한글목차

개요

세계 세포 및 유전자 치료 시장은 2023년 165억 달러, 2031년에는 701억 달러에 달할 것으로 예상되며, 2024-2031년의 예측 기간 동안 18.6%의 연평균 복합 성장률(CAGR)로 성장할 것으로 예상됩니다.

세포치료는 살아있는 세포를 환자에게 이식하여 질병을 치료하거나 예방하는 것입니다. 이 세포는 자가 세포이거나 동종 세포 일 수 있으며 다양한 유형의 세포로 변할 수 있습니다. 다능성 세포는 모든 세포 유형으로 변할 수 있지만, 다능성 세포는 레퍼토리가 제한되어 있습니다. 분화세포와 원시세포는 고정형이며, 투여되는 세포의 유형은 치료법에 따라 달라집니다.

유전자 치료는 세포 내 단일 또는 한 그룹의 단백질 생산을 변화시킴으로써 유전 물질을 사용하여 질병을 치료하거나 예방하는 것입니다. 질병을 유발하는 단백질의 수준을 낮추거나, 질병과 싸우는 단백질의 생산을 증가시키거나, 새로운 단백질이나 변형된 단백질을 생산할 수 있습니다.

시장 역학 :

성장 촉진요인과 저해요인

새로운 유전자 치료제 개발을 위한 주요 시장 기업들 간의 협력 관계 발전

세포 및 유전자 치료 시장은 생명공학 기업, 제약사, 학술 기관들의 전략적 제휴를 통해 성장하고 있습니다. 이러한 제휴는 전문 지식, 기술, 자원을 공유하여 임상시험과 규제 당국의 승인을 신속하게 진행할 수 있도록 돕고 있습니다. 또한, 복잡한 유전성 질환에 보다 효과적으로 대응하고 파이프라인을 확장하는 데에도 도움이 되고 있습니다.

예를 들어, 2023년 2월 15일, 제약회사 Charles River Laboratories International, Inc와 신장 질환 치료의 혁신에 주력하는 유전자 치료의 선구자 퓨어스프링 테라퓨틱스(Purespring Therapeutics)는 플라스미드 DNA의 개발 및 CDMO CDMO(위탁개발 및 제조 수탁기관) 제휴를 발표했습니다. 이번 제휴는 찰스 리버가 구축한 플라스미드 플랫폼 eXpDNA와 자사의 플라스미드 DNA 제조 우수성 센터(Center of Excellence)의 수십 년간의 경험을 활용하여 신장 질환을 타겟으로 하는 유전자 치료 플랫폼을 구축할 예정입니다.

세포-유전자 치료 관련 과제

CAR T 세포 치료제의 개발은 안전성 및 유효성 문제, 임상시험 프로토콜, 엄격한 규제 프레임워크, 높은 비용 등의 문제에 직면해 있습니다. 중요한 요소로는 다양한 세포를 이용한 이종 시스템 치료, 높은 유전자 도입 효율, 낮은 세포 독성, 단일 세포 특이성 등을 들 수 있습니다.

그러나 이런 치료를 받을 자격이 있는 암 환자 5명 중 1명은 생산 시설을 기다리다 사망하고 있습니다. 이러한 어려움에도 불구하고, 현재 2주 이내에 생산이 가능한 자가 세포 제품도 있습니다. 의료 분야에서는 CAR을 이용한 보다 정교한 유전자 도입 도구의 개발, 채취 및 재관류 등 표준화된 아페레시스 센터를 통한 집중적인 조직화를 모색하고 있습니다.

