首页 > 市场调查报告书 > 製药

基因治疗学和RNAi 以基因为基之药物开发

市场调查报告书

商品编码

1529409

CRISPR Cas9全球市场:市场机会及临床试验预测(2029年)

Global CRISPR Cas9 Market Opportunity & Clinical Trials Outlook 2029

出版日期: 2024年08月01日 | 出版商:

KuicK Research | 英文 180 Pages | 商品交期: 最快1-2个工作天内

价格

简介目录图表

以前，包括化疗和标靶治疗在内的传统治疗方法被用来治疗癌症、自体免疫疾病、发炎性疾病和其他疾病，但临床研究人员一直在坚持不懈地致力于发现尖端技术的假设。 CRISPR/Cas9 疗法的发现是由于遗传疾病在世界范围内的惊人传播以及对有效治疗的需求不断增长。自 2024 年 7 月起，CRISPR/Cas9 疗法已被批准用于治疗 β 地中海贫血和镰状细胞性贫血，并在美国、欧盟、英国、沙乌地阿拉伯和巴林上市。

未来五年，第一波 CRISPR/Cas9 疗法预计将获得监管部门批准并进入市场。这是因为，随着这些疗法在现实环境中表现出有效性和安全性，这将为基于 CRISPR/Cas9 的疗法的广泛接受和应用铺平道路。除此之外，这项突破性基因编辑技术的潜在应用将大大扩展到遗传性疾病和癌症之外，扩展到心血管、神经退化性疾病和中枢神经系统疾病的治疗。

迄今为止，CRISPR/Cas9疗法的全球临床管线逐年快速成长，多种疗法进入临床试验，其他CRISPR/Cas9候选疗法处于临床前阶段。许多正在进行的 CRISPR/Cas9 临床试验已达到开发后期阶段，预计将在五到七年内渗透到市场。例如，Intellia Therapeutics 评估了单剂量NTLA-2001（一种CRISPR/Cas9 疗法）与安慰剂相比，对转甲状腺素蛋白淀粉样变性心肌病变(ATTR-CM) 患者的疗效和安全性，我们已经启动了一项多国III 期临床试验。该试验（MAGNITUDE）将于 2023 年 12 月开始，预计 2028 年 4 月完成。

本报告提供全球CRISPR Cas9市场相关调查，提供市场概要，以及药物趋势，临床试验趋势，提供着各地区趋势，及加入此市场的主要企业竞争情形等资讯。

Global & Regional: Commercial & Clinical Outlook
Global CRISPR-Cas9 Therapy Clinical Trials By Company, County, Indication & Phase
Number Of CRISPR-Cas9 Therapies In Trials: > 25 Therapies
Approved CRISPR-Cas9 Therapy: Casgevy (Exagamglogene Autotemcel) Therapy Clinical Insight
Approved CRISPR-Cas9 Therapy Pricing & Dosing Analysis
Technology Platforms For Advancing CRISPR-Cas9 Therapy
Delivery Methodologies For CRISPR-Cas9
Competitive Landscape

Antecedently, out-of-date bestseller conventional therapies, including chemotherapy or targeted therapies, were utilized for the management of cancer, autoimmune, inflammation and other diseases; yet, clinical researchers were occupied tenaciously with the hypothesis of discovering other cutting edge technologies. The growing rampant of genetic disorders across the globe at a frightening gait as well as the need for an effective cure has led to the discovery of CRISPR/Cas9 therapy. Build on the triumph of gene therapies, these therapies clenches gargantuan plausibility with only one CRISPR/Cas9 therapy; Casgevy, approved into the commercial market, as of July 2024, to treat sickle cell anemia along with beta thalassemia and is currently accessible in US, EU, UK, Saudi Arabia and Bahrain; proposing novel opportunity in the approaching years.

With respect to KuicK Research statistics, we anticipate to perceive the first wave of CRISPR/Cas9 therapies receiving regulatory approvals over and above entering the market in upcoming 5 years due to the fact that as these therapies demonstrate their effectiveness in addition to safety in real-world settings, they will pave the way for broader acceptance and application of CRISPR/Cas9 based treatments. Coupled with this, the potential applications of this groundbreaking gene-editing technology are expected to expand dramatically beyond genetic disorders and cancers and will be utilized to cure cardiovascular, neurodegenerative together with CNS diseases as evident from rising preclinical as well as clinical studies, touching fundamentally every area of medicine and biotechnology.

