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Genetic Modification Therapies Clinical Applications: Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing

Genetic Modification Therapies Clinical Applications: Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing

  • August 2018
  • 211 pages
  • ID: 5501535
  • Format: PDF
  • By BCC Research


Table of Contents

Report Scope:
The study scope includes clinical therapies using gene editing, gene therapy, genetically modified cell therapy (chimeric antigen receptor T-cells, or CAR T-cells) or RNA therapy platforms.Delivery technologies covered include AAV, adenovirus, lentivirus, retrovirus, other viral and nonviral.

Disease areas covered include cardiology, oncology, ophthalmology, hematology, musculoskeletal, neurology, rare diseases and others. Therapies that are already commercialized or are forecast to be commercialized with the next five years are included.

We analyze key genetic modification therapy technologies and products to determine present and future market status and forecasted growth from 2018 through 2023. We also discuss strategic alliances, industry structures, competitive dynamics, patents and market driving forces.

BCC Research examines the genetic modification therapies industry by market segment, including the following segments: gene editing, gene therapy, CAR T-cell therapy, RNA therapy and lentiviral production.The geographic market for genetic modification therapy products by platform technology is analyzed.

Regions include North America, Europe, Asia-Pacific and the rest of the world.

The role of key strategic alliances and acquisitions from Jan. 2016 through June 2018 is discussed.

The challenges involved with pricing for genetic modification therapies that can offer one-time cures for diseases is discussed.Emerging markets for clinical gene editing are analyzed.

More than 145 companies in the genetic modification therapies industry are profiled.

Report Includes:
- 30 data tables and 56 additional tables
- An overview of the global market for genetic modification therapies
- Analyses of global market trends with data from 2017, estimates for 2018, and projections of compound annual growth rates (CAGRs) through 2023
- Information on the genetic variation analysis and genetic modification technologies
- Evaluation of key growth driving forces, acquisitions and strategic alliances related to the market
- Detailed description of the criteria for selecting gene modification therapy applications
- Review of patents for gene editing, gene therapy, RNA therapy and gene modification therapies
- Profiles of the key companies in the market, including 4d Molecular Therapeutics LLC, Abeona Therapeutics LLC, Acer Therapeutics Inc., Allergan Plc and American Gene Technologies International Inc.

Genetic modification therapies are changing the landscape of medicine due to their unprecedented ability to offer a one-time cure for severe, debilitating diseases that were previously untreatable. Genetic modification therapies are advanced therapies that use genetic material (i.e., DNA or RNA) to treat diseases.

The four main genetic modification therapy platform technologies covered in this report include gene therapies, genetically modified cell therapies, RNA therapies and gene editing.

In Dec. 2017, Luxturna became the first gene therapy approved for treating a genetic disorder. Luxturna is indicated for treating a severe inherited retinal disorder caused by a mutation in a single gene. Luxturna meets a clear and pressing medical need of restoring functional vision to a patient.

Besides Luxturna, several genetic modification therapies have been approved, including Kymarih and Yescarta (genetically modified cell therapy), Spinraza (RNA therapy) and others, setting the stage for strong market growth in the next five years.

Reasons for Doing the Study

Genetic modification therapies, particularly gene therapy and RNA therapy, have existed for many years, with little clinical success. However, recent improvements in these therapies, including better delivery systems, more efficient and durable gene expression constructs, precise DNA editing tools, have brought this industry to the forefront, and it is now poised for explosive growth in the coming years.

Because of the potentially curative nature of these medicines there is enormous potential in many applications, ranging from cancer to neurology to rare diseases.Genetic modification therapies represent the next wave of medicines with enormous potential for treating and curing debilitating and serious diseases.

Because of its wide scope, genetic modification therapy will play an important role in the future global medical economy.

Continuing advances in key technologies such as DNA editing, viral design and production, and gene expression, as well as a pressing medical need in many serious and debilitating disorders, are driving the growth of the market for genetic modification therapies.Developments in these multidisciplinary fields promise to advance the genetic modification therapies industry and create unique market opportunities.

This report analyzes these trends and their impact on the future markets for genetic modification therapy products.

Based on these market and technology dynamics, it is especially timely to comprehensively examine the current and future markets for genetic modification therapies.

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