목차

제1장 조사 방법과 조사 범위

제2장 정의와 개요

제3장 주요 요약

제4장 시장 역학

• 영향요인
  • 성장 촉진요인
  • 성장 억제요인
  • 기회
  • 영향 분석

제5장 산업 분석

• Porter의 Five Forces 분석
• 공급망 분석
• 가격 분석
• 규제 분석
• 특허 분석
• PESTLE 분석
• SWOT 분석
• DMI의 견해

제6장 치료 유형별

• 세포치료
• 유전자 치료

제7장 치료 영역별

• 암
• 희귀 질환
• 심혈관 질환
• 신경 질환
• 안과
• 정형외과 질환
• 혈액 질환
• 기타

제8장 투여 형태별

• 생체 내
• 생체 외

제9장 최종사용자별

• 병원 및 클리닉
• 연구기관
• 바이오 제약회사
• 학술기관

제10장 지역별

• 북미
  • 미국
  • 캐나다
  • 멕시코
• 유럽
  • 독일
  • 영국
  • 프랑스
  • 이탈리아
  • 스페인
  • 기타 유럽
• 남미
  • 브라질
  • 아르헨티나
  • 기타 남미
• 아시아태평양
  • 중국
  • 인도
  • 일본
  • 한국
  • 기타 아시아태평양
• 중동 및 아프리카

제11장 경쟁 구도

• 경쟁 시나리오
• 시장 현황/점유율 분석
• 인수합병(M&A) 분석

제12장 기업 개요

• Novartis
  • 기업 개요
  • 제품 포트폴리오와 설명
  • 재무 개요
  • 주요 발전
• Gilead Sciences(Kite Pharma)
• Bristol-Myers Squibb
• Bluebird Bio
• Spark Therapeutics(Roche)
• Sarepta Therapeutics
• UniQure
• Biomarin Pharmaceutical
• Fate Therapeutics
• Cellectis

제13장 부록

영문목차

Overview

Global Cell and Gene Therapy Market reached US$ 16.5 billion in 2023 and is expected to reach US$ 70.1 billion by 2031, growing at a CAGR of 18.6% during the forecast period 2024-2031.

Cell therapy involves transferring live cells to a patient to treat or prevent diseases. These cells can be autologous or allogeneic, and can transform into different cell types. Pluripotent cells can transform into any cell type, while multipotent cells have a more limited repertoire. Differentiated or primary cells are fixed types, and the type of cells administered depends on the treatment.

Gene therapy is the use of genetic material to treat or prevent diseases by altering the production of a single or group of proteins in cells. It can reduce disease-causing protein levels, increase disease-fighting protein production, or produce new or modified proteins.

Market Dynamics: Drivers & Restraints

Increasing collaboration between the key market players to develop new gene therapies

The cell and gene therapy market is growing due to strategic alliances among biotechnology firms, pharmaceutical companies, and academic institutions. These partnerships enable sharing of expertise, technology, and resources, facilitating faster clinical trials and regulatory approvals. They also help companies address complex genetic disorders more effectively and expand their pipelines.

For instance, on February 15, 2023, Charles River Laboratories International, Inc., a pharmaceutical company, and Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration. Under the collaboration, the gene therapy platform targeting renal diseases, using Charles River's established plasmid platform, eXpDNA, and decades of experience at the company's plasmid DNA manufacturing center of excellence.

Challenges associated with the cell and gene therapy

The development of CAR T-cell therapies faces challenges such as safety and efficacy issues, clinical study protocols, strict regulatory frameworks, and high costs. Key elements include treating heterogeneous systems with various cells, high gene transfer efficiency, low cell toxicity, and single cell specificity.

However, one in five cancer patients who qualify for these therapies die waiting for production facilities. Despite these challenges, some autologous products can now be produced in less than two weeks. The health sector is exploring the development of more sophisticated gene-transfer tools with CARs and centralized organization with standardized apheresis centers, such as collection and reinfusion.

Segment Analysis

The global cell and gene therapy market is segmented based on therapy type, therapeutic area, mode of delivery, end user and region.