Until now, the clinical pipeline for global CRISPR/Cas9 therapy is up surging year by year with multiple therapies have entered into clinical trials and other CRISPR/Cas9 candidates are lately in the preclinical stage. Many of ongoing CRISPR/Cas9 clinical trials have reached late stage of development and it is predicable that they will penetrate into the market in the imminent 5-7 years. For instance, Intellia Therapeutics has begun a phase III, multinational, multicenter, double-blind, placebo-controlled study is ongoing which aims to evaluate the efficacy and safety of a single dose of NTLA-2001, a CRISPR/Cas9 therapy, compared to placebo in participants with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The study (MAGNITUDE) was begun in December 2023 and is anticipated to be complete by April 2028 coupled with clenches an enrollment of 765 participants.

Just as importantly, the over-all market for CRISPR/Cas9 therapies is estimated to grow substantively and will become a multi-billion-dollar industry over the impending eons, driven by the snowballing number of preclinical and clinical trials, the likely regulatory approvals, in conjugation with the increase of indications. Currently, the US dominates the CRISPR/Cas9 sphere in terms of research and development activities, but EU is correspondingly far-sighted momentous evolution in this field.

As well as, the current approved CRISPR/Cas9 therapy; Casgevy, developed by CRISPR Therapeutics and Vertex Pharmaceutical, is launched into the market at an average cost of roughly US$ 2 Million per single course of treatment according to Casgevy developer company SEC filings. Additionally, the anticipated high prices of these therapies, justified by their potential curative nature, are expected to further drive significant market growth in terms of revenue, even with relatively small patient populations.

As a final point, all elucidations aforesaid epitomize that the global CRISPR/Cas9 therapy is growing at a trailblazing gait and is predictable to multiply additional in the forthcoming 10 years. The expanding clinical application as well as together with indication of CRISPR/Cas9, mounting preclinical and clinical trials, rise in technological advancement, presences of key players like Intellia Therapeutics, CRISPR Therapeutics, Editas Medicine, TransCode Therapeutics and many more, combination studies of CRISPR/Cas9 with other therapies coupled with augment in investment, collaborations and government bestow are driving the market penetration, offering opportunism to pharma and biotech companies in future.

1. Synopsis To CRISPR-Cas9 Therapy

1.1 Overview
1.2 Approved CRISPR-Cas9 Therapies

2. Delivery Methods For CRISPR-Cas9

2.1 Delivery of CRISPR-Cas9 Components Through Physical Methods
- 2.1.1 Microinjection
- 2.1.2 Electroporation
- 2.1.3 Hydrodynamic Delivery
2.2 Delivery of CRISPR-Cas9 Components through Viral Vectors
- 2.2.1 Adeno-Associated Virus (AAV)
- 2.2.2 Lentivirus & Adenovirus
2.3 Delivery of CRISPR-Cas9 Components through Non-Viral Vectors (Biomaterials)
- 2.3.1 Liposomes
- 2.3.2 Gold Nanoparticles
- 2.3.3 DNA Nanoclew

3. CRISPR-Cas9: A Home To Variety Of Applications

3.1 Advantages Accompanying CRISPR-Cas9 Technology
3.2 CRISPR-Cas9 Comparison with other Genome Editing Tools

4. Global CRISPR-Cas9 Therapy Clinical Trials Overview

4.1 By Company
4.2 By Country
4.3 By Indication
4.4 By Phase
4.5 By Priority Status

5. Global CRISPR-Cas9 Therapy Clinical Trials By Company, County, Indication & Phase

5.1 Research
5.2 Preclinical
5.3 Phase I
5.4 Phase I/II
5.5 Phase II

6. Approved CRISPR - Cas9 Therapy: Casgevy (Exagamglogene Autotemcel) Therapy Clinical Insight

7. Approved CRISPR-Cas9 Therapy - Overview, Pricing & Dosing Analysis

7.1 Casgevy (Exagamglogene Autotemcel)
- 7.1.1 Overview
- 7.1.2 Pricing & Dosing

8. Global CRISPR-Cas9 Therapy Current Market Trend & Developments

8.1 Current Market Outline
8.2 Future Market Outlook

9. CRISPR-Cas9 Therapy Market Trend Analysis By Region

9.1 US
9.2 UK
9.3 EU
9.4 China
9.5 Japan
9.6 South Korea

10. CRISPR-Cas9 Therapy Market Opportunities & Clinical Trial Statistics: By Indications

10.1 Inherited Blood Disorders
10.2 Genetic Disorders
10.3 Cancer
10.4 Autoimmune & Inflammatory Disorders
10.5 Microbial Infections
10.6 Cardiovascular Illnesses
10.7 Eye Disorders, Brain Diseases, Metabolic Illness & Other Conditions

