アデノ随伴ウイルスベクター/AAVベクター市場:AAVベースの遺伝子治療とAAV製造にフォーカス:治療の種類(遺伝子増強療法、免疫療法、その他)、使用される遺伝子導入方法の種類(生体外および生体内)、標的治療領域(遺伝子疾患、血液疾患、感染症、代謝疾患、応用分野(遺伝子治療、細胞治療、ワクチン)、事業規模(前臨床、臨床、商業)、地域(北米、欧州、アジア太平洋、中東・アフリカ、中南米、その他の地域):業界動向と世界予測、2022-2035年Adeno-Associated Viral Vectors / AAV Vector Market: Focus on AAV Based Gene Therapy and AAV Manufacturing by Type of Therapy (Gene Augmentation, Immunotherapy and Others), Type of Gene Delivery Method Used (Ex vivo and In vivo), Target Therapeutic Area (Genetic Disorders, Hematological Disorders, Infectious Diseases, Metabolic Disorders, Muscle Disorders, Ophthalmic Disorders, Neurological Disorders and Others), Application Area (Gene Therapy, Cell Therapy and Vaccine), Scale of Operation (Preclinical, Clinical and Commercial) and Geographical Regions (North America, Europe, Asia Pacific, MENA, Latin America and Rest of the World): Industry Trends and Global Forecasts, 2022-2035 世界のAAVベクター市場は2022年に19億米ドルに達すると予測され、予測期間2022-2035年のCAGRは14%で成長すると予測されている。 近年、細胞レベルで病気の原因を狙い撃ちする可能性を活用した遺伝子治療への関... もっと見る
サマリー世界のAAVベクター市場は2022年に19億米ドルに達すると予測され、予測期間2022-2035年のCAGRは14%で成長すると予測されている。近年、細胞レベルで病気の原因を狙い撃ちする可能性を活用した遺伝子治療への関心と需要が急増している。現在、285以上の遺伝子治療薬がさまざまな臨床開発段階で評価されている。このような需要の高まりは多額の資金流入につながり、遺伝子治療開発企業は2021年だけで100億米ドルを超える資金を調達している。このような急成長を背景に、革新的な送達方法に対するニーズが高まっている。利用可能な遺伝子導入ベクターの中で、アデノ随伴ウイルスベクター(AAV)は非常に効率的であることが証明されている。現在までに6つのAAVベースの遺伝子治療が承認されており、そのうち5つが米国FDAによって承認され、最新のものは2023年6月に承認された。同時に、多数のAAVベースの治療法がさまざまな臨床試験で評価中である。近年、AAVベースの遺伝子治療を評価する臨床試験は30%という驚異的な伸びを示しており、今後3年以内に50以上の臨床試験が終了すると予想されている。 この需要の高まりに対応するため、AAVベースの遺伝子治療の開発と製造に注力する企業が世界中で100社近く出現している。これらの企業の多くは、アデノ随伴ウイルスベクターや関連療法を小規模から大規模まで様々な事業規模で製造できる高度なAAV技術プラットフォームを提供している。AAVベースの遺伝子治療開発企業の市場は、予測期間中に力強い成長が見込まれる。この軌跡は、AAV製造企業や高度なAAV技術プラットフォームのプロバイダーからの共同支援によって推進されるでしょう。安全で効果的な遺伝子治療デリバリーオプションとしてAAVベクターが広く認知され採用されていることが、この有望な市場展望を後押しする主要因となっています。 レポート範囲 当レポートでは、AAVベクター市場を分析し、治療タイプ、遺伝子導入方法、治療ターゲット、用途、事業規模、地域などの要因を検証しています。 市場成長の促進要因、阻害要因、機会、課題などを評価します。 主要市場プレイヤーの潜在的な利点、障壁、競争環境の評価を提供します。 主要6地域にわたる市場セグメントの収益予測を提示しています。 簡潔な概要で、アデノ随伴ウイルスベクター市場の現状と中長期的に予想される進化を概説。 アデノ随伴ウイルスベクターの構造設計、ライフサイクル・ダイナミクス、応用を包括的に論じ、その固有の利点と課題の分析で結んでいる。 AAVベースの遺伝子治療市場の詳細な調査には、開発段階、治療領域、標的遺伝子、治療タイプ、送達方法、投与経路、特効薬指定が含まれる。 AAV製造に携わる企業の調査には、設立の詳細、企業規模、本社、製造製品、施設所在地、製造タイプ、事業規模、応用分野が含まれます。 AAV市場に従事する企業が提供する技術を、種類、事業規模、応用分野、著名な市場プレイヤーの特定ごとに分類してレビュー。 上市済みおよび開発段階にある AAV ベースの遺伝子治療薬の詳細なプロフィールは、開発スケジュール、現況、作用機序、関連 AAV 技術、特許ポートフォリオ、用法・用量、製造の詳細、開発企業情報を網羅しています。 AAV製造企業の概要、財務実績(入手可能な場合)、ベクター製造能力、将来展望を含む、厳選されたAAV製造企業のプロフィールを集計。 サプライヤーの強み、製造能力、サービス範囲、企業規模を考慮した 4 次元バブル表現を用いて、地域横断的な AAV 製造における企業の競争力を分析。 サプライヤーの強み、技術目的、事業規模、応用分野を考慮した AAV ベクター技術プラットフォームの詳細評価。 治験登録年、フェーズ、治療領域、地域、スポンサー、治療部位、登録患者集団などのパラメータを評価した、完了済み、進行中、および計画中の臨床試験の調査。 AAV製造に焦点を当てた2017年以降の提携およびパートナーシップの評価、パートナーシップの種類、治療領域、パートナーの種類、地域分布の調査。 開発者の強み、製品ポートフォリオ、治療フォーカス、パイプラインの強みを考慮した、AAVおよび遺伝子治療製品メーカーとのパートナーシップの可能性に関する洞察。 特許の種類、公開年、地域的適用可能性、特許出願における業界リーダー、特許評価など、2017年以降にAAVベースの療法に対して出願/付与された特許の包括的分析。 AAVベースの遺伝子治療を開発する新興企業を分析し、その開発段階、特許、パートナーシップ、主要指標を評価。 AAVベースの遺伝子治療開発企業が、自社製造か受託製造機関(CMO)のどちらを採用するかを決定する際に考慮すべき要素を、中小企業、中堅企業、大企業のパラメータを考慮しながら詳細に分析。 主要市場企業 アベオナ・セラピューティクス アルデブロン(ダナハーが買収) オックスフォード・バイオメディカ サノフィ(CEPiA、サノフィ・パスツール、ジェンザイム) WuXi AppTec YPOSKESI 目次1. PREFACE1.1. Scope of the Report 1.2. Market Segmentation 1.3. Research Methodology 1.4. Key Questions Answered 1.5. Chapter Outlines 2. EXECUTIVE SUMMARY 3. INTRODUCTION 3.1. Chapter Overview 3.2. Viral and Non-Viral Vectors 3.2.1. Viral Vectors 3.2.1.1. Adenovirus Vectors 3.2.1.2. Adeno-Associated Viral Vectors 3.2.1.3. Lentivirus Vectors 3.2.1.4. Retrovirus Vectors 3.2.1.5. Other Viral Vectors 3.2.1.5.1. Alphavirus 3.2.1.5.2. Foamy Virus 3.2.1.5.3. Simian Virus 3.2.1.5.4. Vaccinia Virus 3.2.1.5.5. Chimeric Viral Vectors 3.2.1.5.6. Herpes Simplex Virus 3.2.1.5.7. Sendai Virus 3.2.2. Non-Viral Vectors 3.2.2.1. Plasmid DNA 3.2.2.2. Oligonucleotides 3.2.2.3. Liposomes, Lipoplexes, and Polyplexes 3.2.2.4. Other Non-Viral Vectors 3.3. Adeno-Associated Viral Vectors 3.3.1. Structure and Design 3.3.2. Adeno-Associated Viral Vector Life Cycle 3.3.3. Applications of Adeno-Associated Viral Vectors 3.3.3.1. Gene Therapy 3.3.3.2. Vaccination 3.3.4. Advantages of Adeno-Associated Viral Vectors 3.3.5. Challenges Related to Adeno-Associated Viral Vectors 3.4. Concluding Remarks 4. AAV