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리소좀 축적 질환 치료제 시장 - 세계 산업 규모, 점유율, 동향, 기회, 예측 : 치료별, 적응증별, 최종사용자별, 지역별, 경쟁별(2020-2030년)
Lysosomal Storage Diseases Therapeutics Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Treatment, By Indication, By End User, By Region & Competition, 2020-2030F
상품코드 : 1691803
리서치사 : TechSci Research
발행일 : 2025년 03월
페이지 정보 : 영문 180 Pages
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한글목차

세계 리소좀 축적 질환 치료제 시장은 2024년 76억 달러로 평가되며, 2030년까지 연평균 7.20%의 CAGR로 예측 기간 동안 괄목할 만한 성장을 보일 것으로 예상됩니다.

리소좀 축적 질환(LSD)은 리소좀 저장 장애로도 알려져 있으며, 리소좀의 이상을 특징으로 하는 희귀한 유전성 질환군입니다. 리소좀은 다양한 분자와 세포의 노폐물을 분해하는 세포 소기관입니다. 유전자 돌연변이로 인해 리소좀이 기능 장애를 일으키면 정상적인 기능을 효과적으로 발휘하지 못하게 됩니다. 그 결과, 소화되지 않은 물질이 리소좀 내에 축적되어 세포의 과정을 방해하게 됩니다. 이러한 축적은 다양한 증상과 장기 손상을 유발하고 신체의 여러 시스템에 영향을 미칩니다. 대부분의 LSD는 특정 리소좀 효소의 결핍에 기인합니다. 이 효소는 지질(지방), 당단백질, 뮤코다당류와 같은 복잡한 분자를 분해하는 역할을 합니다. 특정 효소가 결핍되거나 결핍되면 해당 기질이 리소좀에 축적되며, 50여 종의 LSD가 있으며, 각각 특정 리소좀 효소 결핍과 관련이 있으며, LSD의 예로는 고셔병, 테이-삭스병, 파브리병, 폼페병, 니만-픽병, 뮤코다당증(MPS) 등이 있습니다. 각각의 LSD는 독특한 임상적 특징과 질병 경과를 가지고 있습니다. 예를 들어, 미국 국립보건원(NIH)에 따르면, 2022년 4월 현재 폼페병은 미국에서 약 4만 명 중 1명이 앓고 있습니다.

시장 개요
예측 기간 2026-2030년
시장 규모 : 2024년 76억 달러
시장 규모 : 2030년 114억 4,000만 달러
CAGR : 2025-2030년 7.20%
급성장 부문 병원
최대 시장 북미