11. Combination Approaches For CRISPR-Cas9 Therapy

12. Platforms For Advancing CRISPR-Cas9 Therapy

12.1 CRISPR-GNDM Modalis Therapeutics Platform
12.2 Intellia Therapeutics - Modular Platform
12.3 Editas Medicine CRISPR Gene Editing Platform
12.4 CRISPR-Cas9 Genome Editing Technology - ERS Genomics
12.5 CRISTARS Gene Editing Technology Innovation Platform- BRL Medicine
12.6 Gene Editing-BDmRNA Platform Shanghai BDgene
12.7 Kamau Therapeutics Next-Generation Gene Editing Platform
12.8 Excision BioTherapeutics CRISPR Gene Editing Technology

13. Global CRISPR-Cas9 Therapy Market Dynamics

13.1 Market Drivers
13.2 Market Achilles Heels

14. Competitive Landscape

14.1 4D Molecular Therapeutics
14.2 Arbor Biotechnologies
14.3 ASC Therapeutics
14.4 C4U Corporation
14.5 Casebia Therapeutics
14.6 CRISPR Therapeutics
14.7 Editas Medicine
14.8 Intellia Therapeutics
14.9 Nkarta Therapeutics
14.10 Vertex Pharmaceuticals

简介目录图表

List of Tables

Table 1-1: Global - Approved CRISPR-Cas9 Therapies
Table 9-1: US - Ongoing CRISPR-Cas9 Clinical Trials
Table 9-2: CRISPR-Cas9 Candidates Granted FDA Designations
Table 9-3: EU - Ongoing Clinical Trials for CRISPR/Cas9-based Therapeutics, July'2024
Table 9-4: ERS Genomics - Collaborations
Table 10-1: Ongoing CRISPR-Cas9 Clinical Trials for Inherited Blood Disorders
Table 10-2: Ongoing CRISPR-Cas9 Clinical Trials for Cancer Disorders
Table 10-3: Collaboration Deals For Cancer CRISPR-Cas9 Therapy, July 2024
Table 11-1: Ongoing Preclinical & Clinical trials for Combination of CRISPR-Cas9 Therapy