BASED GENE THERAPY: MARKET LANDSCAPE 4.1. Chapter Overview 4.2. AAV Based Gene Therapy: Overall Market Landscape 4.2.1. Analysis by Phase of Development 4.2.2. Analysis by Therapeutic Area(s) 4.2.3. Analysis by Type of Gene / Molecule Targeted 4.2.4. Analysis by Type of Therapy 4.2.5. Analysis by Type of Gene Delivery Method Used 4.2.6. Analysis by Route of Administration 4.3. AAV Based Gene Therapy Candidates: Special Designations 4.3.1. Analysis by Special Designation(s) Awarded 4.4. AAV Based Gene Therapy: List of Developers 4.4.1. Analysis by Year of Establishment 4.4.2. Analysis by Company Size 4.4.3. Analysis by Location of Headquarters 4.4.4. AAV Based Gene Therapy: Leading Developers 5. AAV MANUFACTURING COMPANIES: MARKET LANDSCAPE 5.1. Chapter Overview 5.2. AAV Manufacturing Companies: Overall Market Landscape 5.2.1. Analysis by Year of Establishment 5.2.2. Analysis by Company Size 5.2.3. Analysis by Location of Headquarters 5.2.4. Analysis by Type of Product(s) Manufactured 5.2.5. Analysis by Location of Vector Manufacturing Facilities 5.2.6. Analysis by Type of Manufacturer and Company Size 5.2.7. Analysis by Scale of Operation 5.2.8. Analysis by Location of Headquarters and Scale of Operation 5.2.9. Analysis by Application Area(s) 6. AAV TECHNOLOGY: MARKET LANDSCAPE 6.1. Chapter Overview 6.2. AAV Technology 6.2.1. Analysis by Type of Technology 6.2.2. Analysis by Scale of Operation 6.2.3. Analysis by Application Area(s) 6.2.4. Analysis by Therapeutic Area 6.3. AAV Technology: List of Developers 6.3.1. Analysis by Year of Establishment 6.3.2. Analysis by Company Size 6.3.3. Analysis by Location of Headquarters 6.3.4. Analysis by Company Size and Location of Headquarters 6.3.5. AAV Technology: Leading Developers 7. DRUG PROFILES 7.1. Chapter Overview 7.2. Marketed AAV Based Gene Therapies 7.2.1. LUXTURNA® (Spark Therapeutics) 7.2.1.1. Company Overview 7.2.1.2. Development Timeline 7.2.1.3. Mechanism of Action 7.2.1.4. Target Indication(s) 7.2.1.5. Current Status of Development 7.2.1.6. Manufacturing, Dosage and Sales 7.2.2. ZOLGENSMA® (Novartis) 7.2.2.1. Company Overview 7.2.2.2. Development Timeline 7.2.2.3. Mechanism of Action 7.2.2.4. Target Indication(s) 7.2.2.5. Current Status of Development 7.2.2.6. Manufacturing, Dosage and Sales 7.3.1. HEMGENIX® (CSL Behring / UniQure) 7.3.1.1. Company Overview 7.3.1.2. Development Timeline 7.3.1.3. Mechanism of Action 7.3.1.4. Target Indication(s) 7.3.1.5. Current Status of Development 7.3.1.6. Manufacturing, Dosage and Sales 7.4.1. ROCTAVIAN® (BioMarin Pharmaceuticals) 7.4.1.1. Company Overview 7.4.1.2. Development Timeline 7.4.1.3. Mechanism of Action 7.4.1.4. Target Indication(s) 7.4.1.5. Current Status of Development 7.4.1.6. Manufacturing, Dosage and Sales 7.5.1. ELEVIDYS® (Sarepta Therapeutics) 7.5.1.1. Company Overview 7.5.1.2. Development Timeline 7.5.1.3. Mechanism of Action 7.5.1.4. Target Indication(s) 7.5.1.5. Current Status of Development 7.5.1.6. Manufacturing, Dosage and Sales 7.6.1. UPSTAZA® (PTC Therapeutics) 7.6.1.1. Company Overview 7.6.1.2. Development Timeline 7.6.1.3. Mechanism of Action 7.6.1.4. Target Indication(s) 7.6.1.5. Current Status of Development 7.6.1.6. Manufacturing, Dosage and Sales 7.6. Late Stage (Phase II/III and Above) Gene Therapies 7.6.1. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.2. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.3. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.4. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.5. OAV-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.6. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.7. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.8. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.9. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 8. COMPANY PROFILES 8.1. Chapter Overview 8.2. Abeona Therapeutics 8.2.1. Company Overview 8.2.2. Financial Information 8.2.3. AAV Manufacturing Related Capabilities 8.2.4. Recent Developments and Future Outlook 8.3 Aldevron (Acquired by Danaher) 8.3.1. Company Overview 8.3.2. Financial Information 8.3.3. AAV Manufacturing Related Capabilities 8.3.4. Recent Developments and Future Outlook 8.4 Oxford BioMedica 8.4.1. Company Overview 8.4.2. Financial Information 8.4.3. AAV Manufacturing Related Capabilities 8.4.4. Recent Developments and Future Outlook 8.5 Sanofi (CEPiA, Sanofi Pasteur, Genzyme) 8.5.1. Company Overview 8.5.2. Financial Information 8.5.3. AAV Manufacturing Related Capabilities 8.5.4. Recent Developments and Future Outlook 8.6. WuXi AppTec 8.6.1. Company Overview 8.6.2. Financial Information 8.6.3. AAV Manufacturing Related Capabilities 8.6.4. Recent Developments and Future Outlook 8.7. YPOSKESI 8.7.1. Company Overview 8.7.2. AAV Manufacturing Related Capabilities 8.7.3. Recent Developments and Future Outlook 9. COMPANY COMPETITIVENESS ANALYSIS 9.1. Chapter Overview 9.2. Methodology and Key Parameters 9.3. AAV Manufacturing Companies: In-house Manufacturers 9.3.1. Players based in North America 9.3.2. Players based in Europe 9.4. AAV Manufacturing Companies: Contract Manufacturing Organizations 9.4.1. Players based in North America 9.4.2. Players based in Europe 9.4.3. Players based in Asia-Pacific 9.5. AAV Manufacturing Companies: Both In-House and Contract Manufacturing Organizations 9.5.1. Players based in North America 9.5.2. Players based in Europe 9.5.3. Players based in Asia-Pacific 10. TECHNOLOGY COMPETITIVENESS ANALYSIS 10.1. Chapter Overview 10.2. Methodology and Key Parameters 10.3. AAV Technology Platforms 10.3.1. AAV Technology Platforms Developed by Companies based in North America 10.3.2. AAV Technology Platforms Developed by Companies based in Europe and Asia-Pacific 11. CLINICAL TRIAL ANALYSIS 11.1. Chapter Overview 11.2. Scope and Methodology 11.3. AAV Based Gene Therapy: Clinical Trial Analysis 11.3.1. Analysis by Trial Registration Year 11.3.2. Analysis by Trial Registration Year and Enrolled Patient Population 11.3.3. Analysis by Trial Phase and Enrolled Patient Population 11.3.4. Analysis by Trial Status 11.3.5. Analysis by Study Design 11.3.6. Analysis by Therapeutic Area 11.3.7. Analysis by Target Disease Indication 11.3.8. Analysis by Type of Sponsor / Collaborator 11.3.9. Leading Industry Players: Analysis by Number of Registered Trials 11.3.10. Regional Analysis 11.3.10.1. Analysis by Number of Registered Trials 11.3.10.2. Analysis by Enrolled Patient Population 12. PARTNERSHIPS AND COLLABORATIONS 12.1. Chapter Overview 12.2. Partnership Models 12.3. AAV Based Gene Therapy: List of Partnerships and Collaborations 12.3.1. Analysis by Year of Partnership 12.3.2. Analysis by Type of Partnership 12.3.3. Analysis by Year of Partnership and Type of Partner 12.3.4. Analysis by Type of Partnership and Type of Partner 12.3.5. Analysis by Therapeutic Area 12.3.6. Most Active Players: Analysis by Number of Partnerships 12.3.7. Regional Analysis 12.3.7.1. Intercontinental and Intracontinental Agreements 12.4. AAV Manufacturing Companies: List of Partnerships and Collaborations 12.4.1. Analysis by Year of Partnership 12.4.2. Analysis by Type of Partnership 12.4.3. Analysis by Year of Partnership and Type of Partner 12.4.4. Analysis by Type of Partnership and Type of Partner 12.4.5. Analysis by Therapeutic Area 12.4.6. Most Active Players: Analysis by Number of Partnerships 12.4.7. Regional Analysis 12.4.7.1. Intercontinental and Intracontinental Agreements 13. STRATEGIC PARTNER ANALYSIS 13.1. Chapter Overview 13.2. AAV Manufacturing Companies: Potential Strategic Partners 13.2.1. Methodology and Key Parameters 13.2.2. AAV Manufacturing Companies: Strategic Partner Analysis 13.2.2.1. Most Likely Partners 13.2.2.2. Likely Partners 13.2.2.3. Less Likely Partners 13.2.2.4. Least Likely Partners 14. PATENT ANALYSIS 14.1. Chapter Overview 14.2. Scope and Methodology 14.3. Adeno-Associated Viral Vectors: Patent Analysis 14.3.1. Analysis by Publication Year 14.3.2. Analysis by Annual Patent Application(s) 14.3.3. Analysis by Annual Granted Patents 14.3.4. Analysis by Geography 14.3.5. Analysis by CPC Symbols 14.3.6. Analysis by Type of Applicant 14.3.7. Leading Industry Players: Analysis by Number of Patents 14.4. Adeno Associated Viral Vector Based Market: Patent Benchmarking 14.4.1. Analysis by Patent Characteristics 14.5. Adeno-Associated Viral Vectors: Patent Valuation 14.6. Leading Patents: Analysis by Number of Citations 15. START-UP HEALTH INDEXING 15.1. Chapter Overview 15.2. Start-ups Developing Adeno-Associated