世界各国のリアルタイムなデータ・インテリジェンスで皆様をお手伝い

新しい遺伝子治療:技術、臨床、法規制、競争の動向

Emerging Gene Therapies – Trends within the Technological, Clinical, Regulatory and Competitive Landscape

 

出版社 出版年月電子版価格 図表数
CBR Pharma Insights
CBRファーマインサイツ
2019年1月US$2,995
シングルユーザライセンス(PDF)
68

サマリー

米国調査会社CBRファーマインサイツ(CBR Pharma Insights)の調査レポート「新しい遺伝子治療:技術、臨床、法規制、競争の動向」は、新しい遺伝子治療の市場を概説している。ゲノム編集に関連する遺伝子治療や技術を論じ、その利点や限界について述べ、開発中のプラットフォームに関する最新のエビデンスを記載している。特定の治療適応症のそれぞれの臨床研究についてや、現在の課題、眼、肝臓、血液などの治療事例にも注目している。

Description

GBI Research’s latest report “Emerging Gene Therapies – Trends within the Technological, Clinical, Regulatory and Competitive Landscape” provides a comprehensive overview of the emerging gene therapy market. The report discusses gene therapy and the technology behind gene editing, outlining the advantages, limitations and current evidence for the platforms under development. The report discusses relevant clinical studies targeting specific therapeutic indications and highlights examples of current challenges within the field, with a focus on therapies that target the eye, liver, and blood.

Additionally, the report provides a background to the CRISPR patent litigation, a key factor within the gene editing company landscape. It provides profiles of six companies developing gene editing platforms, considers the gene therapy interests of the main pharmaceutical companies, and discusses current regulatory trends in the development of gene therapies.

The report explores how emerging gene editing products will compete with established products, their relative competitive strengths, and upcoming value inflection points within the field.

Scope

  • What are the key emerging products within the gene therapy landscape?

  • Which companies have the strongest pipeline of innovative products?

  • How will gene editing disrupt existing gene therapy products?

  • What are the regulatory trends for emerging gene therapies?

  • What are the interests of pharmaceutical companies within the field?

Reasons To Buy

  • Achieve an up-to-date understanding of the area, with a comprehensive reference of key products within the gene therapy landscape, compared across technology-specific relevant characteristics such as editing mechanism and delivery vector.

  • Conduct competitive analysis using indication-specific, side-by-side comparisons of the latest data for key gene therapy products in the strategically relevant areas of eye, blood, and liver.

  • Conduct strategic analysis using an overview of gene therapy specific considerations for evaluating and developing gene therapy products – the CRISPR patent space, emerging regulatory trends, innovation leaders and the interests of pharma in gene therapy.



