Global Trends in Gene Therapy 2016 - Research and Markets

DUBLIN--()--Research and Markets has announced the addition of the "Trends in Gene Therapy" report to their offering.

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?

Key Topics Covered:

EXECUTIVE SUMMARY

- The number of gene therapy products in development has doubled since 2012

- Most products in development are in vivo therapies, except in oncology

- The adeno-associated virus is the most popular viral vector

- Cancer is the most common target for gene therapies in development, followed by monogenic diseases

- Most products in advanced clinical development target cancer indications

- Immunotherapy and oncolytic virotherapy are promising approaches in cancer

- Regulatory and reimbursement strategies will be key to the success of new therapies

- Gene therapy of cancer is the most active area of commercial deal-making

GENE THERAPY STRATEGIES

- Introduction to gene therapy

- Bibliography

GENE THERAPY PRODUCTS IN COMMERCIAL DEVELOPMENT IN 2015

- Cancer is the most common target for products, followed by monogenic diseases

INNOVATIONS IN GENE DELIVERY TECHNOLOGIES

- Viruses are efficient gene delivery vectors, but pose several challenges

- Viral vectors can stimulate the host's immune system with undesirable effects

- Plasmids as gene vectors

- Bacteria as gene vectors

- Cells as gene vectors

- Vectors used in in vivo therapies in commercial development in 2015

- Bibliography

GENE THERAPIES FOR CANCER

- Conventional cancer treatment has limited long-term success

- A total of 201 cancer gene therapy products are in commercial development

- Immunotherapy is a popular broad anticancer strategy

- Other approaches to cancer gene therapy

- Targeted destruction of tumors encompasses a variety of approaches

- Oncolytic virotherapy offers hope to patients with inoperable tumors

- Anti-angiogenic gene therapies offer an alternative approach

- Bibliography

GENE THERAPIES FOR MONOGENIC DISEASES

- There are 102 gene therapy products in commercial development

- Lipoprotein lipase deficiency

- Adenosine deaminase deficiency

- Inherited retinal dystrophies

- X-linked childhood cerebral adrenoleukodystrophy

- Hemophilia

- Muscular dystrophies

- Bibliography

GENE THERAPIES FOR ACQUIRED DISEASES OTHER THAN CANCER

- Infectious diseases

- Cardiovascular disease

- Sensory diseases

- Neurological disease

- Other diseases

- Bibliography

REGULATORY ISSUES

- Introduction

- Regulatory framework in the EU

- Regulatory framework in the US

- Bibliography

REIMBURSEMENT ISSUES

- In rare diseases, return on investment is typically realized through repeated drug administration

- An alternative to a high single payment may be annuity payments for effective treatment

- Pay for-performance models may be suitable for gene therapy reimbursement

- Payers are not ready, but gene therapies may drive rethinking of drug pricing in general

- Glybera's reimbursement struggles reveal uncertainties around long-term effects to be a key concern for payers

- Imlygic struggles to gain reimbursement amid increased competition within melanoma

- GlaxoSmithKline to use Strimvelis to test alternative funding mechanisms

- Bibliography

DEALS AND ACQUISITIONS

- Five years of deal-making in the gene therapy area

For more information visit http://www.researchandmarkets.com/research/f37ldb/trends_in_gene

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Laura Wood, Senior Manager
press@researchandmarkets.com
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Contacts

Research and Markets
Laura Wood, Senior Manager
press@researchandmarkets.com
For E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900
U.S. Fax: 646-607-1907
Fax (outside U.S.): +353-1-481-1716