サンプル依頼リスト カート
  • English
  • Korean
  • Traditional Chinese
  • Simplified Chinese
市場調査レポート
商品コード
1529409

CRISPR Cas9の世界市場：市場機会と臨床試験の見通し（2029年）

Global CRISPR Cas9 Market Opportunity & Clinical Trials Outlook 2029

出版日
ページ情報
英文 180 Pages
納期
即日から翌営業日
価格
価格表記: USDを日本円(税抜)に換算
本日の銀行送金レート: 1USD=144.06円

ご注意:　DRM (デジタル著作権管理システム) 付PDFになります。制限内容は、上記ライセンスの[詳細]アイコンをクリックしてご確認ください。

CRISPR Cas9の世界市場：市場機会と臨床試験の見通し（2029年）
出版日: 2024年08月01日
発行: KuicK Research
ページ情報: 英文 180 Pages
納期: 即日から翌営業日
GIIご利用のメリット
  • 全表示
  • 概要
  • 図表
  • 目次
概要

以前は、がん、自己免疫疾患、炎症性疾患、その他の疾患の管理には、化学療法や分子標的治療を含む従来の治療法が利用されていましたが、臨床研究者は他の最先端技術を発見するという仮説に粘り強く取り組んできました。世界中で遺伝性疾患が恐ろしい勢いで蔓延しており、効果的な治療法の必要性が高まっていることから、CRISPR/Cas9療法が発見されました。CRISPR/Cas9療法は、2024年7月現在、βサラセミアと鎌状赤血球貧血の治療薬として承認されており、米国、EU、英国、サウジアラビア、バーレーンで入手可能です。

今後5年間で、CRISPR/Cas9療法の第一波が規制当局の承認を受け、市場に参入することが予想されます。これは、これらの療法が実世界の環境で安全性に加えて有効性を実証するにつれて、CRISPR/Cas9ベースの治療の幅広い受け入れと応用への道が開かれるからです。これと相まって、この画期的な遺伝子編集技術の潜在的応用は、遺伝性疾患やがんを越えて劇的に拡大し、心血管疾患、神経変性疾患、中枢神経系疾患の治療に活用されることが期待されます。

これまで、世界のCRISPR/Cas9療法の臨床パイプラインは年々急増しており、複数の治療法が臨床試験に入り、他のCRISPR/Cas9候補も前臨床段階にあります。現在進行中のCRISPR/Cas9臨床試験の多くは開発後期段階に達しており、5年～7年以内に市場に浸透することが予測されます。例えば、Intellia Therapeuticsは、心筋症を伴うトランスサイレチンアミロイドーシス（ATTR-CM）患者を対象に、CRISPR/Cas9療法であるNTLA-2001の単回投与の有効性と安全性をプラセボと比較して評価することを目的とした第III相、多国籍、多施設、二重盲検、プラセボ対照試験を開始しました。この試験（MAGNITUDE）は2023年12月に開始され、2028年4月までに完了する予定です。

当レポートは、世界のCRISPR Cas9市場について調査し、市場の概要とともに、薬剤動向、臨床試験動向、地域別動向、および市場に参入する企業の競合情勢などを提供しています。

目次

第1章 CRISPR-Cas9療法の要約

第2章 CRISPR-Cas9の送達方法

  • 物理的方法によるCRISPR-Cas9コンポーネントの送達
  • ウイルスベクターによるCRISPR-Cas9コンポーネントの送達
  • 非ウイルスベクター（バイオマテリアル）によるCRISPR-Cas9コンポーネントの送達

第3章 CRISPR-Cas9：多様な用途の場

  • CRISPR-Cas9技術に伴う利点
  • CRISPR-Cas9と他のゲノム編集ツールの比較

第4章 世界のCRISPR-Cas9療法, 臨床試験の概要

  • 企業別
  • 国別
  • 適応症別
  • 相別
  • 優先度別

第5章 世界のCRISPR-Cas9療法臨床試験：企業、国、適応症、段階別

  • 調査
  • 前臨床
  • 第I相
  • 第I/II相
  • 第II相

第6章 承認済みCRISPR-Cas9療法：Casgevy（Exagamglogene Autotemcel）療法の臨床的洞察

第7章 承認済みCRISPR-Cas9療法- 概要、価格設定、投与量分析

  • Casgevy（Exagamglogene Autotemcel）
    • 概要
    • 価格と投与量

第8章 世界のCRISPR-Cas9療法の現在の市場動向と発展

  • 現在の市場の概略
  • 今後の市場見通し

第9章 CRISPR-Cas9療法市場動向分析、地域別

  • 米国
  • 英国
  • 欧州連合
  • 中国
  • 日本
  • 韓国

第10章 CRISPR-Cas9療法の市場機会と臨床試験統計：適応症別

  • 遺伝性血液疾患
  • 遺伝性疾患
  • がん
  • 自己免疫疾患および炎症性疾患
  • 微生物感染症
  • 心臓血管疾患
  • 眼疾患、脳疾患、代謝疾患およびその他の疾患

第11章 CRISPR-Cas9療法の複合アプローチ

第12章 CRISPR-Cas9療法の進歩のためのプラットフォーム

第13章 世界のCRISPR-Cas9療法市場力学

  • 市場促進要因
  • 市場の弱点

第14章 競合情勢

  • 4D Molecular Therapeutics
  • Arbor Biotechnologies
  • ASC Therapeutics
  • C4U Corporation
  • Casebia Therapeutics
  • CRISPR Therapeutics
  • Editas Medicine
  • Intellia Therapeutics
  • Nkarta Therapeutics
  • Vertex Pharmaceuticals
図表

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
目次

Global CRISPR Cas9 Market Opportunity & Clinical Trials Outlook 2029 Report Offering:

  • 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.

Table of Contents

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