目次

1 Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Introduction

2.1 Gene Therapy - Definitions

2.2 Report Coverage – the Emerging Gene Therapy Pipeline

2.3 History of Gene Therapy

2.4 Limitations of Gene Transfer

2.5 The Development of Targeted Gene Editing

2.6 Overview of Gene Editing Platforms

2.6.1 Zinc Fingers (1996)

2.6.2 Transcription Activator-Like Effectors (2011)

2.6.3 The CRISPR/Cas System (2013)

2.6.4 Effectors for Targeting Domains

2.6.5 Comparison of Gene Editing Systems

2.6.6 Summary of Gene Editing Systems

2.7 Overview of In Vivo Gene Therapy

2.7.1 Editing is Dependent on Cell Type, Stage, and Repair Pathway

2.7.2 Delivery

2.7.3 Emerging Safety Concerns with Editing Platforms

2.7.4 Editing Products are Reliant on the Target Cell’s Cycle Stage and DNA Repair Machinery

2.7.5 Advantages of Gene Editing over Gene Transfer

2.7.6 Integration into ‘Safe Harbor’ Sites

2.7.7 The Increasing Complexity of Gene Therapy

2.7.8 Summary of In Vivo Gene Therapy

3 Gene Therapy – Near Term Product Pipeline

3.1 Leber Congenital Amaurosis

3.1.1 Unmet Need

3.1.2 Molecular Genetics

3.1.3 Luxturna (Voretigene neparvovec)

3.1.4 Editas Medicine: EDIT-101

3.1.5 Trial Design

3.1.6 EDIT-101 and Off-Target Effects

3.1.7 The Potential Advantage of EDIT-101 is the Longevity of its Therapeutic Effect

3.1.8 Summary – LCA

3.2 Choroideremia

3.3 Hurler Syndrome (MPS I)

3.3.1 Key Clinical Studies

3.3.2 Regenex: RGX-111

3.3.3 Sangamo Therapeutics: SB-318

3.4 Hunter Syndrome (MPS II)

3.4.1 Unmet Need

3.4.2 Sangamo Therapeutics: SB-913

3.4.3 Immusoft Corporation: Cell Therapy

3.5 Sanfilippo Syndrome (MPS III)

3.5.1 Lysogene: LYS-SAF302

3.6 Summary – MPS Disorders

3.7 Hemophilia

3.7.1 Hemophilia A

3.7.2 Summary – Hemophilia A

3.7.3 Hemophilia B

3.7.4 Summary – Hemophilia B

3.8 Hemoglobinopathies

3.8.1 Beta Thalassemia: Unmet Need

3.8.2 Beta Thalassemia: Molecular Genetics

3.8.3 Sickle Cell Disease: Unmet Need

3.8.4 Sickle Cell Disease: Molecular Genetics

3.9 Cellular Therapies for Hemoglobinopathies

3.9.1 Blue Bird Bio: BB-305 (‘LentiGlobin’)

3.9.2 Sangamo: ST-400

3.9.3 CRISPR Therapeutics: CTX-001

3.9.4 Summary: Cellular Therapies for Hemoglobinopathies

3.1 Duchenne Muscular Dystrophy

3.10.1 Unmet Need

3.10.2 Molecular Genetics

3.10.3 ExonDys 51 – Sarepta Therapeutics

3.10.4 Solid BioSciences: SGT-001

3.10.5 Exonics Therapeutics: CRISPR Approach

3.10.6 Summary – Duchenne Muscular Dystrophy

4 Competitive Landscape

4.1 Regulatory Considerations for Developing Gene Therapy Products

4.1.1 Product Characteristics

4.1.2 Clinical Study Design for Gene Therapy Products

4.1.3 Disease specific guidance

4.1.4 Reimbursement and Payment

4.1.5 Summary – Regulatory Considerations

4.2 Intellectual Property - CRISPR/Cas

4.2.1 Licensing, Exploitation, and MPEG Pool

4.3 Company Analysis: Gene Editing Companies

4.3.1 Sangamo Therapeutics

4.3.2 CRISPR Therapeutics

4.3.3 Casebia Therapeutics

4.3.4 Editas Medicine

4.3.5 Intellia Therapeutics

4.3.6 Homology Medicines

4.4 Company Analysis: Pharma

4.4.1 Amgen

4.4.2 Gilead Sciences

4.4.3 Novartis

4.4.4 Sanofi

4.4.5 GlaxoSmithKline

4.4.6 Pfizer

5 Appendix

5.1 References

5.2 Report Methodology

5.3 About GBI Research

5.4 Disclaimer

 

List of Figures

Figure 1: Nucleases Based on Protein-DNA Interactions

Figure 2: Transcription Activator-Like Effector Nucleases (TALENs)

Figure 3: The CRISPR/Cas System

Figure 4: CRISPR/Cas Binding Mechanism

Figure 5: Cas9 Orthologs

Figure 6: Gene Therapy Clinical Trials Worldwide by Vector

Figure 7: Multiple Sangamo Therapeutics Products Use an Albumin Targeting ‘Safe Harbor’ Approach

Figure 8: Gene Editing Landscape, Progression of Gene Therapy Applications

Figure 9: Gene Editing Landscape, Hemophilia A + B

Figure 10: SPK-9001: Factor IX Activity after SPK-9011 in 8 Participants that did not Show AAV Capsid-Directed Immune Response

Figure 11: Cost of Beta-Thalassemia Major Treatment as of NHS Tariffs at 2013/14 Prices

Figure 12: BCL11A is Involved in Fetal Hemoglobin Silencing

 