Viral Vectors 15.2.1. Analysis by Location of Headquarters 15.3. Benchmarking of Start-ups 15.3.1. Analysis by Pipeline Strength 15.3.2. Analysis by Pipeline Maturity 15.3.3. Analysis by Indication Diversity 15.3.4. Analysis by Number of Patents 15.3.5. Analysis by Partnership Activity 15.3.6. Start-ups Health Indexing: Roots Analysis Perspective 16. OUTSOURCING: GO / NO-GO FRAMEWORK 16.1. Chapter Overview 16.2. Outsourcing: Go / No-Go Framework 16.3. AAV Based Gene Therapy Developers Outsourcing: Go / No-Go Framework 16.3.1. Key Parameters and Assumptions 16.3.2. Methodology 16.3.3. Results and Interpretations 16.3.3.1. Small Companies 16.3.3.2. Mid-sized Companies 16.3.3.3. Large Companies 17. MARKET SIZING AND OPPORTUNITY ANALYSIS 17.1. Chapter Overview 17.2. Methodology and Key Assumptions 17.3. AAV Based Gene Therapy Market, 2022-2035 17.3.1. AAV Based Gene Therapy Market: Distribution by Therapeutic Area 17.3.2. AAV Based Gene Therapy Market: Distribution by Type of Therapy 17.3.3. AAV Based Gene Therapy Market: Distribution by Type of Gene Delivery Method Used 17.3.4. AAV Based Gene Therapy Market: Distribution by Therapeutic Area 17.3.5. AAV Based Gene Therapy Market: Distribution by Route of Administration 17.3.6. AAV Based Gene Therapy Market: Distribution by Therapeutic Area 17.3.7. AAV Based Gene Therapy Market: Distribution by Geography 17.3.8. AAV Based Gene Therapy Market, 2022-2035: Individual Product Sales Forecast 17.3.8.1. LUXTURNA 17.3.8.1.1. Target Patient Population 17.3.8.1.2. Sales Forecast 17.3.8.1.3. Net Present Value 17.3.8.1.4. Value Creation Analysis 17.3.8.2. ZOLGENSMA 17.3.8.2.1. Target Patient Population 17.3.8.2.2. Sales Forecast 17.3.8.2.3. Net Present Value 17.3.8.2.4. Value Creation Analysis 17.3.8.3. HEMGENIX 17.3.8.3.1. Target Patient Population 17.3.8.3.2. Sales Forecast 17.3.8.3.3. Net Present Value 17.3.8.3.4. Value Creation Analysis 17.3.8.4. ROCTAVIAN 17.3.8.4.1. Target Patient Population 17.3.8.4.2. Sales Forecast 17.3.8.4.3. Net Present Value 17.3.8.4.4. Value Creation Analysis 17.3.8.5. UPSTAZA 17.3.8.5.1. Target Patient Population 17.3.8.5.2. Sales Forecast 17.3.8.5.3. Net Present Value 17.3.8.5.4. Value Creation Analysis 17.3.8.6. ELEVIDYS 17.3.8.6.1. Target Patient Population 17.3.8.6.2. Sales Forecast 17.3.8.6.3. Net Present Value 17.3.8.6.4. Value Creation Analysis 17.3.8.7. AGTC 501 17.3.8.7.1. Target Patient Population 17.3.8.7.2. Sales Forecast 17.3.8.7.3. Net Present Value 17.3.8.7.4. Value Creation Analysis 17.3.8.8. LYS-SAF302 17.3.8.8.1. Target Patient Population 17.3.8.8.2. Sales Forecast 17.3.8.8.3. Net Present Value 17.3.8.8.4. Value Creation Analysis 17.3.8.9. NFS-01 17.3.8.9.1. Target Patient Population 17.3.8.9.2. Sales Forecast 17.3.8.9.3. Net Present Value 17.3.8.9.4. Value Creation Analysis 17.3.8.10. OAV-101 17.3.8.10.1. Target Patient Population 17.3.8.10.2. Sales Forecast 17.3.8.10.3. Net Present Value 17.3.8.10.4. Value Creation Analysis 17.3.8.11. PF-06838435 17.3.8.11.1. Target Patient Population 17.3.8.11.2. Sales Forecast 17.3.8.11.3. Net Present Value 17.3.8.11.4. Value Creation Analysis 17.3.8.12. PF-06939926 17.3.8.12.1. Target Patient Population 17.3.8.12.2. Sales Forecast 17.3.8.12.3. Net Present Value 17.3.8.12.4. Value Creation Analysis 17.3.8.13. PF-07055480 17.3.8.13.1. Target Patient Population 17.3.8.13.2. Sales Forecast 17.3.8.13.3. Net Present Value 17.3.8.13.4. Value Creation Analysis 17.3.8.14. RGX-314 17.3.8.14.1. Target Patient Population 17.3.8.14.2. Sales Forecast 17.3.8.14.3. Net Present Value 17.3.8.14.4. Value Creation Analysis 17.3.8.15. SPK-8011 17.3.8.15.1. Target Patient Population 17.3.8.15.2. Sales Forecast 17.3.8.15.3. Net Present Value 17.3.8.15.4. Value Creation Analysis 17.4. AAV Manufacturing Market, 2022-2035 17.4.1. AAV Manufacturing Market, 2022-2035: Distribution by Phase of Development 17.4.2. AAV Manufacturing Market, 2022-2035: Distribution by Therapeutic Area 17.4.3. AAV Manufacturing Market, 2022-2035: Distribution by Application Area 17.4.4. AAV Manufacturing Market, 2022-2035: Distribution by Key Geographical Regions 17.5 Current and Future Market Opportunity for Commercial Products 17.5.1. AAV Manufacturing Market for Commercial Products, 2022-2035: Distribution