List of Figures

Figure 1-1: CRISPR-Cas9 Therapy v/s Conventional Therapies
Figure 2-1: Delivery Methods for CRISPR-Cas9
Figure 2-2: Delivery Pathways for CRISPR-Cas9 Components
Figure 2-3: Physical Mode of Delivery for CRISPR-Cas9 Components
Figure 2-4: Viral Mode of Delivery for CRISPR-Cas9 Components
Figure 2-5: Non-Viral Mode of Delivery for CRISPR-Cas9 Components
Figure 3-1: Advantages of CRISPR-Cas9 Technology
Figure 3-2: Comparison between Different Genome Editing Tools
Figure 4-1: Global - CRISPR-Cas9 Therapy Clinical Trials by Company (Number of Drugs), 2024 till 2029
Figure 4-2: Global - CRISPR-Cas9 Therapy Clinical Trials by Country (Number of Drugs), 2024 till 2029
Figure 4-3: Global - CRISPR-Cas9 Therapy Clinical Trials by Indication (Number of Drugs), 2024 till 2029
Figure 4-4: Global - CRISPR-Cas9 Therapy Clinical Trials by Phase (Number of Drugs), 2024 till 2029
Figure 4-5: Global - CRISPR-Cas9 Therapy Clinical Trials by Priority Status (Number of Drugs), 2024 till 2029
Figure 7-1: Casgevy - Approval Year by Region
Figure 7-2: Casgevy - Patent Expiration Year, July'2024
Figure 7-3: US - Cost per Treatment of Casgevy (US$ in Million), July'2024
Figure 8-1: Global CRISPR-Cas9 Therapy - Important Companies
Figure 8-2: Dominating & Emerging Region in Global CRISPR-Cas9
Figure 8-3: CRISPR-Cas9 - Future Market
Figure 8-4: Global - CRISPR Cas9 Market Opportunity (US$ Million), 2024 -2029
Figure 9-1: CRISPR_SCD001 Phase I/II (NCT04774536) Study - Initiation & Completion Year
Figure 9-2: Intellia Therapeutics - In vivo & Ex vivo CRISPR Approach
Figure 9-3: Crucial US CRISPR-Cas9 Competitor
Figure 9-4: EBT-101 Phase I (NCT05144386) Study - Initiation & Completion Year
Figure 9-5: NTLA-2002 Phase I/II (NCT05120830) Study - Initiation & Completion Year
Figure 9-6: EU Major Hubs in CRISPR-Cas9 Therapy
Figure 9-7: BRL-101 Phase I (NCT06300723) Study - Initiation & Completion Year
Figure 9-8: ET-01 Phase I (NCT04925206) Study - Initiation & Completion Year
Figure 9-9: GRIT Biotechnology Clinical Pipeline
Figure 9-10: GT307 (NCT06453057) Study - Initiation & Completion Year
Figure 9-11: CRISPR-Cas9 - Key Players in China
Figure 9-12: Aspects Influencing Japanese CRISPR-Cas9 Market
Figure 9-13: Modalis Therapeutics & JCR Pharmaceuticals Collaboration
Figure 9-14: ToolGen's TGT-001 - Orphan Drug Designation
Figure 10-1: BRL-101 Phase I (NCT06287086) Study - Initiation & Completion Year
Figure 10-2: Acquisition of Graphite Bio Lead Candidate & CRISPR-Cas9 Platform by Kamau Therapeutics
Figure 10-3: Nula-cel Phase I/II (NCT04819841) Study - Initiation & Completion Year
Figure 10-4 REGV131-LNP1265 Phase I/II (NCT06379789) Study - Initiation & Completion Year
Figure 10-5: Sanofi & Scribe Therapeutics Collaboration
Figure 10-6: Discovery of Innovative CRISPR-Cas9 Approach by University of Trento
Figure 10-7: National Institutes of Health Grants for CRISPR-Cas9 Therapy
Figure 10-8: NTLA-2001 Phase I (NCT04601051) Study - Initiation & Completion Year
Figure 10-9: NTLA-2001 Phase III MAGNITUDE (NCT06128629) Study - Initiation & Completion Year
Figure 10-10: Commercial Genetic Disorder CRISPR-Cas9 Therapy Market Overview
Figure 10-11: CRISPR-Cas9 Approaches To Cure Cancer
Figure 10-12: CRISPR-Cas9 Combinations with Cryotherapy & Others For Cancer Treatment
Figure 10-13: CTX112 Phase I/II (NCT05643742) Study - Initiation & Completion Year
Figure 10-14: MEDiC Life Sciences Collaboration with Bristol Myers Squibb (BMS) on Tumor Target Discovery
Figure 10-15: Innovative Genomics Institute Gene Correction Strategy for Autoimmune Patients
Figure 10-16: BDgene Reported Optimistic Phase I Outcomes of BD111
Figure 10-17: CRISPR Therapeutics Pipeline
Figure 10-18: BD113vLVP (NCT06465537) Study - Initiation & Completion Year
Figure 10-19: ZVS203e Phase I (NCT05805007) Study - Initiation & Completion Year
Figure 10-20: Editas Medicine CRISPR-Cas9 Therapies Synopsis
Figure 10-21: EDIT-101 Phase I/II (NCT03872479) Study - Initiation & Completion Year
Figure 10-22: SNIPR Biome - CRISPR-Cas9 Engineered Microbial Gene Therapy For Various Indications
Figure 12-1: CRISPR-GNDM Modalis Therapeutics Platform
Figure 12-2: Advantage of CRISPR-GNDM
Figure 12-3: Editas Medicine - CRISPR Gene Editing Platform
Figure 12-4: Gene Editing Technology Innovation Platform (CRISTARS) - BRL Medicine
Figure 12-5: Gene Editing-BDmRNA Platform - BDgene
Figure 12-6: Kamau Therapeutics Next Generation Gene Editing Platform
Figure 12-7: Excision's CRISPR Approach
Figure 12-8: Excision BioTherapeutics CRISPR Gene Editing Technology
Figure 13-1: CRISPR-Cas9 Therapy - Market Drivers
Figure 13-2: CRISPR-Cas9 - Market Challenges

02-2729-4219

+886-2-2729-4219

License/价格

CRISPR Cas9全球市场:市场机会及临床试验预测(2029年)

Global CRISPR Cas9 Market Opportunity & Clinical Trials Outlook 2029

目录

第1章 CRISPR-Cas9疗法的摘要

第2章 CRISPR-Cas9通知方法

第3章 CRISPR-Cas9:多种多样的用途的场

第4章全球CRISPR-Cas9疗法,临床试验概要

第5章全球CRISPR-Cas9疗法临床试验:企业，国家，适应症，各阶段

第6章核准的 CRISPR-Cas9 疗法：Casgevy (Exagamglogene Autotemcel) 疗法的临床见解

第7章已通过核准CRISPR-Cas9疗法- 概要，价格设定，剂量分析

第8章全球CRISPR-Cas9疗法的目前市场趋势与发展

第9章 CRISPR-Cas9疗法市场趋势分析，各地区

第10章 CRISPR-Cas9 疗法市场机会与临床试验统计：依适应症分类

第11章 CRISPR-Cas9疗法的复合方法

第12章 CRISPR-Cas9疗法的进步的平台

第13章全球CRISPR-Cas9疗法市场动态

第14章竞争情形