遺伝子編集治療市場

当レポートは遺伝子編集治療市場調査・分析したBCC Researchの市場調査報告書です。

Report Overview

Report Scope:

This report highlights the current and future market potential of gene editing therapeutics and provides a detailed analysis of the drivers, restraints and opportunities in this market. It also surveys the competitive environment, including coverage of the pipeline activities adopted by market players, and it includes market projections for 2029. Also included are company profiles of key players, featuring detailed information regarding each company’s business segments, financials, product portfolios and recent developments.

Report Includes:

– 15 data tables and 42 additional tables
– An analysis of the current and future global markets for gene editing therapeutics
– Analyses of global market trends, with market revenue data (sales figures) for 2021-2023, estimates for 2024, and projected CAGRs through 2029
– Estimates of the market size and revenue forecasts for the global gene editing therapeutics market, with market share analysis by region
– Discussion of the market dynamics, opportunities, and challenges, as well as emerging technologies
– Overview of sustainability trends and ESG developments in the industry, with emphasis on the ESG practices of leading companies, their ESG rankings, and consumer attitudes
– Competitive intelligence, including companies’ market shares, recent M&A activity, and venture funding
– Company profiles of major players within the industry, including CRISPR Therapeutics, Vertex Pharmaceuticals, and Intellia Therapeutics

Executive Summary

Summary:

Gene editing is a technique that precisely alters the genome sequence to introduce insertions, deletions or base substitutions. This technology holds promise for controlling diseases at the genetic level, particularly those genetic disorders caused by mutations in a single gene, as many diseases are associated with changes in gene expression in vivo. The evolution of gene editing technology can be categorized into three main generations: the first generation involves zinc-finger nucleases (ZFNs), the second generation utilizes transcription activator-like effector nucleases (TALENs), and the third and most widely used generation is the CRISPR/Cas9 system. Unlike ZFNs and TALENs, which target DNA strands using proteins, CRISPR technology guides Cas proteins to specific genome locations by altering the base sequence of a guide RNA segment, thereby enhancing gene editing efficiency and broadening the technology’s applicability.

Gene editing therapeutics gained prominence over the last decade. The first therapeutic, Casgevy (exagamglogene autotemcel [exa-cel]), developed jointly by CRISPR Therapeutics and Vertex Pharmaceuticals, was approved in the U.K. in November 2023 and in the U.S. in December 2023. The approval of the first CRISPR/Cas9-based gene editing therapy is a game changer in this field. Casgevy is approved for genetic diseases, including sickle cell disease (SCD) and transfusion dependent beta thalassemia (TDT).

The gene editing therapeutics market consists of numerous small biotech firms that have been conducting research in this field. Approval of the first therapy has provided momentum to many of these companies. Numerous clinical trials are being conducted by various clinical stage biotech firms, but the majority of these trials are in the early stage of development. Approval of these therapies during the forecast period is therefore unlikely. The only product from Intellia Therapeutics, NTLA-2001, used for the treatment of transthyretin amyloidosis with cardiomyopathy (ATTR-CM), is in Phase III stage of development. The trial MAGNITUDE (NCT06128629) has its primary completion date listed as December
2027 and its estimated completion date as April 2028. If the ongoing pivotal trial has a positive outcome, the product will likely enter the market in late 2029 or sometime in 2030.

