それに先立って、化学療法や標的療法などの時代遅れのベストセラーである従来の治療法が、癌、自己免疫、炎症、その他の疾患の管理に利用されました。しかし、臨床研究者は、他の最先端技術を発見するという仮説に固執していました。世界中で恐ろしい歩行で遺伝性疾患が蔓延していることと、効果的な治療法の必要性が、CRISPR/Cas9療法の発見につながっています。遺伝子治療の勝利を基盤に、これらの治療法はたった1つのCRISPR/Cas9療法で巨大な説得力を持ちます。Casgevyは、2024年7月にベータサラセミアとともに鎌状赤血球貧血の治療薬として商業市場に承認され、現在、米国、EU、英国、サウジアラビア、バーレーンで入手可能です。今後数年間に斬新な機会を提案します。
KuicK Researchの統計では、これらの治療法が実際の環境での安全性に加えて有効性を実証することで、CRISPR/Cas9ベースの治療薬のより広範な受容と適用への道を開くという事実から、今後5年以内に規制当局の承認を受ける最初の波が市場に参入すると予想されています。これと相まって、この画期的な遺伝子編集技術の潜在的な応用は、遺伝性疾患や癌を超えて劇的に拡大することが期待されており、前臨床および臨床研究の増加から明らかなように、心血管、神経変性、およびCNS疾患の治療に利用され、医学とバイオテクノロジーの根本的にあらゆる分野に影響を及ぼします。
目次
Table of Content
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 and 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 and 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 and Dosing Analysis
7.1 Casgevy (Exagamglogene Autotemcel)
7.1.1 Overview
7.1.2 Pricing and Dosing
8. Global CRISPR-Cas9 Therapy Current Market Trend and 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 and Clinical Trial Statistics: By Indications
10.1 Inherited Blood Disorders
10.2 Genetic Disorders
10.3 Cancer
10.4 Autoimmune and Inflammatory Disorders
10.5 Microbial Infections
10.6 Cardiovascular Illnesses
10.7 Eye Disorders, Brain Diseases, Metabolic Illness and 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
List of Figures & Tables
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 and 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 and Completion Year
Figure 9-2: Intellia Therapeutics - In vivo and Ex vivo CRISPR Approach
Figure 9-3: Crucial US CRISPR-Cas9 Competitor
Figure 9-4: EBT-101 Phase I (NCT05144386) Study - Initiation and Completion Year
Figure 9-5: NTLA-2002 Phase I/II (NCT05120830) Study - Initiation and Completion Year
Figure 9-6: EU Major Hubs in CRISPR-Cas9 Therapy
Figure 9-7: BRL-101 Phase I (NCT06300723) Study - Initiation and Completion Year
Figure 9-8: ET-01 Phase I (NCT04925206) Study - Initiation and Completion Year
Figure 9-9: GRIT Biotechnology Clinical Pipeline
Figure 9-10: GT307 (NCT06453057) Study - Initiation and 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 and JCR Pharmaceuticals Collaboration
Figure 9-14: ToolGen’s TGT-001 - Orphan Drug Designation
Figure 10-1: BRL-101 Phase I (NCT06287086) Study - Initiation and Completion Year
Figure 10-2: Acquisition of Graphite Bio Lead Candidate and CRISPR-Cas9 Platform by Kamau Therapeutics
Figure 10-3: Nula-cel Phase I/II (NCT04819841) Study - Initiation and Completion Year
Figure 10-4 REGV131-LNP1265 Phase I/II (NCT06379789) Study - Initiation and Completion Year
Figure 10-5: Sanofi and 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 and Completion Year
Figure 10-9: NTLA-2001 Phase III MAGNITUDE (NCT06128629) Study - Initiation and 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 and Others For Cancer Treatment
Figure 10-13: CTX112 Phase I/II (NCT05643742) Study - Initiation and 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 and Completion Year
Figure 10-19: ZVS203e Phase I (NCT05805007) Study - Initiation and Completion Year
Figure 10-20: Editas Medicine CRISPR-Cas9 Therapies Synopsis
Figure 10-21: EDIT-101 Phase I/II (NCT03872479) Study - Initiation and 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
Table 1-1: Globally 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 and Clinical trials for Combination of CRISPR-Cas9 Therapy
