DUBLIN--(BUSINESS WIRE)--The "Gene Therapies: A Growing Pipeline and Approval of CAR-T Cell Therapies Reflect Increasing Commercial Appeal, Despite Difficulties Faced by products such as Glybera and Strimvelis" report has been added to ResearchAndMarkets.com's offering.
This gene therapies report 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?
Key Topics Covered:
1. Table of Contents
2. Gene Therapy Overview
3. Currently Approved Gene Therapies
4. Gene Therapy Production Strategies
5. Challenges to Gene Therapy Development
6. Gene Therapy Pipeline and Emerging Technologies
7. Strategic Consolidations
- Abeona Therapeutics Inc
- Advantagene Inc
- Applied Genetic Technologies Corp
- Arrowhead Pharmaceuticals Inc
- Benitec Biopharma Ltd
- Biomics Biotechnologies Co Ltd
- Bioneer Corp Casebia Therapeutics Excision BioTherapeutics Inc
- BV Dimension Therapeutics Inc
- enGene Inc
- Exi cure Inc
- Genethon SA Spark Therapeutics Inc
- GlaxoSmithKline Plc
- Inovio Pharmaceuticals Inc
- InteRNA Technologies BV
- International Inc
- Ionis Pharmaceuticals Inc
- Myonexus Therapeutics Inc
- NV Quark Pharmaceuticals Inc
- OliPass Corp
- OliX Pharmaceuticals Inc
- Pfizer Inc
- RegenxBio Inc
- Regulus Therapeutics Inc
- Renova Therapeutics Inc
- RXi Pharmaceuticals Corp
- Sangamo Therapeutics Inc
- Sarepta Therapeutics Inc
- Shire Plc
- Silence Therapeutics Plc
- Sirnaomics Inc
- Sylentis SAU
- Voyager Therapeutics Inc
- WAVE Life Sciences Ltd
For more information about this report visit https://www.researchandmarkets.com/research/f2w697/gene_therapy?w=4