시장 촉진요인

연구 개발 진행 상황

주요 시장 과제

질병 메커니즘에 대한 이해의 지연

주요 시장 동향

샤페론 요법

목차

제1장 개요

제2장 조사 방법

제3장 주요 요약

제4장 고객의 소리

제5장 세계의 리소좀 축적 질환 치료제 시장 전망

제6장 아시아태평양의 리소좀 축적 질환 치료제 시장 전망

제7장 유럽의 리소좀 축적 질환 치료제 시장 전망

제8장 북미의 리소좀 축적 질환 치료제 시장 전망

제9장 남미의 리소좀 축적 질환 치료제 시장 전망

제10장 중동 및 아프리카의 리소좀 축적 질환 치료제 시장 전망

제11장 시장 역학

제12장 시장 동향과 발전

제13장 세계의 리소좀 축적 질환 치료제 시장 : SWOT 분석

제14장 Porter's Five Forces 분석

제15장 PESTEL 분석

제16장 경쟁 구도

제17장 전략적 제안

제18장 조사 회사 소개 및 면책사항

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영문 목차

영문목차

Global Lysosomal Storage Diseases Therapeutics Market was valued at USD 7.60 billion in 2024 and is anticipated to witness an impressive growth in the forecast period with a CAGR of 7.20% through 2030. Lysosomal Storage Diseases (LSDs), also known as lysosomal storage disorders, are a group of rare genetic disorders characterized by abnormalities in lysosomes. Lysosomes are cellular organelles responsible for breaking down various molecules and cellular waste products. When lysosomes malfunction due to genetic mutations, they cannot perform their normal functions effectively. As a result, undigested substances accumulate within the lysosomes and disrupt cellular processes. This accumulation leads to a wide range of symptoms and organ damage, affecting multiple systems in the body. Most LSDs result from deficiencies of specific lysosomal enzymes. These enzymes are responsible for breaking down complex molecules, such as lipids (fats), glycoproteins, and mucopolysaccharides. When a particular enzyme is deficient or absent, the corresponding substrate accumulates within lysosomes. There are over 50 different LSDs, each associated with a specific lysosomal enzyme deficiency. Examples of LSDs include Gaucher's disease, Tay-Sachs disease, Fabry disease, Pompe disease, Niemann-Pick disease, and mucopolysaccharidoses (MPS). Each LSD has its unique clinical features and disease course. For instance, according to the National Institutes of Health (NIH), as of April 2022, Pompe disease affects approximately 1 in 40,000 individuals in the United States.

Market Overview
Forecast Period2026-2030
Market Size 2024USD 7.60 Billion
Market Size 2030USD 11.44 Billion
CAGR 2025-20307.20%
Fastest Growing SegmentHospitals
Largest MarketNorth America

Key Market Drivers

Advances in Research and Development

Gene therapy has emerged as a promising approach for treating certain LSDs. Researchers are exploring methods to deliver functional genes into affected cells to restore enzyme production. Clinical trials and studies have shown promising results for diseases like mucopolysaccharidosis type II (Hunter syndrome) and Niemann-Pick disease type A and B. According to a July 2022 study published by the National Library of Medicine, lysosomal storage disorders (LSDs) are more prevalent when considered collectively, with a combined incidence of 1 in 5,000 to 1 in 8,000. The study highlights that ethnicity and geography influence LSD occurrence. For instance, Gaucher disease (GD) affects 1 in 40,000 to 1 in 60,000 in the general population but is significantly higher among Ashkenazi Jews (1 in 800). Similarly, Tay-Sachs disease (1 in 3,900), Niemann-Pick A, and mucolipidosis IV are more common in this group. In Finland, aspartylglucosaminuria occurs in 1 in 18,500 individuals. The increasing burden of LSDs is expected to drive market growth for lysosomal storage disease treatments in the coming years.

Small molecule chaperones are designed to stabilize mutant enzymes, allowing them to function more effectively. These therapies aim to correct the underlying enzymatic defects in LSDs. Some chaperone therapies have received regulatory approval and are available for patients with conditions like Fabry disease. Substrate Reduction Therapy (SRT) involves reducing the production of the toxic substrate that accumulates in lysosomes in LSDs. Medications like miglustat and eliglustat have been developed as SRTs and are used to treat diseases such as Gaucher's disease and Niemann-Pick type C. Ongoing research has led to the development of improved Enzyme Replacement Therapy (ERTs) with enhanced stability, bioavailability, and pharmacokinetics. These advancements aim to increase the effectiveness and convenience of treatment for patients with LSDs. Researchers are investigating the potential benefits of combining different therapeutic approaches, such as ERTs with chaperone therapies or gene therapy with small molecules. These combinations may offer synergistic effects and improved patient outcomes. Advances in biomarker research have led to the identification of specific markers that can aid in the diagnosis, monitoring, and assessment of disease progression in LSDs.