Table of Contents

Table of Contents
Chapter 1 Executive Summary
Market Outlook
Scope of Report
Market Summary
Chapter 2 Market Overview
Introduction
Genome-Editing Tool Types
Meganucleases
Zinc Finger Nucleases
Transcription Activator-like Effector Nucleases
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-Associated Protein 9
Gene editing Therapeutics
Chapter 3 Market Dynamics
Market Dynamics Snapshot
Market Drivers
Increasing Prevalence of Chronic Disease
Increasing Prevalence of Rare Genetic Disorders
Precision Medicine
Increasing Strategic Initiatives
Market Restraints
Stringent Regulatory Frameworks and Time-Consuming Approval Processes
High Cost of Treatment
Adverse Events and Long-Term Monitoring
Lack of Skilled Workforce
Off-Target Impact
Market Challenges
Patent Disputes
Governmental Policies and Regulations
Ethical Issues
Market Opportunities
Chapter 4 Emerging Technologies and Developments
Introduction
Base Editing
Prime Editing
Cas-Clover and Cas-FOKl
CRISPR Interference and CRISPR Activation
RNA Editing
Artificial Intelligence in Genome Editing
Chapter 5 Pipeline Analysis
Overview
Clinical Trial Analysis Based on Technology
Clinical Trial Analysis Based on Diseases
Designations for Pipeline Drugs
Chapter 6 Patent Analysis
Chapter 7 Market Analysis
Introduction
Casgevy
Market Overview
Market Analysis
Market by Region
North America
Europe
Emerging Markets
Chapter 8 Competitive Intelligence
Strategic Initiatives
Competitive Landscape
Chapter 9 Sustainability in the Gene Editing Therapeutics Market: Environmental, Social and Governance (ESG) Perspectives
Introduction
ESG Practices in the Gene Editing Companies
Concluding Remarks from BCC Research
Chapter 10 Appendix
Methodology
Sources
Acronyms
Company Profiles
ALLOGENE THERAPEUTICS
BEAM THERAPEUTICS
CARIBOU BIOSCIENCES INC.
CRISPR THERAPEUTICS
EDITAS MEDICINE
INTELLIA THERAPEUTICS INC.
PRECISION BIOSCIENCES
SANGAMO THERAPEUTICS
VERTEX PHARMACEUTICALS INC.
VERVE THERAPEUTICS INC.
VOR BIO

List of Tables

List of Tables
Summary Table : Global Market for Gene Editing Therapeutics, Through 2029
Table 1 : Comparison of Genome-Editing Tools
Table 2 : U.S. Adults Age 50 Years and Older with Multiple Chronic Conditions, 2020-2050
Table 3 : Global Cases and Deaths Due to Various Cancers, 2022
Table 4 : Gene Editing Index Ratings, 2020
Table 5 : Rating Guide
Table 6 : Comparison of Cas9-derived Proteins with Regular SpCas9
Table 7 : Gene Editing Therapeutic Trials in Phase I, Till May 2024
Table 8 : Gene Editing Therapeutic Trials in Phase I/II, II and III, Till May 2024
Table 9 : Casgevy Label Expansion Studies, Till May 2024
Table 10 : Examples of Some Pipeline Drugs and Their Designations, as of May 2024
Table 11 : Patents on CRISPR Therapeutics, Published in 2021
Table 12 : Patents on CRISPR Therapeutics, Published in 2022
Table 13 : Patents on CRISPR Therapeutics, Published in 2023
Table 14 : Global Market for Gene Editing Therapeutics, Through 2029
Table 15 : Global Market for Gene Editing Therapeutics, by Region, Through 2029
Table 16 : Strategic Initiatives in the Gene Editing Therapeutics Market, 2020–2024
Table 17 : Venture Funding for Startups, 2023
Table 18 : ESG Initiatives/Practices by Various Gene Editing Companies, 2023
Table 19 : ESG Rankings for Companies in the Gene Editing Therapeutics Market, 2023
Table 20 : Report Information Sources
Table 21 : Glossary of Terms Used in This Report
Table 22 : Allogene Therapeutics: Company Snapshot
Table 23 : Allogene Therapeutics: Financial Performance, FY 2022 and 2023
Table 24 : Allogene Therapeutics: News/Key Developments, 2020-2024
Table 25 : Beam Therapeutics: Company Snapshot
Table 26 : Beam Therapeutics: Financial Performance, FY 2022 and 2023
Table 27 : Beam Therapeutics: News/Key Developments, 2021-2023
Table 28 : Caribou Biosciences Inc.: Company Snapshot
Table 29 : Caribou Biosciences Inc.: Financial Performance, FY 2022 and 2023
Table 30 : Caribou Biosciences Inc.: News/Key Developments, 2021-2024
Table 31 : CRISPR Therapeutics: Company Snapshot
Table 32 : CRISPR Therapeutics: Financial Performance, FY 2022 and 2023
Table 33 : CRISPR Therapeutics: Product Portfolio
Table 34 : CRISPR Therapeutics: News/Key Developments, 2021-2024
Table 35 : Editas Medicine: Company Snapshot
Table 36 : Editas Medicine: Financial Performance, FY 2022 and 2023
Table 37 : Editas Medicine: News/Key Developments, 2022-2024
Table 38 : Intellia Therapeutics Inc.: Company Snapshot
Table 39 : Intellia Therapeutics Inc.: Financial Performance, FY 2022 and 2023
Table 40 : Intellia Therapeutics Inc.: News/Key Developments, 2021-2024
Table 41 : Precision Biosciences: Company Snapshot
Table 42 : Precision Biosciences: Financial Performance, FY 2022 and 2023
Table 43 : Precision Biosciences: News/Key Developments, 2020-2024
Table 44 : Sangamo Therapeutics: Company Snapshot
Table 45 : Sangamo Therapeutics: Financial Performance, FY 2022 and 2023
Table 46 : Sangamo Therapeutics: News/Key Developments, 2024
Table 47 : Vertex Pharmaceuticals Inc.: Company Snapshot
Table 48 : Vertex Pharmaceuticals Inc.: Financial Performance, FY 2022 and 2023
Table 49 : Vertex Pharmaceuticals Inc.: Product Portfolio
Table 50 : Vertex Pharmaceuticals Inc.: News/Key Developments, 2023 and 2024
Table 51 : Verve Therapeutics Inc.: Company Snapshot
Table 52 : Verve Therapeutics Inc.: Financial Performance, FY 2022 and 2023
Table 53 : Verve Therapeutics Inc.: News/Key Developments, 2019-2024
Table 54 : Vor Bio: Company Snapshot
Table 55 : Vor Bio: Financial Performance, FY 2022 and 2023
Table 56 : Vor Bio: News/Key Developments, 2020-2024

