Market Research Reports, Inc. has announced the addition of “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” research report to their offering. See more at - http://mrr.cm/UmH

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Category : Pharmacy
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Introduction to Report
 Launch Date: January 1, 2018
 Number of Pages: 79
 Geography Coverage: Global
 Available Format: PDF
Price For Single User License: USD 2,995
Price For Site User License: USD 5,990
Price For Global User License: USD 8,985
Delivery Time: Within 24 Hours (During Working
Days)

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About the Report
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
discusses all gene therapies, including not only
those which involve genetic modification, but also
RNAi, CAR-T cell and aptamer-based therapies.

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Key Highlights:
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 long-term 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.

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Key Highlights: Continues…
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 CAR-T 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.

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Scope:
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?

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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.

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Report Coverage:
1. Gene Therapy Overview
2. Currently Approved Gene Therapies
3. Gene Therapy Production Strategies
4. Challenges to Gene Therapy Development
5. Gene Therapy Pipeline and Emerging
Technologies
6. Strategic Consolidations
7. Conclusion
8. Appendix
For more details regarding Report coverage see the last slide
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