List of Tables

Table 1: Pipeline Products Covered

Table 2: Properties of Standard Gene-Editing Nucleases

Table 3: Registered Clinical Trials of CAR-T Cell Therapies Using Gene-Editing

Table 4: Gene Therapy Clinical Trials Worldwide by Vector

Table 5: Gene Editing Landscape, Vector Differentiation by Target Tissue

Table 6: Gene Editing Landscape, Key Off-Target Effect Studies

Table 7: Pre-existing Immunity to Cas9

Table 8: Characterization of the Rate of Homology Directed Repair in Range of Cell Lines

Table 9: Comparison of Gene Editing and Gene Transfer Approaches

Table 10: Gene Editing Landscape, Pipeline of Targeted Gene Editing Products by Company

Table 11: Luxturna Clinical Studies

Table 12: EDIT-101 – Phase I/II Trial Design and Comparison to Luxturna Efficacy Study

Table 13: Dose Response in CEP290 Gene Editing and CRISPR Expression

Table 14: Gene editing landscape, Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), Diagnosed, Prevalent Cases, 2017-2027

Table 15: Key Phase I/II Studies in MPS I

Table 16: Gene editing landscape, Mucopolysaccharidosis II (MPS II) (Hunter Syndrome), Diagnosed, Prevalent Cases, 2017-2027.

Table 17: SB-913 Trial Design

Table 18: SB-913 – Interim Results

Table 19: LYS-SAF302 Phase II/III Trial Design

Table 20: Gene Editing Landscape, MPS Disorders

Table 21: Gene Editing Landscape, Hemophilia Epidemiology and Forecast, 2016-2026.

Table 22: Scale of Hemophilia Severity

Table 23: Phase I/II Hemophilia A Trials

Table 24: BMN-270 Factor VIII Levels at 1.5 Years (High Dose)

Table 25: Gene Therapy Landscape, Phase I/II Clinical Trial Design for Hemophilia A

Table 26: Gene Therapy Landscape, Summary of Key Pipeline Gene Therapies for Hemophilia A

Table 27: Phase I/II Clinical Studies

Table 28: Gene Therapy Landscape, Overview Of Key Pipeline Products in Hemophilia B

Table 29: Gene editing landscape, Thalassemia, Diagnosed, Prevalent Cases, 2017-2027

Table 30: Frequent Mutations Causing SCD

Table 31: Phase I/II Clinical Studies

Table 32: LentiGlobin - Ongoing Phase III Trials in Beta Thalassemia

Table 33: Results of LentiGlobin trials in Thalassemia and Sickle Cell Disease

Table 34: ST-400 Trial Design Compared with LentiGlobin

Table 35: CTX-001 trial design (Left) against ST-400 and LentiGlobin (Right)

Table 36: LentiGlobin (BB-305) may Achieve Blockbuster Status by 2023

Table 37: Gene Therapy Landscape, DMD, Global Prevalence (%)

Table 38: Exondys 51 – Clinical Studies

Table 39: SGT- 001 Study Design

Table 40: Gene Therapy Landscape, Key Patents in the CRISPR Dispute

Table 41: Gene Therapy Landscape, IP Estates of CRISPR Companies

Table 42: Gene Therapy Landscape, ERS Genomics EU Licensing 2004-2018

Table 43: Sangamo Therapeutics Pipeline, September 2018

Table 44: Sangamo Therapeutics Partnerships

Table 45: Sangamo Therapeutics SWOT

Table 46: CRISPR Therapeutics Pipeline

Table 47: CRISPR Therapeutics Pipeline

Table 48: CRISPR Therapeutics SWOT

Table 49: Casebia Pipeline, September 2018

Table 50: Editas Medicine Pipeline, September 2018

Table 51: Editas Medicine Pipeline, September 2018.

Table 52: Editas Medicine SWOT

Table 53: Intellia Therapeutics Pipeline

Table 54: Intellia Therapeutics SWOT

Table 55: Homology Medicines Pipeline

Table 56: Homology Medicines SWOT

 

ページTOPに戻る

あなたが最近チェックしたレポート一覧

  • 最近チェックしたレポートはありません。

お問合は、お電話またはWEBから承ります。お見積もりの作成もお気軽にご相談ください。

webからのお問合せはこちらのフォームから承ります

このレポートへのお問合せ

03-3582-2531

電話お問合せもお気軽に

<無料>メルマガに登録する

 

 

ページTOPに戻る