by Therapeutic Area 17.5.2. AAV Manufacturing Market for Commercial Products, 2022-2035: Distribution by Application Area 17.5.3. AAV Manufacturing Market for Commercial Products, 2022-2035: Distribution by Key Geographical Regions 17.6. Current and Future Market Opportunity for Clinical Candidates 17.6.1. AAV Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Phase of Development 17.6.2. AAV Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Application Area 17.6.3. AAV Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Key Geographical Regions 17.7. Current and Future Market Opportunity for Preclinical Candidates 17.7.1. AAV Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Type of Animal Model Used 17.7.2. AAV Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Application Area 17.7.3. AAV Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Key Geographical Regions 18. CONCLUDING REMARKS 19. EXECUTIVE INSIGHTS 19.1. Chapter Overview 19.2. Boston College 19.2.1. Organization Snapshot 19.2.2. Interview Transcript: Abhishek Chatterjee, Professor 19.3. University of Florida 19.3.1. Organization Snapshot 19.3.2. Interview Transcript: Arun Srivastava, Professor of Genetics and Chief of Division of Cellular & Molecular Therapy 19.4. Andelyn Biosciences 19.4.1. Organization Snapshot 19.4.2. Interview Transcript: Adam Lauber, Chief Financial Officer 20. APPENDIX 1: TABULATED DATA 21. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
SummaryThe global AAV vector market is expected to reach USD 1.9 billion in 2022 and is anticipated to grow at a CAGR of 14% during the forecast period 2022-2035 Table of Contents1. PREFACE1.1. Scope of the Report 1.2. Market Segmentation 1.3. Research Methodology 1.4. Key Questions Answered 1.5. Chapter Outlines 2. EXECUTIVE SUMMARY 3. INTRODUCTION 3.1. Chapter Overview 3.2. Viral and Non-Viral Vectors 3.2.1. Viral Vectors 3.2.1.1. Adenovirus Vectors 3.2.1.2. Adeno-Associated Viral Vectors 3.2.1.3. Lentivirus Vectors 3.2.1.4. Retrovirus Vectors 3.2.1.5. Other Viral Vectors 3.2.1.5.1. Alphavirus 3.2.1.5.2. Foamy Virus 3.2.1.5.3. Simian Virus 3.2.1.5.4. Vaccinia Virus 3.2.1.5.5. Chimeric Viral Vectors 3.2.1.5.6. Herpes Simplex Virus 3.2.1.5.7. Sendai Virus 3.2.2. Non-Viral Vectors 3.2.2.1. Plasmid DNA 3.2.2.2. Oligonucleotides 3.2.2.3. Liposomes, Lipoplexes, and Polyplexes 3.2.2.4. Other Non-Viral Vectors 3.3. Adeno-Associated Viral Vectors 3.3.1. Structure and Design 3.3.2. Adeno-Associated Viral Vector Life Cycle 3.3.3. Applications of Adeno-Associated Viral Vectors 3.3.3.1. Gene Therapy 3.3.3.2. Vaccination 3.3.4. Advantages of Adeno-Associated Viral Vectors 3.3.5. Challenges Related to Adeno-Associated Viral Vectors 3.4. Concluding Remarks 4. AAV BASED GENE THERAPY: MARKET LANDSCAPE 4.1. Chapter Overview 4.2. AAV Based Gene Therapy: Overall Market Landscape 4.2.1. Analysis by Phase of Development 4.2.2. Analysis by Therapeutic Area(s) 4.2.3. Analysis by Type of Gene / Molecule Targeted 4.2.4. Analysis by Type of Therapy 4.2.5. Analysis by Type of Gene Delivery Method Used 4.2.6. Analysis by Route of Administration 4.3. AAV Based Gene Therapy Candidates: Special Designations 4.3.1. Analysis by Special Designation(s) Awarded 4.4. AAV Based Gene Therapy: List of Developers 4.4.1. Analysis by Year of Establishment 4.4.2. Analysis by Company Size 4.4.3. Analysis by Location of Headquarters 4.4.4. AAV Based Gene Therapy: Leading Developers 5. AAV MANUFACTURING COMPANIES: MARKET LANDSCAPE 5.1. Chapter Overview 5.2. AAV Manufacturing Companies: Overall Market Landscape 5.2.1. Analysis by Year of Establishment 5.2.2. Analysis by Company Size 5.2.3. Analysis by Location of Headquarters 5.2.4. Analysis by Type of Product(s) Manufactured 5.2.5. Analysis by Location of Vector Manufacturing Facilities 5.2.6. Analysis by Type of Manufacturer and Company Size 5.2.7. Analysis by Scale of Operation 5.2.8. Analysis by Location of Headquarters and Scale of Operation 5.2.9. Analysis by Application Area(s) 6. AAV TECHNOLOGY: MARKET LANDSCAPE 6.1. Chapter Overview 6.2. AAV Technology 6.2.1. Analysis by Type of Technology 6.2.2. Analysis by Scale of Operation 6.2.3. Analysis by Application Area(s) 6.2.4. Analysis by Therapeutic Area 6.3. AAV Technology: List of