Market
6.4.4.3.1 UAE Lysosomal Disease Treatment Market by Disease Type
6.4.4.3.2 UAE Lysosomal Disease Treatment Market by Type of Therapy
6.4.4.3.3 UAE Lysosomal Disease Treatment Market by End User
6.4.4.4 Saudi Arabia Lysosomal Disease Treatment Market
6.4.4.4.1 Saudi Arabia Lysosomal Disease Treatment Market by Disease Type
6.4.4.4.2 Saudi Arabia Lysosomal Disease Treatment Market by Type of Therapy
6.4.4.4.3 Saudi Arabia Lysosomal Disease Treatment Market by End User
6.4.4.5 South Africa Lysosomal Disease Treatment Market
6.4.4.5.1 South Africa Lysosomal Disease Treatment Market by Disease Type
6.4.4.5.2 South Africa Lysosomal Disease Treatment Market by Type of Therapy
6.4.4.5.3 South Africa Lysosomal Disease Treatment Market by End User
6.4.4.6 Nigeria Lysosomal Disease Treatment Market
6.4.4.6.1 Nigeria Lysosomal Disease Treatment Market by Disease Type
6.4.4.6.2 Nigeria Lysosomal Disease Treatment Market by Type of Therapy
6.4.4.6.3 Nigeria Lysosomal Disease Treatment Market by End User
6.4.4.7 Rest of LAMEA Lysosomal Disease Treatment Market
6.4.4.7.1 Rest of LAMEA Lysosomal Disease Treatment Market by Disease Type
6.4.4.7.2 Rest of LAMEA Lysosomal Disease Treatment Market by Type of Therapy
6.4.4.7.3 Rest of LAMEA Lysosomal Disease Treatment Market by End User
Chapter 7. Company Profiles
7.1 Merck & Co., Inc.
7.1.1 Company Overview
7.1.2 Financial Analysis
7.1.3 Segmental and Regional Analysis
7.1.4 Research & Development Expenses
7.2 Johnson & Johnson (Janssen Global Services, LLC)
7.2.1 Company Overview
7.2.2 Financial Analysis
7.2.3 Segmental &Regional Analysis
7.2.4 Research & Development Expenses
7.3 Eli Lilly And Company
7.3.1 Company Overview
7.3.2 Financial Analysis
7.3.3 Regional Analysis
7.3.4 Research & Development Expenses
7.3.5 Recent strategies and developments:
7.3.5.1 Acquisition and Mergers:
7.4 Pfizer, Inc.
7.4.1 Company Overview
7.4.2 Financial Analysis
7.4.3 Regional & Segmental Analysis
7.4.4 Research & Development Expense
7.4.5 Recent strategies and developments:
7.4.5.1 Partnerships, Collaborations, and Agreements:
7.5 Takeda Pharmaceutical Company Limited
7.5.1 Company Overview
7.5.2 Financial Analysis
7.5.3 Regional Analysis
7.5.4 Research & Development Expense
7.6 Sanofi S.A.
7.6.1 Company Overview
7.6.2 Financial Analysis
7.6.3 Segmental and Regional Analysis
7.6.4 Research & Development Expense
7.6.5 Recent strategies and developments:
7.6.5.1 Approvals and Trials:
7.7 Novartis AG
7.7.1 Company Overview
7.7.2 Financial Analysis
7.7.3 Segmental and Regional Analysis
7.7.4 Research & Development Expense
7.8 AstraZeneca PLC (Alexion Pharmaceuticals, Inc.)
7.8.1 Company Overview
7.8.2 Financial Analysis
7.8.3 Regional Analysis
7.8.4 Research & Development Expenses
7.9 BioMarin Pharmaceutical Inc.
7.9.1 Company Overview
7.9.2 Financial Analysis
7.9.3 Regional Analysis
7.9.4 Research & Development Expenses
7.9.5 Recent strategies and developments:
7.9.5.1 Approvals and Trials:
7.10. Sigilon Therapeutics, Inc.
7.10.1 Company Overview
7.10.2 Financial Analysis
7.10.3 Research & Development Expenses

Companies Mentioned

  • Merck & Co., Inc.
  • Johnson & Johnson (Janssen Global Services, LLC)
  • Eli Lilly And Company
  • Pfizer, Inc.
  • Takeda Pharmaceutical Company Limited
  • Sanofi S.A.
  • Novartis AG
  • AstraZeneca PLC (Alexion Pharmaceuticals, Inc.)
  • BioMarin Pharmaceutical Inc.
  • Sigilon Therapeutics, Inc.

Methodology

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