The cell therapy from the therapy type segment accounted for approximately 54.3% of the cell and gene therapy market share

The cell therapy from the therapy type segment accounted for approximately 54.3%. implants are vital for osseointegration, a process where the implant acts as an artificial tooth root. Cell therapy is a key player in the cell and gene therapy market, offering transformative treatments for diseases like cancer, autoimmune disorders, and genetic conditions.

Cell-based therapies like CAR-T cells and stem cell therapies repair, replace, or regenerate damaged tissues and organs. The success of CAR-T cell therapies in treating cancer has increased interest and investment in the field. As clinical trials show positive outcomes, cell therapy is seen as a vital component of precision medicine, driving personalized healthcare advancements and expanding market applications.

For instance, in August 2024, the Food and Drug Administration (FDA) approved a cellular therapy called afamitresgene autoleucel, or afami-cel (Tecelra), to treat some people with metastatic synovial sarcoma, a type of soft tissue sarcoma. The decision marks the first time the agency has approved a treatment called a T-cell receptor (TCR) therapy for cancer.

Geographical Analysis

North America is estimated to hold about 38.4% of the total market share throughout the forecast period

North America is estimated to hold about 38.4% of the total market share throughout the forecast period due to rise in the prevalence of cancers, FDA approvals, highest number of gene therapy clinical trials, with over 400 enterprises actively developing cell and gene therapy products for various disorders.

For instance, in April 2024, Walgreens proposed to collaborate with drugmakers to offer cell and gene therapies to US patients as part of its expansion of specialty pharmacy services. The company will open a licensed facility in Pittsburgh for these services, as specialty pharmacies play a crucial role in the US health system, particularly in the fight against chronic diseases.

Market Segmentation

By Therapy Type

• Cell Therapy
• Gene Therapy

By Therapeutic Area

• Oncology (Cancer)
• Rare Diseases
• Cardiovascular Diseases
• Neurological Disorders
• Ophthalmology
• Orthopedic Diseases
• Hematological Disorders
• Others

By Mode of Delivery

• In Vivo
• Ex Vivo

By End-User

• Hospitals & Clinics
• Research Institutes
• Biopharmaceutical Companies
• Academic Institutions

By Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • UK
  • France
  • Italy
  • Spain
  • Rest of Europe
• South America
  • Brazil
  • Argentina
  • Rest of South America
• Asia-Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major global players in the market include Novartis, Gilead Sciences (Kite Pharma), Bristol-Myers Squibb, Bluebird Bio, Spark Therapeutics (Roche), Sarepta Therapeutics, UniQure, Biomarin Pharmaceutical, Fate Therapeutics, Cellectis among others.

Key Developments

• In April 2024, India has launched its first indigenous CAR T-cell therapy, NexCAR19, for cancer treatment. The therapy, developed by IIT Bombay and the Tata Memorial Centre, is a "major breakthrough" that offers a new hope for the fight against the disease. The therapy, which is the first "Made in India" CAR T-cell therapy, will help cure various types of cancer and significantly reduce the cost of cancer treatment. The launch event took place at the Indian Institute of Technology (IIT) Bombay in Mumbai.
• In August 2024, Precigen, Inc., a biopharmaceutical company, has announced a strategic reprioritization of its clinical portfolio and resource streamlining, including a 20% workforce reduction, to focus on commercializing the PRGN-2012 AdenoVerse gene therapy for recurrent respiratory papillomatosis, a treatment that could improve patient lives.

Why Purchase the Report?

