細胞・遺伝子治療の世界市場：2024年～2031年

概要

世界の細胞・遺伝子治療市場は、2023年に165億米ドルに達し、2031年には701億米ドルに達し、予測期間2024-2031年のCAGRは18.6%で成長すると予測されています。

細胞治療では、生きた細胞を患者に移植し、病気の治療や予防を行う。これらの細胞は自家細胞であることも同種細胞であることもあり、様々なタイプの細胞に変化することができます。多能性細胞はどのような細胞型にも変化できるが、多能性細胞はレパートリーが限られています。分化細胞や初代細胞は固定型であり、投与される細胞の種類は治療法によって異なります。

遺伝子治療とは、細胞内の単一または一群のタンパク質の産生を変化させることにより、遺伝物質を用いて病気の治療や予防を行うことです。病気の原因となるタンパク質のレベルを下げたり、病気と闘うタンパク質の生産を増やしたり、新しいタンパク質や改変されたタンパク質を生産したりすることができます。

市場力学：

促進要因と阻害要因

新たな遺伝子治療を開発するための主要市場プレイヤー間の協力関係の発展

細胞・遺伝子治療市場は、バイオテクノロジー企業、製薬企業、学術機関の戦略的提携によって拡大しています。こうした提携により、専門知識、技術、リソースの共有が可能になり、臨床試験や規制当局による承認の迅速化が促進されます。また、複雑な遺伝性疾患により効果的に対処し、パイプラインを拡大することにも役立っています。

例えば、2023年2月15日、製薬会社のCharles River Laboratories International, Incと、腎臓病治療の変革に注力する遺伝子治療のパイオニア企業であるPurespring Therapeuticsは、プラスミドDNAの開発・製造受託機関（CDMO）の提携を発表しました。この提携では、チャールズリバーが確立したプラスミドプラットフォームeXpDNAと、同社のプラスミドDNA製造センター・オブ・エクセレンスにおける数十年の経験を活用し、腎疾患をターゲットとした遺伝子治療プラットフォームを構築します。

細胞・遺伝子治療に関する課題

CAR T細胞療法の開発は、安全性と有効性の問題、臨床試験プロトコル、厳しい規制の枠組み、高コストなどの課題に直面しています。重要な要素としては、様々な細胞による異種システムの治療、高い遺伝子導入効率、低い細胞毒性、単一細胞特異性などが挙げられます。

しかし、このような治療を受ける資格を持つがん患者の5人に1人は、生産施設を待って亡くなっています。このような課題にもかかわらず、現在では2週間以内に製造できる自己細胞製品もあります。医療分野では、CARを用いたより洗練された遺伝子導入ツールの開発や、採取や再灌流など標準化されたアフェレシスセンターによる集中的な組織化を模索しています。

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • 新たな遺伝子治療を開発するための主要市場企業間の協力関係の発展
    • がんの有病率の上昇
  • 抑制要因
    • 細胞・遺伝子治療に伴う課題
  • 機会
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• サプライチェーン分析
• 価格分析
• 規制分析
• 特許分析
• PESTLE分析
• SWOT分析
• DMIオピニオン

第6章 治療タイプ別

• 細胞療法
• 遺伝子治療

第7章 治療領域別

• 癌
• 希少疾患
• 心血管疾患
• 神経疾患
• 眼科
• 整形外科疾患
• 血液疾患
• その他

第8章 投与形態別

• 生体内
• 生体外

第9章 エンドユーザー別

• 病院・クリニック
• 研究機関
• バイオ製薬会社
• 学術機関

第10章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • イタリア
  • スペイン
  • その他欧州
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋
  • 中国
  • インド
  • 日本
  • 韓国
  • その他アジア太平洋地域
• 中東・アフリカ

第11章 競合情勢

• 競合シナリオ
• 市況/シェア分析
• M&A分析

第12章 企業プロファイル

• Novartis
  • 会社概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な発展
• Gilead Sciences（Kite Pharma）
• Bristol-Myers Squibb
• Bluebird Bio
• Spark Therapeutics（Roche）
• Sarepta Therapeutics
• UniQure
• Biomarin Pharmaceutical
• Fate Therapeutics
• Cellectis

第13章 付録

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