Gene Therapies: A Growing Pipeline and Approval of CART Cell Therapies Reflect Increasing Commercial Appeal, Despite Difficulties Faced by products such as Glybera and Strimvelis discusses all gene therapies, including not only those which involve genetic modification, but also RNAi, CART cell and aptamerbased therapies.
In 2017 alone, the marketing approvals of Kymriah, Yescarta and Luxturna added to this growing class of products. However, due to various challenges, progress in developing this technology and achieving commercial uptake over the past few decades has been slow.
In the short term, there is a shortfall in global gene and cell therapy manufacturing capacity, and in the longterm the personalized nature of many gene and cell therapies represents a further challenge that will not be met by the currently used, centralized biopharmaceutical manufacturing model.
Additionally, the very small number of patients in orphan genetic diseases that these therapies are especially well suited for has caused commercial difficulties for drugs such as Glybera and Strimvelis, and the high pricing of these therapies in response to the low patient number presented strong reimbursement difficulties.
However, the pipeline for gene therapies is robust; there are 985 in vivo gene therapies, and a further 354 CART cell therapies currently in pipeline development. Most are at an early stage of development, with 76% at the Discovery or Preclinical stage.
There are also currently 23 gene therapy programs in Phase III development. This report provides a comprehensive view of the clinical, R&D, commercial and competitive landscape of Gene Therapy, and assesses key developments in delivery vector technology, and challenges and advances associated with the production of such vectors.
Why do gene therapies still occupy only a minimal market share in their respective indications?
What can be learned from the gene therapies that have already reached the market, especially from products that have faced commercial difficulties, such as Glybera and Strimvelis?
What are the prospects of recent approvals such as Luxturna, Kymriah and Strimvelis?
What are the relative advantages and disadvantages of each vector type and which hold the most promise?
What proportion of the overall gene therapy R&D pipeline is occupied by each vector type?
How will the entry of gene therapies into the market change the global manufacturing landscape?
Reasons to buy
Understand the current status of the field of therapeutic gene therapies, and the relative clinical and commercial success of currently marketed products.
Assess the pipeline for gene therapies split by therapy area, vector type and intervention type, and stage of development. Additionally, a granular assessment of the pipeline is provided across the four major therapy areas for gene therapy: oncology, central nervous system disorders, ophthalmology, and genetic disorders.
Gain a picture of the current competitive landscape, with a detailed breakdown of companies actively involved in the gene therapy pipeline.
Understand the level of involvement in the landscape on the part of big pharma companies, and the extent to how gene therapies fit into the overall portfolios of companies in this field.
Understand the strategic consolidations landscape in gene therapies across the past decade.
Key Topics Covered:
1. Table of Contents 1.1 List of Tables 1.2 List of Figures
2. Gene Therapy Overview 2.1 Types of Gene Therapy 2.1.1 Types of Intervention 2.1.2 Types of Vector
4. Gene Therapy Production Strategies 4.1 Production of Viral Vectors 4.1.1 Case Study: Challenges in the Manufacture of AAV Vectors 4.2 Production of Cell-based Gene Therapies
5. Challenges to Gene Therapy Development
6. Gene Therapy Pipeline and Emerging Technologies 6.1 Gene Therapy Pipeline by Therapy Area and Stage of Development 6.2 Gene Therapy Pipeline by Intervention and Vector Type 6.3 Pipeline for CAR-T Cell Therapies 6.4 Company Positioning 6.4.1 Companies by Therapy Area 6.4.2 Companies by Stage of Development 6.4.3 Companies by Intervention Type 6.4.4 Companies by Vector Type 6.4.5 Companies Developing CAR-T Cell Therapies by Stage 6.5 Early Genome Editing Technologies 6.5.1 Zinc Finger Nucleases 6.5.2 TALEN 6.5.3 CRISPR-Cas9
7. Strategic Consolidations 7.1 Licensing Deals 7.1.1 Licensing Deals by Region Value and Year 7.1.2 Licensing Deals by Stage of Development and Value 7.1.3 Licensing Deals by Intervention and Vector Type 7.2 Co-development deals 7.2.1 Co-development Deals by Region, Year and Value 7.2.2 Co-development Deals by Stage of Development and Value 7.2.3 Co-development Deals by Intervention and Vector Type