Developers 6.3.1. Analysis by Year of Establishment 6.3.2. Analysis by Company Size 6.3.3. Analysis by Location of Headquarters 6.3.4. Analysis by Company Size and Location of Headquarters 6.3.5. AAV Technology: Leading Developers 7. DRUG PROFILES 7.1. Chapter Overview 7.2. Marketed AAV Based Gene Therapies 7.2.1. LUXTURNA® (Spark Therapeutics) 7.2.1.1. Company Overview 7.2.1.2. Development Timeline 7.2.1.3. Mechanism of Action 7.2.1.4. Target Indication(s) 7.2.1.5. Current Status of Development 7.2.1.6. Manufacturing, Dosage and Sales 7.2.2. ZOLGENSMA® (Novartis) 7.2.2.1. Company Overview 7.2.2.2. Development Timeline 7.2.2.3. Mechanism of Action 7.2.2.4. Target Indication(s) 7.2.2.5. Current Status of Development 7.2.2.6. Manufacturing, Dosage and Sales 7.3.1. HEMGENIX® (CSL Behring / UniQure) 7.3.1.1. Company Overview 7.3.1.2. Development Timeline 7.3.1.3. Mechanism of Action 7.3.1.4. Target Indication(s) 7.3.1.5. Current Status of Development 7.3.1.6. Manufacturing, Dosage and Sales 7.4.1. ROCTAVIAN® (BioMarin Pharmaceuticals) 7.4.1.1. Company Overview 7.4.1.2. Development Timeline 7.4.1.3. Mechanism of Action 7.4.1.4. Target Indication(s) 7.4.1.5. Current Status of Development 7.4.1.6. Manufacturing, Dosage and Sales 7.5.1. ELEVIDYS® (Sarepta Therapeutics) 7.5.1.1. Company Overview 7.5.1.2. Development Timeline 7.5.1.3. Mechanism of Action 7.5.1.4. Target Indication(s) 7.5.1.5. Current Status of Development 7.5.1.6. Manufacturing, Dosage and Sales 7.6.1. UPSTAZA® (PTC Therapeutics) 7.6.1.1. Company Overview 7.6.1.2. Development Timeline 7.6.1.3. Mechanism of Action 7.6.1.4. Target Indication(s) 7.6.1.5. Current Status of Development 7.6.1.6. Manufacturing, Dosage and Sales 7.6. Late Stage (Phase II/III and Above) Gene Therapies 7.6.1. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.2. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.3. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.4. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.5. OAV-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.6. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.7. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.8. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 7.6.9. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results 8. COMPANY PROFILES 8.1. Chapter Overview 8.2. Abeona Therapeutics 8.2.1. Company Overview 8.2.2. Financial Information 8.2.3. AAV Manufacturing Related Capabilities 8.2.4. Recent Developments and Future Outlook 8.3 Aldevron (Acquired by Danaher) 8.3.1. Company Overview 8.3.2. Financial Information 8.3.3. AAV Manufacturing Related Capabilities 8.3.4. Recent Developments and Future Outlook 8.4 Oxford BioMedica 8.4.1. Company Overview 8.4.2. Financial Information 8.4.3. AAV Manufacturing Related Capabilities 8.4.4. Recent Developments and Future Outlook 8.5 Sanofi (CEPiA, Sanofi Pasteur, Genzyme) 8.5.1. Company Overview 8.5.2. Financial Information 8.5.3. AAV Manufacturing Related Capabilities 8.5.4. Recent Developments and Future Outlook 8.6. WuXi AppTec 8.6.1. Company Overview 8.6.2. Financial Information 8.6.3. AAV Manufacturing Related Capabilities 8.6.4. Recent Developments and Future Outlook 8.7. YPOSKESI 8.7.1. Company Overview 8.7.2. AAV Manufacturing Related Capabilities 8.7.3. Recent Developments and Future Outlook 9. COMPANY COMPETITIVENESS ANALYSIS 9.1. Chapter Overview 9.2. Methodology and Key Parameters 9.3. AAV Manufacturing Companies: In-house Manufacturers 9.3.1. Players based in North America 9.3.2. Players based in Europe 9.4. AAV Manufacturing Companies: Contract Manufacturing Organizations 9.4.1. Players based in North America 9.4.2. Players based in Europe 9.4.3. Players based in Asia-Pacific 9.5. AAV Manufacturing Companies: Both In-House and Contract Manufacturing Organizations 9.5.1. Players based in North America 9.5.2. Players based in Europe 9.5.3. Players based in Asia-Pacific 10. TECHNOLOGY COMPETITIVENESS ANALYSIS 10.1. Chapter Overview 10.2. Methodology and Key Parameters 10.3. AAV Technology Platforms 10.3.1. AAV Technology Platforms Developed by Companies based in North America 10.3.2. AAV Technology Platforms Developed by Companies based in Europe and Asia-Pacific 11. CLINICAL TRIAL ANALYSIS 11.1. Chapter Overview 