• To visualize the global cell and gene therapy market segmentation based on therapy type, therapeutic area, mode of delivery, end user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of the cell and gene therapy market level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global cell and gene therapy market report would provide approximately 64 tables, 61 figures and 186 pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Therapy Type
• 3.2. Snippet by Therapeutic Area
• 3.3. Snippet by Mode of Delivery
• 3.4. Snippet by End User
• 3.5. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Increasing collaboration between the key market players to develop new gene therapies
    • 4.1.1.2. Rise in the prevalence of cancers
  • 4.1.2. Restraints
    • 4.1.2.1. Challenges associated with the cell and gene therapy
  • 4.1.3. Opportunity
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Force Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Regulatory Analysis
• 5.5. Patent Analysis
• 5.6. PESTLE Analysis
• 5.7. SWOT Analysis
• 5.8. DMI Opinion

6. By Therapy Type

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
  • 6.1.2. Market Attractiveness Index, By Therapy Type
• 6.2. Cell Therapy*
  • 6.2.1. Introduction
• 6.3. Gene Therapy

7. By Therapeutic Area

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapeutic Area
  • 7.1.2. Market Attractiveness Index, By Therapeutic Area
• 7.2. Oncology (Cancer)*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Rare Diseases
• 7.4. Cardiovascular Diseases
• 7.5. Neurological Disorders
• 7.6. Ophthalmology
• 7.7. Orthopedic Diseases
• 7.8. Hematological Disorders
• 7.9. Others

8. By Mode of Delivery

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Mode of Delivery
  • 8.1.2. Market Attractiveness Index, By Mode of Delivery
• 8.2. In Vivo*
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Ex Vivo

9. By End User

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 9.1.2. Market Attractiveness Index, By End User
• 9.2. Hospitals & Clinics*
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Research Institutes
• 9.4. Biopharmaceutical Companies
• 9.5. Academic Institutions

10. By Region

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 10.1.2. Market Attractiveness Index, By Region
• 10.2. North America
  • 10.2.1. Introduction
  • 10.2.2. Key Region-Specific Dynamics
  • 10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
  • 10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapeutic Area
  • 10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Mode of Delivery
  • 10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 10.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.2.7.1. U.S.
    • 10.2.7.2. Canada
    • 10.2.7.3. Mexico
• 10.3. Europe
  • 10.3.1. Introduction
  • 10.3.2. Key Region-Specific Dynamics
  • 10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
  • 10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapeutic Area
  • 10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Mode of Delivery
  • 10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 10.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.3.7.1. Germany
    • 10.3.7.2. UK
    • 10.3.7.3. France
    • 10.3.7.4. Italy
    • 10.3.7.5. Spain
    • 10.3.7.6. Rest of Europe
• 10.4. South America
  • 10.4.1. Introduction
  • 10.4.2. Key Region-Specific Dynamics
  • 10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
  • 10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapeutic Area
  • 10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Mode of Delivery
  • 10.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 10.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.4.7.1. Brazil
    • 10.4.7.2. Argentina
    • 10.4.7.3. Rest of South America
• 10.5. Asia-Pacific
  • 10.5.1. Introduction
  • 10.5.2. Key Region-Specific Dynamics
  • 10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
  • 10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapeutic Area
  • 10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Mode of Delivery
  • 10.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 10.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.5.7.1. China
    • 10.5.7.2. India
    • 10.5.7.3. Japan
    • 10.5.7.4. South Korea
    • 10.5.7.5. Rest of Asia-Pacific
• 10.6. Middle East and Africa
  • 10.6.1. Introduction
  • 10.6.2. Key Region-Specific Dynamics
  • 10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
  • 10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapeutic Area
  • 10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Mode of Delivery
  • 10.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User

11. Competitive Landscape

• 11.1. Competitive Scenario
• 11.2. Market Positioning/Share Analysis
• 11.3. Mergers and Acquisitions Analysis

12. Company Profiles

• 12.1. Novartis*
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. Gilead Sciences (Kite Pharma)
• 12.3. Bristol-Myers Squibb
• 12.4. Bluebird Bio
• 12.5. Spark Therapeutics (Roche)
• 12.6. Sarepta Therapeutics
• 12.7. UniQure
• 12.8. Biomarin Pharmaceutical
• 12.9. Fate Therapeutics
• 12.10. Cellectis

LIST NOT EXHAUSTIVE

13. Appendix

• 13.1. About Us and Services
• 13.2. Contact Us