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市場調查報告書

基因治療藥趨勢

Trends in Gene Therapy

出版商 Datamonitor Healthcare 商品編碼 365161
出版日期 內容資訊 英文 170 Pages
商品交期: 最快1-2個工作天內
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基因治療藥趨勢 Trends in Gene Therapy
出版日期: 2016年07月22日 內容資訊: 英文 170 Pages
簡介

本報告提供基因治療藥的開發趨勢調查,各對象疾病的藥物開發趨勢,開發計劃,各種遞送方法,美國及歐洲的法律規章上的必要條件,同2個地區的核準的差異,償付相關趨勢,企業間的各種聯盟、收購契約等趨勢彙整。

摘要整理

基因治療策略

  • 簡介:基因治療
  • 參考文獻

基因治療產品的商業性開發趨勢

  • 癌症是最常見的標的、其次是單一遺傳基因疾病

基因轉移技術的革新

  • 病毒:是有效的基因轉移媒介但也有幾個課題
  • 病毒載體:能刺激本體的免疫系但也有不利影響
  • 遺傳基因媒介的質體
  • 遺傳基因媒介的細菌
  • 遺傳基因媒介的細胞
  • 商業性開發中的體內治療藥所使用的媒介
  • 參考文獻

癌症的遺傳基因療法

  • 傳統的癌症治療:特定的長期成功
  • 總合201種致癌基因治療藥現在進行商業性開發中
  • 免疫療法:一般的抗癌策略
  • 癌症的基因治療的其他方法
  • 腫瘤溶解性病毒療法:向無法手術的癌症患者展現希望
  • 血管新生抑制遺傳基因療法:出示替代性方法
  • 參考文獻等

單一遺傳基因疾病的遺傳基因療法

  • 102種基因治療藥現在進行商業性開發中
  • 脂蛋白解脂酵素缺損症
  • 腺核苷去胺酶缺損症
  • 遺傳性視網膜失養症
  • X連鎖性兒童腎上腺腦白質營養不良
  • 血友病
  • 肌肉萎縮症
  • 參考文獻

對癌症以外的後天性疾病的遺傳基因療法

  • 感染疾病
  • 心血管疾病
  • 感覺性疾病
  • 神經疾病
  • 其他
  • 參考文獻

法律規章上的課題

  • 簡介
  • EU
  • 美國
  • 參考文獻

償付上的課題

各種交易、收購

  • 5年契約動向

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目錄
Product Code: DMKC0162772

The number of gene therapy products in preclinical to Phase III and beyond stages of development doubled between 2012 and

2015. Additionally, three gene therapy products have received regulatory approval in Europe. While these approvals give some validation to gene therapy as a therapeutic strategy, doubts remain around their return on investment.

This report addresses the following questions:

  • Which therapy areas are gene therapies targeting and which programs are most advanced?
  • Which delivery systems are used the most and how do the approaches differ by disease?
  • What are the key features of the regulatory requirements for gene therapy products in the US and Europe?
  • What differences have been observed in the approvals for gene therapy products between the US and Europe?
  • What patient access obstacles have approved gene therapy products faced so far and how will current funding models have to change to facilitate access to gene therapies in the future?
  • How are acquisitions and partnerships changing the gene therapy landscape?

TABLE OF CONTENTS

EXECUTIVE SUMMARY

  • 1. The number of gene therapy products in development has doubled since 2012
  • 2. Most products in development are in vivo therapies, except in oncology
  • 3. The adeno-associated virus is the most popular viral vector
  • 4. Cancer is the most common target for gene therapies in development, followed by monogenic diseases
  • 5. Most products in advanced clinical development target cancer indications
  • 6. Immunotherapy and oncolytic virotherapy are promising approaches in cancer
  • 7. Regulatory and reimbursement strategies will be key to the success of new therapies
  • 8. Gene therapy of cancer is the most active area of commercial deal-making

GENE THERAPY STRATEGIES

  • 9. Introduction to gene therapy
  • 10. Bibliography

GENE THERAPY PRODUCTS IN COMMERCIAL DEVELOPMENT IN 2015

  • 11. Cancer is the most common target for products, followed by monogenic diseases

INNOVATIONS IN GENE DELIVERY TECHNOLOGIES

  • 12. Viruses are efficient gene delivery vectors, but pose several challenges
  • 13. Viral vectors can stimulate the host's immune system with undesirable effects
  • 14. Plasmids as gene vectors
  • 15. Bacteria as gene vectors
  • 16. Cells as gene vectors
  • 17. Vectors used in in vivo therapies in commercial development in 2015
  • 18. Bibliography

GENE THERAPIES FOR CANCER

  • 19. Conventional cancer treatment has limited long-term success
  • 20. A total of 201 cancer gene therapy products are in commercial development
  • 21. Immunotherapy is a popular broad anticancer strategy
  • 22. Other approaches to cancer gene therapy
  • 23. Targeted destruction of tumors encompasses a variety of approaches
  • 24. Oncolytic virotherapy offers hope to patients with inoperable tumors
  • 25. Anti-angiogenic gene therapies offer an alternative approach
  • 26. Bibliography

GENE THERAPIES FOR MONOGENIC DISEASES

  • 27. There are 102 gene therapy products in commercial development
  • 28. Lipoprotein lipase deficiency
  • 29. Adenosine deaminase deficiency
  • 30. Inherited retinal dystrophies
  • 31. X-linked childhood cerebral adrenoleukodystrophy
  • 32. Hemophilia
  • 33. Muscular dystrophies
  • 34. Bibliography

GENE THERAPIES FOR ACQUIRED DISEASES OTHER THAN CANCER

  • 35. Infectious diseases
  • 36. Cardiovascular disease
  • 37. Sensory diseases
  • 38. Neurological disease
  • 39. Other diseases
  • 40. Bibliography

REGULATORY ISSUES

  • 41. Introduction
  • 42. Regulatory framework in the EU
  • 43. Regulatory framework in the US
  • 44. Bibliography

REIMBURSEMENT ISSUES

  • 45. In rare diseases, return on investment is typically realized through repeated drug administration
  • 46. An alternative to a high single payment may be annuity payments for effective treatment
  • 47. Pay for-performance models may be suitable for gene therapy reimbursement
  • 48. Payers are not ready, but gene therapies may drive rethinking of drug pricing in general
  • 49. Glybera's reimbursement struggles reveal uncertainties around long-term effects to be a key concern for payers
  • 50. Imlygic struggles to gain reimbursement amid increased competition within melanoma
  • 51. GlaxoSmithKline to use Strimvelis to test alternative funding mechanisms
  • 52. Bibliography

DEALS AND ACQUISITIONS

  • 53. Five years of deal-making in the gene therapy area

APPENDIX

  • 54. About the authors
  • 55. Scope
  • 56. Methodology
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