List of Figures

List of Figures
Summary Figure : Global Market for Gene Editing Therapeutics, 2021-2029
Figure 1 : Genome-Editing Outcomes
Figure 2 : Endonucleases Used in Gene Editing Technologies
Figure 3 : Advances in Therapeutic Strategies to Treat Life-Threatening Diseases
Figure 4 : CRISPR/Cas9 Therapeutics Timeline, Since 1987–Present
Figure 5 : CRISPR/Cas9 Therapeutics Delivery Platform
Figure 6 : Delivery Method
Figure 7 : Market Dynamics of Gene Editing Therapeutics Market
Figure 8 : Genetic Disorders
Figure 9 : Emerging Technologies in the Genome-Editing Therapeutics Market
Figure 10 : Emerging Therapeutics Applications for RNA Editing by Cas Enzymes
Figure 11 : Articles on Gene Editing Therapeutics in PubMed, 2004-2024
Figure 12 : Share of Clinical Trials on the Genome-Editing Market, by Technology, Through May 2024
Figure 13 : Genome-Editing Clinical Trials, by Diseases
Figure 14 : Genome-Editing Clinical Trials, by Trial Phase
Figure 15 : Genome-Editing Clinical Trials, by Trial Status
Figure 16 : Patents Documented, by Top Owners, 2021-2024
Figure 17 : Patents Documented, by Type, 2021-2024
Figure 18 : Patents Documented, by Type, 2021-2024
Figure 19 : Common Strategies Proposed for the Therapeutic Intervention for β-thalassemia and Sickle Cell Disease
Figure 20 : Caribou Biosciences Inc.: Revenue Share, by Country/Region, FY 2023
Figure 21 : Vertex Pharmaceuticals Inc.: Revenue Share, by Business Unit, FY 2023
Figure 22 : Vertex Pharmaceuticals Inc.: Revenue Share, by Country/Region, FY 2023

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