Key Market Challenges

Limited Understanding of Disease Mechanisms

In many LSDs, the underlying genetic and molecular mechanisms are complex and not fully understood. This complexity makes it challenging to develop targeted therapies that address the root cause of the disease. Without a comprehensive understanding of the disease mechanisms, it is difficult to identify specific drug targets and design effective treatments. LSDs encompass a wide range of rare genetic disorders, each with its unique pathophysiology. Understanding the variations in disease mechanisms among different LSDs is essential for developing tailored treatments. Limited knowledge of these variations can hinder therapeutic development efforts. Biomarkers are crucial for disease diagnosis, monitoring, and assessing treatment efficacy. However, without a deep understanding of disease mechanisms, it can be challenging to identify reliable biomarkers for LSDs, which are necessary for clinical trials and personalized medicine approaches. The lack of insight into disease mechanisms contributes to a high failure rate in drug development for LSDs. Many potential drug candidates do not progress past preclinical or early clinical stages because they do not effectively target the underlying disease processes. In the absence of a clear understanding of disease mechanisms, drug developers may face challenges related to off-target effects. These unintended consequences can lead to safety concerns and hinder the development of safe and effective therapies. The complexity of LSDs and the limited understanding of their mechanisms can make it difficult to secure research funding. Potential investors and grant providers may be hesitant to fund projects without a clear path to success, leading to underfunding of critical research efforts.

Key Market Trends

Chaperone Therapies

Chaperone therapies involve the use of small molecules that can stabilize and enhance the activity of misfolded or unstable lysosomal enzymes in LSDs. These molecules act as chaperones by assisting in the correct folding and trafficking of the enzyme to its target location within the lysosome. Chaperone therapies are designed to address the specific genetic mutations that lead to enzyme misfolding and dysfunction in LSDs. They target the underlying cause of the disease by helping the enzyme reach its active form, which is essential for substrate degradation. Many chaperone therapies are administered orally, which is a more convenient and patient-friendly route of administration compared to intravenous infusions or other invasive methods. This can improve treatment adherence and patient quality of life. Chaperone therapies have been developed and tested for various LSDs, including Fabry disease, Pompe disease, Gaucher's disease, and others. This broad applicability makes them relevant to multiple LSD subtypes. Some chaperone therapies have demonstrated clinical success and received regulatory approvals in different regions. For example, migalastat has been approved for the treatment of Fabry disease. Chaperone therapies hold the potential to modify the course of the disease by restoring enzyme activity and reducing substrate accumulation. This can lead to improvements in clinical outcomes and the prevention of disease progression. Researchers are exploring the possibility of combining chaperone therapies with other treatment approaches, such as enzyme replacement therapy (ERT) or gene therapy. These combination therapies may offer synergistic benefits and enhanced treatment efficacy. Chaperone therapies align with the trend toward personalized and precision medicine, where treatments are tailored to individual patients based on their specific genetic mutations and disease manifestations.

Key Market Players

Report Scope:

In this report, the Global Lysosomal Storage Diseases Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Lysosomal Storage Diseases Therapeutics Market, By Treatment:

Lysosomal Storage Diseases Therapeutics Market, By Indication:

Lysosomal Storage Diseases Therapeutics Market, By End-User:

Lysosomal Storage Diseases Therapeutics Market, By region:

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Lysosomal Storage Diseases Therapeutics Market.

Available Customizations:

Global Lysosomal Storage Diseases Therapeutics Market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

Company Information

Table of Contents

1. Product Overview

2. Research Methodology

3. Executive Summary

4. Voice of Customer

5. Global Lysosomal Storage Diseases Therapeutics Market Outlook

6. Asia Pacific Lysosomal Storage Diseases Therapeutics Market Outlook

7. Europe Lysosomal Storage Diseases Therapeutics Market Outlook

8. North America Lysosomal Storage Diseases Therapeutics Market Outlook

9. South America Lysosomal Storage Diseases Therapeutics Market Outlook

10. Middle East and Africa Lysosomal Storage Diseases Therapeutics Market Outlook

11. Market Dynamics

12. Market Trends & Developments

13. Global Lysosomal Storage Diseases Therapeutics Market: SWOT Analysis

14. Porter's Five Forces Analysis

15. PESTLE Analysis

16. Competitive Landscape

17. Strategic Recommendations

18. About Us & Disclaimer

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