11.2. Scope and Methodology 11.3. AAV Based Gene Therapy: Clinical Trial Analysis 11.3.1. Analysis by Trial Registration Year 11.3.2. Analysis by Trial Registration Year and Enrolled Patient Population 11.3.3. Analysis by Trial Phase and Enrolled Patient Population 11.3.4. Analysis by Trial Status 11.3.5. Analysis by Study Design 11.3.6. Analysis by Therapeutic Area 11.3.7. Analysis by Target Disease Indication 11.3.8. Analysis by Type of Sponsor / Collaborator 11.3.9. Leading Industry Players: Analysis by Number of Registered Trials 11.3.10. Regional Analysis 11.3.10.1. Analysis by Number of Registered Trials 11.3.10.2. Analysis by Enrolled Patient Population 12. PARTNERSHIPS AND COLLABORATIONS 12.1. Chapter Overview 12.2. Partnership Models 12.3. AAV Based Gene Therapy: List of Partnerships and Collaborations 12.3.1. Analysis by Year of Partnership 12.3.2. Analysis by Type of Partnership 12.3.3. Analysis by Year of Partnership and Type of Partner 12.3.4. Analysis by Type of Partnership and Type of Partner 12.3.5. Analysis by Therapeutic Area 12.3.6. Most Active Players: Analysis by Number of Partnerships 12.3.7. Regional Analysis 12.3.7.1. Intercontinental and Intracontinental Agreements 12.4. AAV Manufacturing Companies: List of Partnerships and Collaborations 12.4.1. Analysis by Year of Partnership 12.4.2. Analysis by Type of Partnership 12.4.3. Analysis by Year of Partnership and Type of Partner 12.4.4. Analysis by Type of Partnership and Type of Partner 12.4.5. Analysis by Therapeutic Area 12.4.6. Most Active Players: Analysis by Number of Partnerships 12.4.7. Regional Analysis 12.4.7.1. Intercontinental and Intracontinental Agreements 13. STRATEGIC PARTNER ANALYSIS 13.1. Chapter Overview 13.2. AAV Manufacturing Companies: Potential Strategic Partners 13.2.1. Methodology and Key Parameters 13.2.2. AAV Manufacturing Companies: Strategic Partner Analysis 13.2.2.1. Most Likely Partners 13.2.2.2. Likely Partners 13.2.2.3. Less Likely Partners 13.2.2.4. Least Likely Partners 14. PATENT ANALYSIS 14.1. Chapter Overview 14.2. Scope and Methodology 14.3. Adeno-Associated Viral Vectors: Patent Analysis 14.3.1. Analysis by Publication Year 14.3.2. Analysis by Annual Patent Application(s) 14.3.3. Analysis by Annual Granted Patents 14.3.4. Analysis by Geography 14.3.5. Analysis by CPC Symbols 14.3.6. Analysis by Type of Applicant 14.3.7. Leading Industry Players: Analysis by Number of Patents 14.4. Adeno Associated Viral Vector Based Market: Patent Benchmarking 14.4.1. Analysis by Patent Characteristics 14.5. Adeno-Associated Viral Vectors: Patent Valuation 14.6. Leading Patents: Analysis by Number of Citations 15. START-UP HEALTH INDEXING 15.1. Chapter Overview 15.2. Start-ups Developing Adeno-Associated Viral Vectors 15.2.1. Analysis by Location of Headquarters 15.3. Benchmarking of Start-ups 15.3.1. Analysis by Pipeline Strength 15.3.2. Analysis by Pipeline Maturity 15.3.3. Analysis by Indication Diversity 15.3.4. Analysis by Number of Patents 15.3.5. Analysis by Partnership Activity 15.3.6. Start-ups Health Indexing: Roots Analysis Perspective 16. OUTSOURCING: GO / NO-GO FRAMEWORK 16.1. Chapter Overview 16.2. Outsourcing: Go / No-Go Framework 16.3. AAV Based Gene Therapy Developers Outsourcing: Go / No-Go Framework 16.3.1. Key Parameters and Assumptions 16.3.2. Methodology 16.3.3. Results and Interpretations 16.3.3.1. Small Companies 16.3.3.2. Mid-sized Companies 16.3.3.3. Large Companies 17. MARKET SIZING AND OPPORTUNITY ANALYSIS 17.1. Chapter Overview 17.2. Methodology and Key Assumptions 17.3. AAV Based Gene Therapy Market, 2022-2035 17.3.1. AAV Based Gene Therapy Market: Distribution by Therapeutic Area 17.3.2. AAV Based Gene Therapy Market: Distribution by Type of Therapy 17.3.3. AAV Based Gene Therapy Market: Distribution by Type of Gene Delivery Method Used 17.3.4. AAV Based Gene Therapy Market: Distribution by Therapeutic Area 17.3.5. AAV Based Gene Therapy Market: Distribution by Route of Administration 17.3.6. AAV Based Gene Therapy Market: Distribution by Therapeutic Area 17.3.7. AAV Based Gene Therapy Market: Distribution by Geography 17.3.8. AAV Based Gene Therapy Market, 2022-2035: Individual Product Sales Forecast 17.3.8.1. LUXTURNA 17.3.8.1.1. Target Patient Population 17.3.8.1.2. Sales Forecast 17.3.8.1.3. Net Present Value 17.3.8.1.4. Value Creation Analysis 17.3.8.2. ZOLGENSMA 17.3.8.2.1. Target Patient Population 17.3.8.2.2. Sales Forecast 17.3.8.2.3. Net Present Value 17.3.8.2.4. Value Creation Analysis 17.3.8.3. HEMGENIX 17.3.8.3.1. Target Patient Population 17.3.8.3.2. Sales Forecast 17.3.8.3.3. Net Present Value 17.3.8.3.4. Value Creation Analysis 17.3.8.4. ROCTAVIAN 17.3.8.4.1. Target Patient Population 17.3.8.4.2. Sales Forecast 17.3.8.4.3. Net Present Value 17.3.8.4.4. Value Creation Analysis 17.3.8.5. UPSTAZA 17.3.8.5.1. Target Patient Population 17.3.8.5.2. Sales Forecast 17.3.8.5.3. Net Present Value 17.3.8.5.4. Value Creation Analysis 17.3.8.6. ELEVIDYS 17.3.8.6.1. Target Patient Population 17.3.8.6.2. Sales Forecast 17.3.8.6.3. Net Present Value 17.3.8.6.4. Value Creation Analysis 17.3.8.7. AGTC 501 17.3.8.7.1. Target Patient Population 17.3.8.7.2. Sales Forecast 17.3.8.7.3. Net Present Value 17.3.8.7.4. Value Creation Analysis 17.3.8.8. LYS-SAF302 17.3.8.8.1. Target Patient Population 17.3.8.8.2. Sales Forecast 17.3.8.8.3. Net Present Value 17.3.8.8.4. Value Creation Analysis 17.3.8.9. NFS-01 17.3.8.9.1. Target Patient Population 17.3.8.9.2. Sales Forecast 17.3.8.9.3. Net Present Value 17.3.8.9.4. Value Creation Analysis 17.3.8.10. OAV-101 17.3.8.10.1. Target Patient Population 17.3.8.10.2. Sales Forecast 17.3.8.10.3. Net Present Value 17.3.8.10.4. Value Creation Analysis 17.3.8.11. PF-06838435 17.3.8.11.1. Target Patient Population 17.3.8.11.2. Sales Forecast 17.3.8.11.3. Net Present Value 17.3.8.11.4. Value Creation Analysis 17.3.8.12. PF-06939926 17.3.8.12.1. Target Patient Population 17.3.8.12.2. Sales Forecast 17.3.8.12.3. Net Present Value 17.3.8.12.4. Value Creation Analysis 17.3.8.13. PF-07055480 17.3.8.13.1. Target Patient Population 17.3.8.13.2. Sales Forecast 17.3.8.13.3. Net Present Value 17.3.8.13.4. Value Creation Analysis 17.3.8.14. RGX-314 17.3.8.14.1. Target Patient Population 17.3.8.14.2. Sales Forecast 17.3.8.14.3. Net Present Value 17.3.8.14.4. Value Creation Analysis 17.3.8.15. SPK-8011 17.3.8.15.1. Target Patient Population 17.3.8.15.2. Sales Forecast 17.3.8.15.3. Net Present Value 17.3.8.15.4. Value Creation Analysis 17.4. AAV Manufacturing Market, 2022-2035 17.4.1. AAV Manufacturing Market, 2022-2035: Distribution by Phase of Development 17.4.2. AAV Manufacturing Market, 2022-2035: Distribution by Therapeutic Area 17.4.3. AAV Manufacturing Market, 2022-2035: Distribution by Application Area 17.4.4. AAV Manufacturing Market, 2022-2035: Distribution by Key Geographical Regions 17.5 Current and Future Market Opportunity for Commercial Products 17.5.1. AAV Manufacturing Market for Commercial Products, 2022-2035: Distribution by Therapeutic Area 17.5.2. AAV Manufacturing Market for Commercial Products, 2022-2035: Distribution by Application Area 17.5.3. AAV Manufacturing Market for Commercial Products, 2022-2035: Distribution by Key Geographical Regions 17.6. Current and Future Market Opportunity for Clinical Candidates 17.6.1. AAV Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Phase of Development 17.6.2. AAV Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Application Area 17.6.3. AAV Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Key Geographical Regions 17.7. Current and Future Market Opportunity for Preclinical Candidates 17.7.1. AAV Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Type of Animal Model Used 17.7.2. AAV Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Application Area 17.7.3. AAV Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Key Geographical Regions 18. CONCLUDING REMARKS 19. EXECUTIVE INSIGHTS 19.1. Chapter Overview 19.2. Boston College 19.2.1. Organization Snapshot 19.2.2. Interview Transcript: Abhishek Chatterjee, Professor 19.3. University of Florida 19.3.1. Organization Snapshot 19.3.2. Interview Transcript: Arun Srivastava, Professor of Genetics and Chief of Division of Cellular & Molecular Therapy 19.4. Andelyn Biosciences 19.4.1. Organization Snapshot 19.4.2. Interview Transcript: Adam Lauber, Chief Financial Officer 20. APPENDIX 1: TABULATED DATA 21. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
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